Congruence Therapeutics today announced that it has entered into a research collaboration agreement with Ono Pharmaceutical Co., Ltd. (Ono) to discover novel drug candidates leveraging Congruence’s proprietary drug discovery platform, Revenir™.  Congruence will apply its technology and expertise toward multiple cancer protein targets.  Once the discovery effort advances to a prespecified stage, Ono has the option to acquire exclusive worldwide rights to further develop and commercialize small molecule correctors generated during the collaboration.

“We believe that this collaboration with Congruence may help generating novel small molecule correctors for validated targets in the oncology area by leveraging their own technologies in protein dynamics and computational biology, leading to our development pipeline,” said Seishi Katsumata, PhD, Corporate Officer / Executive Director, Discovery & Research of Ono. “We will be committed to delivering innovative new drugs to cancer patients as soon as possible.”

The collaboration will leverage Congruence’s purpose-built computational drug discovery engine called Revenir™, which captures the biophysical changes caused by mutations in proteins.  By examining surface features and numerous biophysical descriptors of both the mutated and wild-type proteins, Congruence can derive novel insights regarding protein defects and how to correct them.  Congruence’s current pipeline consists of both first-in-class and best-in-class potential therapeutic candidates that address significant unmet medical need in a number of high value indications.  

“Congruence is thrilled to partner with Ono, which has established itself as a global leader in drug development, particularly in the oncology space. We believe that our Revenir™ platform and capabilities in protein dynamics will accelerate the discovery of novel therapies for compelling targets of interest to both companies,” said Sharath Hegde PhD, Chief Scientific Officer of Congruence.

Under the terms of the collaboration, Congruence will receive an undisclosed upfront payment and is eligible to receive milestone payments upon the achievement of certain discovery, development, approval and sales events – as well as tiered royalties based on annual net sales of related products.  Ono will additionally reimburse Congruence for the research costs it incurs in connection with the collaboration. Ono has the option to take an exclusive worldwide license to development candidates discovered under the collaboration.

About Revenir™ Drug Discovery Platform

Revenir™, Congruence’s proprietary computational drug discovery platform, captures the dynamic biophysical changes caused by mutations in proteins, offering unique insights into protein defects and their correction. By examining surface features and a spectrum of biophysical descriptors across an ensemble of protein conformers, Revenir™ predicts small molecule induced correction of the underlying defect.

About Congruence Therapeutics

Congruence is a computationally-driven biotechnology company building a unique pipeline of transformative small molecule correctors rationally designed to rescue aberrant protein function. Our proprietary scalable platform, Revenir™, captures the biophysical features of proteins across their conformational ensembles, in order to identify novel allosteric and cryptic pockets which are virtually screened to generate novel chemical matter.

For more information, please visit www.congruencetx.com.

Company Contact
Charles Grubsztajn
Chief Operating Officer
cgrubsztajn@congruencetx.com

Media Contact
Amy Conrad
Juniper Point
amy@juniper-point.com 
858-366-3243

COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced a strategic collaboration and licensing agreement with Genentech, a member of the Roche Group, for the development and commercialization of COUR’s proprietary tolerogenic nanoparticle treatments for an autoimmune disease.

“Genentech has a long history of innovation, and we are excited to collaborate on the development of disease-modifying therapies leveraging COUR’s proprietary tolerance platform for the treatment of an autoimmune disease,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “We see Genentech as an ideal partner with world-class scientific expertise and together have an opportunity to build upon COUR’s clinical success to date in autoimmune diseases. Through this partnership, we believe we have the potential to have a significant impact on an important patient community.”

“We have a long and successful legacy of advancing innovative science and developing transformative medicines for people diagnosed with autoimmune diseases,” said Boris L. Zaïtra, Head of Roche Corporate Business Development. “Collaborating with innovative partners such as COUR supports our rigorous commitment to pioneering science in pursuit of advances in research, such as novel tolerogenic nanoparticle treatments, to deliver the most impactful medicines for patients with significant unmet needs.”

Under the terms of the agreement, COUR is eligible for upfront and near-term milestones of $40 million, plus additional development, commercial and net sales milestone payments that could exceed $900 million, as well as tiered royalties on net sales. Under the collaboration agreement with Genentech, COUR is responsible for the preclinical development and technical transfer of manufacturing. Genentech will be responsible for clinical development, regulatory filing, and commercialization.

About COUR Pharmaceuticals:

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.

COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.

For more information, please visit www.courpharma.com

Contacts
For Investor Relations
Brian Bock, Chief Financial Officer
bbock@courpharma.com

For Media
Jason Braco
jbraco@lifescicomms.com

KisoJi Biotechnology Inc., a company focused on the discovery and development of transformative antibody therapeutics, announces that it has raised $41 million in equity, including the conversion of previously funded convertible debentures.  These funds will be used to advance its lead asset, KJ-103, the first potent naked antibody against TROP2, into the clinic to treat solid tumours.  Funds will also be used to deploy its cutting-edge antibody discovery platform towards new multi-specific therapeutic antibody drugs in cardiometabolic disease, as well as immunology and inflammation.  The financing was led by Investissement Quebec and Lumira Ventures with participation by Fonds de solidarite FTQ, adMare BioInnovations, and Remiges Ventures.

As previously announced, KisoJi has established a clinical development partnership with Cancer Research UK to bring KisoJi’s lead asset, KJ-103, into a first-in-human clinical trial of approximately 100 patients.  KJ-103 is highly differentiated from existing TROP2-targeting drugs on the market or in development, which are antibody drug conjugates (ADCs).  KJ-103 does not require a cytotoxic payload but instead functions by recruiting immune cells to kill tumour cells. 

The financing will also support the deployment of KisoJi’s cutting-edge antibody discovery platform, including the first of its kind antibody paratope map (KisoSeek^TM) that effectively visualizes an immune response against a given target.  The Company’s sampling tools allow it to maximize paratope diversity in a directed iterative strategy to focus on distinct antibodies and uncover novel biology, including functional GPCR antibodies.

KisoSeek^TM is more efficient and provides greater insights than traditional antibody discovery methods and can integrate seamlessly into existing workflows.  All existing data, whether positive or negative, can be used to augment a target specific paratope to supercharge antibody discovery instead of relying on traditional methods of random screening.

KisoJi has also developed a suite of camelid transgenic mice (KisoMouse®) that generate single domain antibodies to continue populating the world’s largest single domain antibody database created by KisoJi over the past 8 years.  The paratope map and antibody database can be used to generate first-in-class and best-in-class multi-specific antibodies using KisoJi’s modular multi-specific antibody format (KisoBody^TM).  The KisoBody^TM is easily customizable and manufacturable with tri-specific antibody titers at greater than 6 g/L in commercial production cell lines.

Bicha Ngo, President and CEO, Investissement Québec, said: “By contributing to KisoJi Biotechnology’s financing round, Investissement Québec is reaffirming its commitment to supporting innovative companies in the life sciences industry, a key sector for the Québec economy. This funding will allow KisoJi Biotechnology to hit critical milestones in its operations, from completing preclinical activities to launching clinical trials, notably through a landmark agreement with Cancer Research UK, one of the world’s largest funders of cancer research, all the while helping move innovations from the R&D stage to the marketplace.”

Daniel Hétu, Managing Director, Lumira Ventures, said: “KisoJi has built a truly differentiated, AI enabled and comprehensive antibody discovery platform leveraging the deep expertise of their team that spans transgenic mice, single-domain antibodies and AI. Lumira is proud to have supported the development of KisoJi’s innovative platform from its inception and we look forward to continuing to work with the company and our co-investors as KisoJi transitions its propriety programs to the clinical stage.

Maxime Pesant, Vice-President Private Equity and Impact Investing | Life Sciences, Fonds de solidarité FTQ, said: “The Fonds de solidarité FTQ is proud to reiterate its commitment to KisoJi, an innovative company of which we have been partners from the very beginning alongside Lumira. Our strategic investments in the sector help propel high-potential biotechs to new heights. With major expertise in the life sciences field and with our long-term presence, the Fund continues to support high-impact companies like KisoJi, paving the way for groundbreaking innovations and significant medical advancements.”

David Young, co-founder and CEO of KisoJi said: “Through the support of our new and existing investors, this financing will enable KisoJi to continue its development of pipeline assets in oncology, cardiometabolic and immunology indications using our cutting-edge platform and AI tools.”

Evolution Venture Partners acted as the exclusive strategic advisor to KisoJi in connection with the financing.

About KisoJi Biotechnology 
KisoJi is a Canadian biotechnology company that deploys the latest scientific and AI tools to conduct therapeutic antibody discovery in a fundamentally new way. KisoJi has developed a multi-species transgenic mouse to generate highly diverse single domain antibodies, as well as a modular multi-specific antibody scaffold with high stability and productivity. Most recently, KisoJi has used advanced AI tools to visualise the universe of all antibodies against a target in order to uncover novel biology and new therapeutic antibody capabilities for its partners and its own pipeline.

www.kisojibiotech.com

About KJ-103 
KJ-103 is a single domain antibody that binds to a unique epitope on TROP2. It acts via effector cell mechanisms, including the activation of macrophages, to kill tumour cells. KJ-103 has been shown to have significant anti-tumour potency with no evidence of toxicity or resistance across a number of preclinical solid tumour models. KJ-103 was humanised by LifeArc.

KisoJi Biotechnology Contact:
Investors and Media
Argot Partners
Sam Martin/Cameron Willis
KisoJi@argotpartners.com
+1 212 600 1902

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced the acquisition of a portfolio of novel nucleoside analogs from PrimeFour Therapeutics, Inc., a biotechnology company focused on cancer chemotherapeutic agents.

The portfolio of novel nucleosides consists of a chemically diverse library of unique analogs. The most advanced lead compounds are nucleosides that cause “synthetic lethality” in cancers characterized by distinct tumor-defined genetic signatures, allowing them to be precisely targeted to the most susceptible cancers with reduced toxicity. These compounds can be targeted towards cancers with newly discovered genetic biomarkers that are present in pancreatic and certain other solid tumors as well as in hematologic cancers, allowing for personalized therapy in those patients most likely to respond to treatment.

“This transaction allows us to build upon our leadership in developing first-in-class and best-inclass nucleoside reverse transcriptase inhibitors for neurodegenerative and autoimmune diseases, including ALS, PSP and Alzheimer’s disease. We are excited to expand our pipeline to include this portfolio of potent nucleoside chemotherapeutics with significant therapeutic potential against a variety of cancers,” said Eckard Weber, M.D., founder and Chief Innovation Officer of Transposon. “We look forward to advancing the lead compounds into preclinical development to target DNA-damage repair deficient cancers, such as pancreatic and colorectal cancers.”

About Transposon Transposon Therapeutics, Inc.

is a clinical-stage biopharmaceutical company developing a platform of novel nucleoside reverse transcriptase inhibitors for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. Transposon is advancing TPN-101 into Phase 3 registration studies for the treatment amyotrophic lateral sclerosis (ALS) and progressive supranuclear palsy (PSP), and a Phase 2 proof-of-concept study in Alzheimer’s disease. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications

About PrimeFour

PrimeFour discovered and developed a class of novel nucleoside analogs that are selectively incorporated into cancer cells with biomarker-identifiable DNA damage repair deficiencies and that, once incorporated into some cancer cells, cause irreversible DNA damage and cancer cell death.

AmacaThera today announced results from its Phase 1 clinical trial of AMT-143, an innovative, non-opioid anesthetic, formulated with the company’s patented AmacaGel™ platform, demonstrating sustained anesthetic release out to 14 days.  AMT-143 is intended to deliver extended, localized pain relief without the need for opioids, significantly reducing the risks associated with opioid addiction and side effects in order to improve patient outcomes post-surgery.

“We are excited by the promise of these results as they show a substantial differentiation above and beyond the currently available products and herald a next generation of post-surgical pain relief alternatives,” said Dr. Mike Cooke, Chief Executive Officer, and Co-Founder of AmacaThera, “We look forward to advancing this important product into a Phase 2 study.”

The recent clinical data has strengthened support for AmacaThera with lead investor of AmacaThera’s recently completed Series A extension Paul Austin saying “The progress shown in the Phase 1 study demonstrates the tremendous potential of AMT-143 to transform post-operative pain management.  Based on these strong results, we are increasing our investment to accelerate its continuing development.”

The urgent need for non-opioid, long-acting anesthetics stems from the ongoing opioid crisis, where opioid-based pain management serves as a gateway to opioid abuse.  While effective for acute pain relief, opioids can result in addiction, overdose, and adverse side effects, creating a pressing need for safer alternatives.  Non-opioid long-acting anesthetics offer a viable solution by providing effective pain relief without the risk of dependency or the severe side effects commonly associated with opioids.

Dr. Molly Shoichet, Chief Scientific Officer and Co-Founder of AmacaThera: “The Phase 1 clinical data, in combination with our optimized release kinetics, cost effective manufacturing, and ease of application comprise the solution physicians want for their patients.”

About AmacaThera

AmacaThera is a venture backed clinical stage biotechnology company supported by institutional investors including; Lumira Ventures, BDC Capital’s Women in Technology Venture Fund, Inveready, StandUp Ventures, MaRS IAF, and Changrong Capital.

AmacaThera is specializing in the development of advanced sustained release hydrogel formulations to solve key therapeutic delivery challenges with both off-patent and proprietary payloads. AmacaThera is focused on developing an internal pipeline of pain management and oncology assets; and partnering to solve key delivery challenges with advanced proprietary therapeutic modalities.

AmacaThera’s platform technology, AmacaGel^TM, is a fast-gelling physical hydrogel blend of two well-established polymers.  It has been designed to liquify under shear force and to be delivered by a conventional syringe, thereby rapidly forming a depot as it warms to body temperature. AMT-143, the platform’s lead asset, is a slow-release non-opioid, local anesthetic leverages the AmacaGel^TM platform to provide long-acting, post-operative pain relief.

With the global market for non-opioid pain management expected to exceed $96 Billion by 2034¹.  AmacaThera will address a critical gap in post-operative care with AMT-143.

With strong early data, AmacaThera is actively seeking strategic partners to expand the clinical program and accelerate market entry for AMT-143 and related products.

For more information, visit www.amacathera.ca

Thryv Therapeutics Inc., a clinical-stage biotechnology company pioneering novel therapies for cardiovascular diseases, is pleased to announce the appointment of Amy Sehnert, MD, as Chief Medical Officer (CMO). Dr. Sehnert will lead the clinical development and strategy for Thryv Therapeutics’ expanding cardiometabolic product portfolio targeting serum glucocorticoid inducible kinase 1 (SGK1) – a kinase implicated in adverse electrical and structural remodeling of the heart.

Dr. Sehnert is a pediatric cardiologist with 20 years of experience as a serial and founding medical lead at innovative biotech companies introducing precision diagnostics and therapeutics to the clinic. Dr. Sehnert joins Thryv Therapeutics from Bristol Myers Squibb (BMS), where she held leadership roles of increasing responsibility and most recently served as Vice President and Global Program Lead for cardiovascular assets transitioning from early to late development. Prior to her time at BMS, Dr. Sehnert served as Vice President, Clinical Science at MyoKardia, Inc., where she played a pivotal role in the development and approval of Camzyos® – a groundbreaking treatment for hypertrophic cardiomyopathy (HCM) – contributing to the $14 billion acquisition of MyoKardia by BMS in 2020.

“I am thrilled to join Thryv Therapeutics at such an important time in the company’s journey,” said Dr. Sehnert. “Thryv Therapeutics’ cutting-edge approach to cardiovascular diseases aligns with my passion for advancing precision medicine, and I look forward to working with the team to deliver transformative therapies to patients worldwide.”

Dr. Sehnert has also made notable contributions in other fields of medicine, such as maternal-fetal health, oncology, and genomics, leading early efforts in non-invasive prenatal testing (NIPT) and cell-free DNA applications now in wide clinical use. She holds a Doctor of Medicine degree from the University of Minnesota, completed Pediatrics Residency at University of Colorado, and Fellowship in Pediatric Cardiology at UCSF followed by faculty appointments at UCSF and Stanford University. In 2022 she was recognized as one of the Most Influential Women in Business by the San Francisco Business Times and among the Top 25 Healthcare Technology Leaders of San Francisco. She has co-authored over 60 peer-reviewed publications and has been involved in numerous international collaborations, making her an internationally recognized expert in her field.

“On behalf of Thryv Therapeutics, I am excited to welcome Amy to the team,” said Paul F. Truex, CEO of Thryv Therapeutics. “Dr. Sehnert brings a wealth of medical and clinical development knowledge in the field of cardiometabolic and genetic diseases. Her extensive experience in designing and executing late-stage clinical trials for genetic and broader cardiovascular diseases and leadership qualities will be invaluable as we enter the next phase of clinical development of our SGK1 portfolio.”

Dr. Philip Sager, Thryv Therapeutics’ founding Chief Medical Officer, will continue to provide strategic clinical and regulatory guidance as the company’s portfolio advances into later stage clinical studies.

“Philip and his collaborators originally developed unique insights into the clinical potential for SGK1 in various cardiometabolic diseases and in particular, Long QT Syndrome. His tireless leadership and relentless passion for exploring these initial ideas advanced Thryv Therapeutics’ portfolio to its current stage,” said Paul F. Truex. “We’re thankful for his continued, on-going contributions in his new role to all of our clinical programs and we are inspired by his commitment to finding treatments for these patient communities.”

About Thryv Therapeutics Inc.

Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop potent and highly selective serum glucocorticoid inducible kinase 1 (SGK1) inhibitors to treat heart failure, atrial fibrillation, Long QT Syndrome and other cardiometabolic and cardiorenal diseases. Numerous animal models have demonstrated that activation of SGK1 leads to inappropriate electrical and structural remodeling of the heart contributing to adverse outcomes in heart failure, atrial fibrillation, and genetic channelopathies, such as Long QT Syndrome. For more information, please visit www.thryvtrx.com.

Media Inquiries

brittany@thryvtrx.com | 514 973 0915

HistoSonics, the developer and manufacturer of the Edison® Histotripsy System, announced today the first patients were enrolled in their novel prospective master protocol study, called BOOMBOX, designed to evaluate the real-world use of histotripsy for treatment of liver tumors. BOOMBOX is a comprehensive, post-market master study that will provide important data for up to 5,000 patients treated across a growing global base of multi-disciplinary histotripsy providers. This master study design is unique in the medical device industry, as it captures treatment data across diverse tumor types and disease stages in patients with varying interventional needs, and amongst multiple clinical specialties.

“Traditionally, this complexity would require multiple individual study protocols over a prolonged period of time, delaying access to histotripsy for patients in need,” said Amanda Cafaro, VP of Clinical Affairs, HistoSonics. “We designed this post-market master protocol study and its accompanying sub-studies to accelerate our understanding of a wide range of clinically relevant research questions, expediting access to this innovative therapy.”

The primary endpoint for BOOMBOX will examine histotripsy’s capability as a versatile tool and the subsequent sub-studies will evaluate additional endpoints to demonstrate increasingly specific treatment goals: as a monotherapy, a combination therapy, for curative intent as well as priming for additional treatments. Enrollment has begun for the BOOMBOX study across multiple US histotripsy clinical treatment centers and the company expects participation from both US and global histotripsy sites.

“Histotripsy represents a novel treatment for my patients suffering with liver tumors and has already demonstrated an enhanced benefit-risk profile in a clinically meaningful way,” said Juan P. Rocca, M.D., MHA, surgical director for the New York-Presbyterian and Weill Cornell Medicine Liver Cancer Program, one of the centers around the country to offer histotripsy. “By participating in the BOOMBOX study, we are engaging in practical, real-world research that will drive the advancement of this innovative platform and explore new ways to utilize histotripsy in the future. Participating in BOOMBOX is exactly the type of practical research that will continue to advance this groundbreaking new platform and help us find new and innovative ways to use histotripsy going forward.”

“The depth and breadth of data we will capture as part of the BOOMBOX study will provide us and our histotripsy program partners with insight on how histotripsy may impact patients across many diseases and patient conditions. BOOMBOX is our long-term commitment to learning how histotripsy can best improve the lives of patients suffering from liver tumors and the debilitating symptoms these patients face,” said Joe Herman, MD, VP of Medical Affairs, HistoSonics.

The BOOMBOX study begins HistoSonics’ post-market evidence generation efforts in the liver after the recent publication of the company’s pivotal clinical #HOPE4LIVER trials which were submitted to the US Food and Drug Administration (FDA) as part of HistoSonics market access submission resulting in FDA De Novo grant in October 2023 for the non-invasive destruction of liver tumors using histotripsy. In addition to the BOOMBOX: Master Study, HistoSonics has established an ongoing Investigator Sponsored Research program designed to support independent research with histotripsy providers through grants, advancing the safe and effective use of histotripsy.

The Edison System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any disease including, but not limited to, cancer or evaluated any specific cancer outcomes (such as local tumor progression, 5-year survival or overall survival). The System should only be used by physicians who have completed training performed by HistoSonics, and its use guided by the clinical judgment of an appropriately trained physician. Refer to the device Instructions for Use for a complete list of warnings, precautions, and a summary of clinical trial results, including reported adverse events.

About HistoSonics

HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison System in the US and select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

For more information on the BOMBOX study please reference NCT06486454 at BOOMBOX Study Information at ClinicalTrials.gov. For information on the Edison Histotripsy System please visit: www.histosonics.com/. For patient related information please visit: www.myhistotripsy.com/.

Contacts

Media contacts:
Josh King
Vice President of Global Market Access
Joshua.king@histosonics.com
608-332-8124

Nocion Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions”, that selectively silence actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain, today announced that the first patient has been dosed in the Phase 2b ASPIRE clinical trial of taplucainium for the treatment of chronic cough.

ASPIRE is a Phase 2b, randomized, double-blind, placebo-controlled study investigating the efficacy, safety, and tolerability of Taplucainium Inhalation Powder (NOC-110) once daily in adults with refractory or unexplained chronic cough. Approximately 325 patients will be enrolled in the study at over 100 sites across the US, Canada, UK and Europe.

“Dosing of the first patient in the ASPIRE clinical study is a significant milestone for Nocion,” said Rick Batycky, CEO of Nocion Therapeutics. “Taplucainium represents a novel approach to the treatment of chronic cough with the potential to deliver enhanced efficacy and reduced side effects to more than 8 million patients per year just in the United States.”

ABOUT TAPLUCAINIUM
Taplucainium (formerly NTX-1175) is a proprietary molecule in the novel class of charged sodium channel blockers that allows for specific silencing of activated/inflamed nociceptors while having minimal local off-target effects or systemic exposure. Unlike other investigative cough therapies, such as P2X3 antagonists, which target a specific large pore channel, taplucainium is formulated into a dry powder for inhalation, and once inhaled, gains access to the pulmonary nociceptors through any open large pore channel including P2X, TRPV and TRPA channels whereupon it inhibits the sodium channels responsible for initiating the pathological cough response. The broader mechanism of taplucainium should translate into an efficacious, long-lasting, antitussive that is fast acting in a number of cough conditions. Combined with good preliminary safety and efficacy data from earlier stage clinical work, this forms the basis for its use in cough indications beyond chronic cough. For more information, visit https://www.aspirechroniccoughstudy.com/#!/

ABOUT NOCION
Nocion Therapeutics is a biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions” that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain. The company’s mission is to safely alleviate suffering for millions of patients with conditions arising from activated sensory neurons. Working with Harvard’s Office of Technology Development, Nocion was founded on an exclusive license to foundational intellectual property from Harvard University and Boston Children’s Hospital. Venture investors in Nocion include Arkin Bio Capital, Canaan Partners, F-Prime Capital, Lumira Ventures, Mass General Brigham Ventures, Mission BioCapital, Monograph Capital, Morningside and Osage University Partners. For more information, visit: www.nociontx.com.

COMPANY CONTACT
Stephanie Gillis
pr@nociontx.com

Thryv Therapeutics Inc., a biotechnology company pioneering treatments for genetic and cardiometabolic conditions, including congenital Long QT Syndrome, heart failure, and atrial fibrillation, has completed phase 1 dosing of its second SGK1 inhibitor.  This study was a first-in-human, randomized, double-blind, placebo-controlled, single and multiple ascending dose study to assess the safety, tolerability, pharmacokinetics, and food effect of THRV-1268 in healthy adult subjects.  The completion of dosing marks a significant milestone in the development of THRV-1268, laying the groundwork for future clinical investigations in heart failure and atrial fibrillation. 

“Completion of this first-in-human study is the initial clinical step with THRV-1268, providing a path towards addressing cardiometabolic diseases where both structural and electrical remodeling lead to adverse CV outcomes in patients with atrial fibrillation and heart failure,” stated Philip Sager, MD, Chief Medical Officer of Thryv Therapeutics. “Building on our initial clinical work with LQT-1213 for Long QT Syndrome, we now have an opportunity to address both the pro-arrhythmic and metabolic pathways of heart failure and atrial fibrillation in patients with underlying metabolic disease.  We look forward to beginning our proof-of-concept studies next year.” 

THRV-1268 is a novel and potent inhibitor of the Serum Glucocorticoid Kinase 1 (SGK1). SGK1 is a PI3-kinase-dependent kinase with its expression and activation regulated by several metabolic signaling pathways that are crucial in cardiometabolic diseases. Thryv Therapeutic’s first SGK1 inhibitor, LQT-1213, repeatedly demonstrated reductions in QTc interval in pre-clinical studies and in patients with the genetic and acquired Long QT Syndrome. SGK1 has been implicated in various pathological conditions, including QTc prolongation, arrhythmias, fibrosis, and heart failure. SGK1 dysfunction is also linked to cardiometabolic stressors such as obesity and hypertension, which are common comorbidities in heart failure.    

Genetic and pharmacological inhibition of SGK1 has been demonstrated to have a protective effect in a mouse model of obesity-related atrial fibrillation. In several preclinical models of heart failure, THRV-1268 mitigated the development of heart failure and fibrosis, and reduced heart failure biomarkers associated with adverse changes in hemodynamic and functional cardiovascular outputs.  These findings demonstrate the potential of SGK1 inhibition in addressing the pathogenesis of heart failure and related cardiometabolic conditions, including atrial fibrillation.  Results from the THRV-1268 non-clinical efficacy program are to be presented at the American Heart Association meeting on November 16 – 18, 2024 in Chicago, Illinois. 

Heart Failure 

Heart failure is a complex clinical syndrome characterized by the heart’s inability to pump sufficient blood to meet the body’s needs. It can manifest in two primary forms: Heart Failure with Reduced Ejection Fraction (HFrEF), where the heart muscle is weakened and cannot contract effectively, leading to a reduced ejection fraction; and Heart Failure with Preserved Ejection Fraction (HFpEF), where the heart muscle contracts normally but is stiff and unable to relax properly, resulting in impaired filling and increased pressure in the heart.  Often coexisting with atrial fibrillation, heart failure can result from various underlying conditions such as coronary artery disease, hypertension and previous heart attacks.  If left untreated, heart failure leads to symptoms like fatigue and shortness of breath that profoundly impact a patient’s quality of life, and significantly increases the risk of severe complications and mortality. 

Atrial Fibrillation 

Atrial fibrillation (AF or AFib) is a prevalent cardiac arrhythmia characterized by irregular and often rapid heartbeats.  Affecting nearly 40 million individuals worldwide, AF significantly diminishes quality of life and heightens the risk of stroke, heart failure and other cardiovascular complications.  Obesity and metabolic disorders are key risk factors for AF, contributing to its rising prevalence.  Effective treatment strategies aim to control heart rate, restore normal heart rhythm and mitigate complications.

About Thryv Therapeutics Inc.

Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop potent and highly selective inhibitors of serum glucocorticoid inducible kinase 1 (SGK1) to treat Long QT Syndrome, heart failure, and atrial fibrillation.  For more information, please visit www.thryvtrx.com. 

COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced positive 120-day data from its Phase 2a study evaluating CNP-104 as a potential treatment for patients with Primary Biliary Cholangitis (PBC), a chronic liver disease that is estimated to affect approximately 130,000 individuals in the U.S.

“These data highlight the potential of CNP-104 to be the first disease-modifying treatment for people living with PBC,” said Paul M. Peloso, M.D., Chief Medical Officer of COUR Pharmaceuticals. “In addition to safety and tolerability data supporting further studies, we observed multiple immunological and clinical measurements supporting mechanistic proof of concept for CNP-104. Additionally, we observed positive clinical endpoints such as a reduction in liver stiffness measured on Fibroscan in the active arms compared to placebo, with Fibroscan being a surrogate for reductions in fibrosis. These data suggest that CNP-104 has the potential to halt disease progression which would be a transformational advancement for people living with PBC. We look forward to presenting these encouraging data at an upcoming scientific meeting.”

Data Highlights:

• CNP-104 led to a slowing of disease progression in liver stiffness on Fibroscan, reaching a statistically significant decrease on day 120 in the active arms compared to placebo (p = 0.011)
• Study subjects in the placebo arm experienced a greater trending decrease in albumin levels compared with CNP-104 treated patients
• CNP-104 demonstrated a favorable T cell response in pathogenic CD4 T cell populations and tolerance inducing CD8 T cells
• Notably, Th17 T cells decreased in both number and percentage mean change, with statistically significant higher rates of response in the active arms versus placebo at day-120 (p = 0.0037)
• CNP-104 was safe and well tolerated, and all drug related adverse events (AEs) were mild (Grade 1 or 2) and no drug related severe adverse events (SAEs) were reported

The Phase 2a first-in-human, proof-of-concept, randomized clinical trial (NCT05104853) is designed to assess the safety, tolerability, pharmacodynamics, and efficacy of CNP-104 in patients aged 18-75, unresponsive to treatment with ursodeoxycholic acid (UDCA) and/or obeticholic acid (OCA). Participants received 4 mg/kg or 8 mg/kg of CNP-104 or placebo on Days 1 and 8 with all patients receiving two I.V. loading doses administered one week apart. Of the 41 subjects dosed, the final randomization ratio approximated 1:1:1 placebo to 4 mg/kg CNP-104 to 8 mg/kg CNP-104. This study report considers the 120-day primary study period while the 20-month long-term safety evaluation is ongoing.

Christopher Bowlus, M.D., the Lena Valente Professor and Chief, Division of Gastroenterology and Hepatology at UC Davis, and Principal Investigator for the study, added, “Despite the recent evolution of the PBC treatment landscape, there remains a significant unmet need for new treatments. Unlike CNP-104, current therapies do not address the root cause of the disease. While these data are early and from a small number of patients, they are incredibly promising and merit future investigation.”

Detailed results from the Phase 2a study of CNP-104 will be submitted for presentation at a future scientific conference.

About CNP-104:

CNP-104 is a biodegradable nanoparticle encapsulating the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), a key autoantigen in PBC. CNP-104 aims to address the root cause of PBC by inducing tolerance to pathogenic activated PDC-E2 T-cells that drive inflammation in bile ducts, leading to improvement in clinical outcomes of liver health. In January 2022, CNP-104 received Fast Track Designation from the United States Food and Drug Administration (FDA), making it potentially eligible for Accelerated Approval and Priority Review, if relevant criteria are met.

About PBC:

Primary biliary cholangitis (PBC) is a chronic, life-threatening autoimmune disease of the liver that primarily affects women between 40 and 60 years old. PBC is one of the leading causes of liver transplant in women. People with PBC experience impaired bile flow (cholestasis) and accumulation of toxic bile acids in the liver, which can progress to fibrosis, cirrhosis and liver failure. Increasing fibrosis results in cirrhosis and eventual liver failure and the need for a transplant. Additional symptoms of PBC include fatigue and pruritus, which can have a significant impact on the quality of life for patients. While the exact cause of PBC is unknown, the presence of antimitochondrial antibodies (AMA) which target the E2 component of the mitochondrial pyruvate dehydrogenase complex (PDC), are highly associated with the development of the disease.

About COUR Pharmaceuticals:

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.

COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.

For more information, please visit www.courpharma.com

Edesa Biotech, Inc. (Nasdaq:EDSA), a clinical-stage biopharmaceutical company focused on developing host-directed therapeutics for immuno-inflammatory diseases, today announced that the company has entered into a purchase agreement with an entity affiliated with Par Nijhawan, MD, Edesa’s Chief Executive Officer and Founder, to invest up to $5.0 million in the company, including an immediate investment of approximately $1.5 million.

The entity will purchase shares of the company’s Series A-1 Convertible Preferred Shares, as amended (the “Series A-1 Preferred Shares”), having a stated value of $10,000 per share, and warrants (the “Warrants”) to purchase the company’s common shares in a transaction structured as an at-the-market issuance under Nasdaq rules. The Series A-1 Preferred Shares will be convertible into the company’s common shares at a conversion price of $3.445. The Warrants will be exercisable for a number of common shares equal to 75% of the common shares initially issuable upon the conversion of the Series A-1 Preferred Shares.

Commenting on today’s announcement, Dr. Nijhawan said “I am pleased to demonstrate my strong belief in Edesa’s future growth opportunities and my continuing commitment to lead the company’s strategic initiatives. I believe Edesa has a strong development pipeline, and I am confident that we can continue to build on our operational and clinical success.”

Subject to certain exceptions and adjustments for share splits, each Series A-1 Preferred Share is convertible into a number of Edesa’s common shares calculated by dividing (i) the sum of the stated value of the Series A-1 Shares being converted plus a return equal to 10% of such stated value per annum by (ii) the conversion price. The Warrants will have an exercise price of $3.445 per share, will be exercisable immediately upon issuance and will expire five years from the date of issuance. Under applicable Canadian securities laws, purchases of Series A-1 Preferred Shares under the purchase agreement in an aggregate amount greater than $2.0 million will be subject to the prior approval of the company’s shareholders, excluding Dr. Nijhawan. The company also announced that in connection with the transactions contemplated by the purchase agreement, the $10 million revolving credit agreement previously entered into with the purchaser has been terminated. The company did not draw any funds from the facility.

The securities described above have not been and will not be registered under the Securities Act of 1933, as amended, and may not be offered or sold in the United States absent registration or an applicable exemption from registration requirements.

About Edesa Biotech, Inc.

Edesa Biotech, Inc. (Nasdaq: EDSA) is a clinical-stage biopharmaceutical company developing innovative ways to treat inflammatory and immune-related diseases. Its clinical pipeline is focused on two therapeutic areas: Medical Dermatology and Respiratory. In Medical Dermatology, Edesa is developing EB06, an anti-CXCL10 monoclonal antibody candidate, as therapy for vitiligo, a common autoimmune disorder that causes skin to lose its color in patches. Its medical dermatology assets also include EB01 (1.0% daniluromer cream), a Phase 3-ready asset developed for use as a potential therapy for moderate-to-severe chronic Allergic Contact Dermatitis (ACD), a common occupational skin condition. The company’s most advanced Respiratory drug candidate is EB05 (paridiprubart), which is being evaluated in a U.S. government-funded platform study as a treatment for Acute Respiratory Distress Syndrome (ARDS), a life-threatening form of respiratory failure. The EB05 program has been the recipient of two funding awards from the Government of Canada to support the further development of this asset. In addition to EB05, Edesa is preparing an investigational new drug application (IND) in the United States for EB07 (paridiprubart) to conduct a future Phase 2 study in patients with pulmonary fibrosis. Sign up for news alerts. Connect with us on X and LinkedIn.

Edesa Forward-Looking Statements

This press release may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements may be identified by the use of words such as “anticipate,” “believe,” “plan,” “estimate,” “expect,” “intend,” “may,” “will,” “would,” “could,” “should,” “might,” “potential,” or “continue” and variations or similar expressions, including statements related to: the company’s belief that Dr. Nijhawan’s investment is strategic and demonstrates a strong belief in Edesa’s future growth opportunities and a continuing commitment to lead the company’s strategic initiatives; the company’s belief that it has a strong development pipeline; the company’s confident belief that it can continue to build on its operational and clinical success; and the company’s timing and plans regarding its clinical studies in general. Readers should not unduly rely on these forward-looking statements, which are not a guarantee of future performance. There can be no assurance that forward-looking statements will prove to be accurate, as all such forward-looking statements involve known and unknown risks, uncertainties and other factors which may cause actual results or future events to differ materially from the forward-looking statements. Such risks include: the ability of Edesa to obtain regulatory approval for or successfully commercialize any of its product candidates, the risk that access to sufficient capital to fund Edesa’s operations may not be available or may be available on terms that are not commercially favorable to Edesa, the risk that Edesa’s product candidates may not be effective against the diseases tested in its clinical trials, the risk that Edesa fails to comply with the terms of license agreements with third parties and as a result loses the right to use key intellectual property in its business, Edesa’s ability to protect its intellectual property, the timing and success of submission, acceptance and approval of regulatory filings, and the impacts of public health crises. Many of these factors that will determine actual results are beyond the company’s ability to control or predict. For a discussion of further risks and uncertainties related to Edesa’s business, please refer to Edesa’s public company reports filed with the U.S. Securities and Exchange Commission and the British Columbia Securities Commission. All forward-looking statements are made as of the date hereof and are subject to change. Except as required by law, Edesa assumes no obligation to update such statements.

Contact:
Gary Koppenjan
Edesa Biotech, Inc.
(289) 800-9600

COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that the first patient has been dosed in its Phase 1b/2a proof-of-concept clinical trial evaluating CNP-106 as a potential treatment for people living with generalized myasthenia gravis (gMG).

“Currently approved treatments for gMG work via broad immunosuppression and do not address the underlying autoimmunity of the disease,” said Dannielle Appelhans, President and Chief Executive Officer of COUR. “In preclinical studies to date, CNP-106 has demonstrated a unique ability to induce tolerance to the acetylcholine receptor (AChR), leading to improved muscle function in an experimental model of autoimmune myasthenia gravis. These results indicate that CNP-106 has the potential to be a first-in-class treatment that offers people living with gMG a disease modifying therapy without burdensome immune system suppression.”

The Phase 1b/2a double-blind, randomized, placebo-controlled clinical trial (NCT06106672) will enroll up to 54 patients with gMG, aged 18-75, to evaluate the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106.

About CNP-106

CNP-106 is a biodegradable nanoparticle that encapsulates acetylcholine receptor (AChR). gMG is primarily driven by the production of antibodies against AChR in the bloodstream. These antibodies either block or destroy the receptors for acetylcholine, which is a crucial neurotransmitter for muscle contraction. As gMG progresses, it may lead to serious health issues, such as myasthenic crisis, tumors of the thymus gland, thyroid imbalances, and other autoimmune diseases. CNP-106 is designed to potentially induce the downregulation of AChR T cell populations and AChR antibodies, leading to improved muscle function.

About COUR Pharmaceuticals:

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune diseases.

COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having completed first-in-human clinical studies in Celiac Disease (partnered with Takeda Pharmaceuticals), and Primary Biliary Cholangitis.

For more information, please visit www.courpharma.com 

Axonis Therapeutics, a biotechnology company focused on the development of novel neuromedicines, announced today the successful completion of an oversubscribed $115 million Series A financing. Proceeds from the financing will be used to advance Axonis’ lead development candidate, AXN-027, through clinical proof-of-concept in patients. AXN-027 is a first-in-class oral small molecule designed to potentiate the function of KCC2, a major CNS chloride transporter essential for inhibitory neurotransmission, for the treatment of epilepsy and pain. Proceeds will also enable the development of next generation compounds targeting KCC2 in these and other indications, including psychiatric and neurodevelopmental disorders.

Cormorant Asset Management and venBio Partners co-led the financing with significant investments from Sofinnova Investments, MRL Ventures Fund, Perceptive Advisors, Lumira Ventures and Solasta Ventures. In conjunction with this financing, Axonis named Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant Asset Management and Jonathan Leff, M.D., Executive Partner, Private Equity, from Sofinnova Investments to its Board of Directors. Eos Bioinnovation, Hatteras Venture Partners, SCI Ventures, Alexandria Venture Investments, Tachyon Ventures, BoxOne, Iaso Ventures and Pathway Bioventures also participated in the financing.

“Axonis’ vision is to advance a pipeline of oral KCC2 therapeutics to restore functional inhibition in the CNS to treat neurological disorders without disabling side effects,” said Joanna Stanicka, Ph.D., Chief Executive Officer, President, and co-founder of Axonis. “As we head into the clinic later this year, this financing is a significant milestone for Axonis as we translate our exciting science into efficacious and well-tolerated treatments for patients suffering from neurological disorders.”

“We’re delighted that this accomplished group of investors share our mission to translate Axonis’ science into impactful medicines,” said Corey Goodman, Ph.D., Executive Chair and co-founder of Axonis.  “I’m pleased to see how far the science has progressed at Axonis; what the team has accomplished is remarkable. We are now well positioned to bring our lead therapeutic program through clinical proof of concept and expand into related arenas and build a leading biotechnology company.”

Despite the approval of several drugs for epilepsy and pain, many patients do not tolerate or respond to current first-line therapies due to impaired synaptic inhibition, which can contribute to drug resistance. Axonis’ oral KCC2 potentiators are designed to restore functional inhibition and overcome drug resistance in patients.

“I’m enthusiastic about the potential of Axonis’ novel precision approach to re-shape how we treat patients with neurological disorders, particularly epilepsy and pain where modulation of synaptic inhibition has great therapeutic promise,” said Raymond J. Kelleher, M.D., Ph.D., Managing Director at Cormorant. “Axonis’ scientists have done amazing work. We’re very excited about what this program could mean for patients and their families.”

Leadership Team

Axonis is led by Joanna Stanicka, Ph.D., co-founder and co-inventor of Axonis’ underlying IP. She is a Harvard-trained scientist, drug hunter, and entrepreneur, and a recipient of the Massachusetts Next Generation Award, a prize in leadership for woman-CEO in life sciences. Shane Hegarty, Ph.D., is the company’s Chief Scientific Officer, co-founder, and IP co-inventor. He was previously a Research Fellow at Harvard Medical School and Boston Children’s Hospital, faculty member in Neuroscience Department of University College Cork, and recipient of the Neuroscience Ireland Investigator Award.

In conjunction with the financing, Axonis also announces the appointments of Donald Manning, MD, Ph.D., as Chief Medical Officer and Jeff Imbaro, M.B.A., as Chief Operating Officer. Dr. Manning is a clinician-scientist with comprehensive experience in acute and chronic clinical practice, research and management. He brings to Axonis over 25 years of executive experience, having overseen the filings of seven INDs and five NDAs that have led to nine approved products. Mr. Imbaro brings over 20 years of company building expertise, having led Business Development and General and Administrative functions of several successful biotech and pharmaceutical companies. He was a member of the founding team of Semma Therapeutics, which was later acquired by Vertex Pharmaceuticals in 2019 for nearly $1 billion. 

About Axonis Therapeutics

Axonis Therapeutics is a neuro-focused biotechnology company developing first- and best-in-class medicines targeting KCC2, the critical mediator of inhibitory neurotransmission within the brain, by translating breakthrough discoveries spun out from Boston Children’s Hospital, Harvard and Université Laval. The company has built a proprietary KCC2 discovery engine, based on several years of world-leading know-how, to become the leaders in this potential blockbuster drug space.  Axonis is grateful for grant awards and support received from National Institutes of Health, Department of Defense SCIRP, ISS National Lab, CURE Epilepsy, Wings for Life, Massachusetts Life Center, SynGAP Research Fund, Praxis Spinal Cord Institute, Creative Destruction Lab, Springboard Enterprises and MassChallenge. The company is headquartered in Boston, MA. For more information, visit www.axonis.us.

About venBio Partners

Since inception in 2011, venBio has raised roughly $2 billion in capital commitments and led investment rounds in 40+ companies, including Labrys Biologics (acquired by Teva), Aragon Pharmaceuticals (acquired by Johnson & Johnson), Seragon Pharmaceuticals (acquired by Roche), Aurinia Pharmaceuticals (NASDAQ: AUPH), Apellis Pharmaceuticals (NASDAQ: APLS), Checkmate Pharmaceuticals (NASDAQ: CMPI, acquired by Regeneron), Turning Point Therapeutics (NASDAQ: TPTX, acquired by BMS), Akero Therapeutics (NASDAQ: AKRO), Harmony Biosciences (NASDAQ: HRMY), Pharvaris (NASDAQ: PHVS), CinCor Pharma (NASDAQ: CINC, acquired by Astra Zeneca), RayzeBio (NASDAQ: RYZB, acquired by BMS), and many others. For more information, visit the venBio website at www.venbio.com and follow on LinkedIn.

About Cormorant Asset Management

Founded in 2013, Cormorant Asset Management, LP (“Cormorant”) is a leading life sciences-focused investment firm with over $3 billion of assets under management. Cormorant is driven by a deep focus on fundamental scientific principles and provides financial resources to support the most innovative and promising publicly traded and private companies in biotech and allied sectors. Cormorant is among the most active crossover investors in life sciences, with investments in over 100 privately held, life science-focused companies during the past decade. 

HistoSonics, (www.histosonics.com), the developer and manufacturer of the Edison Histotripsy System, announced today they have been awarded an exclusive contract to provide Veteran’s Affairs hospitals across the United States access to their novel non-invasive tumor liquefying platforms. The contract, the work of the Veterans Health Administration (VHA) and the Strategic Acquisition Center (SAC), establishes a $90 million ongoing schedule to develop histotripsy programs at key VA Hospitals. The announced VA contract is the culmination of a multi-year effort by HistoSonics, the company called Project Hero, to ensure meaningful value for Veterans and their families, VHA clinicians, administrators, and caregivers.

Histotripsy is a novel form of focused ultrasound that uses high amplitude, very short pulses to create a “bubble cloud” that is designed to mechanically destroy and liquefy targeted liver tumors. These bubble clouds form and collapse in microseconds, creating mechanical forces strong enough to destroy tissue at cellular and sub-cellular levels in a non-invasive and non-thermal method. Histotripsy is a single-setting, outpatient procedure for most patients and offers a promising treatment option for patients with liver tumors, avoiding complications like infection and toxicity which are common for invasive surgical and radiation procedures. The company believes that the novel mechanism of action of their proprietary technology may provide significant advantages to patients, including the ability of the treatment site to recover and resorb quickly.

“We are pleased that Veterans suffering with tumors will soon have access to this innovative, non-invasive treatment at select VA facilities with interdisciplinary expertise,” said Patrick Malloy MD, Executive Director of the VHA National Radiology Program. Mark Wilson, MD, VHA National Director of Surgery, added, “Histotripsy is an innovative tool that will further enable VA specialists to continue to provide a full continuum of care to Veterans.”

Receiving FDA “De Novo” clearance in October 2023, the Edison Histotripsy System has been successfully put into clinical practice at leading US academic centers and integrated health systems, as well as many community hospitals seeking to offer breakthrough histotripsy treatment for their patients. Michael Kelly MD, Executive Director of the VHA National Oncology Program, acknowledged the promise histotripsy has within the VA system and in conjunction with ongoing Federal oncology initiatives, “As the largest integrated oncology provider in the United States, Veterans Affairs remains committed to improving the lives of Veterans and supports the goals of collaborative programs like the Cancer Moonshot Initiative (https://www.cancer.va.gov/cancer-moonshot.html),”.

“Too frequently, Veterans do not have early access to meaningful innovation in health care, so it has been a mission for our team to partner with the VA in bringing histotripsy to our Veterans as soon as possible,” said Mike Blue HistoSonics CEO and President. Blue adds, “Histotripsy’s unique mechanism of action and non-invasive approach have demonstrated a patient friendly procedure for destroying liver tumors allowing many patients to return home and, ideally, to their lives quickly after the procedure. We believe that histotripsy is especially well-suited as a therapy for those who served with honor and are due the best healthcare we can provide.” All participating VA hospitals will be eligible to enroll patients in HistoSonics’ prospective study and post market clinical program, called BOOMBOX, which aims to collect data across a broad number of clinical use cases, and liver tumor pathologies, observing the use of histotripsy across all stages of liver disease.

HistoSonics is proud to partner with FIDELIS Sustainability as the SDVOSB partner and exclusive distributor of histotripsy systems to the federal government. “This is an absolute medical technology landmark for US patient care, and we are honored to be a part of bringing the Edison System to the Veteran Hospitals & DoD medical centers around the world,” said Dustin Lee Fidelis President & CEO.

The Edison System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any disease including, but not limited to, cancer or evaluated any specific cancer outcomes (such as local tumor progression, 5-year survival or overall survival). The System should only be used by physicians who have completed training performed by HistoSonics, and its use guided by the clinical judgment of an appropriately trained physician. Refer to the device Instructions for Use for a complete list of warnings, precautions, and a summary of clinical trial results, including reported adverse events.

About HistoSonics

HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison Histotripsy System in the US and the HistoSonics Histotripsy System in select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

For more information on the Edison Histotripsy System please visit: www.histosonics.com/. For patient related information please visit: www.myhistotripsy.com/.

Cancer Research UK, one of the world’s largest funders of cancer research, and KisoJi Biotechnology Inc., a company focussed on the discovery and development of antibody therapeutics, have signed a landmark agreement to bring KisoJi’s lead asset, KJ-103, into a first-in-human clinical trial. KJ-103 is a naked anti-TROP2 antibody that has been created by KisoJi using its proprietary antibody technology. 

Under the agreement, Cancer Research UK’s Centre for Drug Development (CDD) will sponsor, design and deliver a Phase 1/2a clinical trial of KJ-103, in selected TROP2 expressing solid tumours. KisoJi will supply the antibody for the clinical trial and work with CDD to complete the preclinical package. Cancer Research Horizons, Cancer Research UK’s innovation arm, will manage the commercial relationship.

Unlike antibody drug conjugates (ADCs), KJ-103 does not require a cytotoxic payload but instead functions by recruiting immune cells to kill tumour cells. KJ-103 binds to TROP2 in a location distinct from where current TROP2 ADCs bind. It uses TROP2 as a way of directing macrophage activation and phagocytosis of the tumour cells expressing it, leading to tumour cell death. 

KJ-103 may provide an alternative treatment option for TROP2-expressing cancers in which TROP2 ADCs have proven ineffective or are not suitable due to their toxicity profile. Tumour types expressing TROP2 include: colorectal, head and neck, ovarian, breast, bladder and lung cancers. 

Lars Erwig, Director of the CDD, said: “We are excited to collaborate with KisoJi to bring KJ-103 into clinical development. This partnership aligns with our mission to explore innovative therapeutic approaches for hard-to-treat cancers. With KJ-103’s unique mechanism of action, which harnesses the body’s immune system without the potential toxicity of a drug conjugate, we hope to offer new treatment options for patients with TROP2-expressing solid tumours.” 

David Young, co-founder and CEO of KisoJi said: “We are thrilled to be advancing KJ-103 into the clinic in partnership with Cancer Research UK. As the first naked antibody to target TROP2 cancers, KJ-103 is the first of a new wave of antibodies to come from our modernised technology platform that leverages AI grounded in biology to create transformative antibody therapeutics.” 

About Cancer Research UK’s Centre for Drug Development 
Cancer Research UK has an impressive record of developing novel treatments for cancer. The Cancer Research UK Centre for Drug Development has been pioneering the development of new cancer treatments for 30 years, taking over 160 potential new anti-cancer agents into clinical trials in patients. Six of these new agents have made it to market, including temozolomide for brain cancer, abiraterone for prostate cancer and rucaparib for ovarian cancer. Two other drugs are in late development Phase 3 trials. Thirteen agents remain in active development with the potential to reach the market. It currently has a portfolio of 16 projects in preclinical development, Phase 1 or early Phase 2 clinical trials.

www.cruk.org.uk/cdd 

About KisoJi Biotechnology
KisoJi is a Canadian biotechnology company that deploys the latest scientific and AI tools to conduct therapeutic antibody discovery in a fundamentally new way. KisoJi has developed a multi-species transgenic mouse to generate highly diverse single domain antibodies, as well as a modular multi-specific antibody scaffold with high stability and productivity. Most recently, KisoJi has used advanced AI tools to visualise the universe of all antibodies against a target in order to uncover novel biology and new therapeutic antibody capabilities for its partners and its own pipeline.

www.kisojibiotech.com

About KJ-103 
KJ-103 is a single domain antibody that binds to a unique epitope on TROP2. It acts via effector cell mechanisms, including the activation of macrophages, to kill tumour cells. KJ-103 has been shown to have significant anti-tumour potency with no evidence of toxicity or resistance across a number of preclinical solid tumor models. KJ-103 was humanised by LifeArc.

Cancer Research UK Contact:
Tim Bodicoat
tim.bodicoat@cancer.org.uk
+44 203 469 8300
+44 203 469 8301 (out of hours)

KisoJi Biotechnology Contact:
Investors and Media
Argot Partners
Sam Martin/Cameron Willis
KisoJi@argotpartners.com
+1 212 600 1902

SOURCE KisoJi Biotechnology Inc.; Cancer Research UK

Alpha-9 Oncology Inc. (Alpha-9 or the Company), a clinical stage company developing radiopharmaceuticals to meaningfully improve the treatment of people living with cancer, today announced an oversubscribed $175 million Series C financing to support the progression of its pipeline. The financing was led by Lightspeed Venture Partners and Ascenta Capital. A selected syndicate of new investors – General Catalyst, a16z Bio + Health, RA Capital Management, Janus Henderson Investors, Delos Capital, Digitalis Ventures, Lumira Ventures and a healthcare fund managed by abrdn Inc. – joined the round, in addition to existing investors Frazier Life Sciences, Longitude Capital, Nextech Invest, BVF Partners LP, and Samsara BioCapital. Shelley Chu, head of Lightspeed Venture Partners’ healthcare team and Evan Rachlin, co-founder and managing partner of Ascenta Capital will join the Company’s Board of Directors.

Alpha-9 has built a diversified portfolio of clinical and discovery assets across both validated and novel targets. The Series C will fund human studies for the clinical stage assets and advancement of discovery stage assets to clinic-ready development candidates. Furthermore, the Series C will fund expanded R&D capabilities and continued investment in CMC and supply chain.

“Over the last few years, Alpha-9 has built a leading radiopharmaceutical company with a deep pipeline and robust infrastructure,” said Alpha-9 CEO, David Hirsch, MD, PhD. “The Series C is an exciting, significant milestone for us and will greatly accelerate our growth. We are thrilled to have the backing of a top-tier investor syndicate who share our belief in the potential of radiopharmaceuticals.”

“Alpha-9 is developing a differentiated portfolio that includes multiple radiopharmaceuticals with first in-class and best-in-class potential,” said Shelley Chu, head of Lightspeed Venture Partners’ healthcare team. “We are impressed with the team’s progress to date and are proud to support the advancement of these programs.”

Alpha-9’s approach to designing bespoke molecules is systematic and data-driven. The Company has a differentiated toolbox of binders, linkers, chelators and radioisotopes – elements that each play an integral role in radiopharmaceutical development. Alpha-9 designs each component of the radiopharmaceutical for optimal selectivity, stability and payload delivery. The Alpha-9 approach is rigorous, fast and capital efficient, generating best-in-class compounds for rapid clinical development.

“We have been following this space for a long time. What differentiated Alpha-9 was its effective approach to molecule design as well as its thoughtful strategy on infrastructure expansion,” said Evan Rachlin, MD, managing partner of Ascenta Capital. “We are pleased to support the Company’s continued progress as it strives to deliver on the promise of radiopharmaceuticals.”

To support its endeavors, Alpha-9 has purpose-built research facilities in Vancouver, which were completed last year and have been operating at scale. These facilities help to accelerate drug development by streamlining discovery processes. Alpha-9 has also partnered with isotope suppliers and CDMOs to support its ongoing clinical trials. Alpha-9’s commitment to continue building robust infrastructure and world class capabilities underscores the company’s mission to provide effective treatments for patients worldwide.

About Alpha-9 Oncology Inc.

Alpha-9 Oncology Inc. is a clinical stage radiopharmaceutical company developing differentiated and highly targeted radiopharmaceuticals with the potential to meaningfully improve the treatment of people living with cancer. Applying proprietary technologies and deep foundational expertise, Alpha-9 is on the forefront of engineering bespoke radiopharmaceuticals that are optimized to selectively deliver radiation to tumor sites while minimizing off-target effects. Alpha-9 is advancing a robust pipeline of novel radiopharmaceuticals with a systematic approach to molecule design that offers broad potential for expansion into several validated oncology targets. For more information, please visit www.a9oncology.com.

About Lightspeed Venture Partners

Lightspeed Venture Partners is a multi-stage venture capital firm focused on accelerating disruptive innovations and trends in the Enterprise, Healthcare, Consumer and Fintech sectors. Over the past two decades, the Lightspeed team has backed hundreds of entrepreneurs and helped build more than 500 companies globally including biotechnology companies such as Abata, Ancora, Diagonal, Enlaza, Forty Seven, Personalis, Pheon, Teneobio, Triana, Scorpion, Seismic, Ultima Genomics, Xaira, ZagBio, and more. Lightspeed and its global team currently manage $25B in AUM across the Lightspeed platform, with investment professionals and advisors in the U.S., Europe, India, Israel, and Southeast Asia. For more information, please visit www.lsvp.com.

About Ascenta Capital

Ascenta Capital is a biotech venture fund, co-founded in 2023 by Dr. Evan Rachlin and Dr. Lorence Kim. The fund is focused on a concentrated portfolio of investments in multi-product companies. For more information about Ascenta Capital, visit www.ascentacap.com.

Notice Regarding Forward-Looking Statements:

This news release contains forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and forward-looking information within the meaning of applicable Canadian securities laws (collectively, “forward-looking statements”). The words “may”, “will”, “potential”, “believes” and “if” are intended to identify forward-looking statements, although not all forward-looking statements contain such terms. You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause actual results, future circumstances, or events to differ materially from those projected in the forward-looking statements. These risks include but are not limited to: those associated with the success of research and development programs, the ability to raise additional funding, and the need to obtain regulatory approval. Forward-looking statements are made as of the date hereof and Alpha-9 disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

Alpha-9 Contact
Corporate Communications
Stephen Mitchener
Phone: +1 617 865 1004
Email: smitchener@a9oncology.com

enGene Holdings Inc. (Nasdaq: ENGN or “enGene” or the “Company”), a clinical-stage genetic medicines company whose non-viral lead investigational product detalimogene voraplasmid (also known as detalimogene, and previously EG-70) is in an ongoing pivotal study in patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (Cis), today announced the appointment of Joan Connolly as Chief Technology Officer (CTO) and member of the corporate leadership team. In addition, enGene Co-Founder and former CTO, Anthony Cheung, Ph.D., will transition to the role of Chief Scientific Officer, succeeding James Sullivan, MSc, Ph.D.

“We are thrilled to welcome Joan to the enGene team. She brings significant experience in managing the technical operations required to drive a late-stage clinical asset through to commercialization, which will be critical as we complete the pivotal portion of the LEGEND study of detalimogene and anticipate filing our BLA in mid-2026,” said Ron Cooper, Chief Executive Officer of enGene.

“I am excited to join enGene at this critical juncture. Detalimogene’s highly differentiated, non-viral DDX formulation and easily scalable manufacturing process are designed to offer significant advantages over other therapies available and in development,” said Ms. Connolly. “Recent preliminary data from LEGEND’s pivotal arm underscore detalimogene’s future potential as a foundational therapy for the many NMIBC patients in need of new therapeutic options.”

Dr. Cheung is a co-founder of enGene and has served in various capacities since the Company’s inception. He has been central to the discovery and development of the Company’s novel and proprietary Dually Derivatized Oligochitosan (DDX) platform and in the creation of detalimogene. He has co-authored numerous book chapters, review articles and peer-reviewed journals, and is a named inventor on numerous patents in the areas of gene therapy and polymer chemistry.

“Having developed enGene’s DDX technology from the ground up, Anthony is intimately familiar with the science and well-suited to drive future expansion of our DDX platform,” said Ron Cooper. “We are grateful to Dr. Sullivan for his meaningful contributions in advancing detalimogene and wish him luck in his future endeavors.”

About enGene

enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene’s lead program is detalimogene voraplasmid, (also known as detalimogene, and previously EG-70) for patients with Non-Muscle Invasive Bladder Cancer (NMIBC) – a disease with a high clinical burden. Detalimogene is being evaluated in the ongoing multi-cohort LEGEND Phase 2 study, which includes a pivotal cohort studying detalimogene in Bacillus Calmette-Guérin (BCG)-unresponsive patients with carcinoma in situ (Cis). Detalimogene was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA. For more information, visit enGene.com.

Forward-Looking Statements

Some of the statements contained in this press release may constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). enGene’s forward-looking statements include, but are not limited to, statements regarding enGene’s expectations, hopes, beliefs, intentions, goals, strategies, forecasts and projections. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about: our expectations regarding regulatory submissions, our beliefs as to the potential benefits of detalimogene, and our expectations regarding the potential broad adoption of detalimogene.

Many factors, risks, uncertainties and assumptions could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, that preliminary clinical data may not accurately reflect the complete results of a particular study and remain subject to audit and verification, and final data may differ materially from preliminary data; the Company’s ability to recruit and retain qualified scientific and management personnel; establish clinical trial sites and enroll patients in its clinical trials; execute on the Company’s clinical development plans and ability to secure regulatory approval on anticipated timelines; and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2023 and most recent Quarterly Report on Form 10-Q for the fiscal quarter ended July 31, 2024 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov).

You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved.

Contacts

For media: media@engene.com
For investors: investors@engene.com

Endogenex, Inc., a clinical-stage medical device company focused on improving outcomes for patients with type 2 diabetes (T2D), today announced the enrollment of the first patient in the ReCET Clinical Study. This multicenter, prospective, randomized, double-blinded, sham-controlled trial is designed to assess the safety and effectiveness of the ReCET™ System in adult patients whose T2D is inadequately controlled with non-insulin, glucose-lowering medications.

“This milestone is a major step forward in our mission to transform T2D treatment by addressing the underlying causes impacting millions of patients living with T2D,” stated Stacey Pugh, CEO of Endogenex. “We are thrilled to have completed the first enrollment and look forward to continued collaboration with our ReCET Clinical Study sites in advancing study enrollment.”

The first patient was successfully enrolled at Orlando Health, Orlando, FL, under the leadership of Dr. Andre Teixeira, Medical Director of the Orlando Health Weight Loss and Bariatric Surgery Institute. “We are excited to be part of this landmark study,” stated Dr. Teixeira. “Clinical evidence continues to evolve, supporting the central role of the duodenum in regulating glucose metabolism. Therapeutic interventions targeting the upper gastrointestinal tract are demonstrating improved outcomes for patients with type 2 diabetes. The ability to reset pathologic signaling in the duodenum using the ReCET Technology, delivered through an endoscopic procedure, holds great promise for transforming care for T2D patients.”

“We are proud to have reached this important milestone in the ReCET pivotal study,” stated Dr Richard Pratley, Medical Director of the Advent Health Diabetes Institute in Orlando, FL, and Co-Principal Investigator for the ReCET Study.  “The ReCET Study offers new hope for advancing treatment options and the potential to provide patients a better opportunity to manage their diabetes more effectively.”

The ReCET Study is a pivotal trial approved by the U.S. Food and Drug Administration (FDA) under the Investigational Device Exemption (IDE). The study aims to enroll up to 350 patients at approximately 40 sites in the United States and Australia.

About the ReCET™ Procedure
ReCET is a novel, endoscopic outpatient procedure that targets the cellular pathology of the duodenum. This pathology may contribute to the development and progression of type 2 diabetes.

The ReCET System aims to initiate the body’s natural regenerative process by applying highly controlled, non-thermal pulsed electric fields to the mucosa and sub-mucosa duodenal tissue. This approach may help restore proper cellular signaling from the duodenum and improve metabolic function, including better control of blood glucose levels. 

The ReCET System has been evaluated in feasibility clinical studies, such as REGENT-1 US, REGENT-1 Australia, and EMINENT in the Netherlands. These studies assessed the safety and efficacy of the treatment in adults with type 2 diabetes whose blood glucose levels were inadequately controlled despite using insulin and non-insulin medications. Preliminary outcomes from these studies have been presented at medical conferences globally.

About Endogenex
Endogenex, founded in partnership with Mayo Clinic, aims to revolutionize treatment options for individuals with type 2 diabetes (T2D). The company’s innovations focus on resetting the body’s metabolic signaling system by harnessing its natural regenerative capabilities to improve metabolic function. Through the development of the ReCET System and the novel application of precise, controlled, non-thermal pulsed electric fields, Endogenex is establishing a new era in T2D therapy, helping patients regain control of their blood glucose levels and slow disease progression.

For more information, please visit www.endogenex.com or www.recetstudy.com.

Media Inquiries:
Krissy Wright
CFO
kwright@endogenex.com
+1 651-329-5413

SOURCE Endogenex

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease, today announced that results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD) have been accepted for both oral and poster presentation at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis Consortium (NEALS) Meeting. The hybrid meeting will take place October 21-24, 2024, in Clearwater, Florida, and virtually.

“Given the effects of TPN-101 on key biomarkers of neurodegeneration and neuroinflammation, including NfL and IL-6, and clinical outcome measures of disease progression and respiratory function in patients with C9orf72-related ALS, we are very pleased these data were accepted for presentation at NEALS,” said Andrew Satlin, M.D., Chief Medical Officer at Transposon. “Based on these promising results, we are advancing TPN-101 into a Phase 3 registration study for the treatment of C9orf72-related ALS, while also continuing to develop TPN-101 for other neurodegenerative diseases.”

About the Phase 2 Study in C9orf72-related ALS/FTD
The Phase 2 study in patients with C9orf72-related ALS/FTD was a multi-center, randomized, double-blind, placebo-controlled parallel-group, two-arm study with an open-label treatment period. Participants (n=42) were randomized 3:2 to receive daily doses of 400 mg of TPN-101 or placebo. The study included a six-week screening period, a 24-week double-blind treatment period, a 24-week open-label treatment period, and a follow-up visit four weeks post-treatment. Further information on the study can be accessed at ClinicalTrials.gov.

About TPN-101
TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 cDNA, triggering innate immune responses that contribute to neurodegenerative, neuroinflammatory, and aging-related disease pathology.

About ALS and FTD
ALS is a neurodegenerative disease characterized by progressive muscle weakness, and loss of ability to speak, eat, move or breathe. FTD is a progressive frontal/temporal cortex disease associated with behavior and personality changes, emotional problems, and difficulty walking, communicating, or working. A C9orf72 hexanucleotide repeat mutation accounts for 10-15% of both disorders. With onset commonly in middle age or earlier, patients with ALS have a mean survival of two to three years. Patients with FTD have a mean survival of nine years.

About Transposon
Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications.

Contact:
Rick Orr
Transposon Therapeutics, Inc.
(858) 535-4821
rorr@transposonrx.com

Thryv Therapeutics Inc., a clinical-stage biotechnology company, received positive top-line results from the Wave I Part 2 proof-of-concept clinical study evaluating LQT-1213 for the treatment of congenital Long QT Syndrome (cLQTS) Types 2 and 3. LQT-1213, a potent and selective serum glucocorticoid inducible kinase 1 (SGK1) inhibitor, demonstrated clinically meaningful and statistically significant reductions in the QTcF interval (a measure of how long it takes the heart to “reset” between beats) after only two days of dosing in patients with cLQTS Types 2 and 3.  LQT-1213 appeared to be generally well-tolerated in the study with no drug-related adverse events (AEs) reported.

Congenital LQTS is a rare genetic condition that affects the heart’s electrical system, leading to an increased risk of life-threatening arrhythmias in children and adults. While Wave I Part 1 evaluated the effect of LQT-1213 in a model of LQTS induced by the drug dofetilide, Wave 1 Part 2 included, for the first time, patients with genetically confirmed congenital LQTS Types 2 and 3, providing direct clinical insights into the acute efficacy of LQT-1213 in the targeted patient population.  It is believed that reductions in QTcF will reduce arrhythmic risk in pediatric and adult patients with prolonged QTc intervals.

“We are excited about the positive top-line results from the Wave I Part 2 study in cLQTS Types 2 and 3 patients. Two days of treatment with LQT-1213 demonstrated rapid and clinically meaningful reductions in QTcF.  As well, a clear dose level effect was seen with the highest dose of LQT-1213 reducing QTcF for all key endpoints of the study,” said Philip Sager, MD, FAHA, Chief Medical Officer of Thryv Therapeutics. “The totality of LQT-1213 treatment effects, which are on top of beta blocker therapy, has allowed us to engage regulatory agencies about our next steps towards a pivotal program in patients with congenital Long QT Syndrome.”

Study Highlights

Wave I is a multi-part, proof-of-concept clinical study designed to evaluate the safety and efficacy of LQT-1213 in reducing QT in patients with both acquired and congenital forms of Long QT Syndrome.  Further details about the study can be found at clinicaltrials.gov (NCT05906732).

Full data from the Wave I Part 2 study in congenital LQTS Types 2 and 3 will be presented at an upcoming scientific conference in 2025. Part 3 of the Wave I study will enroll patients with cLQTS Type 1 who demonstrate continued prolongation of their QT interval despite standard of care.

About LQT-1213

LQT-1213 is a potent and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor being developed as an investigational therapeutic to treat Long QT Syndrome Types 1, 2, and 3. By targeting SGK1, LQT-1213 aims to reduce the prolonged QTc interval in children and adults with LQTS, thereby decreasing the risk of life-threatening arrhythmias. Currently, LQT-1213 is being evaluated in clinical studies for its efficacy and safety in treating both congenital and acquired LQTS.   LQT-1213 has been granted Orphan Drug Designation by the US FDA for the treatment of congenital Long QT Syndrome.

About Long QT Syndrome

LQTS, is a disorder of the electrical system that results in the heartbeat taking longer than usual to “reset” between beats, measured on the ECG as the QTc interval. LQTS can be either congenital or acquired due to certain drugs. Congenital LQTS is a set of rare orphan diseases in which people are genetically predisposed to chronic prolongation of their QTc interval (commonly more than 480 milliseconds), leading to increased risk of torsades de pointes, a lethal cardiac arrhythmia that can cause sudden cardiac death. Acquired Long QT may develop from the administration of therapies which block electrical pathways in the heart, leading to a similar mechanistic prolongation of QTc and risk of sudden cardiac death.

About Thryv Therapeutics

Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors to treat Long QT Syndrome, atrial fibrillation and heart failure, with potent and selective inhibitors of serum glucocorticoid inducible kinase. For more information, please visit www.thryvtrx.com. 

SOURCE Thryv Therapeutics Inc.

Thryv Therapeutics Inc., a clinical stage biotech company dedicated to developing treatments for rare and life-threatening cardiovascular indications, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to LQT-1213 for the treatment of Long QT Syndrome (LQTS).  LQT-1213 is a novel, first-in-class SGK1 inhibitor specifically designed to treat congenital LQTS.  Thryv has developed a series of SGK1 inhibitors for the treatment of cardiometabolic stress associated with various arrhythmic diseases including LQTS, atrial fibrillation, and heart failure.

“We are pleased with the FDA’s Orphan Drug Designation for LQT-1213 based partly on emerging positive clinical data from our Wave 1 clinical study in patients with Long QT Syndrome” said Debra Odink, PhD, President and Chief Development Officer of Thryv Therapeutics. “People with Long QT Syndrome deserve a properly studied and FDA-approved therapy to help in their battle against this potentially lethal genetic disease. There is currently no FDA-approved therapy for people with Long QT Syndrome. This designation reinforces the potential of LQT-1213 to fulfill this unmet need and provides critical incentives for Thryv to accelerate our efforts to deploy prospectively designed, pivotal efficacy studies in people with congenital Long QT Syndrome.  We remain focused on bringing innovative treatments to adults and children with rare diseases who have limited options.” 

Following a request from the FDA, clinical data from the ongoing Wave I clinical study in people with congenital LQTS was provided to the Office of Orphan Products Designation.  Shortly thereafter, the company received notification that its request for Orphan Drug Designation was approved. 

The FDA Orphan Drug Designation is a special status granted to drugs and biologics targeting rare diseases, defined as those affecting fewer than 200,000 people in the United States.  This designation offers numerous benefits to support the development of treatments for rare conditions, including tax credits for clinical trial costs, exemption from certain FDA fees, and up to seven years of market exclusivity following FDA approval. 

About LQT-1213 

LQT-1213 is a potent and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor being developed as an investigational therapeutic to treat Long QT Syndrome Type 1, 2, and 3.  By targeting SGK1, LQT-1213 aims to reduce the prolonged QTc interval in people with LQTS, thereby decreasing the risk of life-threatening arrhythmias.  Currently, LQT-1213 is being evaluated in clinical studies for its efficacy and safety in treating congenital LQTS.   

Additional information about the on-going Wave I clinical study is available at www.clinicaltrials.gov using identifier: NCT05906732. 

About Thryv Therapeutics Inc.

Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors to treat Long QT Syndrome, atrial fibrillation and heart failure, with potent and selective inhibitors of Serum Glucocorticoid inducible Kinase. For more information, please visit www.thryvtrx.com.

Media Inquiries

brittany@thryvtrx.com | 514 973 0915

SpectraWAVE, Inc., a medical imaging company focused on improving the treatment and outcomes for patients with coronary artery disease (CAD), today announced a $50M Series B funding round led by Johnson and Johnson Innovation – JJDC, Inc. joined by S3 Ventures, Lumira Ventures, SV Health Investors, Deerfield Management, NovaVenture, Heartwork Capital, and undisclosed parties. The funding round will advance commercial expansion and product additions to the Company’s 510k-cleared HyperVue^™ Imaging System.

“Intravascular imaging guidance is backed by a wealth of randomized clinical trials (RCT) and recently collated in a meta-analysis of 22 RCT studies and approximately 16,000 patients published this year in The Lancet, demonstrating significantly improved outcomes for patients undergoing coronary stenting,” said Eman Namati, Ph.D., Chief Executive Officer of

SpectraWAVE. “This evidence and broad recognition of impact has recently moved intravascular imaging to a 1A guideline recommendation in Europe, with an expectation that the United States will follow in due time. Increasing both the capabilities and the ease of use of intravascular imaging systems is now critical to expand the use of imaging and improve care for these patients. That is our mission with HyperVue – simpler, faster, and better imaging to drive optimized stenting procedures for improved patient outcomes. The reception to the product during our initial U.S. launch has been fantastic, and this financing, supported by leading MedTech investors, will support our commercial expansion to bring HyperVue to more patients.”

Intravascular imaging is an essential tool to optimize coronary stenting procedures, providing key insights into plaque morphology, plaque modification decisions, stent and balloon sizing and landing zone selection, confirmation of treatment optimization, and future adverse event risk. Prior intravascular imaging technologies offer compromises in image resolution, image depth, and ease of use. HyperVue is the first intravascular imaging technology to combine two important imaging technologies, DeepOCT and NIRS, while optimizing for image quality and procedural efficiency in the cath lab, including no-flush catheter prep, fast and long pullbacks designed to reduce and remove the use of contrast, and the most comprehensive AI-driven workflow and image analysis offering.

Namati continued, “In addition to our commercial intravascular imaging system, we are developing a wire-free physiology software add-on to allow physicians rapid assessment of pressure drops in the coronaries using the same HyperVue hardware. This is an important step in our journey to enhance the clinical decision-making for these patients and establish an anchor point for future innovation in the cath lab.”

About SpectraWAVE, Inc.

SpectraWAVE, located in Bedford, Mass., is a privately held medical device company founded in 2017 to provide unrivaled optical and computational insights to improve the treatment and outcomes for patients with coronary artery disease (CAD). CAD, the buildup of plaque in the wall of the arteries that supply blood to the heart, affects 20.5 million adults aged 20 and older. In CAD patients that undergo percutaneous stent placement, it is estimated that one in five patients experience adverse events within two years. SpectraWAVE’s flagship HyperVue^TM Imaging System combines next generation DeepOCT^TM images and near infrared spectroscopy (NIRS) with workflows optimized for the cardiac catheterization lab, and serves as a central hub for future enhancements that will continue to empower interventionalists in their treatment decision making and optimization. The HyperVue Imaging System is intended for the imaging of coronary arteries and is indicated in patients who are candidates for transluminal interventional procedures. The NIRS capability of the system is intended for the identification of patients and plaques at increased risk of major adverse cardiac events. For more information and complete indications for use, please visit www.spectrawave.com.

HyperVue, Starlight, and DeepOCT are commercial trademarks cleared for sale in the U.S.A.

Media Contact: SpectraWAVE, Inc.

Jason Tucker-Schwartz, PhD jason@spectrawave.com

HistoSonics, (www.histosonics.com), the developer and manufacturer of the Edison™ Histotripsy System, announced today the company’s initial partnership in Asia with Hong Kong University receiving their first histotripsy system after a generous donation from the Li Ka Shing Foundation. The Foundation, led by its founder Mr. Li Ka-shing, is a prolific global philanthropic organization with a focused effort in Hong Kong, and is responsible to date for over hk$30 billion in projects involving education, medical services, charity, and anti-poverty programs. As part of the Foundation’s focus on bringing innovative medical advancements to Hong Kong, the Foundation provided the capital to acquire two initial histotripsy systems to the leading public hospitals in Hong Kong, one each to the University of Hong Kong and the Chinese University of Hong Kong. Chinese University of Hong Kong will receive the second histotripsy system early next year to complete the initial gift from Mr. Li.

Experts in liver treatment from the University of Hong Kong and the Chinese University of Hong Kong recently underwent training at HistoSonics headquarters in Minneapolis, MN and expect to begin treating patients as early as this week. “We could not be more excited to partner with these two prestigious university hospitals to be the first in Hong Kong to offer our novel therapy to their patients,” said Mike Blue, HistoSonics CEO and President. “Unfortunately, Asia carries a substantial proportion of the world’s population suffering from diseases that cause liver tumors and based on the experiences from our U.S. launch we are confident that we can begin to make an immediate impact on the quality of life of many of the patients who need it most in this region. We are very grateful for the Foundation’s very generous gift and Mr. Li’s personal interest in helping patients in Hong Kong,” added Blue. Both Hong Kong based hospitals will be eligible to enroll patients in HistoSonics’ prospective study and post market clinical program, called BOOMBOX, which aims to collect data across a broad number of clinical use cases, and liver tumor pathologies, observing the use of histotripsy across all stages of liver disease.

The Edison Histotripsy System is indicated for the non-invasive destruction of liver tumors, including unresectable liver tumors, using a non-thermal, mechanical process of focused ultrasound.

About HistoSonics

HistoSonics is a privately held medical device company developing a non-invasive platform and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison Histotripsy System in the US and the HistoSonics Histotripsy System in select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, and others. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

For more information on the Edison Histotripsy System please visit: www.histosonics.com/. For patient related information please visit: www.myhistotripsy.com/.

“The pooled #HOPE4LIVER single-arm pivotal trial for histotripsy of primary and metastatic liver tumors” published in Radiology by Mishal Mendiratta-Lala, et.al. is licensed under CC BY 4.0.

Contacts

For Media Inquiries
Media contact:
Josh King
Vice President of Marketing
Email : Joshua.king@histosonics.com
Phone : 608.332.8124

HistoSonics, the manufacturer of the Edison® Histotripsy System and novel histotripsy therapy platforms, announced today the completion of an oversubscribed $102 million Series D financing. The round was led by Alpha Wave Ventures, a world leader in growth stage investments, with participation from new investors Amzak Health and HealthQuest Capital, and existing investors Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Venture Investors, Lumira Ventures, Yonjin Venture, the State of Wisconsin Investment Board, and others.
Histotripsy is a novel form of focused ultrasound that uses high amplitude, very short pulses to create a “bubble cloud” that is designed to mechanically destroy and liquefy targeted liver tumors. These bubble clouds form and collapse in microseconds, creating mechanical forces strong enough to destroy tissue at cellular and sub-cellular levels in a non-invasive and non-thermal method. Histotripsy offers a promising alternative to treatments such as surgery, radiation and chemotherapy, which often have significant side effects.
The Series D funding will be used to accelerate category defining advancements to its non-invasive histotripsy platforms, support commercial growth in the US and in planned global markets, and initiate the company’s innovative, prospective BOOMBOX Master Study that will evaluate HistoSonics’ Edison System for the treatment of liver tumors across multidisciplinary users. The Edison Histotripsy System was granted FDA De Novo clearance in October 2023. HistoSonics is currently partnering with leading institutions across the US and internationally in developing multi-disciplinary histotripsy liver programs.
“Histotripsy is a paradigm-changing treatment option for patients who want a non-invasive approach to target and destroy tumors without the need for needles or incisions,” said Mike Blue, President & CEO, HistoSonics. “We’re thrilled to announce this top-tier investor syndicate led by Alpha Wave, which reinforces the confidence in our mission to impact patients’ lives with our current liver application and expanded use in kidney, pancreas, prostate, brain and other tumor types. This funding will accelerate key projects designed to enhance core technical capabilities impacting current and future platforms, and support collaboration with physicians and researchers studying innovative ways to use histotripsy’s unique mechanism of action to improve patient outcomes. By pioneering a new category of medicine with histotripsy, HistoSonics is redefining liver tumor treatment and providing hope for millions of people worldwide who desperately need new options.”
“Alpha Wave is proud to lead the investment in HistoSonics, a pioneer in the use of histotripsy,” said Chris Dimitropoulos, Managing Director, Healthcare Investments at Alpha Wave Global. “Histotripsy’s unique non-invasive approach uses focused ultrasound to precisely target and destroy diseased tissue without damaging surrounding healthy tissue. This groundbreaking technology has the potential to transform the treatment landscape for a variety of challenging clinical conditions, offering patients improved outcomes and recovery times. The level of adoption we’ve seen from leading hospital centers across the country and overseas highlights the huge unmet medical need. We are excited to support HistoSonics in accelerating the development and availability of this innovative therapy.” In connection with this financing, Mr. Dimitropoulos will join the HistoSonics Board of Directors.
HistoSonics’ Edison System uses proprietary technology and advanced imaging to deliver personalized, non-invasive histotripsy treatments with precision and control. The novel mechanism of action of this proprietary technology may provide significant patient advantages, including the ability of the treatment site to recover and resorb quickly. HistoSonics’ platform also provides physicians the ability to monitor the destruction of tissue under continuous real-time visualization and control, unlike any modality that exists today.
About HistoSonics
HistoSonics is a privately held medical device company developing non-invasive platforms and proprietary sonic beam therapy utilizing the science of histotripsy, a novel mechanism of action that uses focused ultrasound to mechanically destroy and liquify unwanted tissue and tumors. The company is currently focused on commercializing their Edison System in the US and select global markets for liver treatment while expanding histotripsy applications into other organs like kidney, pancreas, prostate, brain, and others. HistoSonics has offices in Ann Arbor, Michigan, and Minneapolis, Minnesota. The Edison® System is intended for the non-invasive mechanical destruction of liver tumors, including the partial or complete destruction of unresectable liver tumors via histotripsy. The FDA has not evaluated the Edison System for the treatment of any specific disease or condition. Patients seeking information on histotripsy and if they may be a candidate for histotripsy can learn more at www.myhistotripsy.com.
Use of the Edison System in kidney applications is limited by federal law to investigational use. The #HOPE4KIDNEY Trial is designed to support a future expansion of the indication to include the destruction of kidney tissue/tumors.
About Alpha Wave
Alpha Wave is a global investment company with three main verticals: private equity, private credit, and public markets. It is led by Rick Gerson, Navroz Udwadia, and Ryan Khoury. Its flagship global private equity fund, Alpha Wave Ventures, aims to invest in best-in-class growth-stage companies and endeavors to be helpful long-term partners to the founders and management teams.
Alpha Wave has offices in Miami, New York, London, Monaco, Madrid, Abu Dhabi, Tel Aviv, Bangalore, and Sydney. For more information, please visit www.alphawaveglobal.com.
For more information please visit: www.histosonics.com/
Contacts
For Media Inquiries
Media contacts:

Josh King
Vice President of Global Market Access
Email : Joshua.king@histosonics.com
Phone : +1 608.332.8124

Kimberly Ha
KKH Advisors
917-291-5744
kimberly.ha@kkhadvisors.com

G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company focused on delivering next-generation therapies that improve the lives of those affected by cancer, and Pharmacosmos A/S, a leader in the development of innovative treatments for patients suffering from iron deficiency and iron deficiency anemia, today announced that they have entered into a definitive merger agreement under which Pharmacosmos A/S, through its U.S. subsidiary Pharmacosmos Therapeutics Inc., will acquire all outstanding shares of G1 Therapeutics common stock for U.S. $7.15 per share in cash for a total equity value of approximately $405 million, which represents a 68% premium to G1’s closing share price on August 6, 2024 and a 133% premium to G1’s prior 30-day volume weighted average price. The Boards of Directors of the parties have unanimously approved the transaction, which is expected to close late in the third quarter of 2024.

G1’s COSELA is the first and only product approved by the U.S. Food and Drug Administration to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer (ES-SCLC).

Together, Pharmacosmos and G1 Therapeutics will execute on the shared vision to grow and accelerate the availability of COSELA for all appropriate patients with ES-SCLC. G1 brings a well-established and successful commercial, sales, and medical platform to Pharmacosmos, which has complementary expertise in commercializing hematology and supportive care products, a robust global commercial presence, and significant resources to maximize the penetration of COSELA into the ES-SCLC market. Together, the combined company will be able to optimize the commercial reach to oncologists and expand the availability of COSELA among patients living with ES-SCLC.

“G1 and Pharmacosmos have a shared commitment to people living with cancer; the transaction announced today will enable a more rapid uptake of COSELA into the ES-SCLC market to maximize availability for patients who need this important drug,” said Jack Bailey, Chief Executive Officer of G1 Therapeutics. “Importantly, this acquisition delivers significant value to G1’s stakeholders by providing better and broader access to this important product for the cancer patients we seek to treat and a significant premium to our shareholders. I am proud of all that the G1 team has accomplished over the years, thankful for their great effort, and excited about what’s possible by the combined Pharmacosmos/G1 team as we meet the needs of more cancer patients.”

“The acquisition of G1 Therapeutics Inc., its intellectual property, and the addition of COSELA® (trilaciclib) to our portfolio of innovative products is transformative for Pharmacosmos. By combining our existing colleagues with the great team at G1 Therapeutics, we will meaningfully expand our organization serving oncologists in the US. This will enable broader and better access for patients in need of COSELA as well as for our existing FDA approved drug, Monoferric® (ferric derisomaltose),” said Tobias S. Christensen, President and Chief Executive Officer of Pharmacosmos A/S. “COSELA is a first-in-class product that brings important benefits to patients and fits very nicely together with our lead product Monoferric® (ferric derisomaltose). While Monoferric is available around the World, COSELA is so far only approved in the US and in China. It will be a focus for us to bring this important product to more patients both in US and worldwide to help minimize the number of lung cancer patients suffering from myelosuppression after chemotherapy.”

Transaction Terms

Under the terms of the merger agreement, Pharmacosmos has agreed to commence a cash tender offer to acquire all issued and outstanding shares of G1 common stock for US $7.15 per share in cash. The transaction will be fully financed by Pharmacosmos’ existing cash on hand and existing corporate credit facilities.

The closing of the tender offer will be subject to customary conditions, including the tender of shares which represent at least a majority of the total number of G1’s outstanding shares of common stock and the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act. Upon successful completion of the tender offer, Pharmacosmos would acquire all shares not acquired in the tender offer through a second-step merger for the same consideration that the tendering stockholders will receive in the tender offer.

It is anticipated the transaction will close late in the third quarter of 2024. Upon completion of the transaction, G1’s common stock will no longer be publicly listed.

As previously announced, G1 will be releasing its second quarter 2024 financial results and filing its Form 10-Q Quarterly Report tomorrow. However, due to the pending transaction, we will no longer be hosting a conference call at 8:30 am ET, August 8 to review such results.

Advisors

For Pharmacosmos, MTS Health Partners, L.P. is serving as exclusive financial advisor, and Arnold & Porter Kaye Scholer LLP is serving as legal counsel. For G1, Centerview Partners LLC is serving as exclusive financial advisor, and Ropes & Gray LLP and Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C. are serving as legal counsel.

About COSELA^® (trilaciclib) for Injection

COSELA (trilaciclib) was approved by the U.S. Food and Drug Administration on February 12, 2021.

Indication

COSELA^® (trilaciclib) is indicated to decrease the incidence of chemotherapy-induced myelosuppression in adult patients when administered prior to a platinum/etoposide-containing regimen or topotecan-containing regimen for extensive-stage small cell lung cancer.

Important Safety Information

COSELA is contraindicated in patients with a history of serious hypersensitivity reactions to trilaciclib.

Warnings and precautions include injection-site reactions (including phlebitis and thrombophlebitis), acute drug hypersensitivity reactions, interstitial lung disease (pneumonitis), and embryo-fetal toxicity.

The most common adverse reactions (>10%) were fatigue, hypocalcemia, hypokalemia, hypophosphatemia, aspartate aminotransferase increased, headache, and pneumonia.

This information is not comprehensive. Please click here for full Prescribing Information. https://www.g1therapeutics.com/cosela/pi/

To report suspected adverse reactions, contact G1 Therapeutics at 1-800-790-G1TX or call FDA at 1-800-FDA-1088 or visit www.fda.gov/medwatch.

About Monoferric (ferric derisomaltose)

Indication

Monoferric (ferric derisomaltose) is indicated for the treatment of iron deficiency anemia (IDA) in adult patients:

• who have intolerance to oral iron or have had unsatisfactory response to oral iron
• who have non-hemodialysis dependent chronic kidney disease (NDD-CKD)

Important Safety Information

Monoferric is contraindicated in patients with a history of serious hypersensitivity to Monoferric or any of its components. Reactions have included shock, clinically significant hypotension, loss of consciousness, and/or collapse.

Warnings and precautions include serious hypersensitivity reactions, including anaphylactic-type reactions, some of which have been life-threatening and fatal, have been reported in patients receiving Monoferric. Patients may present with shock, clinically significant hypotension, loss of consciousness, and/or collapse. Monitor patients for signs and symptoms of hypersensitivity during and after Monoferric administration for at least 30 minutes and until clinically stable following completion of the infusion. Only administer Monoferric when personnel and therapies are immediately available for the treatment of serious hypersensitivity reactions. Monoferric is contraindicated in patients with prior serious hypersensitivity reactions to Monoferric or any of its components. In clinical trials in patients with IDA and CKD, serious or severe hypersensitivity were reported in 0.3% (6/2008) of the Monoferric treated subjects. These included 3 events of hypersensitivity in 3 patients; 2 events of infusion-related reactions in 2 patients and 1 event of asthma in one patient.

Excessive therapy with parenteral iron can lead to excess iron storage and possibly iatrogenic hemosiderosis or hemochromatosis. Monitor the hematologic response (hemoglobin and hematocrit) and iron parameters (serum ferritin and transferrin saturation) during parenteral iron therapy. Do not administer Monoferric to patients with iron overload.

Adverse reactions were reported in 8.6% (172/2008) of patients treated with Monoferric. Adverse reactions related to treatment and reported by ≥1% of the treated patients were nausea (1.2%) and rash (1%). Adjudicated serious or severe hypersensitivity reactions were reported in 6/2008 (0.3%) patients in the Monoferric group. Hypophosphatemia (serum phosphate <2.0 mg/dL) was reported in 3.5% of Monoferric-treated patients in Trials 1 & 2.

To report adverse events, please contact Pharmacosmos at 1-888-828-0655. You may also contact the FDA at www.fda.gov/medwatch or 1-800-FDA-1088.

This information is not comprehensive. Please click here for full Prescribing Information.

Pharmacosmos Group

Pharmacosmos A/S, headquartered in Holbaek, Denmark, and founded in 1965, is a highly specialised company focused on carbohydrate chemistry and a global leader in the development of innovative treatments for patients suffering from iron deficiency and iron deficiency anaemia. With companies in the UK, Ireland, Nordics, Germany, the USA, and China, as well as through partners, Pharmacosmos markets its products around the world. With a strong and ongoing commitment to R&D, Pharmacosmos is able to leverage a unique carbohydrate production platform along with deep expertise in the synthesis of iron-carbohydrate complexes. The Pharmacosmos Group has more than 500 employees.

About G1 Therapeutics

G1 Therapeutics, Inc. is a commercial-stage oncology biopharmaceutical company whose mission is to develop and deliver next-generation therapies that improve the lives of those affected by cancer, including the Company’s first commercial product, COSELA® (trilaciclib). G1’s goal is to provide innovative therapeutic advances for people living with cancer. G1 is based in Research Triangle Park, N.C. For additional information, please visit www.g1therapeutics.com and follow us on X (formerly known as Twitter) @G1Therapeutics and LinkedIn.

G1 Therapeutics^® and the G1 Therapeutics logo and COSELA^® and the COSELA logo are trademarks of G1 Therapeutics, Inc.

Forward-Looking Statements

This document contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements regarding the proposed acquisition of G1 by Pharmacosmos, the expected timetable for completing the transaction, and G1’s future financial or operating performance. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “seek,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this document are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation: (i) risks associated with the timing of the closing of the proposed transaction, including the risks that a condition to closing would not be satisfied within the expected timeframe or at all or that the closing of the proposed transaction will not occur; (ii) uncertainties as to how many of G1’s stockholders will tender their shares in the offer; (iii) the possibility that a governmental entity may prohibit, delay or refuse to grant approval for the consummation of the transaction; (iv) the possibility that competing offers will be made; (v) the outcome of any legal proceedings that may be instituted against the parties and others related to the merger agreement; (vi) unanticipated difficulties or expenditures relating to the proposed transaction, the response of business partners and competitors to the announcement of the proposed transaction, and/or potential difficulties in employee retention as a result of the announcement and pendency of the proposed transaction; (vii) G1’s ability to successfully demonstrate the efficacy and safety of its drug or drug candidates, and the preclinical or clinical results for its product candidates, which may not support further development of such product candidates; (viii) comments, feedback and actions of regulatory agencies; (ix) G1’s dependence on the commercial success of COSELA (trilaciclib); (x) the inherent uncertainties associated with developing new products or technologies and operating as commercial stage company; (xi) chemotherapy shortages; and (xii) other risks identified in G1’s SEC filings, including G1’s Annual Report on Form 10-K for the year ended December 31, 2023, and subsequent filings with the SEC. G1 cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. G1 disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

Additional Information and Where to Find It

The tender offer referred to in this document has not yet commenced. This document is for informational purposes only and is neither an offer to purchase nor a solicitation of an offer to sell shares, nor is it a substitute for the tender offer materials that Pharmacosmos and its acquisition subsidiary will file with the SEC upon commencement of the tender offer. At the time the tender offer is commenced, Pharmacosmos and its acquisition subsidiary will cause to be filed a tender offer statement on Schedule TO with the SEC, and G1 will file a solicitation/recommendation statement on Schedule 14D-9 with respect to the tender offer. THE TENDER OFFER STATEMENT (INCLUDING AN OFFER TO PURCHASE, A RELATED LETTER OF TRANSMITTAL AND OTHER OFFER DOCUMENTS) AND THE SOLICITATION/RECOMMENDATION STATEMENT WILL CONTAIN IMPORTANT INFORMATION THAT SHOULD BE READ CAREFULLY AND CONSIDERED BY G1’S STOCKHOLDERS BEFORE ANY DECISION IS MADE WITH RESPECT TO THE TENDER OFFER. Both the tender offer statement and the solicitation/recommendation statement will be mailed to G1’s stockholders free of charge. A free copy of the tender offer statement and the solicitation/recommendation statement will also be made available to all stockholders of G1 by accessing https://investor.g1therapeutics.com/ or by contacting Investor Relations at ir@g1therapeutics.com. In addition, the tender offer statement and the solicitation/recommendation statement (and all other documents filed with the SEC) will be available at no charge on the SEC’s website: www.sec.gov, upon filing with the SEC.

G1’S STOCKHOLDERS ARE ADVISED TO READ THE SCHEDULE TO AND THE SCHEDULE 14D-9, AS EACH MAY BE AMENDED OR SUPPLEMENTED FROM TIME TO TIME, AND ANY OTHER RELEVANT DOCUMENTS FILED WITH THE SEC WHEN THEY BECOME AVAILABLE BEFORE THEY MAKE ANY DECISION WITH RESPECT TO THE TENDER OFFER, BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT THE PROPOSED TRANSACTION AND THE PARTIES THERETO.

Pharmacosmos A/S Contact:

Christian Lundquist Madsen
VP Global Marketing & Communication
+45 5948 5959
clm@pharmacosmos.com

G1 Therapeutics Contacts:

John W. Umstead V
Chief Financial Officer
919-747-8419
jumstead@g1therapeutics.com

Will Roberts
Communications Officer
Vice President, Investor Relations and Corporate Communications
(919) 907-1944
wroberts@g1therapeutics.com

One of our Fierce 15 honorees dove deep into the ocean for inspiration from the sea squirt to develop new human therapeutics. Another thought the adorable 13-lined ground squirrel, with its polka dots and hibernation habits, might provide a clue to new treatments.

And there’s the biotech that looked at the Human Genome Project and thought, what else can it do?

There’s a company looking at psychedelics in a whole new way, rising on the recent interest in repurposing the illicit substances for something better, like improving mental health.

And then, over in New Zealand, one tiny company is levying a fight against viral infections. It has been told its mission is outrageous, but we don’t think so.

Welcome to the 2024 Fierce 15. The Fierce Biotech editorial team is proud to present this class of the best in biotech. These 15 companies were carefully selected from hundreds of nominees.

This is the hardest project we do all year—to pick just 15 companies breaking barriers and defining the future of the industry. But it’s also the most fulfilling when we’re done and the most exciting to publish. So read on to find out who we picked this year.

Cour Pharmaceuticals

Retraining the culprits at the center of autoimmune diseases 

Founded: 2013 
Based: Skokie, Illinois
Clinical focus: Autoimmune diseases

What makes Cour fierce: Cour Pharmaceuticals hopes to bring more than four decades’ worth of research into the immune system to fruition with a novel type of reverse vaccine that aims to reprogram the body’s aggressive—if a touch overzealous—white blood cells and ultimately convince them to not attack otherwise healthy organs. 

The startup’s goal is to build a targeted, platform-based approach that can address debilitating autoimmune diseases without blunting a patient’s ability to fight off an invading infection—and one that could potentially be tailored for a variety of different conditions. 

With technology originally developed at the Northwestern University Feinberg School of Medicine in Chicago, Cour employs biodegradable nanoparticles that encapsulate certain antigens—specifically, whichever peptides or proteins are needed to match the ones responsible for triggering the chain reaction that drives T cells to destroy the body’s own tissues.  

These nanoparticles hitch a ride on white blood cells known as monocytes as they travel to the spleen and liver. When those cells are recycled by the body, the antigens are released in a way that makes them look like normal dead cellular debris, which the immune system then learns to recognize. 

Where a traditional vaccine would instruct these types of cells to attack a particular intruder, Cour’s strategy is to assure the immune system that they’re allowed to pass by unharmed. 

“It was a 40-year endeavor trying to figure out how you get the immune system to be retrained so that it won’t attack the antigen of interest,” CEO John Puisis said in an interview with Fierce. “We are just very fortunate to hit the crossroads of a serendipitous scientific medical discovery.” 

In the earliest stages of the company, the tactic was studied as a way to tamp down the dangerous inflammatory responses seen in people with severe peanut allergies. Today, Cour is focused solely on autoimmunity, with a pipeline that includes candidates targeting Type 1 diabetes, celiac disease and the muscle-weakening condition myasthenia gravis. And, so far, early results have been promising, according to the CEO. 

“We can actually abrogate the disease,” Puisis said. “That’s what our data shows, which is really profound.” 

In Type 1 diabetes, where T cells assault the insulin-producing islet cells of the pancreas, Cour plans to halt the progression of the disease.  

The biotech’s CNP-103 program aims to present the body with 120 antigen epitopes by encapsulating four different recombinant proteins at once—and it’s slated to make a first-in-human debut next year through Cour’s phase 1b/2a trial.  

The plan is to start the study with participants in the advanced stages of the disease—namely those with established losses of pancreatic beta cells who may be considering a tissue transplant, despite still having the autoimmunity—before developing it into a treatment for early diagnoses. 

“Type 1 diabetes is still not terribly well managed today, despite improvements and continuous glucose monitoring,” said Chief Operating Officer Dannielle Appelhans. “But for addressing the underlying cause, there’s really nothing available.”  

Meanwhile, Cour’s CNP-106 treatment for myasthenia gravis has cleared the proof-of-concept stage and is enrolling adults in a similar early to midphase study.  

For celiac disease, the company has found a partner in Takeda, with a global licensing agreement inked in 2019 that includes a commitment of up to $420 million in potential milestone and royalty payments. That therapy—known as CNP-101 or TAK-101, depending on who you ask—smuggles in the gliadin proteins that serve as one of the two major components of gluten compounds. 

In a previous first-in-human clinical trial of about 30 patients, the company demonstrated that within the weeks following two infusions, measures of inflammatory cytokine biomarkers as well as T-cell activation and infiltration into the small intestine were greatly diminished, even when study participants were subsequently given large doses of gluten. 

“Doctors who diagnose patients with celiac disease give them a gluten challenge, and then they go look at the T cells,” Puisis said.  

“What a few doctors told us was that, with our treatment, that person would no longer have been diagnosed. So take that for what it’s worth, but that’s a pretty profound change, and it was significant with a very small population,” he said. “I’m being cautiously exuberant.” 

At the same time, Cour expects its products will be cheaper compared to gene therapies, despite falling under the same regulatory category as other cell-programming tech.  

“What’s really interesting about what we’re doing is that we have this platform, but 98% of it is a polymer, and then the proteins are only 1% to 2%,” said Appelhans, who previously served as COO and CEO of cell therapy developer Rubius Therapeutics and as chief technical officer of Novartis’ gene therapy division. Appelhans will take over the CEO role from Puisis effective Sept. 1. 

“So what really excites me is the potential to reach many patients across the world, because, while being an advanced modality, it doesn’t have the same cost structure associated with those.” 

Investors: Lumira Ventures, Bristol Myers Squibb, Roche Venture Fund, Pfizer Breakthrough Growth Initiative, Alpha Wave Ventures

COUR Pharmaceuticals, a clinical-stage biotechnology company developing first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced that as part of a leadership succession plan, John J. Puisis will transition his responsibilities as President and Chief Executive Officer of COUR to the Company’s Chief Operating Officer, Dannielle Appelhans, effective September 1, 2024. Mr. Puisis, who founded and led COUR through multiple INDs, clinical studies and strategic partnerships, will continue to serve in his role on the Company’s Board of Directors and will be an advisor to COUR through the end of 2024. Ms. Appelhans has also been appointed to the COUR Board of Directors.

“Leading COUR since its inception over a decade ago has been a highlight of my career,” stated Mr. Puisis. “With the support of industry-leading partners and top-tier investors, the COUR team has transformed a serendipitous discovery in the labs at Northwestern University into a clinically validated nanoparticle platform poised to deliver first-in-class, disease-modifying treatments for autoimmune and inflammatory diseases. Since securing our $105 million Series A earlier this year, we’ve solidified the Company’s foundation by filling critical management roles, expanding our board of directors, and fortifying key partnerships. The Company is now stronger than ever before. Given Dannielle’s significant contributions since joining COUR in 2023, and her extensive experience in the biopharmaceutical industry, I am confident in her vision for COUR’s future and her ability to lead the Company as CEO through the next phases of growth and expansion. I look forward to collaborating with Dannielle to ensure a seamless transition of leadership responsibilities and am excited to serve alongside her on COUR’s Board of Directors.”

Ms. Appelhans joined COUR as COO in 2023 and has since led R&D, technical development, clinical operations, quality, technical operations and has played a pivotal role in shaping recent corporate strategy. Before joining COUR, she served as CEO of Rubius Therapeutics, where she previously held the position of COO. Since 2022, she has served on the Board of Directors and as a member of the Nominating and Governance Committee for Generation Bio Co. Prior to Rubius, Ms. Appelhans held progressive roles at Novartis, culminating in her tenure as Senior Vice President, Head of Global Supply Chain across all divisions, and later as Senior Vice President of Technical Operations and Chief Technical Officer at Novartis Gene Therapies, where she oversaw late-stage development, clinical and commercial manufacturing, supply chain, and all supporting operational functions for the gene therapy unit. Earlier in her career, she served as a Senior Engagement Manager at McKinsey & Company and held roles of increasing responsibility at Eli Lilly and Company.

Ms. Appelhans earned a Master of Science in mechanical engineering from Massachusetts Institute of Technology’s School of Engineering, an MBA from Massachusetts Institute of Technology’s Sloan School of Management, and a Bachelor of Science in Engineering in mechanical engineering from the University of Michigan.

Ms. Appelhans remarked, “I joined COUR in 2023 because I saw tremendous potential in the Company’s platform technology. After over a year of working closely with the team, it’s evident to me that our nanoparticle approach to antigen-specific immune tolerance is at the forefront of the industry. As CEO, I am eager to build upon the momentum of our recent achievements, expanding COUR’s portfolio and advancing our clinical programs to pivotal stages, with the ultimate aim of delivering new life-changing treatments to patients.”

“On behalf of the COUR Board of Directors, I extend heartfelt gratitude to John for his more than 10 years of dedicated service to COUR Pharmaceuticals. Under his leadership, COUR has established extensive capabilities and assembled a strong leadership team laying the groundwork for future success. John’s contributions have been instrumental in shaping COUR into the organization it is today,” said Robert F. Carey, Lead Independent Director of the COUR Board of Directors. “Looking ahead, we are excited to announce Ms. Appelhans’ transition to the role of CEO. With our recent fundraising success and the anticipated clinical progress of our wholly owned programs for Myasthenia Gravis and Type 1 Diabetes, we believe COUR is well-positioned for continued success.”

About COUR Pharmaceuticals:

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune and inflammatory diseases.

COUR is currently enrolling patients in a phase 1b/2a double-blind, placebo-controlled, multicenter clinical study in Myasthenia Gravis and developing a product candidate in Type 1 Diabetes in addition to having partnered products in Celiac Disease (with Takeda Pharmaceuticals), and Primary Biliary Cholangitis (with Ironwood Pharmaceuticals).

For more information, please visit www.courpharma.com

Contacts
For Investor Relations
Brian Bock, Chief Financial Officer
bbock@courpharma.com

For Media
Jason Braco
jbraco@lifescicomms.com

X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced positive new clinical data from its ongoing Phase 2 clinical trial evaluating the safety and efficacy of mavorixafor, an oral CXCR4 antagonist, in the treatment of people with chronic neutropenia (CN). An interim analysis of data from the ongoing six-month study showed that once-daily oral mavorixafor was generally well tolerated and durably increased participants’ absolute neutrophil counts (ANC) both as a monotherapy and in combination with stable doses of injectable granulocyte colony-stimulating factor (G-CSF), the only therapy approved in the U.S. for severe chronic neutropenia.

Today the company also announced that it is currently screening patients for enrollment into its global, pivotal Phase 3 clinical trial, the 4WARD study, evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without stable doses of G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic CN who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants.

“For the first time, we have demonstrated the ability of mavorixafor monotherapy to durably and meaningfully increase ANC in people living with chronic neutropenia,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “In addition, we were pleased to see that once-daily mavorixafor used in combination with G-CSF also led to meaningful and sustained ANC increases and was generally well tolerated, further supporting the design of our newly initiated Phase 3 4WARD clinical trial, which will study the use of mavorixafor alone and with G-CSF in people with CN dealing with recurrent and/or serious infections. Today’s exciting interim results build on the momentum we established in April with mavorixafor’s first approval in the U.S., and we look forward to quickly advancing the 4WARD trial as we pursue a second indication to deliver for more patients in need.”

Interim Analysis of Data from Phase 2 Clinical Study of Mavorixafor in CN
The Phase 2 study of mavorixafor is a six-month, open-label clinical trial that enrolled a total of 23 participants diagnosed with idiopathic, congenital, or cyclic CN. The interim analysis included results from the two treatment groups in the study (mavorixafor monotherapy and mavorixafor with stable-dose G-CSF) that most closely mirror the participant population of the newly initiated Phase 3 4WARD trial. Fifteen participants were enrolled across these two groups and, as of the May 14, 2024 interim analysis data cut-off date, seven had completed the study, and five remain ongoing. Data from a third treatment group of eight participants receiving mavorixafor and dose-adjusted G-CSF are expected to be presented later this year.

The mavorixafor monotherapy group included 10 participants and the mavorixafor with stable-dose G-CSF group included five participants. As of the data cut-off date, findings from the interim analysis show:

• 100% (6/6) of evaluable participants who had completed the six-month study achieved target ANC increase (ANC >500 cells/µL) at Months 3 and 6 on once-daily mavorixafor therapy with or without stable-dose G-CSF.
• Participants on mavorixafor monotherapy achieved mean ANC levels above the lower limit of normal for CN (≥1,500 cells/µL) at Month 3 (n=8) and Month 6 (n=3).
  • Mavorixafor monotherapy also durably increased ANC in participants with severe CN (ANC<500 cells/µL at baseline), achieving mean ANC of ~800-1,000 cells/µL (ANC range targeted by experts) at Months 1, 3, and 6 (n=5, 3, and 2, respectively).
• Participants on mavorixafor in combination with stable-dose G-CSF experienced increases in mean ANC of >1,000 cells/µL at Months 1, 3, and 6 (n=4, 4, and 3, respectively) versus baseline.

Across the 23 participants enrolled in the study, mavorixafor was generally well tolerated as a monotherapy and in combination with G-CSF, with no drug-related serious adverse events reported, as of the interim analysis data cut-off date. Of the 23 participants, three discontinued due to non-serious adverse events. The overall safety profile remains consistent with previous clinical studies.

Jean Donadieu, M.D., Ph.D., pediatrician in the hemato-oncology department of Trousseau Hospital in Paris, coordinator of the French registry for chronic neutropenia, and coordinator of the French chronic neutropenia reference center, commented on the results: “I am pleased to see that these interim data are consistent with the previous results of the Phase 1b study, but now with durability of effect and a good tolerability profile out to six months of treatment. This patient group has only one currently approved treatment option – one that is injectable and that has dose-related, dose-limiting, and challenging side effects and risks. The results from this interim analysis offer a sound and compelling rationale for mavorixafor’s evaluation in a Phase 3 trial, which is very good news for my patients with chronic neutropenia who, I believe, would benefit from an oral therapy to help reduce recurring or severe infections.”

Investor Webinar Details:
X4 will host an investor webinar to present and discuss the new data today at 8:00 am ET. To register for the event, click here. A live Q&A will follow the formal presentation. Following the conclusion of the live webcast, a replay of the event and the presented slides will be available within the investors’ section of the X4 Pharmaceuticals website at www.x4pharma.com.

About Chronic Neutropenia and Mavorixafor
Chronic neutropenia is a rare blood condition lasting more than three months, persistently or intermittently, and characterized by increased risk of infections and reduced quality of life due to abnormally low levels of neutrophils circulating in the blood. Neutrophils are retained in the bone marrow by the CXCR4/CXCL12 axis, creating a reserve of cells. Downregulation of the CXCR4 receptor by mavorixafor, an orally active CXCR4 antagonist, has been shown to mobilize neutrophils from the bone marrow into the peripheral blood across multiple disease states. The level of circulating neutrophils is typically measured by drawing blood to determine the absolute neutrophil count (ANC).

About the Phase 1b/Phase 2 Chronic Neutropenia Trial
The Phase 1b/Phase 2 clinical trial (NCT04154488) is a proof-of-concept, open-label, multicenter study designed to assess the safety and tolerability of oral mavorixafor, with or without injectable G-CSF, in participants with chronic neutropenic disorders, including idiopathic, cyclic, and congenital neutropenia. In the Phase 1b portion of the study, participants received one dose of oral mavorixafor and were assessed for magnitude of absolute neutrophil count (ANC) response and tolerability. In this initial portion of the study, 100% of participants (n=25) responded to treatment and mavorixafor was generally well tolerated alone or dosed concurrently with G-CSF. The ongoing Phase 2 portion of the trial (n=23 fully enrolled) is assessing the safety, tolerability, and the impact on participants’ neutropenia of oral, once-daily mavorixafor with and without concurrent injectable G-CSF therapy over a six-month period.

About the 4WARD Global, Pivotal, Phase 3 Clinical Trial
The 4WARD trial is a global, pivotal Phase 3 clinical trial (NCT06056297) evaluating the efficacy, safety, and tolerability of oral, once-daily mavorixafor (with or without G-CSF) in people with congenital, acquired primary autoimmune, or idiopathic chronic neutropenia who are experiencing recurrent and/or serious infections. The 52-week trial is a randomized, double-blind, placebo-controlled, multicenter study aiming to enroll 150 participants with confirmed trough ANC levels less than 1,500 cells per microliter at baseline screening and histories of two or more serious and/or recurrent infections in the prior year. The primary endpoint of the trial is based on two outcome measures: annualized infection rate and positive ANC response.

About X4 Pharmaceuticals
X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI™ (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding the potential therapeutic benefit of mavorixafor; the initiation, timing, progress, and results of our current and future studies and clinical trials, including the Phase 2 clinical trial in chronic neutropenia and the Phase 3 4WARD clinical trial and related preparatory work and the period during which the results of the trials will become available; and the mission and goals for our business. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: we may encounter adverse events for mavorixafor at any stage that negatively impact development and/or commercialization; the expected availability, content, and timing of clinical data from our ongoing clinical trials of mavorixafor may be delayed or unavailable, including clinical results from our ongoing Phase 2 clinical trial and the announced Phase 3 4WARD trial; the trials and studies may not have satisfactory outcomes; the outcomes of preclinical studies or earlier clinical trials will not be predictive of later clinical trial results; the design and rate of enrollment for clinical trials, including the current design of our Phase 3 clinical trial evaluating mavorixafor in certain chronic neutropenic disorders may not enable successful completion of the trial(s); we may be unable to obtain and maintain regulatory approvals; uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development; initial or interim results from a clinical trial may not be predictive of the final results of the trial or the results of future trials; the potential adverse safety effects arising from the testing or use of our product and product candidates may negatively impact development and/or commercialization; there will be changes in expected or existing competition; there will be changes in the regulatory environment; our business may be adversely affected and our costs may increase if any of our key collaborators fails to perform its obligations or terminates our collaboration; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected which may cause us to use cash more quickly than we expect or to change or curtail some of our plans or both; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2024, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.

Company Contact:
José Juves
Head of Corporate & Patient Affairs
jose.juves@x4pharma.com

Investor Contact:
Daniel Ferry
Managing Director, LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease, today announced final results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD). Final results from this study confirmed the excellent safety profile of TPN-101 and showed clinical signs of disease-modifying effects in patients with these disorders, which align with previously reported safety and therapeutic activity in patients with Progressive Supranuclear Palsy (PSP). These findings are consistent with TPN-101’s reduction of neuroinflammation and neurodegeneration through blocking the activity of LINE-1, a human-specific retrotransposon that is no longer adequately suppressed in many neurodegenerative disorders and aging.

ALS is a progressive and uniformly fatal neurodegenerative disease with a mean survival of two-to-three years. Respiratory failure is the most common cause of death for people with ALS. Vital Capacity (VC) is an objective measure of respiratory function that correlates with mortality in these patients. After 24 weeks, C9-ALS participants treated with TPN-101 experienced approximately 50% less decline in VC compared to those treated with placebo (LS mean change -8.4% vs -16.5%). When the participants in the placebo group were switched to TPN-101 during the open-label period, the decline in VC over the following 24 weeks (-7.2%) was less than half that while on placebo and comparable to that in the original TPN-101 group during the double-blind period. Overall, the 48-week changes in both groups were lower than expected based on natural history in similar study populations.

The decline on the Revised ALS Functional Rating Scale (ALSFRS-R) was comparable between the TPN-101 and placebo groups during the 24-week double-blind period (LS mean -7.2 points vs -6.7). However, during the 24-week open-label extension period, the decline on the ALSFRS-R in the original TPN-101 group was less than half that during the initial 24-week period and less than half that of the placebo group during both study periods. The decline in ALSFRS-R in the original TPN-101 group over the entire 48 weeks was approximately 40% less than expected based on natural history data, indicating a global clinical benefit with longer treatment.

“The effects of TPN-101 across multiple key endpoints in this study are encouraging and represent an important step forward in finding a potential treatment for this serious illness,” said Merit Cudkowicz, M.D., Chair of the Massachusetts General Hospital Department of Neurology, Julieanne Dorn Professor of Neurology at Harvard Medical School, and principal investigator in the Phase 2 study of TPN-101 for C9orf72-related ALS/FTD. “I look forward to advancing the development of TPN-101 and what that could mean for people living with C9-ALS.”

In participants with C9-ALS, those treated with TPN-101 had lower levels of neurofilament light chain (NfL) compared with placebo at the end of the double-blind period. NfL is the primary biomarker of neurodegeneration, and the NfL results at both Weeks 24 and 48 are consistent with findings from the company’s Phase 2 study of TPN-101 for the treatment of PSP. TPN-101 also had lowering effects on additional biomarkers of neurodegeneration and neuroinflammation, including neurofilament heavy chain (NfH), interleukin 6 (IL-6), neopterin, and osteopontin.

A meta-analysis of the combined C9-ALS and PSP populations from the two Phase 2 studies of TPN-101 showed a statistically significant NfL-lowering effect of TPN-101 versus placebo at Week 24 (p = 0.034). The consistency of biomarker improvements in these two studies evaluating patients with different clinical conditions supports the treatment hypothesis that these diseases share a common LINE-1-related pathophysiology.

“Today there are very limited treatment options for ALS patients and based on these results that show patients treated with TPN-101 experience impactful benefits across multiple functional and biomarker measures, we plan to rapidly advance TPN-101 into a Phase 3 registration study for the treatment of C9-ALS,” said Dennis Podlesak, Chairman and Chief Executive Officer of Transposon. “In addition to ALS, we are committed to advancing TPN-101 for the treatment of PSP, Alzheimer’s disease and other neurodegenerative and autoimmune disorders with the goal of providing new and innovative therapies that can significantly improve the lives of those battling these devastating diseases.”

Transposon intends to present detailed data from this study at upcoming scientific meetings.

About the Phase 2 Study in C9orf72-related ALS/FTD

The Phase 2 study in C9orf72-related ALS/FTD is multi-center, randomized, double-blind, placebo-controlled parallel-group, two-arm study with an open-label treatment phase in patients with C9orf72-related ALS and/or FTD. Participants (n=42) were randomized to receive daily doses of 400 mg of TPN-101 or placebo. The study includes a six-week screening period, a 24-week double-blind treatment period, a 24-week open label treatment period, and a follow-up visit four weeks post-treatment. All phases of the study, including the 24-week open label treatment period, have been completed. Further information on the study can be accessed at ClinicalTrials.gov.

About TPN-101

TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 DNA, triggering innate immune responses that contribute to neurodegenerative, autoimmune and aging-related disease pathology.

About ALS and FTD

ALS is a neurodegenerative disease characterized by progressive muscle weakness, loss of ability to speak, eat, move or breathe. FTD is a progressive frontal / temporal cortex disease associated with behavior and personality changes, emotional problems, and difficulty walking, communicating or working. With onset commonly in middle age or earlier, patients with ALS have a mean survival of two-to-three years. Patients with FTD have a mean survival of nine years.

About Transposon

Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease. The company’s lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications.

Contact:
Rick Orr
Transposon Therapeutics, Inc.
(858) 535-4821
rorr@transposonrx.com

SOURCE Transposon Therapeutics

Edwards Lifesciences (NYSE: EW) today announced investments that reflect the company’s deep commitment to advancing patient care through structural heart innovation, addressing large unmet patient needs and supporting sustainable long-term growth.

Edwards has entered into an agreement to acquire JenaValve Technology, a pioneer in the transcatheter treatment of aortic regurgitation (AR), a deadly disease that impacts a significant and growing population and is largely untreated today. JenaValve presented positive results of its U.S. pivotal trial for the treatment of symptomatic, severe AR in high-risk patients late last year. As the pioneer in valve innovation for more than 60 years, Edwards believes it is uniquely positioned to lead this next frontier of aortic valve disease treatment. Edwards anticipates FDA approval of the JenaValve Trilogy Heart Valve System in late 2025, which will represent the first approved therapy for patients suffering from AR.

Building on an investment made in 2016, Edwards has exercised its option to acquire Endotronix, a leader in heart failure (HF) management solutions. Many structural heart patients Edwards serves today also suffer from HF with limited options. This acquisition will expand Edwards’ structural heart portfolio into a new therapeutic area to address the large unmet needs of patients suffering from HF. Last month, Endotronix received FDA approval for Cordella, an implantable pulmonary artery pressure sensor allowing early, targeted therapeutic intervention. A CMS national coverage determination is expected in early 2025.

“These acquisitions expand our opportunities to address the unmet needs of aortic regurgitation and heart failure patients around the world,” said Bernard Zovighian, Edwards’ CEO. “We are pleased to enter these structural heart therapeutic areas with innovation, world-class science and clinical evidence to provide access to life-saving technologies for patients around the world.”

Edwards anticipates these investments will strengthen its leadership in structural heart innovation and represent long-term growth opportunities. Edwards expects minimal revenue contribution from these acquisitions in 2025. The aggregate upfront purchase price for these strategic investments is approximately $1.2 billion. The acquisitions are subject to the satisfaction of certain closing conditions, including the receipt of required antitrust and foreign investment approvals.

About Edwards Lifesciences

Edwards Lifesciences is the global leader of patient-focused innovations for structural heart disease and critical care monitoring. We are driven by a passion for patients, dedicated to improving and enhancing lives through partnerships with clinicians and stakeholders across the global healthcare landscape. For more information, visit www.edwards.com and follow us on Facebook, Instagram, LinkedIn, X and YouTube.

This news release includes forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. We intend the forward-looking statements contained in this press release to be covered by the safe harbor provisions of such Acts. These forward-looking statements can sometimes be identified by the use of forward-looking words, such as “may,” “might,” “believe,” “will,” “expect,” “project,” “estimate,” “should,” “anticipate,” “plan,” “goal,” “continue,” “seek,” “intend,” “optimistic,” “aspire,” “confident” and other forms of these words and include, but are not limited to, statements made by Mr. Zovighian and statements regarding our expected continued performance of Edwards; performance of the Edwards, JenaValve or Endotronix technologies; product and therapy benefits; patient access and outcomes; size of treatable population; leading position; growth opportunities; unmet needs in structural heart, aortic regurgitation, and heart failure therapeutic areas; probability of approval by the FDA and in the anticipating timeline; probability of a positive NCD by the CMS and in the anticipated timeline; synergies between the technologies, business, and operations of each of JenaValve and Endotronix and Edwards’ technologies, products, portfolio, expertise, and operations; ability to leverage the technology or innovation from these acquisitions or cause or ensure accelerated access to life-saving technologies for patients or development of novel technologies as a result of these acquisitions; commitment to expand opportunities in structural heart innovation, address large unmet patient needs, and support sustainable long-term growth; objective to expand Edwards’ portfolio into new structural heart therapeutic areas; opportunities and revenue return on these acquisitions and their contribution to Edwards’ growth and performance, as well as the expectations on timing of such returns and contributions; therapy approval pipeline for patients suffering from AR; probability of the closing of the two acquisitions; other objectives and expectations; and other statements that are not historical facts. Forward-looking statements are based on estimates and assumptions made by management of the company and are believed to be reasonable, though they are inherently uncertain and difficult to predict. Our forward-looking statements speak only as of the date on which they are made, and we do not undertake any obligation to update any forward-looking statement to reflect events or circumstances after the date of the statement. Investors are cautioned not to unduly rely on such forward-looking statements.

Forward-looking statements involve risks and uncertainties that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements. Factors that could cause actual results or experience to differ materially from that expressed or implied by the forward-looking statements include, but are not limited to: (i) Edwards may be unable to close the acquisitions of each of JenaValve and Endotronix, which may materially and adversely affect Edwards’ business and the price of Edwards’ common stock; (ii) the occurrence of any event, change or other circumstance that could cause Edwards to abandon the acquisitions of either or both of JenaValve and Endotronix; (iii) risks related to disruption of management’s attention from Edwards’ ongoing business operations; (iv) the effect of the announcement or the pendency of the acquisitions on Edwards’ relationships with its customers, operating results and business generally; (v) potential significant transaction costs associated with either or both acquisitions; (vi) the outcome of any legal proceedings or regulatory actions to the extent initiated against Edwards or others related to either or both acquisitions; (vii) the ability of Edwards to execute on its strategy and achieve its goals and other expectations after the closing of either or both acquisitions; (viii) legal, regulatory, tax and economic developments affecting Edwards’ business; (ix) the unpredictability and severity of catastrophic events, including, but not limited to, acts of terrorism, outbreak of war or hostilities or current or future pandemics or epidemics, as well as Edwards’ response to any of the aforementioned factors; and (x) other risks detailed in Edwards’ filings with the SEC, which may be found at edwards.com.

Edwards, Edwards Lifesciences, and the stylized E logo are trademarks of Edwards Lifesciences Corporation or its affiliates. All other trademarks are the property of their respective owners.

Contacts

Media Contact: Amy Hytowitz, 949-250-4009
Investor Contact: Mark Wilterding, 949-250-6826

enGene Holdings Inc. (Nasdaq: ENGN, “enGene” or the “Company”), a clinical-stage genetic medicines company whose non-viral, intravesical lead product candidate, EG-70, is in a pivotal study for BCG-unresponsive high-risk Non-Muscle Invasive Bladder Cancer (NMIBC), today announced that Ron Cooper has joined the Company as Chief Executive Officer and member of the Board of Directors, effective July 22, 2024. This transition follows a previously announced succession plan for Jason Hanson, who will remain in service to the Company as a strategic advisor.

“The Board of Directors and I would like to warmly welcome Ron Cooper, who brings significant discovery, development, and commercial launch expertise,” said Dr. Richard Glickman, Chairman of the Board of Directors. “Given his proven record of effectively developing and commercializing transformative therapeutics, Mr. Cooper is an excellent fit to lead enGene forward as the Company continues to advance EG-70 as an innovative and differentiated therapeutic option for patients with high-risk NMIBC.”

“The unmet need for patients with bladder cancer is significant,” said Mr. Cooper. “EG-70 has the potential to be the most practical treatment option for high-risk NMIBC based on a unique combination of efficacy, tolerability and ease of administration. Under Mr. Hanson’s leadership, the enGene team has developed an innovative platform to deliver genetic medicines through mucosal tissues, which I believe has underappreciated potential.”

Mr. Cooper most recently served as President and Chief Executive Officer of Albireo Pharma, a fully integrated global commercial biopharmaceutical company that Ipsen acquired in 2023. While at Albireo Pharma, Mr. Cooper took the company public, created a new corporate strategy to focus development on building a rare pediatric company while monetizing other pipeline assets, and guided Bylvay® through three Phase 3 programs, regulatory approvals, and a global commercial launch. Earlier in his career, Mr. Cooper spent nearly 30 years at Bristol-Myers Squibb (BMS) in roles of increasing responsibility in sales, marketing and general management, most recently serving as President, Europe. While at BMS, he played a leadership role in numerous successful product launches. Mr. Cooper is currently Chairman of the Board of Directors at C4 Therapeutics and serves on the Board of Generation Bio. He is a graduate of St. Francis Xavier University.

The Company also announced the promotion of Dr. Raj Pruthi to Chief Medical Officer, succeeding Dr. Richard Bryce. Dr. Pruthi, who joined enGene in April 2024, is a thought leader in the urological community with over 25 years of experience providing patient care and advancing the development of new therapeutics.

“On behalf of the Board of Directors, we would like to sincerely thank Mr. Hanson for his service to enGene,” said Dr. Glickman. “Mr. Hanson successfully led the Company’s evolution from an early-stage research organization to a well-capitalized, publicly traded, pivotal-stage company poised to make an impactful difference for patients living with NMIBC. We wish him all the best in his future endeavors.”

About enGene

enGene is a clinical-stage biotechnology company mainstreaming genetic medicines through the delivery of therapeutics to mucosal tissues and other organs, with the goal of creating new ways to address diseases with high clinical needs. enGene’s lead program is EG-70 for patients with Non-Muscle Invasive Bladder Cancer (NMIBC) – a disease with a high clinical burden. EG-70 is being evaluated in the ongoing multi-cohort LEGEND Phase 2 study, which includes a registrational cohort studying EG-70 in Bacillus Calmette Guérin (BCG)-unresponsive patients with carcinoma in situ (Cis). EG-70 was developed using enGene’s proprietary Dually Derivatized Oligochitosan (DDX) platform, which enables penetration of mucosal tissues and delivery of a wide range of sizes and types of cargo, including DNA and various forms of RNA.

Forward-Looking Statements

Certain statements contained in this press release may constitute “forward-looking statements” within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995, and “forward-looking information” within the meaning of Canadian securities laws (collectively, “forward-looking statements”). enGene’s forward-looking statements include, but are not limited to, statements regarding enGene’s management teams’ expectations, hopes, beliefs, intentions, goals or strategies regarding the future. In addition, any statements that refer to projections, forecasts or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking statements. The words “anticipate”, “appear”, “approximate”, “believe”, “continue”, “could”, “estimate”, “expect”, “foresee”, “intend”, “may”, “might”, “plan”, “possible”, “potential”, “predict”, “project”, “seek”, “should”, “would”, and similar expressions (or the negative version of such words or expressions) may identify forward-looking statements, but the absence of these words does not mean that a statement is not forward-looking. Forward-looking statements may include, for example, statements about the potential of EG-70 and enGene’s proprietary DDX platform.

Many factors, risks, uncertainties and assumptions could cause the Company’s actual results, performance or achievements to differ materially from those expressed or implied by the forward-looking statements, including, without limitation, the Company’s ability to recruit and retain qualified scientific and management personnel; establish clinical trial sites and enroll patients in its clinical trials; execute on the Company’s clinical development plans and ability to secure regulatory approval on anticipated timelines; and other risks and uncertainties detailed in filings with Canadian securities regulators on SEDAR+ and with the U.S. Securities and Exchange Commission (“SEC”) on EDGAR, including those described in the “Risk Factors” sections of the Company’s Annual Report on Form 10-K for the fiscal year ended October 31, 2023, our Quarterly Report on Form 10-Q for the fiscal quarter ended January 31, 2024 and our Quarterly Report on Form 10-Q for the fiscal quarter ended April 30, 2024 (copies of which may be obtained at www.sedarplus.ca or www.sec.gov).

You should not place undue reliance on any forward-looking statements, which speak only as of the date on which they are made. enGene anticipates that subsequent events and developments will cause enGene’s assessments to change. While enGene may elect to update these forward-looking statements at some point in the future, enGene specifically disclaims any obligation to do so, unless required by applicable law. Nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240724237337/en/

For media: media@engene.com

For investors: investors@engene.com

Positive results including complete inhibition of cough response in preclinical models and a favorable safety profile in three clinical studies support the initiation of a Phase 2b clinical trial of taplucainium in 240 subjects in the second half of 2024.

Nocion Therapeutics, Inc., a clinical stage biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions”, that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain, today announced the presentation of progress in the development of taplucainium, a novel, charged sodium channel blocker, as a potential treatment for chronic cough at the 13^th London International Cough Symposium. The presentation and poster will be given by Bernard Silverman, MD., Nocion’s Chief Medical Officer.

“We are excited with the strong progress that we have made developing taplucainium as a treatment for chronic cough with the ability to address this significant unmet medical need,” said Dr. Silverman. “We look forward to the start of our ASPIRE (Phase 2b) clinical trial of taplucainium in 240 chronic cough patients in the second half of 2024.”

ABOUT TAPLUCAINIUM
Taplucainium (formerly NTX-1175) is a proprietary molecule in the novel class of charged sodium channel blockers that allows for specific silencing of activated/inflamed nociceptors while having minimal local off-target effects or systemic exposure. Unlike other investigative cough therapies, such as P2X3-antagonists, TRPA1-antagonists and TRPM8-agonists, which target a specific large pore channel, taplucainium works downstream by selectively targeting the sodium channels in activated nociceptors which are the target of these large pore channels. Taplucainium is formulated into a dry powder for inhalation and, once inhaled, gains access to the pulmonary nociceptors through any open large pore channel including P2X, TRPV, TRPA and TRPM channels whereupon it inhibits the sodium channels responsible for initiating the pathological cough response. The broader mechanism of taplucainium has shown significant antitussive effects in preclinical models of cough. Combined with good preliminary safety and efficacy data from earlier stage clinical work, this forms the basis for its investigation not just in chronic cough but in other cough indications as well.

ABOUT NOCION
Nocion Therapeutics is a biopharmaceutical company developing novel small molecule charged sodium channel blockers called “nocions” that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain. The company’s mission is to safely alleviate suffering for millions of patients with conditions arising from activated sensory neurons. Working with Harvard’s Office of Technology Development, Nocion was founded on an exclusive license to foundational intellectual property from Harvard University and Boston Children’s Hospital. Venture investors in Nocion include Arkin Bio Capital, Canaan Partners, F-Prime Capital, Lumira Ventures, Mass General Brigham Ventures, Mission BioCapital, Monograph Capital, Morningside and Osage University Partners. For more information, visit: www.nociontx.com.

Contacts

Stephanie Gillis
pr@nociontx.com

The Extroducer® Infusion Catheter System ® enables local delivery of XC001 to the heart without the need for surgery.

XC001 has achieved positive Phase 1/2 results in the EXACT Trial validating its transformative potential for treatment of refractory angina in patients who have exhausted available treatment options and have a debilitating quality-of-life.

XyloCor Therapeutics, Inc. (“XyloCor”), a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, and SmartWise, a unit ofSmartCella Holding AB (“SmartCella”), have entered into a licensing agreement under which XyloCor has rights to the Extroducer® Infusion Catheter System ®, a first-in-class endovascular device designed to deliver advanced therapies directly into the heart [and hard-to-reach tissues] . XyloCor plans to deploy the Extroducer to support catheter-based endocardial delivery of its lead gene therapy candidate, XC001 (encoberminogene rezmadenovec), in future clinical studies and commercial use.

“This agreement with SmartCella will enable XyloCor to build upon its robust foundation of efficacy and safety data for XC001 by offering the potential for improved safety and ease of delivery without surgery via this novel catheter,” said Al Gianchetti, President and CEO of XyloCor. “Teaming up with SmartCella will help in our effort to optimize patient safety and tolerability while maintaining accurate delivery of XC001 to target areas in the heart for patients with refractory angina. It also opens up the potential to develop XC001 earlier in the coronary artery disease progression for even larger patient groups.”

XC001 is designed to reduce ischemic burden by creating new blood vessels in the heart through the local expression of multiple isoforms of vascular endothelial growth factor (VEGF). With the use of the Extroducer catheter, XyloCor can offer patients a better delivery option for local administration of XC001 directly to the heart, that is less invasive and eliminates potential risks associated with surgical administration.

“We welcome the Extroducer delivery of XC001 as it offers a more efficient method for gene therapy administration for patients with refractory angina,” said Timothy D. Henry MD, Interventional Cardiologist and Director of the Lindner Center, The Christ Hospital, Cincinnati, Ohio. “Preclinical models provide strong evidence that this approach will maintain, or even improve the efficacy when compared to surgical delivery and it should lower the risk of complications that may arise from surgical administration. I am looking forward to initiating the Phase 2b trial of XC001 in patients with refractory angina using this innovative administration approach.”

The recently published EXACT Phase 1/2 trial assessed the use of one-time gene therapy with XC001 as a new therapeutic approach in refractory angina – a debilitating and chronic condition that impacts over one million people in the United States and is growing in prevalence. In the EXACT trial, 42 patients with class II-IV angina were treated with XC001 directly administered to the heart following minimally invasive surgical access. The results demonstrated that treatment with XC001 can be safely administered and achieve durable clinical improvements of exercise duration, and angina frequency, due to a decrease in ischemic burden, as measured by Positron Emission Tomography (PET) imaging. Notably, six months after treatment 43% of patients had no chest pain with ordinary activities and 58% reported no angina episodes at 12-month clinical follow up. XC001 was well tolerated in the patient population and there were no serious adverse events related to the drug. The Phase 2b trial will be a randomized double-blinded study assessing the safety and efficacy of XC001 administered via the Extroducer® Delivery Catheter in coronary artery disease patients with refractory angina.

“The collaboration underscores the transformative potential of the Extroducer in delivering XC001 therapy for patients with refractory angina. A great example of a powerful combination of delivery system and drug therapy representing a substantial advancement in treatment options. The collaboration with such a distinguished partner as XyloCor marks a significant milestone for our global expansion efforts and will also enable us to further explore and harness the future capabilities of the Extroducer, ultimately expanding the benefits to a greater number of patients in need,“ said Niklas Prager, CEO of SmartCella.

Terms of the agreement include a global license to XyloCor for use of the Extroducer for the administration of XC001 and provide for SmartCella to supply catheters to XyloCor in clinical trials and commercial use in exchange for an upfront payment, clinical, regulatory and commercial milestones and a royalty on sales. Total deal value amounts to approximately USD 130 million and mid-single digit royalties.

About XC001

XC001 is designed to promote new blood vessels in the heart that will bypass diseased blood vessels and improve blood flow. By restoring blood flow, chest pain associated with refractory angina may decrease, potentially improving patients’ quality of life by enabling them to engage in daily physical activities that would otherwise cause pain. XC001 is designed to avoid toxicity issues observed with other gene therapies through a strategy of one‑time, local administration. This approach allows XC001 to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects.

AboutExtroducer® Infusion Catheter System

The Extroducer® Infusion Catheter System is a first-in-class endovascular delivery device which enables direct-to-tissue drug delivery. The Extroducer® addresses a significant unmet need in the field of novel therapies, enabling targeted delivery of a wide range of modalities for solid tumor treatment, genetic disorders and tissue repair, to name but a few. Using standard equipment and routine interventional radiology approaches, the Extroducer provides access to hard-to-reach tissues by safely penetrating the vessel wall and delivering payload directly to the target location. SmartWise received U.S. Food and Drug Administration (FDA) clearance under 510(k) for the Extroducer® delivery catheter in June 2022.

About XyloCor

XyloCor Therapeutics, Inc. is a private, clinical‑stage biopharmaceutical company developing potential best‑in‑class gene therapies to transform outcomes for patients with cardiovascular disease. The Company’s lead product candidate, XC001, is in clinical development to investigate use for patients with refractory angina for whom there are no treatment options. XyloCor has a second preclinical investigational product, XC002, in discovery stage, being developed for the treatment of patients with cardiac tissue damage from heart attacks. The company, which was co‑founded by Ronald Crystal, M.D., and Todd Rosengart, M.D., has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

About SmartCella

SmartCella, founded in 2014, is an innovative biotechnology company based in Stockholm, Sweden. SmartCella’s vision is to combine first-in-class delivery platforms with cutting-edge cell and mRNA therapies to unleash the full potential of targeted therapies. The company has three main business units, Smartwise, SmartCella Solutions and ProCella. For more information, visit www.smartcella.com.

Contacts

Corporate and Investor Relations:
Brian Davis
XyloCor Therapeutics, Inc
brian.davis@xylocor.com
610-541-2056

Media Contact:
Mike Beyer
Sam Brown Inc. Healthcare Communications
mikebeyer@sambrown.com
312-961-2502

SmartCella Contact:
Niklas Prager, CEO, +46 768 117744; niklas.prager@smartcella.com

COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced the appointment of Tim Walbert, an industry veteran with more than 30 years of leadership experience in biotech, to its Board of Directors, effective immediately.

Mr. Walbert joins COUR after a successful 15-year career at Horizon Therapeutics, where he served as president, chief executive officer, and chairman of the company’s board of directors. During his tenure leading Horizon, he grew the company from its founding into a leader in the development and commercialization of treatments for rare diseases. In 2023, Horizon Therapeutics was acquired by Amgen for $28 billion. Mr. Walbert now serves as a senior advisor to Amgen, as well as a director for several publicly traded companies including Sagimet Biosciences, Mirum Pharmaceuticals, and Century Therapeutics.

Prior to Horizon, Mr. Walbert served as president, CEO, and as a director of IDM Pharma. Before this role, he was executive vice president, commercial operations, at NeoPharm. Earlier in his career, he served as divisional vice president and general manager, immunology, at Abbott (AbbVie), where he led the development and launch of HUMIRA, and was also divisional vice president, global cardiovascular strategy. Mr. Walbert’s experience also includes roles of increasing responsibility at CELEBREX North America, G.D. Searle & Company, Merck, and Wyeth.

“The addition of Mr. Walbert to our board of directors dramatically broadens the expertise of our team and further solidifies our position as the industry leader in antigen-specific immune tolerance,” said John J. Puisis, founder, president, and CEO of COUR. “We are thrilled to welcome him to our board, as he offers valuable knowledge in leading companies through discovering, developing, and commercializing novel treatments that provide unique solutions to underserved patient populations. With notable clinical catalysts forthcoming, including the dosing of the first patient in our Phase 2 clinical trial of CNP-106, our investigational treatment for myasthenia gravis, and the filing of our Type 1 diabetes IND, which is anticipated later this year, we are eager for Mr. Walbert to make meaningful contributions to our team.”

Mr. Walbert added, “COUR’s versatile nanoparticle approach offers the unique opportunity to develop a pipeline of disease modifying products out of a robust platform technology, which has been validated both with clinical data and a consortium of high-profile investors and industry partners. I am excited to leverage my experience from leading Horizon Therapeutics to drive value for patients and all other company stakeholders as a part of COUR’s board of directors.”

About COUR Pharmaceuticals:

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune and inflammatory diseases.

COUR is developing product candidates in Myasthenia Gravis and Type 1 Diabetes in addition to having partnered products in Celiac Disease (with Takeda Pharmaceuticals), and Primary Biliary Cholangitis (with Ironwood Pharmaceuticals).

For more information, please visit www.courpharma.com

Contacts
For Investor Relations
Brian Bock, Chief Financial Officer
bbock@courpharma.com

For Media
Owen Blaschak
oblaschak@lifescicomms.com

Oversubscribed financing led by strategic investor, with participation from new and existing investors

Endogenex, a clinical-stage medical device company dedicated to improving outcomes in individuals with type 2 diabetes, announced it has closed an oversubscribed Series C financing totaling $88 million. The new capital will be used to complete the pivotal ReCET Clinical Study, which has been granted an Investigational Device Exemption (IDE) by the U.S. Food and Drug Administration (FDA). A group of new investors, including Hatteras Venture Partners, Lumira Ventures, and Orlando Health Ventures, joined an undisclosed strategic lead investor and existing investors Intuitive Ventures, Longitude Capital, Mayo Clinic, and Santé Ventures in the Series C funding.

“We are excited to take this significant step forward in our mission to transform diabetes care,” said Stacey Pugh, CEO of Endogenex.  “This funding will enable us to complete our pivotal clinical study, bringing us closer to offering a groundbreaking solution for type 2 diabetes patients.  The therapeutic landscape in diabetes continues to evolve, especially around the earlier use of SGLT2i and GLP-1RA. However, there remains a considerable unmet need to address the underlying pathophysiology and progression of the disease.”

“We are very proud of the company’s progress, and this successful funding round is a testament to Endogenex’s innovative technology,” said Juliet Tammenoms Bakker, Endogenex Board Chair and Managing Director at Longitude Capital. “This milestone underscores our investors’ confidence in the company’s vision and the impact it can make in treating the epidemic of type 2 diabetes around the world.”

About the ReCET Clinical Study

The ReCET Clinical Study is a multicenter, prospective, randomized, double-blinded, sham-controlled study assessing the safety and effectiveness of the ReCET System. The pivotal study received IDE approval in November 2023. The study will enroll up to 350 patients at clinical sites in the United States and Australia.

About the ReCET™ Procedure

ReCET is a novel, endoscopic outpatient procedure that targets the cellular pathology of the duodenum. This pathology may contribute to the development and progression of type 2 diabetes.

The ReCET System aims to initiate the body’s natural regenerative process by applying highly controlled, non-thermal pulsed electric fields to the mucosa and sub-mucosa duodenal tissue. This approach may help restore proper cellular signaling from the duodenum and improve metabolic function, including better control of blood glucose levels.

The ReCET System has been evaluated in feasibility clinical studies, such as REGENT-1 US, REGENT-1 Australia, and EMINENT in the Netherlands. These studies assessed the safety and efficacy of the treatment in adults with type 2 diabetes whose blood glucose levels were inadequately controlled despite using insulin and non-insulin medications. Preliminary outcomes from these studies have been presented at medical conferences globally.

The ReCET System has received FDA Breakthrough Device Designation for treating type 2 diabetes in adults inadequately controlled by glucose-lowering medications.

About Endogenex™

Endogenex, founded in partnership with Mayo Clinic, aims to revolutionize treatment options for individuals with type 2 diabetes (T2D). The company’s innovations focus on resetting the body’s metabolic signaling system by harnessing its natural regenerative capabilities to improve metabolic function. Through the development of the ReCET System and the novel application of precise, controlled, non-thermal pulsed electric fields, Endogenex is establishing a new era in T2D therapy, helping patients regain control of their blood glucose levels and slow disease progression.

For more information, please visit www.endogenex.com.

Cordella, which consists of the now PMA-approved Cordella PA Sensor and commercial Cordella™ HF System, enables comprehensive heart failure management in the home using pulmonary artery (PA) pressure, a leading indicator of congestion, and non-invasive vital sign data to improve care decisions.

Backed by definitive clinical evidence from the PROACTIVE-HF pivotal trial, the company is planning a U.S. launch of Cordella this year.

Endotronix, Inc., a privately held company dedicated to advancing the treatment of heart failure (HF) at the intersection of digital health and medtech, today announced Premarket Approval (PMA) from the U.S Food and Drug Administration (FDA) of the company’s Cordella™ Pulmonary Artery (PA) Sensor System for the treatment of New York Heart Failure (NYHA) class III heart failure patients. The Cordella platform is the first and only PA pressure-guided platform to offer comprehensive patient management using daily PA pressure and vital signs from home to guide therapeutic management and improve patient outcomes.

“This approval is very exciting and has the potential to transform care for HF patients. Endotronix’s solution provides a more complete clinical picture of the patient, so providers are able to make informed remote care decisions between office visits,” stated Dr. Liviu Klein, Section Chief of Advanced Heart Failure, Mechanical Circulatory Support, Pulmonary Hypertension, and Heart Transplant at the University of California San Francisco and national principal investigator of the PROACTIVE-HF trial. “PROACTIVE-HF demonstrated that with Cordella clinicians achieved more optimal and timely dosing of key HF medications, significantly improving outcomes. In addition, the easy-to-use platform engages patients to drive consistent daily habits and self-awareness of trends to support sustainable lifestyle changes.”

Cordella is a proactive HF management platform that delivers daily PA pressure and other vital data via an implantable sensor and user-friendly, non-invasive health tools, respectively, to a managing HF clinician for remote patient care. This information guides clinical decision-making and medication dosing while enhancing the adoption of guideline-directed medical therapy (GDMT) to reduce congestion and improve outcomes. Regulatory approval was based on the PROACTIVE-HF trial, which demonstrated a markedly low 0.159 rate of heart failure hospitalization and all-cause mortality at 6 months.

In addition, Cordella enables:

• Seated PA pressure measurements, preferred by most patients, with a handheld reader.
• Patient visibility into key health trends to support healthy lifestyle changes.
• Secure messaging on a tablet between the clinical team, patient, and caregiver to support remote care.
• Reimbursement for implantation and ongoing management through existing reimbursement pathways.

“At Endotronix, we firmly believe that innovation can drive patient care excellence in the home and ultimately change outcomes for patients with heart failure, one of the largest cost categories in healthcare. The FDA’s approval validates this foundational belief and is a major milestone for our company and the field of HF management,” commented Harry Rowland, CEO and co-founder of Endotronix. “With this approval, we will deliver proactive, comprehensive care that extends optimal HF therapeutic management to more patients, keeping them out of the hospital and living more fulfilling lives.”

Endotronix will launch Cordella in the U.S. later this year. The company also shared that they have submitted a dossier for CE Mark review and expect a decision on European market access in 2025.

About Endotronix
Endotronix innovates at the intersection of medtech and digital health to improve care for people living with heart failure (HF). The comprehensive Cordella solution enables proactive, data-driven HF management that engages patients, reduces and prevents congestion, and improves outcomes. The Cordella Sensor is an implantable pulmonary artery (PA) pressure sensor that directly measures the leading indicator of congestion, allowing early, targeted therapy. The Cordella HF System is a patient health management platform, which combines comprehensive vital sign data from non-invasive devices to support patient-clinician engagement and care decisions. Combining trended insights, the versatile and scalable Cordella enhances current clinical practice and supports guideline-based care across the entire HF continuum. Learn more at www.endotronix.com.

In the U.S., the Cordella PA Sensor System is Rx Only. CAUTION: Federal law restricts this device to sale by or on the order of a physician.
In Europe, the Cordella PA Sensor System is Exclusively for Clinical Investigation.
The Cordella HF System is commercially available in the U.S. and Europe.

Cautionary Statement Regarding Forward-Looking Statements

This press release may contain predictions, estimates, or other information that might be considered forward-looking statements. Such forward-looking statements are not a guarantee of future performance.

SOURCE Endotronix, Inc.

The Toronto-based biotech startup has the potential to reshape the way we approach pain relief.

Every 65 minutes and 22 seconds.

That’s how often someone dies from an opioid overdose in Canada. On average, it works out to about 22 people per day. By comparison, at the height of Covid-19, the death toll in 2020 was about 11 per day. All told, opioids—a class of prescription pain-relieving medications that include heroin, fentanyl, morphine, oxycontin, among others—have killed more than 42,000 Canadians since tracking began in 2016.

According to the OECD (Organization for Economic Cooperation and Development), Canada holds the dubious distinction of having the second highest rate of opioid–related deaths, just behind the U.S.

In the face of such brutal numbers, it’s clear that combatting the opioid epidemic requires an all-hands-on-deck approach, integrating everything from health care providers and emergency services to all levels of government. A pair of scientists-turned-startup-entrepreneurs, however, believe that the private sector, specifically their emerging biotech company, can play a critical part in solving this widespread and devastating issue.

Mike Cooke and Molly Shoichet are the co-founders of AmacaThera, a Toronto-based venture offering a novel way to provide pain relief without the use of opioids. Their breakthrough product, which is currently in clinical trials, was spun out of research led by Shoichet from her lab at the University of Toronto, where she’s a professor in the Department of Chemical Engineering and Applied Chemistry. Cooke, who hails from the U.K., worked in the lab studying stem cells while pursuing his PhD. In 2016, the two joined forces (she as chief science officer; he as CEO) with a vision, Cooke says, of “translating their academic research into real-world products” that have the potential to herald a promising avenue for long-term opioid reduction strategies.

The FedEx of therapeutic delivery

At the core of the company’s technology is its proprietary hydrogel platform. The AmacaGel, as it’s known, is a blend of hyaluronic acid (the same ingredient commonly used in anti-aging cosmetics), and methyl cellulose which, when combined with the hyaluronic acid, gives the material its gel consistency. When injected by syringe into a patient’s soft tissue, the gel acts as a kind of carrier system for existing drugs like an anesthetic, or other therapeutics that have been mixed with it, allowing their gradual release into the body.

Shoichet likens AmacaThera to FedEx, whereby it provides the protective packaging (the gel) for what’s inside (the drugs) and then determines where and when they need to be deployed. “We’re not changing the drugs, we’re changing the delivery method,” adds Cooke. “That’s why we call it a platform.”

How it works: a game-changer in pain management

To understand what sets AmacaThera’s technology apart as it relates to the opioid epidemic, is to understand why opioids are often prescribed in the first place. Anyone that’s undergone surgery—from major dental work to having a hip replaced—knows that the recovery period can be painful. During the procedure, an analgesic is typically administered to the area, but tends to wear off within hours, leaving most patients to rely on a prescription narcotic for relief. Studies have shown that this leads to millions of cases of opioid addiction each year.

In contrast, the AmacaGel platform’s controlled release mechanism would ensure a steady supply of opioid-free pain medication over three days, effectively numbing the surgical site for an extended duration, far beyond the 12 to 18 hour window of conventional local anesthetics. Cooke and Shoichet theorize this prolonged-release formulation not only obviates the need for frequent opioid administration, but also alleviates the intensity of postoperative pain, thereby safeguarding individuals from the perils of addiction and overdose. It’s against this backdrop that the company offers a lifeline in the battle against opioid misuse.

“It’s been so exciting to see our research invention of AmacaGel be the basis of AmacaThera and now to see it applied in people,” Shoichet says. “Our goal is to make products that will make a difference in people’s lives. With our first product, we aim to both alleviate pain and put a dent in the opioid crisis, thereby filling unmet medical and societal needs.”

“Our goal is to make products that will make a difference in people’s lives. With our first product, we aim to both alleviate pain and put a dent in the opioid crisis, thereby filling unmet medical and societal needs.”

Moreover, Cooke explains, its efficacy in controlling pain not only fosters optimal healing, but the downstream effect may be a boon to our struggling health system. “If you have that rapid pain control, you’ll get better recovery. You can get people out of the hospital quicker and by reducing their length of stay, you can increase hospital capacity, and serve more patients. These are huge drivers and we’re building the company with that mentality of efficacy and efficiency.”

Beyond the operating room: exploring diverse applications

While the company’s first formulation, AMT-143, is focused on post-surgical pain management, Cooke and Shoichet are betting that AmacaThera’s utility could extend far beyond the confines of the operating room. Consider, for example, the ability to deliver cancer-fighting treatment to otherwise difficult-to-reach areas of the body, like the lungs or the brain. “With this technology, you could inject chemotherapy right into the tumour,” Cooke explains. “Because it’s localized, you could have greater efficacy because more of the drug would infiltrate the tumour directly, so that you’re decreasing the systemic toxicity overall.”

That AmacaThera’s platform is compatible with other materials—small molecules, antibodies, stem cells, proteins, and peptides, to name a few—effectively means patient outcomes could be improved across multiple therapeutic areas. A wide swath of rigorous scientific research has come out of Shoichet’s lab (underscored by over 325 peer-reviewed publications) shows how the technology could be used to treat not just certain cancers, but blindness, spinal cord injuries, and strokes.

Further still, when one considers the possibility of extending the product to patients of the non-human variety (think: dogs and cats, but also farm animals), its versatility could make it a cornerstone in various medical disciplines. “People pay a lot of money to care for their pets,” Cooke points out, “so there are active discussions with animal health pharma companies looking at licensing this technology.”

In this landscape, AmacaThera emerges as a revolutionary solution, offering a paradigm shift in the range of patient treatment options available, while holding the promise of mitigating some of society’s biggest challenges. “I believe in this technology because I’ve seen it work with my own eyes,” says Cooke. “As we learn more about the product itself, the manufacturing, the usages, how it performs as we advance to human trials, and the more positive aspects we’re uncovering, the more exciting it gets.”

Given the potential market opportunity (surgery alone is performed on over 51 million Americans every year, with opioids the primary means of pain control), AmacaThera has, unsurprisingly, created alot of excitement among investors as well. Last November, the company announced it secured a $4 million Series A extension from investors, including Lumira Ventures and StandUp Ventures, both of which RBCx proudly backs as a Limited Partner.

RBCx also supports the young startup with its day-to-day banking needs. “We’ve been with RBCx since the very start and it just makes life a lot simpler and more efficient. The fewer headaches I have, the more time we can devote to focusing on the science,” Cooke says, adding that the opportunity to network at RBCx-hosted client events with our thought leaders is a “big deal…I see that you’ve hired a few people from Silicon Valley Bank, so it will be exciting to see what RBCx does in the future.”

And yet, despite banking support, deep-pocketed investors, a best-in-class product, and Shoichet’s own prior experience launching two other startups, the co-founders acknowledge that entrepreneurship has been anything but easy. “When you start on this journey, you don’t know how hard this is going to be; it takes a certain type of crazy to want to do this because raising capital has been by far the toughest part,” says Cooke.

“When you start on this entrepreneurial journey, you don’t know how hard this is going to be. It takes a certain type of crazy to want to do this because raising capital has been by far the toughest part.”

“The number one comment we’ve had from investors is congratulations on being so relentless. They’re amazed by the tenacity of this company and how it just keeps going,” says Cooke. “And that’s the key: we’re going to take this all the way to the finish line.”

To get there, AmacaThera is using the cash infusion to speed up testing and development of AMT-143, as well as advancing other formulations through their pipeline before going out to fundraise again later this year. And while they’re the first to acknowledge that Canada remains a tough economic environment for tech startups, the duo remain undaunted, driven by a profound commitment to their mission. “In 20 years, we envision a successful company with products that make lives better,” says Shoichet. “There’s nothing more exciting than working toward creating a better tomorrow with innovative ideas.”

“Getting the product in humans and moving it forward is such a huge privilege,” adds Cooke. “We’re privileged to have the investors, we’re privileged to have the team. I never want to lose sight of that. It would be great to have invented something going into potentially millions and millions of people. But, to be honest, I think if we’re able to improve even one person’s life, that would be a huge achievement.”

COUR Pharmaceuticals, a clinical-stage biotechnology company focused on the development of first-in-class, disease-modifying therapies designed to induce antigen-specific tolerance for immune-mediated diseases, today announced the appointment of Paul M. Peloso, MD, a seasoned drug developer with more than 20 years of experience advancing multiple therapeutic candidates through market approval, as chief medical officer.

Prior to joining COUR, Dr. Peloso served as Chief Medical Officer for ACELYRIN, Inc., a biopharma company focused on providing life-changing new treatment options for patients by employing a strategy of identifying, acquiring and accelerating development and commercialization of promising drug candidates. While at ACELYRIN, Dr. Peloso was responsible for global clinical development of the company’s pipeline of products, with an initial focus in immunology.

Before ACELYRIN, Dr. Peloso was vice president and therapeutic head for rheumatology of Horizon Therapeutics, where he was responsible for advancing the company’s pipeline of first-in-class rare disease medicines including, notably KRYSTEXXA^® until its acquisition by Amgen in 2023. Prior to this role, Dr. Peloso served as group medical director for both AbbVie and Merck, where he leveraged his extensive experience in biologics, small molecules, and medical devices.

Among Dr. Peloso’s global clinical and regulatory achievements across his career, he helped develop and ultimately secure approvals for ORILISSA^® (elagolix), ILUMYA™ (tildrakizumab), Arcoxia^® (etoricoxib), and Enbrel^® (etanercept). Dr. Peloso has also led a broad range of successful filings in numerous therapeutic areas that have led to approvals by the U.S. Food and Drug Administration, European Medicines Agency and in the Asian and Latin American markets.

“We are delighted to welcome Dr. Peloso during this time of significant growth at COUR,” said John J. Puisis, founder, president, and chief executive officer of COUR. “The addition of Dr. Peloso to our management team comes on the heels of our successful fundraising earlier this year, which will allow us to further develop our clinical-stage pipeline as we advance into Phase 2 clinical studies with our lead program in Myasthenia Gravis as well as advance our Type 1 Diabetes program into the clinic later this year. We look forward to benefiting from Dr. Peloso’s significant expertise in late-stage clinical development and regulatory approval as we aim to bring transformative solutions to patients in need. I would also like to take this opportunity to express a deep sense of gratitude to Dr. Roy First, who has been COUR’s acting CMO for the past five years. Working closely with the company, Dr. First has been invaluable to COUR’s present success.”

Dr. Peloso added, “I am excited to be joining COUR as the company moves forward into late-stage development. Given the tremendous promise already demonstrated by their nanoparticle technology platform in earlier studies and across other indications, I’m enthusiastic to be at the forefront of a truly innovative solution for patients with a range of autoimmune diseases with key unmet medical needs. My experience in bringing new medicines from bench to bedside will complement an existing strong leadership team at COUR.”

Prior to entering the industry, Dr. Peloso was in academic practice in rheumatology in the U.S. and Canada. He holds two bachelor’s degrees, one in biochemistry and another in sociology, from McMaster University and earned his medical degree from the University of Calgary before training in rheumatology and epidemiology.

About COUR Pharmaceuticals:

COUR Pharmaceuticals is a clinical-stage biotechnology company developing therapies to treat patients with autoimmune and inflammatory diseases. COUR’s first-in-class therapies are based on our proprietary antigen-specific immune tolerance platform and are designed to reprogram the immune system to address the underlying root cause of immune-mediated diseases. Data from multiple clinical and preclinical programs have demonstrated the ability of COUR’s product candidates to induce antigen-specific immune tolerance and have the potential to treat a wide range of autoimmune and inflammatory diseases.

COUR is developing product candidates in Myasthenia Gravis and Type 1 Diabetes in addition to having partnered products in Celiac Disease (with Takeda Pharmaceuticals), and Primary Biliary Cholangitis (with Ironwood Pharmaceuticals).

For more information, please visit www.courpharma.com

Contacts
For Investor Relations
Brian Bock, Chief Financial Officer
bbock@courpharma.com

For Media
Owen Blaschak
oblaschak@lifescicomms.com

AmacaThera Inc, a clinical stage biotechnology company specializing in the development of advanced sustained release hydrogel formulations, has dosed its first human subject with AMT-143, the company’s lead asset in non-opioid acute pain management.     

AMT-143 is a local slow-release non-opioid local anesthetic that would leverage the AmacaGel platform to provide long-acting post-operative pain relief and is the first therapeutic product formulated to be delivered via AmacaGel, the Company’s patented technology platform.

Previously, the Company had tested the AmacaGel platform without any active pharmaceutical agent.  This trial will test the platform combined with a known anesthetic.  The study will assess the tolerability, safety, pharmacokinetic and clinical activity of the AmacaGel platform with a known compound that has a long history of safety and efficacy.

If approved, AMT-143 could one day be used to reduce post-operative pain without the need for opioids. AMT-143 would be applied in the incisional site at the time of surgery to control local pain.

“A successful outcome will be data demonstrating safety of our asset, and release kinetics showing a sustained release longer than the competitive products,” said Dr. Mike Cooke, Chief Executive Officer, and Co-Founder of AmacaThera, “There is a real need for products that provide longer relief than those currently available on the market.”

Dr. Molly Shoichet is the Company’s Chief Scientific Officer and Co-Founder of AmacaThera. The original technology was developed in her laboratory at the University of Toronto. “My laboratory has demonstrated compatibility of AmacaGel with a wide range of therapeutics – from cells to biologics,” said Shoichet, “With this study, our goal is to provide human data on our technology that will help advance AMT-143 to the clinic. At the same time, we are building out the platform technology so that it can be leveraged for other local-release therapeutics.”

About AmacaThera

AmacaThera is a clinical stage biotechnology company specializing in the development of advanced sustained release hydrogel formulations to solve key therapeutics delivery challenges with both off-patent and proprietary payloads.  AmacaThera is focused on developing an internal pipeline of pain management and oncology assets; and partnering to solve key delivery challenges with advanced proprietary therapeutic modalities.

AmacaThera’s platform technology, AmacaGel, is a fast-gelling physical hydrogel blend of two well-established medical-grade polymers.  It has been designed to liquify under shear force to be delivered by a conventional syringe, rapidly forming a depot at the injection site as it warms to body temperature. AMT-143, the platform’s lead asset, is a slow-release non-opioid local anesthetic that leverages the AmacaGel platform to provide long-acting post-operative pain relief.

For more information, visit www.amacathera.ca

SOURCE AmacaThera Inc.

For further information: Media Contact: Abhaya Khulbe, Investor Relations, info@amacathera.ca

X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that it has completed the sale of its Rare Pediatric Disease Priority Review Voucher (PRV) to an undisclosed purchaser for $105 million and that it has drawn an additional tranche of $20 million under its existing loan facility with Hercules Capital, Inc. (NYSE: HTGC). Both transactions result from the U.S. Food and Drug Administration (FDA) approval of the company’s first product, XOLREMDI™ (mavorixafor), in late April.

“This infusion of non-dilutive capital provided by these transactions extends our projected cash runway into late 2025, excluding expected commercial product sales, and provides us with additional financial strength and flexibility as we execute on the XOLREMDI launch in WHIM syndrome and continue to advance mavorixafor into a potential second indication, with the planned initiation of a Phase 3 trial this quarter in certain chronic neutropenic disorders,” said Adam Mostafa, Chief Financial Officer of X4 Pharmaceuticals.

Under the Rare Pediatric Disease program, the FDA awards PRVs to sponsors of rare pediatric disease product applications that meet certain criteria to encourage development of new drugs and biologics for the prevention and treatment of rare pediatric diseases. The term loan facility with Hercules Capital provides for up to $115 million of term loans in the aggregate, available to be funded in multiple tranches, and is in an interest-only period until July 2027. The $105 million of gross funds received from the sale of the PRV and the $20 million drawn from the existing loan facility add to the $82 million in cash, cash equivalents, restricted cash, and short-term marketable securities reported as of March 31, 2024.

About WHIM Syndrome and XOLREMDI™ (mavorixafor)
WHIM (warts, hypogammaglobulinemia, infections and myelokathexis) syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder; people with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections. XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U.S. for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.

About X4 Pharmaceuticals
X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI™ (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding X4’s plans with respect to the use of the PRV sale proceeds and the drawdown from the loan facility with Hercules; X4’s expected cash runway with the PRV sale proceeds and the drawdown from the loan facility with Hercules and its existing cash balance as of March 31, 2024; X4’s commercialization plans and ongoing efforts with respect to XOLREMDI and the expected timing thereof; X4’s continued advancement of mavorixafor into a second indication, including timing of the initiation of a Phase 3 clinical trial in the second quarter of 2024 in certain chronic neutropenic disorders; and other statements regarding X4’s future operations, financial performance, financial position, prospectus, objectives and other future events. Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: unanticipated costs and expenses may be greater than anticipated; the company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; delays, interruptions or failures in the manufacture and supply of our products; the company’s ability to obtain additional funding to support its clinical development and commercial programs; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on May 7, 2024, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.

Company Contact:
José Juves
Head of Corporate & Patient Affairs
jose.juves@x4pharma.com

Investor Contact:
Daniel Ferry
Managing Director, LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576

X4 Pharmaceuticals (Nasdaq: XFOR), a company driven to improve the lives of people with rare diseases of the immune system, today announced that the U.S. Food and Drug Administration (FDA) has approved XOLREMDI™ (mavorixafor) capsules for use in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.

XOLREMDI, a selective CXC chemokine receptor 4 (CXCR4) antagonist, is the first therapy specifically indicated in patients with WHIM syndrome, a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 pathway dysfunction. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia) and experience serious and/or frequent infections. The FDA granted Breakthrough Therapy Designation to mavorixafor in WHIM syndrome and evaluated the New Drug Application (NDA) under Priority Review, a designation for therapies that have the potential to provide significant improvement in the treatment, diagnosis, or prevention of serious conditions.

“The approval of XOLREMDI is a transformational milestone both for X4 and, more importantly, for the WHIM syndrome community,” said Paula Ragan, Ph.D., President and Chief Executive Officer of X4 Pharmaceuticals. “We are incredibly grateful to the people living with WHIM syndrome, their families, and the investigators who took part in our clinical program, to U.S. regulators for their continued focus on rare-disease treatment development, and to our dedicated employees for making this targeted breakthrough therapy a reality.”

“Effective and innovative treatments are critical for those diagnosed with a primary immunodeficiency. The approval of XOLREMDI marks an important advancement for people living with WHIM syndrome, who are susceptible to serious and frequent infections,” said Jorey Berry, President and Chief Executive Officer of the Immune Deficiency Foundation (IDF). “We are very pleased to have been a partner to X4 in their journey to bring this much-needed treatment to this underserved rare disease community.”

Teresa K. Tarrant, M.D., Associate Professor of Medicine, Rheumatology, and Immunology at Duke University School of Medicine and a principal investigator in the 4WHIM trial, commented on the news: “Until now, supportive care for people with WHIM syndrome has focused on symptom management and not the underlying cause of disease — the dysfunction of the CXCR4 pathway. I am thrilled that with the approval of XOLREMDI, a therapy designed to address dysregulated CXCR4 pathway signaling, we now have a targeted treatment that has demonstrated the ability to elevate absolute neutrophil and lymphocyte counts, increasing WHIM patients’ ability to fight infections.”

The FDA approval of XOLREMDI was based on results of the pivotal, 4WHIM Phase 3 clinical trial, a global, randomized, double-blind, placebo-controlled, 52-week multicenter study that evaluated the efficacy and safety of XOLREMDI in 31 people aged 12 years and older diagnosed with WHIM syndrome. The efficacy of XOLREMDI was determined by improvement in absolute neutrophil counts (ANC), improvement in absolute lymphocyte counts (ALC), and a reduction in infections. In the 4WHIM trial, XOLREMDI treatment demonstrated increased time above threshold (≥500 cells/microliter) for absolute neutrophil count (TAT-ANC) vs. placebo (p<0.0001) and increased time above threshold (≥1000 cells/microliter) for absolute lymphocyte count (TAT-ALC) v. placebo (p<0.0001). The efficacy of XOLREMDI was further assessed in a composite endpoint consisting of total infection score and total wart change score using a Win-Ratio method. Analyses of the individual components of this composite endpoint showed an approximate 40% reduction in total infection score, weighted by infection severity, in XOLREMDI-treated patients compared with placebo-treated patients. There was no difference in total wart change scores between the XOLREMDI and placebo treatment arms over the 52-week period. Treatment with XOLREMDI also resulted in a 60% reduction in the annualized infection rate compared with placebo-treated patients. The most common adverse reactions reported in the 4WHIM trial (≥10% and more frequently reported than placebo) were: thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.

With the FDA approval of XOLREMDI, X4 has received a Rare Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent application or sold to another drug sponsor.

X4Connect™ Offers Disease and Treatment-Related Support
X4 is committed to helping people with WHIM syndrome access XOLREMDI and announced today the launch of X4Connect, offering eligible U.S. patients dedicated support throughout their XOLREMDI treatment journey, including disease and treatment-related resources, help navigating insurance coverage, and copay assistance. For additional information about X4Connect, call 1-844-X4CNNCT (844-942-6628), Monday-Friday, 8am-8pm ET or visit https://www.xolremdihcp.com/access-and-support.

XOLREMDI will be commercially available in the U.S. through X4’s specialty pharmacy partner PANTHERx^® Rare.

Conference Call and Webcast
The company will host a conference call and webcast today at 8:30 am ET. The conference call can be accessed by dialing 1-877-451-6152 from the United States or 1-201-389-0879 internationally, followed by the conference ID: 13746357. The live webcast will be accessible through the investor relations section of X4 Pharmaceuticals’ website at www.x4pharma.com. Following the completion of the call, a webcast replay will be available on the website.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION

XOLREMDI is contraindicated with drugs highly dependent on CYP2D6 for clearance.

WARNINGS AND PRECAUTIONS

Embryo-Fetal Toxicity: Based on its mechanism of action, XOLREMDI is expected to cause fetal harm. Verify pregnancy status of female patients of reproductive potential prior to starting XOLREMDI. Advise females of reproductive potential to use effective contraception during treatment with XOLREMDI and for three weeks after the final dose.

QTc Interval Prolongation: XOLREMDI causes concentration-dependent QTc prolongation. Correct any modifiable risk factors for QTc prolongation, assess QTc at baseline, and monitor QTc during treatment as clinically indicated in patients with risk factors for QTc prolongation or receiving concomitant medications that increase XOLREMDI exposure and/or drugs with a known potential to prolong the QTc interval. Dose reduction or discontinuation of XOLREMDI may be required.

ADVERSE REACTIONS

The most common adverse reactions (in ≥10% patients and more frequently reported than placebo) were thrombocytopenia, pityriasis, rash, rhinitis, epistaxis, vomiting, and dizziness.

DRUG-DRUG INTERACTIONS

Avoid co-administration of XOLREMDI and strong CYP3A4 inducers. Reduce XOLREMDI daily dosage when administered with strong CYP3A4 inhibitors. Monitor more frequently for adverse reactions associated with an increase in exposure of XOLREMDI when used concomitantly with moderate CYP3A4 inhibitors or P-gp inhibitors and reduce XOLREMDI daily dosage if necessary.

USE IN SPECIFIC POPULATIONS

• Advise females that breastfeeding is not recommended during treatment with XOLREMDI and for three weeks after the final dose.
• The safety and effectiveness of XOLREMDI have not been established in pediatric patients younger than 12 years of age.
• XOLREMDI is not recommended in patients with severe renal impairment, end-stage renal disease, or moderate to severe hepatic impairment.

To report suspected adverse reactions, contact X4 Pharmaceuticals at 1-866-MED-X4MI (1-866-633-9464) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see the full Prescribing Information for XOLREMDI.

About WHIM Syndrome
WHIM syndrome is a rare, combined primary immunodeficiency and chronic neutropenic disorder caused by CXCR4 receptor dysfunction that results in impaired mobilization of white blood cells from the bone marrow into peripheral circulation. WHIM syndrome is named for its four classic manifestations: warts, hypogammaglobulinemia, infections, and myelokathexis, although only a minority of patients experience all four manifestations in the acronym. People with WHIM syndrome characteristically have low blood levels of neutrophils (neutropenia) and lymphocytes (lymphopenia), and as a result, experience serious and/or frequent infections. It is estimated that at least 1,000 people are currently diagnosed with WHIM syndrome in the U.S.

About XOLREMDI™ (mavorixafor)
XOLREMDI (mavorixafor) is a selective CXCR4 receptor antagonist approved in the U.S. for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. CXCR4 receptor stimulation by its ligand, CXCL12, has been shown to play a key role in the movement of white blood cells (leukocytes) to and from the bone marrow compartment. Treatment with XOLREMDI results in increased mobilization of neutrophils and lymphocytes from the bone marrow into peripheral circulation.

About X4 Pharmaceuticals
X4 is delivering progress for patients by developing and commercializing innovative therapies for those with rare diseases of the immune system and significant unmet needs. Leveraging our expertise in CXCR4 and immune system biology, we have successfully developed mavorixafor, which has received U.S. approval as XOLREMDI™ (mavorixafor) capsules in its first indication. We are also evaluating the use of mavorixafor in additional potential indications. X4 corporate headquarters are in Boston, Massachusetts and our research center of excellence is in Vienna, Austria. For more information, please visit our website at www.x4pharma.com.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of applicable securities laws, including the Private Securities Litigation Reform Act of 1995, as amended. These statements may be identified by the words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” or other similar terms or expressions that concern X4’s expectations, strategy, plans, or intentions. Forward-looking statements include, without limitation, implied or express statements regarding X4’s expectations as to the timing of commencement of planned launch, availability, and commercialization of XOLREMDI, which is approved in the U.S. for use in patients 12 years of age and older with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes; X4’s plans for commercial launch of XOLREMDI in this indication, including its planned commercial launch in the U.S. through PANTHERx Rare; X4’s belief in its readiness for commercial launch of XOLREMDI; the potential benefit of XOLREMDI in the indicated patient population; the potential number of patients with WHIM syndrome and the potential market for XOLREMDI; the anticipated timing for completion of commercial drug product manufacturing; and the mission and goals for our business.

Any forward-looking statements in this press release are based on management’s current expectations and beliefs. These forward-looking statements are neither promises nor guarantees of future performance, and are subject to a variety of risks and uncertainties, many of which are beyond X4’s control, which could cause actual results to differ materially from those contemplated in these forward-looking statements, including the risks that: X4’s launch and commercialization efforts in the U.S. with respect to XOLREMDI may not be successful, and X4 may be unable to generate revenues at the levels or on the timing we expect or at levels or on the timing necessary to support our goals; the number of patients with WHIM syndrome, the unmet need for additional treatment options, and the potential market for XOLREMDI may be significantly smaller than we expect; XOLREMDI may not achieve the clinical benefit, clinical use, or market acceptance we expect or we may encounter reimbursement-related or other market-related issues that impact the success of our commercialization efforts; we may encounter adverse events for XOLREMDI at any stage that negatively impact commercialization; the need to align with our collaborators may hamper or delay our development and commercialization efforts or increase our costs; our business may be adversely affected and our costs may increase if any of our key collaborators fails to perform its obligations or terminates our collaboration; the internal and external costs required for our ongoing and planned activities, and the resulting impact on expense and use of cash, may be higher than expected, which may cause us to use cash more quickly than we expect or to change or curtail some of our plans or both; and other risks and uncertainties, including those described in the section entitled “Risk Factors” in X4’s Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) on March 21, 2024, and in other filings X4 makes with the SEC from time to time. X4 undertakes no obligation to update the information contained in this press release to reflect new events or circumstances, except as required by law.

Investor Contact:
Daniel Ferry
Managing Director, LifeSci Advisors
daniel@lifesciadvisors.com
(617) 430-7576

Media Contact:
Kit Rodophele
Account Director, Ten Bridge Communications
krodophele@tenbridgecommunications.com

TORONTO, ON / ACCESSWIRE / APRIL 2, 2024 / Lumira Ventures is delighted to announce its latest investment in Nocion Therapeutics, a clinical-stage biopharmaceutical company developing first and potentially best-in-class, small molecule charged sodium channel blockers (CSCBs), called Nocions. These innovative compounds are designed to selectively silence activated nociceptors for the treatment of serious conditions involving cough, itch, and pain. Proceeds from the financing will be used to evaluate the lead program, Taplucanium Dry Powder for Inhalation, in a Phase 2b study in Chronic Cough patients.

“With a highly differentiated mechanism of action delivered via a targeted approach, we are extremely excited about Taplucanium as a potential new treatment modality for refractory chronic cough patients with very limited options today.” Said Lu Han, Ph.D., Partner, Lumira Ventures.

The Series B financing was led by Arkin Bio Capital and Monograph Capital, with participation from additional new investors as well as existing investors. Lumira Ventures is proud to support Nocion Therapeutics alongside this distinguished group of syndicates including a strategic investor with deep domain expertise in inhaled respiratory therapeutic drug development.

Benjamin “Beni” Rovinski, Ph.D., Managing Director at Lumira Ventures added, “With this financing, and together with a broad syndicate of sophisticated investors, we look forward to supporting a strong management team with deep domain expertise in the respiratory field to develop innovative treatments targeting major unmet needs.”

In conjunction with the Series B investment, Lu Han, Ph.D., of Lumira Ventures will join Nocion’s Board as an observer.

“We are excited to have Lumira join our Series B syndicate. Their participation, along with the rest of the syndicate, closes our Series B raise of $70M” said Rick Batycky, Ph.D., CEO of Nocion Therapeutics. “This robust raise will allow for a comprehensive evaluation of Taplucanium in Chronic Cough patients, and completion of additional activities to advance this program to Phase 3 readiness, as we diligently work to get this potentially transformative treatment to patient in a timely fashion.”

For further information, please contact:

Rick Batycky

rick@nociontx.com

About Lumira Ventures:

Founded in 2007, Lumira Ventures is a multi-stage life sciences investment firm with offices in Toronto, Montréal, Vancouver, and Boston. We partner with mission-driven entrepreneurs building companies that harness rapidly evolving advancements in medicine to develop transformative products for patients while delivering strong financial returns for our investors and meaningful economic value and impact for society. For more information, visit www.lumiraventures.com

About Nocion Therapeutics:

Nocion Therapeutics is a biopharmaceutical company developing novel small molecule CSCBs, “nocions,” that selectively affect actively firing nociceptors for the treatment of serious conditions involving cough, itch, and pain. The company’s mission is to safely alleviate suffering for millions of patients with conditions arising from activated sensory neurons. Working with Harvard’s Office of Technology Development, Nocion was founded on an exclusive license to foundational intellectual property from Harvard University and Boston Children’s Hospital. For more information, visit: www.nociontx.com.

HAMILTON, ON and BOSTON, March 19, 2024 /PRNewswire/ — Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radioconjugates (RCs) as precision medicines, today announced the Company has entered into a definitive agreement to be acquired by AstraZeneca. The acquisition marks a major step forward in AstraZeneca delivering on its ambition to transform cancer treatment and outcomes for patients by replacing traditional regimens like chemotherapy and radiotherapy with more targeted treatments.

RCs have emerged as a promising modality in cancer treatment over recent years. These medicines deliver a radioactive isotope directly to cancer cells through precise targeting using molecules such as antibodies, peptides or small molecules. This approach has many potential advantages compared to traditional radiotherapy, including minimizing damage to healthy cells and enabling access to tumors not reachable through external beam radiation.

This acquisition complements AstraZeneca’s leading oncology portfolio with the addition of the Fusion pipeline of RCs, including the Company’s most advanced program, FPI-2265, a potential new treatment for patients with metastatic castration-resistant prostate cancer (mCRPC). FPI-2265 targets prostate-specific membrane antigen (PSMA), a protein that is highly expressed in mCRPC, and is currently in a Phase 2 trial.

The acquisition brings new expertise and pioneering R&D, manufacturing and supply chain capabilities in actinium-based RCs to AstraZeneca. It also strengthens their presence in and commitment to Canada.

Fusion Chief Executive Officer John Valliant, Ph.D., said: “This acquisition combines Fusion’s expertise and capabilities in radioconjugates, including our industry-leading radiopharmaceutical R&D, pipeline, manufacturing and actinium-225 supply chain, with AstraZeneca’s leadership in small molecules and biologics engineering to develop novel radioconjugates. Expanding on our existing collaboration with AstraZeneca where we have advanced FPI-2068, an EGFR-cMET targeted radioconjugate into Phase I clinical trials, gives us a unique opportunity to accelerate the development of next-generation radioconjugates with the aim of transforming patient outcomes.”

Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “Between thirty and fifty per cent of patients with cancer today receive radiotherapy at some point during treatment, and the acquisition of Fusion furthers our ambition to transform this aspect of care with next-generation radioconjugates. Together with Fusion, we have an opportunity to accelerate the development of FPI-2265 as a potential new treatment for prostate cancer, and to harness their innovative actinium-based platform to develop radioconjugates as foundational regimens.”

Fusion President and Chief Business Officer Mohit Rawat, said: “Fusion has differentiated itself in the growing radioconjugate space by assembling an industry-leading team with deep expertise and infrastructure to support bringing these much-needed therapies to cancer patients. Together we look forward to building upon our work to impact the landscape of cancer therapy. Deepening our collaboration with AstraZeneca presents an exciting opportunity for the Fusion team.”

Fusion will become a wholly owned subsidiary of AstraZeneca, with operations continuing in Canada and the US.

Financial Considerations

Under the terms of the definitive agreement, AstraZeneca, through a subsidiary, will acquire all of Fusion’s outstanding shares pursuant to a plan of arrangement for a price of $21.00 per share in cash at closing plus a non-transferable contingent value right (CVR) of $3.00 per share in cash payable upon the achievement of a specified regulatory milestone.

The upfront cash portion of the consideration represents a transaction value of approximately $2 billion, a 97% premium to Fusion’s closing market price of $10.64 on March 18, 2024, and an 85% premium to the 30-day volume-weighted average price (VWAP) of $11.37 before this announcement. Combined, the upfront and maximum potential contingent value payments represent, if achieved, a transaction value of approximately $2.4 billion, a 126% premium to Fusion’s closing market price on March 18, 2024, and a 111% premium to the 30-day VWAP. As part of the transaction, AstraZeneca will acquire the cash, cash equivalents and short-term investments on Fusion’s balance sheet, which totaled $234 million as of December 31, 2023.

The proposed acquisition of Fusion is to be completed by way of a statutory plan of arrangement under the Canada Business Corporations Act and subject to customary closing conditions, including approval of (i) 66⅔% of the votes cast by Fusion shareholders and (ii) a simple majority of the votes cast by Fusion shareholders (excluding certain persons required to be excluded in accordance with Multilateral Instrument 61-101 of the Canadian Securities Administrators), in each case, at a special meeting of Fusion shareholders. The transaction is expected to close in the second quarter of 2024, subject to customary closing conditions, including the approval of Fusion shareholders and regulatory clearances, as noted above.

Centerview Partners LLC is serving as exclusive financial advisor to Fusion and Goodwin Procter LLP is serving as legal counsel, with Osler, Hoskin & Harcourt LLP serving as Canadian legal counsel.

Radioconjugates in oncology
RCs combine the precise targeting of antibodies, small molecules or peptides with potent medical radioisotopes to deliver radiation directly to cancer cells. By seeking out cancer cells, RCs provide a more precise mechanism of cancer cell killing compared with traditional radiation therapy, with the goal of improving efficacy while minimizing toxicity on healthy cells. RCs are administered via systemic delivery, which enables their use in tumor types not accessible to external beam radiation and the targeting of cancer cells that have spread from the main tumor to other sites in the body.

About FPI-2265
FPI-2265 is an actinium-225 based PSMA targeting RC, for mCRPC, currently in a Phase II trial. Actinium-225 emits alpha particles and holds the promise of being a next-generation radioisotope in cancer treatment. By delivering a greater radiation dose over a shorter distance, alpha particles such as actinium-225 have the potential for more potent cancer cell killing, and targeted delivery, thereby minimizing damage to surrounding healthy tissue.

About Fusion 
Fusion Pharmaceuticals is a clinical-stage oncology company focused on developing next-generation RCs. Fusion connects alpha particle emitting isotopes to various targeting molecules in order to selectively deliver the alpha emitting payloads to tumors. Fusion’s clinical-stage development portfolio includes lead program, FPI-2265, targeting PSMA for mCRPC and novel RCs targeting solid tumors.

Cautionary Note Regarding Forward-Looking Statements

To the extent any statements made in this communication contain information that is not historical, these statements are forward-looking statements within the meaning of Section 27A of the U.S. Securities Act of 1933, as amended, and Section 21E of the U.S. Securities Exchange Act of 1934, as amended, and forward-looking information under Canadian securities law (collectively, “forward-looking statements”). Certain statements in this communication may constitute forward-looking statements, which reflect the expectations of Fusion’s management regarding the business prospects and opportunities of Fusion and the proposed transaction. The use of words such as “may,” “will,” “could,” “should,” “expects,” “intends,” “plans,” “anticipates,” “believes,” “estimates,” “predicts,” “projects,” “seeks,” “endeavor,” “potential,” “continue” or the negative of such words or other similar expressions can be used to identify forward-looking statements. More particularly and without limitation, this communication contains forward-looking statements and information regarding whether the proposed transaction will be completed, whether the CVR Agreement will be entered into, the anticipated benefits of the proposed transaction for Fusion and its Shareholders, whether approval will be received under the U.S. Hart-Scott-Rodino Antitrust Improvements Act of 1976 and the Competition Act (Canada), each as amended, and whether the milestone under the CVR Agreement will be achieved.

Fusion’s actual results could differ materially from those stated or implied in forward-looking statements due to a number of factors including but not limited to risks related to the satisfaction or waiver of the conditions to closing the proposed transaction (including the failure to obtain necessary regulatory, court and Fusion’s shareholder approvals) in the anticipated timeframe or at all, including the possibility that the proposed transaction does not close; the response of business partners and competitors to the announcement of the proposed transaction, and/or potential difficulties in employee retention as a result of the announcement and pendency of the proposed transaction; significant transaction costs; the failure to realize the expected benefits of the proposed transaction; risks associated with the disruption of management’s attention from ongoing business operations due to the proposed transaction; and unknown liabilities and the risk of litigation and/or regulatory actions related to the proposed transaction. Please also refer to the factors discussed under “Risk Factors” and “Special Note Regarding Forward-looking Information” in Fusion’s Annual Report on Form 10-K for the year ended December 31, 2022, with the U.S. Securities Exchange Commission (“SEC”), each as updated by Fusion’s continuous disclosure filings, which are available at www.sec.gov and at www.sedarplus.com.

Forward-looking statements involve significant risks and uncertainties, should not be read as guarantees of future performance or results, and will not necessarily be accurate indications of whether or not or the times at or by which such performance or results will be achieved. All forward-looking statements herein are qualified in their entirety by its cautionary statement and are made as of the date of this document. Fusion disclaims any obligation to revise or update any such forward-looking statements or to publicly announce the result of any revisions to any of the forward-looking statements contained herein to reflect future results, events or developments, except as required by law.

Additional Information about the Proposed Transaction and Where to Find It

This communication is not intended to and does not constitute an offer to sell or the solicitation of an offer to subscribe for or buy or an invitation to purchase or subscribe for any securities or the solicitation of any vote, consent or approval in any jurisdiction, nor shall there by any sale, issuance or transfer of securities in any jurisdiction in contravention of applicable law. This communication has been prepared in respect of the transaction involving Fusion, AstraZeneca and Purchaser (as defined in the Arrangement Agreement) pursuant to the terms of the Arrangement Agreement, and may be deemed to be soliciting material relating to the transaction. In connection with the transaction, Fusion will file a management information circular and proxy statement on Schedule 14A relating to a special meeting of the shareholders with the SEC and Canadian Securities Administrators (“CSA”). Additionally, Fusion will file other relevant materials in connection with the transaction with the SEC and the CSA. Shareholders of Fusion are urged to read the management information circular and proxy statement and/or consent solicitation documents regarding the transaction and any other relevant materials carefully in their entirety when they become available before making any voting or investment decision with respect to the transaction because they will contain important information about the transaction and the parties to the Arrangement Agreement. The definitive management information circular and proxy statement will be mailed to holders of Fusion’s shares. Shareholders will be able to obtain a copy of the management information circular and proxy statement, as well as other filings containing information about the transaction and the parties to the Arrangement Agreement made by Fusion with the SEC and CSA free of charge on EDGAR at www.sec.gov, on SEDAR+ at www.sedarplus.com, or on Fusion’s website at www.fusionpharma.com. Information contained on, or that may be accessed through, the websites referenced in this communication is not incorporated into and does not constitute a part of this document. We have included these website addresses only as inactive textual references and do not intend them to be active links.

Participants in the Solicitation

Fusion and its directors and executive officers may be deemed to be participants in the solicitation of proxies from the shareholders of Fusion in respect of the transaction. Information about Fusion’s directors and executive officers is set forth in the proxy statement and management information circular for Fusion’s Annual General Meeting of Shareholders, which was filed with the SEC and CSA on April 27, 2023. Investors may obtain additional information regarding the interest of such participants by reading the management information circular and proxy statement regarding the proposed transaction when it becomes available.

Contact:
Amanda Cray
Senior Director of Investor Relations & Corporate Communications
(617) 967-0207
cray@fusionpharma.com

SOURCE Fusion Pharmaceuticals

TORONTO, ON / ACCESSWIRE / March 7, 2024 / Lumira Ventures, a life sciences investment firm dedicated to supporting healthcare innovation and patient impact, is proud to announce several significant advancements and promotions within its investment team. “As we commemorate International Women’s Day on March 8^th, 2024, it is crucial to recognize the invaluable contributions of women in the workforce,” said Peter van der Velden, Managing Director at Lumira Ventures. “Lumira was among the first VC firms in Canada with a female partner and these team promotions along with the recent new addition to our team showcase the indispensable contributions and influence of women in leadership roles shaping the professional landscape within venture capital.”

Lumira Ventures commends the dedication and accomplishments of three deserving employees who have been promoted to elevated leadership positions. These promotions underscore Lumira’s commitment to nurturing and empowering talent from within our organization. The following team members, all talented women, have been promoted to positions of new responsibility within the firm.

Suman Rao, Ph.D. has been promoted to Senior Associate. Suman has continuously demonstrated strong leadership and strategic insight since joining the Lumira Ventures investment team in 2021. In her new role, she will take on additional responsibilities for sourcing, evaluating and closing new investments and serving on the boards of Lumira portfolio companies. As well she will continue being a core member of the team developing and promoting new strategies with our partner Angelini Ventures to pursue the growing opportunity we see from our investment approach in Europe.

Baye Galligan, MA, HBA, has been promoted to Director of Research. Since joining Lumira in 2017, Baye has been an integral part of our investment team. In 2021 she became one of the internal leads on supporting our Venture Innovation Program, which mentors current Ph.D., M.D. or MBA students through a 6-12 month in-house fellowship in venture capital and entrepreneurship. As this program expanded, and as Lumira continued to deepen its pro-active internal research, it presented a perfect opportunity for Baye to take the lead with respect to prioritizing and developing the research to support new investment themes and portfolio company value creation through the application of traditional and AI-enabled research tools.

Alyssia Watkin has been promoted to Director of Communications and Investor Relations. After joining Lumira first as an intern and then as our first dedicated internal marketing resource in 2021, Alyssia has played a pivotal role in transforming our marketing and communications functions in support of our investor community and external stakeholders. In her new position, she will take on additional responsibilities related to the firm’s fundraising initiatives and limited partner relationship management, as well as coordinating Lumira’s communications initiatives supporting our portfolio companies.

Along with these promotions, Lumira Ventures welcomes the new addition of:

Isabelle Harris, HBA to our team, as an Analyst. Isabelle brings a rich background in life sciences and strategy consulting to the Lumira team. She holds a B.S in genetics from Western University and an HBA from Ivey Business School. In her new role, she will collaborate with the investment team and support the evaluation of new investment opportunities, utilizing her understanding of industry dynamics and analytical acumen to help drive investment decisions across our various investment funds.

Lumira Ventures is excited to have Isabel join the team and looks forward to the leadership of the newly promoted team members. Their diverse backgrounds, distinctive perspectives, and commitment to excellence embody the essence of International Women’s Day. As we join the world in celebrating this important day, Lumira Ventures reaffirms its dedication to fostering gender equality, empowerment, and an inclusive workplace where everyone has the opportunity to flourish, not just today, but every day.

For more information, please visit www.lumiraventures.com

Media Inquiries: info@lumira.vc

www.lumiraventures.com

About Lumira Ventures

Founded in 2007, Lumira Ventures is a multi-stage life sciences investment firm with offices in Toronto, Montréal, Vancouver, and Boston. We partner with mission-driven entrepreneurs building companies that harness rapidly evolving advancements in medicine to develop transformative products for patients while delivering strong financial returns for our investors and meaningful economic value and impact for society.

SOURCE: Lumira Ventures