• Financing Round Led by OrbiMed with participation from Lumira Ventures, Harrington Discovery Institute and existing investors Advent Life Sciences and Belinda Termeer

• Therapy development focused on addressing a fundamental mechanism in cancer-driving oncogenes

October 19, 2022 – PIC Therapeutics, Inc., a biopharmaceutical company dedicated to developing life-changing medicines for patients with cancer, today announced the closing of a $35 million Series A financing led by OrbiMed. Other new investors participating in this financing include Lumira Ventures and Harrington Discovery Institute. The company’s existing investors including Advent Life Sciences and Belinda Termeer also participated and provided initial seed financing instrumental to meeting key milestones.

Proceeds from the financing will be used to advance the company’s development-stage small molecule drug, an allosteric protein translation modulator targeting eIF4E, into first-in-man, first-in-mechanism clinical studies in advanced metastatic breast cancer. The proceeds will also support expansion of the company’s pipeline of emerging oncology indications.

PIC Therapeutics is targeting a fundamental mechanism at the convergence of many oncogenic signaling pathways that results in apoptotic cancer cell death while sparing normal cells. Allosteric modulation of eIF4E offers many advantages to previous approaches, and simultaneously addresses multiple drivers of pharmacology, allowing the company’s small molecules to truly drive differential CAP dependent translation in target cells.

Preclinical studies show that PIC compounds modulate, but do not block, protein translation. PIC compounds mechanistically modulate cellular proteomes, leading to rapid and significant reduction in cancer cell viability via apoptosis. Inducing apoptosis rather than senescence is an important distinguishing feature of PIC’s approach to this elusive target.

“This financing from a committed and distinguished investor syndicate, which includes new and existing investors, underscores the progress we’ve made to advance our lead program toward our goal of cancer therapies that broadly address cancer-driving oncogenes via a fundamental mechanism in protein translational modulation” said Katherine Bowdish, Chief Executive Officer of PIC Therapeutics. “We are well positioned to build a leading mechanistic-based oncology company that brings promising science to cancer patients with drug resistant tumors.”

“We are excited to partner with PIC Therapeutics as they build a differentiated targeted oncology company, and we look forward to supporting the team as they work towards achieving key development goals over the coming years,” said Tal Zaks, Partner of OrbiMed Advisors.

“Founded on the scientific work of Dr. Gerhard Wagner at Harvard University and Dr. Nahum Sonenberg at McGill University, PIC Therapeutics has developed a truly novel approach for eIF4E, an important target in resistant cancers. We are pleased to work with the PIC team and its investors to enable new frontiers in targeted mechanistic oncology with the potential to transform the treatment paradigm for cancer patients” commented Gerry Brunk, Managing Director of Lumira Ventures.

About PIC Therapeutics

PIC Therapeutics is a biotechnology company pioneering the discovery and development of first-in mechanism, first-in-class small molecule medicines focused on fundamentally changing how we treat cancer by developing therapeutics that modulate protein translation. PIC Therapeutics targets a “master switch” of cancer signaling pathways, selectively impacting oncogene protein production by altering the Pre-Initiation Complex (PIC) that drives their mRNA translation. PIC Therapeutics’ selective approach modulates cancer cell proteomes, impacting multiple dysregulated oncogenic drivers leading to a powerful new generation of cancer-treating therapeutics. Our agents offer the opportunity to address both drug resistance and tumor heterogeneity, issues that plague many existing treatments. For more information visit www.pictherapeutics.com. PIC is guided by a dedication to improving cancer patient outcomes and to realizing the potential of our programs to their benefit.

About OrbiMed

OrbiMed is a leading healthcare investment firm, with approximately $18 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, through a range of private equity funds, public equity funds, and royalty/credit funds. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies. OrbiMed’s team of over 130 professionals is based in New York City, San Francisco, Shanghai, Hong Kong, Mumbai, Herzliya, and other key global markets. www.orbimed.com

About Lumira Ventures

Lumira Ventures is a North American healthcare venture capital firm whose companies have brought dozens of biomedical innovations to patients worldwide over the past two decades. Lumira invests across multiple stages, sectors and therapeutics areas, partnering with mission-driven entrepreneurs and co-investors to build transformative healthcare companies. Lumira has offices in Toronto, Montréal, Vancouver and Boston. For more information please visit www.lumiraventures.com

Contacts

Matt Burke

mattdavidburke@gmail.com

TORONTO, Ontario, September 26, 2022 — DAMONA Pharmaceuticals today announced the closing of a seed stage financing round to support the development of its lead therapeutic for the treatment of cognitive symptoms associated with depression and diseases of aging. The syndicated investment was co-led by the Angelini Lumira Biosciences Fund and Noetic Fund and included participation from Toronto Innovation Acceleration Partners (TIAP), and several family offices. In addition to the equity financing, the company has received significant financial support from the Centre for Addiction and Mental Health (CAMH) and the Ontario Brain Institute and additional early stage development programs. Proceeds from these financings will support the development of the company lead, AC-101, which was licensed from CAMH, UWM Research Foundation Inc., University of Tours and University of Belgrade-Faculty of Pharmacy, and additional early stage development programs.

DAMONA’s mission is the discovery and development of first-in-class modulators of GABA neurotransmitters that target and restore impaired brain functions in patients suffering from cognitive symptoms. Promising data generated from testing in preclinical models has demonstrated the ability of AC-101 to specifically target and activate impaired brain receptors and to rapidly improve cognitive symptoms while renewing and repairing underlying brain dysfunctions, thereby offering the potential of reversing the impairment and in doing so resolve previously unresolvable symptoms.

“The Lumira team was first drawn to the DAMONA opportunity by the quality and depth of the science being generated by company founder Dr. Etienne Sibille, a world-leading scientist in the areas of neuroscience and neuropharmacology. Over the past several years, we built a strong working relationship with Dr. Sibille which enabled us to see the terrific progress culminating in the selection of its lead, AC-101, which the company now plans to take into IND enabling studies.” said Jacki Jenuth, Partner and COO of Lumira Ventures.

“Currently, there are no medications available to treat cognitive symptoms such as memory loss and inability to focus that occur in association with depression, aging, and other mental illnesses. DAMONA’s lead program and pre-clinical pipeline offer real hope for patients suffering from these devastating disorders,” said Sri Teja Mullapudi, Principal of Noetic Funds.

“We are grateful for the support from all of our investors and supporters who have brought us to where we are today,” said Dr. Etienne Sibille, Interim CEO and Chief Scientific Officer of DAMONA Pharmaceuticals. “We look forward to working with our new partners and utilizing the
capital from this financing to complete our pre-clinical studies and take our program to the clinic.”

“As a world leader in mental health and brain sciences research, we are proud to work with Angelini, Lumira, Noetic, TIAP, OBI and others to move this critical work to its next stage,” added Dr. Aristotle Voineskos, Vice President of Research and Director of the Campbell Family Mental Health Research Institute at CAMH. “This investment brings us one step closer to helping the millions of people affected by cognitive symptoms associated with depression and diseases of aging.”

About DAMONA Pharmaceuticals
DAMONA is a privately held pre-clinical pharmaceutical company founded by Dr. Etienne Sibille and the Centre for Addiction and Mental Health (CAMH) on a mission to develop small molecule therapeutics that transform the treatment of cognitive symptoms and restore normal life functions for underserved and understudied mental health and aging populations.

About Angelini Lumira Biosciences Fund
Angelini Lumira Biosciences Fund is a venture capital fund established by Angelini Pharma in partnership with Lumira Ventures. The Fund focuses on early stage investments in North American based companies developing products targeted at rare and central nervous system diseases. Angelini is an international pharmaceutical company, part of the Italian privately-owned Angelini Group. Angelini Pharma is committed to helping patients in the therapeutics areas of central nervous system and mental health, rare diseases, and consumer healthcare. Angelini Pharma operates directly in 25 countries employing almost 3,000 people and commercializes its products in more than 70 countries through strategic alliances with leading international pharmaceutical groups. Lumira Ventures is a North American healthcare venture capital firm with two-decades of experience of investing in and helping to build transformative life science based companies. Lumira Ventures manages its activities from offices in Toronto, Montréal, Vancouver and Boston. For more information, please visit www.lumiraventures.com

About Noetic
Noetic was founded in early 2020 with a vision to disrupt how mental health indications are treated and managed. Through its venture capital funds, Noetic invests globally to build emerging technologies in molecular therapeutics/biotech, digital health, medical devices, and wellness with a focus on mental health, pain, and other complications resulting from the central nervous system.

About CAMH
The Centre for Addiction and Mental Health (CAMH) is Canada’s largest mental health and addiction teaching hospital and a world leading research centre in this field. CAMH combines
clinical care, research, education, policy development and health promotion to help transform the lives of people affected by mental illness and addiction. CAMH is fully affiliated with the University of Toronto, and is a Pan American Health Organization/World Health Organization Collaborating Centre. For more information, please visit camh.ca or follow @CAMHnews on Twitter.

For More Information:
DAMONA pharmaceuticals
Etienne Sibille
Etienne.sibille@damonapharma.com

GERMANTOWN, Md., Sept. 8, 2022 /PRNewswire/ — Deka Biosciences (“Deka”), a biotech company focused on the development of novel cytokine therapies to treat cancer and inflammatory diseases, announced today the appointment of Charlotte Moser, MD, PhD, MSc, MBA as Chief Medical Officer (CMO) and Deb Kientop, MBA as Vice President, Clinical Operations.

Dr. Moser joins Deka from Nanocan Therapeutics Corporation  where she served as Chief Scientific Officer. With over 28 years working as an oncologist, 15 of which she has spent specifically in drug development, Dr. Moser has obtained extensive clinical operational and regulatory knowledge and broad clinical trial design and execution experience. “I’m keen to propel Deka from a preclinical to clinical stage company and drive the development of more tolerable and selective immunotherapies at a fast pace,” shared Dr. Moser.

Deb Kientop comes to Deka from ErgoMed, where she served as the Senior Vice President, Oncology Strategy and Innovation. Deb has over 27 years working in oncology drug development, from bench research at the University of Wisconsin, to medical affairs and clinical development roles within pharma and CRO. “I’m excited to be a part of the expert team that is bringing next generation targeted cytokines to the clinic. I look forward to the opportunity to bring precision therapies to those with cancer and autoinflammatory diseases,” shared Ms. Kientop.

About Deka Biosciences

Deka Biosciences is a biotech company focused on the development of novel cytokine therapies to treat cancer and inflammatory diseases such as Crohn’s, psoriasis, rheumatoid arthritis and sepsis. The company is led by entrepreneur Dr. John Mumm, who is backed by a team of experienced academic, biopharma and CDMO innovators with expertise in drug discovery, product development, characterization and testing. Deka has developed disease specific Diakines™ that maximize patient benefits through improved pharmacokinetics / pharmacodynamics (PK/PD) function via targeted delivery of dual and complimentary cytokines to affected tissues or cells. Using precision medicine, Deka will maximize the impact of its Diakines™ by building targeted therapies for every patient. To learn more, visit www.dekabiosciences.com.

SOURCE Deka Biosciences

Satsuma Pharmaceuticals, Inc. (Nasdaq: STSA), a clinical-stage biopharmaceutical company developing STS101 (dihydroergotamine (DHE) nasal powder), a novel investigational therapeutic product candidate for the acute treatment of migraine, today announced completion of subject enrollment in the Company’s ongoing STS101 SUMMIT pivotal Phase 3 efficacy trial, with more than 1,400 subjects randomized. Satsuma expects to announce topline results from the SUMMIT trial in the fourth quarter of 2022.  

“We are pleased to report completion of subject enrollment in the STS101 SUMMIT Phase 3 efficacy trial, the largest-ever randomized and controlled trial of a DHE product,” stated John Kollins, Satsuma’s President and Chief Executive Officer. “This marks an important milestone in our STS101 development program. We look forward to reporting SUMMIT trial topline results in the fourth quarter of 2022, and, in the interim, to reporting further STS101 development program progress and clinical data, including topline results from our ongoing STS101 ASCEND Phase 3, open-label, long-term safety trial.”

STS101 SUMMIT Pivotal Phase 3 Efficacy Trial
The STS101 SUMMIT pivotal Phase 3 efficacy trial is a multi-center, single-dose, randomized, double-blind, placebo-controlled, parallel group study in more than 1,400 subjects with migraine that is being conducted in the United States. The trial is designed in accordance with recommendations contained in the U.S. Food and Drug Administration’s (FDA) current guidance document for industry (Migraine Developing Drugs for Acute Treatment, February 2018) and the International Headache Society’s (IHS) published guidelines for controlled trials of acute treatment of migraine attacks in adults.¹   The SUMMIT trial, the largest-ever randomized and controlled trial of a DHE product, provides a basis for STS101 to become the first and only DHE product to demonstrate efficacy in a randomized and controlled trial on co-primary endpoints (freedom from pain and freedom from most bothersome symptom at two hours post-treatment) currently recommended by the FDA and IHS.   Based on Satsuma’s communications with the FDA, including a Type B, clinical pre-NDA videoconference meeting held in May 2022, the Company believes the SUMMIT trial, if successful, will support inclusion of differentiating efficacy claims in the STS101 prescribing information, presuming STS101 is approved for marketing.

After establishing full eligibility, SUMMIT trial participants are randomized (1:1) to receive either STS101 5.2 mg or matching placebo and instructed to treat their next migraine attack of at least moderate pain severity with the allocated blinded study medication. Following randomization, participants have 56 days in which to treat the qualifying migraine attack.

The co-primary endpoints of the SUMMIT trial, to be assessed at two hours after STS101 administration, are freedom from pain and freedom from most bothersome symptom (from among photophobia, phonophobia or nausea). The trial is designed for greater than 99% statistical power for the freedom from pain endpoint and greater than 95% statistical power for the freedom from most bothersome symptom endpoint. In addition, the SUMMIT trial incorporates a number of secondary endpoints and prospective evaluations of the clinical performance of STS101 that could differentiate the clinical profile of STS101.

Satsuma expects to report topline results from the SUMMIT trial in the fourth quarter of 2022.

For further information regarding the STS101 SUMMIT Phase 3 efficacy trial, see www.ClinicalTrials.gov, identifier NCT04940390: A Randomized, Double-Blind, Placebo-Controlled Study to Assess STS101 in the Acute Treatment of Migraine (SUMMIT).

About Satsuma Pharmaceuticals and STS101
Satsuma Pharmaceuticals is a clinical-stage biopharmaceutical company developing a novel therapeutic product, STS101, for the acute treatment of migraine. STS101 is a unique and proprietary nasal powder formulation of the well-established anti-migraine drug, dihydroergotamine mesylate (DHE), administered via Satsuma’s proprietary nasal delivery device.  STS101 is designed to provide significant benefits versus existing acute treatments for migraine, including the combination of quick and convenient self-administration and other clinical advantages, that current DHE liquid nasal spray products and injectable dosage forms lack. Satsuma’s dry powder DHE formulation has demonstrated fast absorption, rapid achievement of high DHE plasma concentrations which Satsuma believes is necessary for early efficacy, and sustained DHE plasma levels over time with low dose-to-dose variability. STS101 also now incorporates an improved 2nd-generation nasal delivery device designed to provide more consistent nasal dosing, irrespective of user administration technique. DHE has long been recommended in published migraine treatment guidelines as a first-line acute treatment option for migraine and has significant advantages versus other anti-migraine treatments for many patients. However, disadvantages of current DHE liquid nasal spray and injectable products, including invasive and burdensome administration and/or sub-optimal clinical performance, have limited the widespread use of DHE. Featuring an easy-to-carry and easy-to-use dosage form, STS101 is designed to overcome these shortcomings and provide patients an improved therapeutic solution for acutely treating migraines that consistently delivers robust clinical performance.

Satsuma is headquartered in South San Francisco, California with operations in both California and Research Triangle Park, North Carolina. For further information, please visit www.satsumarx.com.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements concerning the business, operations and financial performance and condition of Satsuma Pharmaceuticals, Inc. (the “Company”), as well as the Company’s plans, objectives and expectations for its business operations and financial performance and condition. Any statements contained herein that are not statements of historical facts may be deemed to be forward-looking statements. In some cases, you can identify forward-looking statements by terminology such as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “due,” “estimate,” “expect,” “goal,” “intend,” “may,” “objective,” “plan,” “predict,” “potential,” “positioned,” “seek,” “should,” “target,” “will,” “would,” and other similar expressions that are predictions of or indicate future events and future trends, or the negative of these terms or other comparable terminology. These forward-looking statements include, but are not limited to, statements about the Company’s expectations regarding the potential safety and efficacy of STS101, the potential results of the ASCEND and SUMMIT trials, the timing of data readouts for ongoing clinical trials, the anticipated timing for a potential STS101 NDA submission, the potential for STS101 to be an important and differentiated acute treatment option, and the expected cash runway of the Company. In light of these risks and uncertainties, the events or circumstances referred to in the forward-looking statements may not occur. The Company’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 detailed in the Company’s Quarterly Report on Form 10-Q for the quarter ended March 31, 2022, filed with the Securities and Exchange Commission, as well as other documents that may be filed by the Company from time to time. In particular, the following factors, among others, could cause results to differ materially from those expressed or implied by such forward-looking statements: the Company’s ability to demonstrate sufficient evidence of efficacy and safety in its clinical trials of STS101; the results of preclinical and clinical studies may not be predictive of future results; and the risk that the COVID-19 worldwide pandemic may negatively impact the Company’s business, operations, clinical trials or ability to raise capital. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the events and circumstances reflected in the forward-looking statements will be achieved or occur, and the timing of events and circumstances and actual results could differ materially from those projected in the forward-looking statements. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and the Company undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise.

This press release discusses STS101, a product candidate that is in clinical development, and which has not yet been approved for marketing by the U.S. Food and Drug Administration. No representation is made as to the safety or effectiveness of STS101 for the therapeutic use for which STS101 is being studied.

INVESTOR AND CORPORATE CONTACTS:         

Corey Davis, PhD
LifeSci Advisors, LLC
cdavis@lifesciadvisors.com

Tom O’Neil, Chief Financial Officer
Satsuma Pharmaceuticals, Inc.
tom@satsumarx.com

¹ Diener et al., Guidelines of the International Headache Society for controlled trials of acute treatment of migraine attacks in adults: Fourth Edition, Cephalalgia, 2019

X4 Pharmaceuticals, Inc. (Nasdaq: XFOR), a leader in the discovery and development of novel CXCR4 targeted small molecule therapeutics to benefit people with rare immune system disorders, today announced that it has agreed to sell an aggregate of 50,925,365 shares of common stock (or pre-funded warrants in lieu thereof) and warrants to purchase an aggregate of 50,925,365 shares of common stock, to certain institutional accredited investors in a private investment in public equity (PIPE) financing. X4 anticipates that gross proceeds from the PIPE will be approximately $55 million, before deducting fees to the placement agent and other estimated offering expenses payable by the Company. The closing of the financing is expected to occur on or about July 6, 2022, subject to customary closing conditions.

In addition, on June 30, 2022, contemporaneous with the execution of the PIPE, X4 entered into an amendment to its loan and security agreement with Hercules Capital Inc. to extend the interest-only period of its loan facility by up to twelve months (into 2024), subject to achieving certain financial and business milestones. This amendment results in a potential reduction of X4’s cash burn by $20 million over the interest-only period.

Pursuant to the terms of the securities purchase agreement, at the closing of the PIPE, X4 will issue an aggregate of 50,925,365 shares of common stock (or pre-funded warrants in lieu thereof) and warrants to purchase an aggregate of 50,925,365 shares of common stock. The purchase price per share and accompanying warrant is $1.095 (or $1.094 per pre-funded warrant and accompanying warrant). The warrants will have a per share exercise price of $1.095 and may be exercised at any time on or after the closing date and through the fifth anniversary of the closing date. The price per share and accompanying warrant was based in part upon the last reported sale price of the common stock on the Nasdaq Capital Market. If exercised for cash, the warrants would result in additional gross proceeds to X4 of up to approximately $55 million.

X4 expects to use these funds for continued clinical development and commercial readiness of its lead candidate, mavorixafor, and for business development activities, working capital, and general corporate purposes.

The PIPE financing included participation from new investors including co-lead investor New Enterprise Associates (NEA), Acorn Bioventures and Lumira Ventures, as well as existing investors including co-lead investor Bain Capital Life Sciences, OrbiMed, AXA Investment Managers and Hercules Capital, Inc (NYSE: HTGC). Stifel served as sole placement agent for the financing. B. Riley Securities and H.C. Wainwright & Co. acted as financial advisors to X4 in the financing.

The securities sold in this financing are being made in a transaction not involving a public offering and have not been registered under the Securities Act of 1933, as amended, and may not be offered or sold in the United States except pursuant to an effective registration statement or an applicable exemption from the registration requirements. X4 has agreed to file a registration statement with the Securities and Exchange Commission registering the resale of the shares of common stock and the shares of its common stock underlying the pre-funded warrants and accompanying warrants sold in this financing. In connection with the PIPE, the Company has agreed to convene a special meeting of its stockholders no later than 90 days following the closing to seek approval of an increase in the number of its authorized shares of common stock.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction.

About X4 Pharmaceuticals, Inc.
X4 Pharmaceuticals is a late-stage clinical biopharmaceutical company leading the discovery and development of novel therapies for people with rare diseases of the immune system. The company’s lead candidate is mavorixafor, a first-in-class, small molecule antagonist of chemokine receptor CXCR4 that is being developed as a once-daily oral therapy. Due to mavorixafor’s ability to antagonize CXCR4 and improve the healthy maturation and trafficking of white blood cells, X4 believes that mavorixafor has the potential to provide therapeutic benefit across a wide variety of diseases, including primary immunodeficiencies (PIDs) and certain types of cancer. Mavorixafor has already demonstrated clinical potential in a Phase 2 trial in people with WHIM syndrome, a rare PID. Its efficacy and safety continue to be evaluated in a global Phase 3 clinical trial in WHIM (fully enrolled) and in two Phase 1b clinical trials – one, as monotherapy in people with chronic neutropenia, including Severe Congenital Neutropenia (SCN), and another in combination with ibrutinib in people with Waldenström’s macroglobulinemia (also fully enrolled). X4 is continuing to leverage its insights into CXCR4 biology at its corporate headquarters in Boston, Massachusetts and at its research facility in Vienna, Austria, to discover and develop additional product candidates.

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of 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, statements regarding the timing, use of proceeds and closing of the PIPE. Actual events or results may differ materially from those expressed or implied by any forward-looking statements contained herein, including, without limitation, as a result of market and other conditions; the risk that the conditions to the closing of the proposed PIPE are not satisfied; the risk that the Company fails to timely obtain stockholder approval to increase its authorized share capital, if at all; as well as other risks and uncertainties described in the section entitled “Risk Factors” in X4’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on May 12, 2022, 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.

Contacts:

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

Mónica Rouco Molina, Ph.D. (media)
Account Supervisor, LifeSci Communications
mroucomolina@lifescicomms.com

• New Name Emphasizes Relentless Pursuit of an Expanded and Rare Disease Portfolio
• Oncology Programs added to existing Long QT Syndrome Program (“LQTS”)
• Additional US$15M Financing Provides Fuel for Proof-of-Concept in LQTS

LAVAL, Québec, Canada | 11 July 2022 | Thryv Therapeutics Inc. is excited to announce the evolution of its portfolio to include treatments for resistant and rare cancers. The new name, Thryv Therapeutics Inc., embodies our commitment to relentlessly pursue therapies which provide lifelong benefits to patients, families, doctors, nurses and stakeholders. Recent discoveries implicating Serum Glucocorticoid Kinase 1 (“SGK1”) provided a strong rationale for additional funding, acceleration of a portfolio of compounds to target these new opportunities of high unmet medical need, and a new corporate name – Thryv Therapeutics Inc.

“We are grateful to our investors who recognize the significant potential of our SGK1 platform to treat resistant and rare cancers in addition to the on-going development of our compounds for Long QT Syndrome,” said Debra Odink, PhD, Chief Development Officer. “Recent internal research combined with our collaborators results confirms what various researchers have demonstrated. SGK1 is a novel, yet unexplored target that may provide an opportunity to address treatment resistance in oncology.”

In various genetic and acquired disease states, SGK1 may be mutated and overexpressed leading to the development of serious resistance to existing therapies. The development of SGK1 inhibitors represents a novel approach to target treatment-resistant cancers, and complement existing therapies.

Ongoing external collaborations with leading academic institutions are evaluating Thryv Therapeutics’ internally developed SGK1 inhibitors as potential treatments for patients with treatment-resistant breast, prostate and anaplastic thyroid cancer. This strategic evolution triggered a significant new investment from investors allowing for the completion of a proof-of-concept study in patients with Long QT Syndrome, acceleration of several in vivo proof of concept studies in cancer and Long QT, and submission of an IND for the treatment of resistant cancers.

About Thryv Therapeutics

Thryv Therapeutics Inc., previously called LQT Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. They have been pioneering a precision medicine approach to treat Long QT Syndrome via SGK1 inhibition since 2019 and have since evolved their portfolio to include the treatment of resistant and rare cancers. The development of SGK1 inhibitors represents a novel approach to target treatment-resistant cancers, and complement existing therapies. This strategic evolution has secured an additional US$15M financing fueling Thryv to complete a proof-of-concept study for LQTS, and submit an IND for the treatment of resistant cancer.

Media Inquiries:

Daphne Doucet | admin@thryvtrx.com | +1 (514) 973-0915

• Positive Phase 1 results reported at the American Association for Thoracic Surgery (AATS) and the American Society of Gene and Cell Therapy (ASGCT) revealed XC001 is well tolerated at all dose levels
• Phase I data support XC001 therapeutic effect and potential dose response
• Topline Phase 2 data readout expected in February 2023 with interim results in the second half of this year

XyloCor Therapeutics, a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced it has achieved enrollment of target number of subjects in the Phase 2 portion of its ongoing Phase 1/2 clinical trial (EXACT) for refractory angina. Topline results from the Phase 2 study are expected in February 2023 with interim results in the second half of this year.

“Achievement of this important milestone in the Phase 2 portion of the study is a testament to the clinical need in this patient population and I am eager to see the Phase 2 results as they emerge,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “Patients with refractory angina have no treatment options, and the results from the Phase 1 portion of the EXACT trial suggest a dose response and therapeutic potential which is encouraging for the development of XC001 as a treatment to improve these patients’ quality of life. We are very excited to see this more definitive evaluation of the safety and efficacy of this approach.”

“We are pleased to announce this important milestone in enrollment for our Phase 2 study especially during this unprecedented and challenging time,” said Al Gianchetti, President and CEO of XyloCor. “An estimated one million people suffer from refractory angina in the United States, and we are encouraged that XC001 may address the high unmet need in this patient group. XyloCor also plans to study XC001 in other patient groups as well, including as adjunctive therapy in patients undergoing bypass surgery.”

Individuals with refractory angina experience pressure or intense pain in the chest due to insufficient blood flow to the heart muscle. These symptoms can severely impact quality of life and may worsen comorbidities.

XyloCor’s lead investigational drug, XC001 (encoberminogene rezmadenovec) is a locally administered, single-dose gene therapy currently in development as a novel approach to treating patients with refractory angina who have no other medical and surgical options. The treatment strategy is to use local administration to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects. 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.

About the EXACT Study

The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open-label, single-arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, followed by an expansion phase of the trial with target enrollment of 27 additional subjects at the highest tolerated dose (1 x 10¹¹ vps, the highest tested dose). The investigational gene therapy is administered directly to the heart muscle through a mini-thoracotomy by an experienced cardiac surgeon at top cardiovascular research sites across the United States.

About Chronic Refractory Angina

In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain.

About XyloCor

XyloCor Therapeutics 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 which 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.

Contacts

Medexus Pharmaceuticals (Medexus) (TSX: MDP) (OTCQX: MEDXF) today announced its operating and financial results and provided a business update for the company’s fourth fiscal quarter and fiscal year ended March 31, 2022. All dollar amounts in this press release are in United States dollars unless specified otherwise.

Financial Highlights

• Delivered total revenue of $20.3 million in fiscal Q4 2022, an increase of 15% compared to fiscal Q4 2021 and, as expected, a decrease of 5% compared to fiscal Q3 2022 partly due to a large order in late fiscal Q3 2022 originally anticipated in fiscal Q4 2022.
• Generated total revenue of $76.7 million in fiscal year 2022, a decrease of 4% compared to fiscal year 2021.
• Achieved adjusted EBITDA* of $1.1 million in fiscal Q4 2022 compared to $(1.6) million in fiscal Q4 2021 and $1.9 million in fiscal Q3 2022.
• Generated adjusted EBITDA* of $(3.9) million in fiscal year 2022 compared to $8.2 million in fiscal year 2021.
• Produced net loss of $5.3 million in fiscal Q4 2022 and $2.9 million in fiscal year 2022 compared to $10.5 million in fiscal Q4 2021 and $28.3 million in fiscal year 2021.
• Generated adjusted net loss* of $4.6 million in fiscal Q4 2022 and $24.0 million in fiscal year 2022 compared to $5.2 million in fiscal Q4 2021 and $7.6 million in fiscal year 2021.
• Cash and cash equivalents of $10.0 million (with $11.2 million of total available liquidity) at end of fiscal Q4 2022.

*  Refer to “Non-GAAP Measures” at the end of this press release for information about adjusted EBITDA and adjusted net income (loss).

Ken d’Entremont, Chief Executive Officer of Medexus, commented, “We are very pleased with the strength and stability we have seen in our base business. We continue to see strong performance in Rasuvo, and Rupall has continued to gain market share and benefit from another strong Canadian allergy season. IXINITY sales have normalized and, with the manufacturing improvements Medexus and our contract manufacturer are undertaking, we expect to realize operational efficiencies going forward that will positively impact our margins down the road.”

Mr d’Entremont continued, “Looking forward at our growth products, the transition for Gleolan in the U.S. is going better than expected and we will begin recognizing product revenue partway through fiscal Q2 and fully in fiscal Q3. We previously estimated that Gleolan had generated US$3 million to US$4 million in revenue in the last full quarter before we licensed the product, and we hope to continue that strong performance following our U.S. relaunch of this product over the coming months. Turning to treosulfan, our partner medac’s collection of data recently requested by the FDA is progressing well, and we are confident we will see an NDA resubmission in July. The review clock for the NDA resubmission, which will run for up to six months, will start after the response is considered complete by the FDA. An FDA approval would then allow a commercial launch of treosulfan in the U.S. in the first half of calendar year 2023.”

Operational Highlights

Operational highlights for the three-month period ended March 31, 2022 and subsequent period include:

• Treosulfan: medac, a strategic partner of Medexus, continues to collect data requested by the FDA in May 2022 to complete the resubmission of medac’s New Drug Application (NDA) for treosulfan. The data collection process is progressing well, and medac continues to expect to respond to the FDA’s information requests in July 2022.
• Gleolan: In March 2022, Medexus acquired the exclusive right to commercialize Gleolan in the United States. Medexus expects to complete the transition of full responsibility for commercializing Gleolan in the United States, and begin recognizing product revenue, in fiscal Q2 2023.
• IXINITY: Unit demand for IXINITY continues to grow, and net sales of IXINITY increased in fiscal Q4 2022 as pharmacy and wholesale customers have now worked through much of their accrued inventory and return to buying patterns better aligned with patient unit demand. Medexus continues to invest in an ongoing initiative to improve the IXINITY manufacturing process. Medexus expects the resulting operational efficiencies to ultimately improve IXINITY gross margins over the coming quarters.
• Rupall: Increasingly severe allergy seasons across Canada and successful sustained execution of sales and marketing initiatives yielded continued strong growth in Rupall sales in fiscal year 2022. This performance continues to position Rupall as one of the fastest-growing antihistamines in the Canadian prescription market. (Source: IQVIA CDH units – Drugstores and hospitals purchases, MAT March 2022.)

Operating and Financial Results Summary for Fiscal Q4 2022

Total revenue reached $20.3 million for fiscal Q4 2022, compared to revenue of $17.6 million for fiscal Q4 2021. The $2.6 million increase is primarily attributable to an increase in net sales of IXINITY during the quarter, and also the continued strong performance of Rasuvo, which is efficiently supported by a moderate allocation of sales personnel, and Rupall.

Operating loss for fiscal Q4 2022 was $2.5 million, compared to an operating loss of $4.6 million for fiscal Q4 2021.

Adjusted EBITDA was $1.1 million for fiscal Q4 2022, compared to $(1.6) million for fiscal Q4 2021. The $2.7 million increase is primarily attributable to the increase in net sales of IXINITY in fiscal Q4 2022 and a $0.9 million expense related to a one-time destruction of IXINITY inventory in fiscal Q4 2021.

Net loss was $5.3 million for fiscal Q4 2022, compared to $10.5 million for fiscal Q4 2021.

Operating and Financial Results Summary for Fiscal Year 2022

Total revenue reached $76.7 million for fiscal year 2022, a decrease of $3.0 million, or 3.8%, compared to fiscal year 2021. The decrease year over year was primarily attributable to a drop in net sales of IXINITY which was partially offset by strong Rupall sales. Unit demand for IXINITY continues to grow, but net sales were lower in fiscal year 2022 as pharmacy and wholesale customers continued to work through inventory on hand for much of the year.

Operating loss for fiscal year 2022 was $15.0 million compared to $1.4 million for fiscal year 2021.

Adjusted EBITDA was $(3.9) million for fiscal year 2022, compared to $8.2 million for fiscal year 2021. The decrease was primarily attributed to significant investments in personnel and infrastructure to support Medexus’s anticipated future growth, the reduction in IXINITY net sales discussed above, and a $1.9 million increase in cost of goods sold related to previously disclosed failed batches during the IXINITY manufacturing process earlier in the fiscal year.

Net loss was $2.9 million for fiscal year 2022, compared to $28.3 million for fiscal year 2021.

Additional Information

Medexus’s financial statements and management’s discussion and analysis for the fiscal year ended March 31, 2022 are available on Medexus’s corporate website at www.medexus.com and in the company’s corporate filings on SEDAR at www.sedar.com.

Conference Call Details

Medexus will host a conference call at 8:00 AM Eastern Time on Thursday, June 23, 2022, to discuss the company’s operating and financial results and corporate updates for fiscal Q4 2022 and fiscal year 2022.

A replay of the call will be available approximately one hour following the end of the call through Thursday, June 30, 2022. To access the replay, please dial the following numbers:

877-481-4010 for Canadian and U.S. callers
+1 919-882-2331 for international callers

Conference ID: 45811

A replay of the webcast will be available on the Investors—News & Events—IR Calendar section of Medexus’s corporate website until Friday, June 23, 2023.

About Medexus

Medexus is a leader in innovative rare disease treatment solutions with a strong North American commercial platform and a portfolio of proven best-in-class products. Our current focus is on the therapeutic areas of hematology, auto-immune diseases, and allergy. We continue to build a highly differentiated company with a growing portfolio of innovative and high-value orphan and rare disease products that will underpin our growth for the next decade.

Our current leading products are Rasuvo™ and Metoject®, a unique formulation of methotrexate (auto-pen and pre-filled syringe) designed to treat rheumatoid arthritis and other auto-immune diseases; IXINITY®, an intravenous recombinant factor IX therapeutic for use in patients 12 years of age or older with Hemophilia B (a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood, which is necessary to control bleeding); and Rupall®, an innovative prescription allergy medication with a unique mode of action. We also hold exclusive US and Canadian rights to commercialize Gleolan™ (aminolevulinic acid hydrochloride or ALA HCl), an FDA-approved, orphan drug designated optical imaging agent currently indicated in patients with glioma (suspected World Health Organization Grades III or IV on preoperative imaging) as an adjunct for the visualization of malignant tissue during surgery.

We have also licensed treosulfan, part of a preparative regimen for allogeneic hematopoietic stem cell transplantation to be used in combination with fludarabine, for commercialization in the United States and Canada. Treosulfan was approved by Health Canada in June 2021 and is marketed in Canada as Trecondyv®. Treosulfan is currently the subject of a regulatory review process with the U.S. Food and Drug Administration.

Our mission is to provide the best healthcare products to healthcare professionals and patients. We strive to deliver on this mission by acting on our core values: Quality, Innovation, Customer Service, and Collaboration.

Contacts

For more information, please contact any of the following:

Forward-Looking Statements

Certain statements made in this press release contain forward-looking information within the meaning of applicable securities laws (forward-looking statements). The words “anticipates”, “believes”, “expects”, “will”, “plans”, “potential”, and similar words or expressions are often intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Specific forward-looking statements contained in this news release include, but are not limited to, statements regarding Medexus’s business strategy or outlook and future growth plans, expectations regarding future financial or operating performance (including with respect to the expected benefits of improvements made to the IXINITY manufacturing process and expected results from sales of Gleolan in the United States), ability to obtain FDA and other regulatory approvals for treosulfan and other product candidates, the timing of treosulfan launch in the United States, and competitive position of and anticipated trends and challenges in the company’s business and the markets in which it operates, among others. These statements are based on factors or assumptions that were applied in drawing a conclusion or making a forecast or projection, including assumptions based on historical trends, current conditions and expected future developments. Since forward-looking statements relate to future events and conditions, by their very nature they require making assumptions and involve inherent risks and uncertainties. Medexus cautions that although it is believed that the assumptions are reasonable in the circumstances, these risks and uncertainties give rise to the possibility that actual results may differ materially from the expectations set out in the forward-looking statements. Material risk factors include those set out in Medexus’s materials filed with the Canadian securities regulatory authorities from time to time, including Medexus’s most recent annual information form and management’s discussion and analysis; future capital requirements and dilution; intellectual property protection and infringement risks; competition (including potential for generic competition); reliance on key management personnel; Medexus’s ability to implement its business plan; Medexus’s ability to leverage its U.S. and Canadian infrastructure to promote additional growth; regulatory approval by relevant health authorities, including the FDA; product reimbursement by third party payers; litigation or expiry with respect to patents or other intellectual property rights; litigation risk; stock price volatility; government regulation; and potential third party claims. Given these risks, undue reliance should not be placed on these forward-looking statements, which are made only as of the date hereof. Other than as specifically required by law, Medexus undertakes no obligation to update any forward-looking statements to reflect new information, subsequent or otherwise.

Trademarks and trade names

This press release contains references to trademarks and service marks, including those belonging to other companies, persons, or entities. Solely for convenience, trademarks and trade names referred to in this document may appear without the “®” or “™” symbols. Each such reference should be read as though it appears with the relevant symbol. Any such references are not intended to indicate, in any way, that the holder or holders of the relevant intellectual property rights will not assert, to the fullest extent under applicable law, its rights to these trademarks and trade names.

Non-GAAP measures

Company management uses, and this press release refers to, financial measures that are not recognized under IFRS and do not have a standard meaning prescribed by generally accepted accounting principles (GAAP) in accordance with IFRS or other financial or accounting authorities (non-GAAP measures). These non-GAAP measures may include “non-GAAP financial measures” and “non-GAAP ratios” (each defined in National Instrument 52-112, Non-GAAP and Other Financial Measures Disclosure). Medexus’s method for calculating these measures may differ from methods used by other companies and therefore these measures are unlikely to be comparable to similarly-designated measures used or presented by other companies.

In particular, management uses Adjusted Net Income (Loss) and Adjusted EBITDA as measures of Medexus’s performance. Adjusted Net Income (Loss), EBITDA (earnings before interest, taxes, depreciation, and amortization) and Adjusted EBITDA are non-GAAP financial measures. In addition, Adjusted Net Income (Loss) may be presented on a per share basis.

An explanation and discussion of each of these non-GAAP measures, including their limitations, is set out under the heading “Preliminary Notes—Non-GAAP measures” in Medexus’s most recent management’s discussion and analysis. A reconciliation of each of these non-GAAP measures to the most directly comparable IFRS measure can be found under the heading “Reconciliation of Adjusted Net Income (Loss) and Adjusted EBITDA to Net Income (Loss)” below.

Reconciliation of Adjusted Net Income (Loss) and Adjusted EBITDA to Net Income (Loss) The following tables are derived from and should be read together with Medexus’s consolidated statement of operations for the three- and 12-month periods ended March 31, 2022. This supplementary disclosure is intended to more fully explain disclosures related to Adjusted Net Income (Loss) and Adjusted EBITDA and provides additional information related to Medexus’s operating performance. However, Medexus’s non-GAAP measures have limitations as analytical tools and should not be considered in isolation or as a substitute for analysis of Medexus’s financial information as reported under IFRS.

Existing investor syndicate supports atrial shunting as effective therapy for heart failure patients

TEWKSBURY, Mass., June 9, 2022 /PRNewswire/ — Corvia Medical, Inc, a company dedicated to transforming the treatment of heart failure (HF), today announced the closing of a $54 million equity financing from its existing investor syndicate. Funds will be utilized to support a confirmatory trial that builds on the extensive data and progressive learnings from the REDUCE LAP-HF II clinical trial of the Corvia® Atrial Shunt in heart failure patients with preserved (HFpEF) or mildly reduced (HFmrEF) ejection fraction.

“We are grateful to have the ongoing support of our investors as we continue the work to expand access to this novel therapy,” said George Fazio, CEO of Corvia Medical, “Corvia is dedicated to bringing atrial shunt therapy to the millions of heart failure patients who might benefit, and this funding allows us to continue advancing toward this important goal.”

REDUCE LAP-HF II is the largest randomized controlled trial of device-based therapy for HFpEF patients and, as recently published in Circulation,¹ is the only study of an implantable therapeutic device to show clinical benefit in this population. 626 patients were randomized at 89 centers across the US, Canada, Europe, Australia, and Japan. Results showed that within a large responder population, representing 50% of study patients, treatment with the Corvia Atrial Shunt resulted in a 45% reduction in HF events and a 55% greater improvement in quality of life compared to sham control.

“We are committed to ongoing support for the only interventional therapy that has demonstrated significant clinical benefit for a large group of heart failure patients,” commented Paul LaViolette, Board Chairman for Corvia Medical. “Corvia’s institutional and strategic investors continue to believe in the benefits of atrial shunt therapy and feel strongly that that the Corvia Atrial Shunt has the potential to change the way heart failure patients are treated.”

More than 26 million people worldwide have HF,² and over half those have HFpEF,³ which has been described as the largest unmet clinical need in cardiovascular medicine. The Corvia Atrial Shunt is designed to reduce elevated left atrial pressure (LAP), the primary contributor of HF symptoms in HFpEF patients. The shunt is placed via catheter between the left and right atria, forming a passage that allows blood to flow from the high pressure left atrium to the lower pressure right atrium, with the aim of reducing HF symptoms and events and improving quality of life.

About Corvia Medical, Inc.

Corvia Medical, Inc. is revolutionizing the treatment of heart failure through novel transcatheter cardiovascular devices. Founded in 2009 and headquartered in Tewksbury, MA, Corvia is dedicated to transforming the standard of care for heart failure treatment, enabling patients to reclaim their lives. The Corvia Atrial Shunt was granted Breakthrough Device designation by the FDA in 2019.  Privately held, the company is backed by Third Rock Ventures, General Catalyst Partners, AccelMed, Lumira Ventures, Edwards Lifesciences, and an undisclosed strategic investor. Visit https://corviamedical.com/.

MEDIA CONTACT:
Lisa Ensz
+1 978-654-6120
lensz@corviamedical.com
https://corviamedical.com/newsroom/

1.     Borlaug BA et al. Latent Pulmonary Vascular Disease May Alter the Response to Therapeutic Atrial Shunt Device in Heart Failure. Circulation. 2022;10.1161.
2.     Savarese G, Lund LH. Global Public Health Burden of Heart Failure. Card Fail Rev. 2017;3(1):7-11.
3.     Owan TE et al. Trends in prevalence and outcome of heart failure with preserved ejection fraction. N Engl J Med. 2006;355:251-259.

SOURCE Corvia Medical, Inc.

Data Presented at ASCO Demonstrate Trilaciclib Helps Protect Against Severe Neutropenia, Severe Anemia, and Severe Thrombocytopenia When Given to Extensive-Stage Small Cell Lung Cancer (ES-SCLC) Patients Prior to Chemotherapy

• Patients Receiving Trilaciclib Experienced a Lower Incidence of Single-Lineage and Multilineage Chemotherapy-Induced Myelosuppressive Events Compared with Patients Receiving Placebo
• Total Number of Patients Experiencing a Myelosuppressive Event was Lower with Trilaciclib Compared to Placebo

G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, announced on June 02, 2022, results of a post-hoc study analysis showing that ES-SCLC patients who received trilaciclib prior to chemotherapy had a lower incidence of single- and multilineage myelosuppressive events—fewer cases of severe neutropenia, severe anemia, and severe thrombocytopenia—compared to patients receiving placebo. Moreover, the proportion of patients who experienced at least one multilineage myelosuppressive event was lower in the trilaciclib arm compared to the placebo arm. The data, derived from a post-hoc analysis of Phase 2 trials, were presented in a poster at the 2022 American Society of Clinical Oncology (ASCO) annual meeting.

“Myelosuppression is a major toxicity of chemotherapy treatment for patients with extensive-stage small cell lung cancer that often results in chemotherapy dose delays and dose reductions, both of which can compromise clinical outcomes,” said Jerome Goldschmidt, M.D., medical oncologist with Blue Ridge Cancer Care in Blacksburg, VA, and lead author of the poster. “Both the patients and the healthcare system at large bear the complications of myelosuppressive events such as neutropenia, anemia, and thrombocytopenia, so it is imperative that we achieve clinically meaningful reductions in myelosuppression in multiple cell lineages and its consequences utilizing novel therapies such as trilaciclib.”

In the analysis, the researchers calculated the number of patients who experienced single lineage and multilineage myelosuppressive events as well as the total number of events each person experienced in both first-line and second/third-line chemotherapy settings. Only severe grade events (grade ≥ 3 per the National Cancer Institute) were included in the analysis, and 75 percent of patients were in the first-line setting.

Results of the analysis showed that throughout cycles one through four of first-line therapy, fewer patients treated with trilaciclib experienced single-lineage (neutrophil, red blood cell or platelet lineages) and multilineage myelosuppressive events—and fewer events occurred per person—than patients who received placebo.

Specifically, analyses of the pooled data showed that patients receiving trilaciclib in the first-line setting experienced fewer single-lineage myelosuppressive events, including:

• a 75% reduction (56.7% to 14.4%) in severe neutropenia compared to patients receiving placebo
• a 50% reduction (17.8% to 8.9%) in severe anemia compared to patients receiving placebo
• a 100% reduction (12.2% to 0.0%) in severe thrombocytopenia compared to patients receiving placebo

Additionally, analyses of the pooled data showed that patients receiving trilaciclib in the first-line setting experienced fewer concurrent, multilineage myelosuppressive events, including:

• a 100% reduction (2.2% to 0.0%) in concurrent severe anemia, severe neutropenia, severe thrombocytopenia compared to patients receiving placebo.
• a 100% reduction (13.3% to 0.0%) in concurrent severe neutropenia and severe thrombocytopenia compared to patients receiving placebo
• a 50% reduction (4.4% to 2.2%) in concurrent severe neutropenia and severe anemia compared to patients receiving placebo
• a 33% reduction (3.3% to 2.2%) in concurrent severe anemia and severe thrombocytopenia compared to patients receiving placebo

Concurrent events were defined as having two or three lineage-specific myelosuppressive events overlap for at least one day.

The ASCO poster, titled, “Impact of Trilaciclib on Multilineage Chemotherapy-Induced Myelosuppression Events in Patients with Extensive-Stage Small-Cell Lung Cancer: Post-Hoc Analyses of Data from Randomized Clinical Trials,” can be found here.

About Small Cell Lung Cancer

In the United States, approximately 30,000 small cell lung cancer patients are treated annually. SCLC, one of the two main types of lung cancer, accounts for about 10% to 15% of all lung cancers. SCLC is an aggressive disease and tends to grow and spread faster than NSCLC. It is usually asymptomatic; once symptoms do appear, it often indicates that the cancer has spread to other parts of the body. About 70% of people with SCLC will have cancer that has metastasized at the time they are diagnosed. The severity of symptoms usually increases with increased cancer growth and spread. From the time of diagnosis, the general 5-year survival rate for people with SCLC is 6%. The five-year survival rates for limited-stage (the cancer is confined to one side of the chest) SCLC is 12% to 15%, and for extensive stage (cancer has spread to the other lung and beyond), survival rates are less than 2%. Chemotherapy is the most common treatment for ES-SCLC. A majority (>90%) of ES-SCLC patients receive first-line chemotherapy at the time of treatment initiation.

About G1 Therapeutics
G1 Therapeutics, Inc. is a commercial-stage biopharmaceutical company focused on the development and commercialization of next generation therapies that improve the lives of those affected by cancer, including the Company’s first commercial product, COSELA® (trilaciclib). G1 has a deep clinical pipeline and is executing a tumor-agnostic development plan evaluating trilaciclib in a variety of solid tumors, including colorectal, breast, lung, and bladder cancers. G1 Therapeutics is based in Research Triangle Park, N.C. For additional information, please visit www.g1therapeutics.com and follow us on Twitter @G1Therapeutics.

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

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “expect,” “plan,” “anticipate,” “estimate,” “intend” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. Forward-looking statements in this press release include, but are not limited to the potential value of, and need for, myeloprotective interventions such as trilaciclib in the management of multilineage myelosuppression, are based on the company’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Factors that may cause the company’s actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in the company’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” sections contained therein and include, but are not limited to, the company’s ability to complete a successful commercial launch for COSELA (trilaciclib); the company’s ability to complete clinical trials for, obtain approvals for and commercialize any of its product candidates other than COSELA (trilaciclib); the company’s initial success in ongoing clinical trials may not be indicative of results obtained when these trials are completed or in later stage trials; the inherent uncertainties associated with developing new products or technologies and operating as a commercial-stage company; and market conditions. Except as required by law, the company assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.

G1 Therapeutics Contacts:

Will Roberts
Vice President, Investor Relations & Corporate Communications
919-907-1944
wroberts@g1therapeutics.com

Rebecca Levine
Director, Corporate Communications and Public Relations
(919) 667-8711
rlevine@g1therapeutics.com

Company Continues to Build as It Prepares for FDA Submission and Clearance of Its Breakthrough Therapy

HistoSonics, the developer and manufacturer of a non-invasive platform and novel sonic beam therapy called histotripsy, announced today the fulfillment of patient enrollment required for regulatory submission of their Edison™ system to the US Food and Drug Administration (FDA). The company plans to use safety and efficacy data from combined parallel company-sponsored trials in an upcoming FDA submission. The ongoing studies providing these data, #HOPE4LIVER US and #HOPE4LIVER Europe, are designed to evaluate the safety and efficacy of using image guided histotripsy to destroy primary and secondary tumors of the liver.

“We are incredibly excited to have achieved this pivotal enrollment milestone in the #HOPE4LIVER clinical trials,” stated Mike Blue, President and CEO of HistoSonics. “It’s a significant achievement for our entire organization and team, including many who have spent the last 20 years advancing the science of histotripsy, and most recently, the development of our novel new platform. Additionally, I want to thank all the trials’ clinical investigators, their teams, and the patients who have entrusted us in these trials as we aim to demonstrate that histotripsy can be safely and effectively used in the liver”, continued Mr. Blue. HistoSonics plans on filing a De Novo submission with the FDA for the destruction of liver tissue using histotripsy later this year in anticipation of achieving US marketing authorization in 2023.

HistoSonics’ image guided sonic beam therapy system uses advanced imaging and proprietary sensing technology to deliver non-invasive, personalized treatments with precision and control. The science of histotripsy uses focused sound energy to produce controlled acoustic cavitation that mechanically destroys and liquifies targeted liver tissue at sub-cellular levels. The company believes that the novel mechanism of action of their proprietary technology provides significant advantages to patients, including the ability of the treatment site to recover and resorb quickly. Uniquely, the 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.

This significant company milestone builds upon strategic accomplishments achieved over the past year including receipt of Breakthrough Device Designation by the FDA, awarding of a unique Category III CPT® Code for histotripsy of the liver by the AMA, and a published payment rate by CMS for the inpatient and outpatient use of histotripsy in the liver.

In addition to the clinical trial milestone, and in preparation for long term growth, HistoSonics has recently expanded its advanced research and development facility in Ann Arbor and is completing final renovations of its 30,000 sq. ft. Minneapolis headquarters. The company is also in the process of adding key personnel across most of the organization and has initiated discussions with the FDA on the next applications of its histotripsy platform.   

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 the continued development of its Edison™ Platform, global clinical studies, and new strategic projects including future clinical applications and platforms. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

For more information please visit: www.histosonics.com/

SOURCE HistoSonics, Inc.

MINNEAPOLIS, May 23, 2022 — HistoSonics Inc., developer of a completely non-invasive platform using the science of histotripsy, today announced an agreement formalizing ongoing efforts to use GE Healthcare’s LOGIQ E10 Series ultrasound imaging platform to power the real time visualization features of HistoSonics’ novel sonic beam therapy.  As part of the agreement and upon market authorization, HistoSonics will distribute GE Healthcare’s LOGIQ E10 Series on a one-to-one basis with its breakthrough liver therapy system.

HistoSonics’ Edison™ system, currently in development, uses the novel science of histotripsy to non-invasively destroy targeted liver tissue. HistoSonics intends to utilize GE Healthcare’s LOGIQ E10 Series, currently the most technologically advanced ultrasound platform for guiding radiology interventions, to provide treating physicians with continuous visualization for key and unique elements of the histotripsy therapy procedure, including planning, monitoring, and immediate post-treatment verification. This agreement is aimed to support HistoSonics’ efforts to launch their Edison^TM system leveraging their deep domain expertise along with GE Healthcare’s world-class ultrasound imaging technologies, digital infrastructure, data analytics and clinical decision support capabilities.

“We are very excited to formalize our imaging partnership with GE Healthcare, which is a key part of bringing our transformative therapy platform, and an entirely new treatment option, to the clinic and to patients,” said Josh Stopek, HistoSonics Vice President of R&D.  “We’ve developed a very collaborative relationship with GE Healthcare and look forward to expanding our efforts to realize the full potential of histotripsy across clinical applications, specialties, and care settings.”

HistoSonics’ non-invasive platform combines advanced imaging and proprietary software to deliver patient specific treatments using histotripsy to mechanically destroy and liquify targeted tissues at a sub-cellular level.  The company believes the novel mechanism of action of their proprietary technology may offer significant advantages to patients, including precise and predictable treatment zones with equivalent treatment effect throughout the entire treated volume. Early clinical and pre-clinical results also suggest that histotripsy largely preserves critical structures such as the liver capsule, and larger vessels and bile ducts within or adjacent to the treated volume of tissue. Additionally, histotripsy enables the treating physicians to monitor the destruction of tissue under continuous real-time visualization and control, unlike any modality that exists today.

The agreement between GE Healthcare and HistoSonics comes as HistoSonics continues enrollment in their U.S. and European #HOPE4LIVER Trials, evaluating the safety and efficacy of histotripsy for the destruction of targeted primary or metastatic liver tumors.  Additionally, the company recently was awarded “Breakthrough Device Designation” by the FDA for histotripsy of liver tissue, validating the company’s vision that histotripsy has the potential to provide advantages over existing therapies such as surgery, radiation therapy and thermal ablation.

The HistoSonics System is investigational and is not available for sale in the United States or Europe. It is limited to investigational use in the approved IDE and European studies.

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 the continued development of its Edison^TM Platform, global clinical studies, and new strategic projects including future clinical applications and platforms. HistoSonics has offices in Ann Arbor, Michigan and Minneapolis, MN.

For more information please visit: www.histosonics.com/

SOURCE HistoSonics, Inc.

• Data from the Phase 1 dose-escalation portion of the Phase 1/2 EXACT study demonstrate XC001 was well-tolerated at all dose levels tested; highest dose level evaluated selected for ongoing Phase 2 portion of the study
• Preliminary efficacy data highlight XC001 potential for patients with refractory angina with no other treatment options
• Treatment strategy is to use local administration to achieve higher gene expression in the heart while minimizing systemic vector circulation and associated side effects
• Completion of Phase 2 enrollment is expected by the end of May 2022

Wayne, PA, May 18, 2022 — XyloCor Therapeutics, a clinical-stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced presentation of initial clinical data from the Phase 1 portion of its ongoing Phase 1/2 clinical trial (EXACT) for refractory angina at the American Association for Thoracic Surgery (AATS) Annual Meeting on May 15, 2022, and at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting on May 18, 2022.

XC001: Locally administered, single-dose gene therapy candidate to address the unmet need in refractory angina
XyloCor’s lead investigational drug, XC001 (encoberminogene rezmadenovec), is under development as a novel approach to treating patients with refractory angina who have exhausted other medical and surgical options. This investigational gene therapy is designed to activate naturally occurring biological pathways by creating new vessels to improve blood flow to areas of the heart not receiving adequate blood supply. This restored blood supply could potentially improve patients’ quality of life by enabling them to resume physical activities and it could reduce episodes of chest pain associated with refractory angina.

In the Phase 1 portion of the EXACT study, 12 subjects with Canadian Cardiovascular Society (CCS) angina class 2-4 without revascularization options were divided into four escalating dose groups with three subjects each. Each subject received 15 epicardial injections of XC001 at one of the four dosage levels. Safety, efficacy and tolerability evaluations were measured as adverse events (AEs), serious adverse events (SAEs) and change from baseline from three to six months post-treatment in exercise capacity, ischemic burden by positron emission tomography (PET) imaging, and patient-reported symptomatology.

No drug-related SAEs, bleeding complications or ventricular arrhythmias were observed in this Phase 1 dose-escalation study. Over a six-month follow up there were a total of 17 SAEs in seven subjects. Eleven SAEs were related to the underlying disease process or other causes. The other six SAEs which occurred in four subjects were judged to be related to the administration procedure, with none of those being unexpected nor resulting in patient death.

“The administration of XC001 appears to have been well-tolerated at all tested doses,” said Nahush Mokadam, M.D., presenting author at AATS, Division Director, Cardiac Surgery, The Ohio State University Wexner Medical Center and Associate Director of the Heart and Vascular Center and site Principal Investigator for this study. “Objective criteria, including results from exercise tolerance tests and PET scans, suggest therapeutic potential.”

Although the Phase 1 portion of the study was primarily focused on safety and Phase 2 dose selection, initial clinical efficacy data appeared promising. Notably, the data showed positive trends in total exercise duration and reductions in patient symptoms and ischemic burden. Although patient numbers are small, preliminary data suggest that response may be correlated to administered dose.

“The preliminary efficacy evaluation suggests a dose response which is encouraging for the development of XC001 as a therapeutic strategy,” said Thomas Povsic, M.D., Ph.D., Professor of Medicine, Duke University School of Medicine and National Principal Investigator for the EXACT study. “We anticipate that the Phase 2 expansion portion of this study, which is testing the highest and most efficacious dose from Phase I, will complete enrollment this month. We are incredibly excited by the potential for this investigational therapy to improve the quality of life for these cardiac patients.”

“In many other gene therapy trials, safety concerns arose due to systemic administration of high viral particle loads,” added Dr. Povsic. “In contrast, because we can inject XC001 directly into the heart, we can dramatically reduce overall viral particle loads and systemic exposure while increasing efficacy.”

EXACT Phase 1/2 Study Data Presentations at AATS and ASGCT
Lead author, Dr. Mokadam presented three-month data from the Phase 1 study in the presentation, Dose Escalation Study of Encoberminogene Rezmadenovec (Adenoviral Vector with Multiple Isoforms of Vascular Endothelial Growth Factor) in Refractory Angina: Phase 1 Results at the AATS Annual Meeting.

Dr. Povsic will present six-month data from the Phase 1 study in the presentation, Preliminary Safety, Tolerability and Efficacy of Direct Epicardial Administration of Encoberminogene Rezmadenovec to Ischemic Myocardium in Patients with Refractory Angina: Six Month Phase 1 Data at the ASGCT 25th Annual Meeting.

About the EXACT Study
The Epicardial Delivery of XC001 Gene Therapy for Refractory Angina Coronary Treatment (EXACT) clinical trial is a Phase 1/2 multicenter, open-label, single-arm trial. Twelve subjects (n=3 per dose cohort) who have refractory angina were enrolled into four ascending dose groups, to be followed by an expansion phase of the trial with 27 additional subjects at the highest tolerated dose. The trial is designed to assess the preliminary safety and efficacy of XC001. The investigational gene therapy is administered directly to the heart muscle through a mini-thoracotomy by an experienced cardiac surgeon. The EXACT trial is being conducted at top cardiovascular research sites across the United
States.

About Chronic Refractory Angina
In the United States, coronary artery disease is a leading cause of death and disability. Chronic angina pectoris occurs when the heart muscle does not receive sufficient oxygen resulting in chest pain. This is usually due to atherosclerotic plaques that block the coronary arteries. Refractory angina is a growing problem that occurs in patients with chronic angina who are symptomatic despite optimal medical therapy and are no longer eligible for mechanical interventions like percutaneous coronary intervention (PCI) and coronary artery bypass grafting (CABG). These patients currently have no treatment options and are frequently highly symptomatic, which severely impacts their quality of life, and may exacerbate comorbidities and cause further deterioration of their health status. Refractory angina results in significant consumption of healthcare resources, including visits to the emergency department as a result of patients’ chest pain. An estimated one million people suffer from refractory angina in the United States.

About XyloCor
XyloCor Therapeutics 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 which 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.

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

OncoMyx Therapeutics, a privately-held oncolytic virus immunotherapy company, today announced the appointment of Robert Williamson as Chief Business and Chief Financial Officer. Mr. Williamson has more than two decades of experience in building, financing, and leading private and public biotechnology companies through critical phases of growth. At OncoMyx, Mr. Williamson will lead the business development and financing strategies to maximize the company’s pipeline of multi-armed myxoma immunotherapies for the treatment of solid tumors and hematological malignancies.

“Rob has a proven track record of establishing successful partnerships and executing financings that propel companies to the next stage,” said Steve Potts, Ph.D., MBA, cofounder and Chief Executive Officer of OncoMyx. “The addition of Rob to the team comes at a key moment for OncoMyx, as we look to advance our lead multi-armed myxoma immunotherapy into clinical trials as a novel platform to deliver multiple cancer-killing payloads in one therapeutic. I look forward to working closely with him to realize the potential of OncoMyx’s unique myxoma platform to become best-in-class components of novel immunotherapeutic regimens and to build OncoMyx into the next great oncology company that delivers lifesaving medicines for many difficult-to-treat cancers.”

“I’m impressed not only with the potential of OncoMyx’s platform to systemically deliver multiple immune-activating and anti-tumor agents, but also the team, who are industry leaders with the experience needed to advance novel targeted treatments for cancer patients into and through the clinic,” said Mr. Williamson. “Furthermore, the compelling preclinical data demonstrating efficacy and safety of the myxoma platform across a broad range of solid and liquid tumors suggests that OncoMyx has the opportunity to make significant breakthroughs in cancer therapy.”

Prior to joining OncoMyx, Mr. Williamson was the Chief Executive Officer of BioTheryX, a protein degradation therapeutics company, where he raised a $100 million crossover round and positioned the company for an initial public offering. Prior to joining BioTheryX, he served as Chief Executive Officer of both PharmAkea (acquired by Galecto) and ATXCo (acquired by Blade Therapeutics), oncology and fibrosis drug development companies financed through a partnership with Celgene. Prior to those companies, Mr. Williamson was Chief Executive Officer of Arriva Pharmaceuticals, President and Chief Operating Officer of Eos Biotechnology (acquired by Protein Design Labs), and Chief Operating Officer of DoubleTwist (acquired in part by Merck and Hitachi). Notably, Mr. Williamson served as an early Director of Pharmasset, where he helped finance, grow, and advance the company into the public markets and through its acquisition by Gilead in 2011 for $11 billion.

About OncoMyx’s Myxoma Immunotherapy Platform

OncoMyx’s multi-armed myxoma virus delivers multiple antitumor immunomodulatory proteins that target critical points in the cancer immunity cycle to modulate the tumor microenvironment and stimulate a robust anti-tumor response. Myxoma is a natural oncolytic virus, selectively infecting and killing a wide range of cancer cell types. It is also inherently highly immuno-interactive, and as a large dsDNA poxvirus, can be engineered to express multiple payloads to treat cancer. Because myxoma is not pathogenic to humans, there is no pre-existing immunity, making it highly amenable to intravenous and repeat dosing.

About OncoMyx Therapeutics

OncoMyx Therapeutics is advancing multi-armed, systemic immunotherapies with the potential to be broadly effective in treating solid tumors and heme cancers. Successful immuno-oncology cancer treatment generally requires combination therapy, and OncoMyx’s myxoma platform is designed to deliver multiple cancer-killing payloads in one therapeutic and orchestrate an immune response capable of treating a wide range of cancers. OncoMyx has assembled a top immuno-oncology team to develop next generation immunotherapies that can have the greatest therapeutic benefit for more cancer patients. The company’s myxoma virus platform is poised to be a best-in-class oncolytic virus approach and was developed based on breakthrough research from Dr. Grant McFadden’s lab that was exclusively licensed from Arizona State University.

Media Contact: Jessica Yingling, Ph.D., Little Dog Communications Inc., jessica@litldog.com , +1.858.344.8091

enGene Inc., a clinical-stage biotechnology company pioneering novel non-viral gene therapies for local administration into mucosal tissues enabled by its proprietary DDX platform, today announced positive results from its LEGEND study, a first-in-human Phase 1/2 clinical trial of EG-70 for the treatment of high-grade Non-Muscle Invasive Bladder Cancer (NMIBC) in patients with carcinoma in situ (CIS) that are BCG-unresponsive.

For over 30 years, the standard treatment for NMIBC has been BCG immunotherapy. However, the 60% disease recurrence and progression rates associated with BCG treatment have created an urgent unmet medical need for NMIBC patients who are left with very few options. Ultimately, the majority of NMIBC patients that do not respond to BCG will undergo radical cystectomy, a procedure in which the bladder is removed concurrently with other surrounding organs.

The results from LEGEND study to-date have shown that enGene’s novel intravesical monotherapy, EG-70, is safe and well tolerated, with encouraging clinical efficacy in high-grade NMIBC patients with CIS in whom BCG has failed. Of the six patients that have reached the 3-month efficacy assessment, five have achieved complete response (CR). This marks an 83% CR rate that is trending best-in-class for an intravesical monotherapy at the end of the first treatment cycle.

“The responses observed with EG-70 are an important step towards NMIBC patients avoiding radical cystectomy,” said Dr. Gary Steinberg, Professor and Director at the Perlmutter Cancer Center and Department of Urology Goldstein Urology Bladder Cancer Program of NYU Langone Health, and lead Principal Investigator for the LEGEND study. “If similar results are observed as the study progresses, I am confident that this novel intravesical monotherapy will make a meaningful impact in the lives of patients with high risk NMIBC.”

“We are thrilled to announce the safety and clinical efficacy of EG-70 in patients with BCG-unresponsive NMIBC who are facing bladder removal as an alternative,” said Jason Hanson, Chief Executive Officer at enGene. “EG-70 was designed ab initio as an intravesical monotherapy that would slot directly into current medical practice to provide ease-of-use and increased access to patients. With the initial results providing a clear best-in-class trajectory, we are confident that EG-70 will be the organ-sparing solution that is a game-changer for patients and physicians.”

Summary of Clinical Results

• The analysis reported that 83% of patients evaluable for efficacy have achieved CR (N=6) at 3-months
• There have been no DLTs or SAEs reported to date
• AEs were limited to transient Grade 1 and Grade 2 Aes such as intermittent pain when urinating, urinary tract pain, bilateral serous otitis, increased nocturia, restlessness, and low back pain

The open label trial has a primary endpoint of safety and tolerability and a key secondary endpoint of CR rate at 3-months. With these initial data, the LEGEND study is slated to successfully meet both primary and secondary endpoints.

About enGene’s DDX platform – EG-70
Based on enGene’s DDX platform, EG-70 is a novel non-viral gene therapy encoding two RIG-I agonists to stimulate the innate immune system, and IL-12 to stimulate the adaptive immune system. By stimulating both arms of the immune system, intravesically administered EG-70 yields remarkable tumor regression in preclinical models of bladder cancer, with induction of effective immunological memory and minimal signs of toxicity.

About the LEGEND study
The LEGEND study, both first-in-human and first-in-class, is an open-label, monotherapy, multi-center, dose-escalation trial evaluating safety and tolerability, pharmacokinetics, pharmacodynamics, and efficacy of EG-70 administered by intravesical instillation. To learn more about the first-in-human clinical trial of EG-70 in BCG-unresponsive NMIBC, please visit ClinicalTrials.gov. For additional information about the LEGEND study, please visit thelegendstudy.com.

About enGene Inc.
enGene Inc. is a clinical-stage biotechnology company developing non-viral gene therapies based on localized delivery of nucleic acid payloads to mucosal tissues. The dually derived chitosan (DDX) platform has a high-degree of payload flexibility including DNA and various forms of RNA with broad tissue and disease applications. In preclinical models, enGene’s DDX technology has been demonstrated to effectively induce expression of therapeutic genes following delivery to the lung, gastrointestinal tract and urinary bladder. Engene has developed scalable GMP-compliant manufacturing of DDX products. http://www.engene.com 

Note regarding forward-looking statements
This press release contains certain “forward-looking statements” that reflect the Company’s beliefs and assumptions based on currently available data and information. These forward-looking statements fall within the meaning of the “safe harbor” provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: “target,” “believe,” “expect,” “will,” “may,” “anticipate,” “estimate,” “would,” “positioned,” “future,” and other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on enGene’s current beliefs, expectations, and assumptions that by definition involve risks, uncertainties, that are difficult to predict and are subject to factors outside of management’s control and that could cause actual results to differ substantially from statements made including but not limited to: risks associated with the success of preclinical studies, clinical trials, research and development programs, as well as regulatory approval processes. Actual results and outcomes may differ materially from those indicated in the forward-looking statements. enGene has no approved drugs available for sale marketing at this time and may never have an approved drug. You are cautioned not to rely on enGene’s forward looking statements, which are only made as of the date hereof. The Company is under no obligation to update these statements.

SOURCE enGene

Congruence Therapeutics, a biotechnology company working at the interface of computational and experimental drug discovery to design novel small molecules for diseases of protein misfolding, announced today the closing of a US$50 million, tranched, series A financing. The financing was led by Amplitude Ventures and Fonds de solidarité FTQ, with participation from Lumira Ventures, Investissement Quebec, OrbiMed Advisors, Driehaus Capital Management, and others.

“Our industry faces a watershed moment where the application of novel computational tools, including machine learning, is poised to disrupt traditional drug discovery,” commented Dr. Clarissa Desjardins. “Our proprietary computational platform, Revenir™, has already demonstrated its ability to uncover novel biophysical features of certain proteins that underpin disease. This financing will allow us to build out a world-class team of ‘drug hunters’ to efficiently design novel small molecules for rare diseases of high unmet medical need, and progress them toward and into the clinic. We are grateful for the strong commitment provided by our syndicate of investors.”

“We are delighted to back Clarissa and the Congruence team as we see the potential of computational approaches to design better drugs faster,” said Nancy Harrison, Venture Partner at Amplitude Ventures.

David Bonita, MD, General Partner of OrbiMed Advisors commented, “We are pleased to support this experienced team and their unique computational tools toward the goal of developing novel, next-generation pharmacological stabilizers, a well validated and commercially successful class of drugs for rare diseases.”

The cornerstone of the Congruence strategy is its proprietary computational platform, Revenir™, a tool for the rational design of stabilizers that circumvents the target validation, protein purification, high throughput screening and hit triage stages. This purpose-built technology uses mathematical modeling, physics, and machine learning to characterize the biophysical defects of misfolded proteins that cause disease, then designs compounds to rescue the mutated proteins by correcting these defects in silico. Congruence is currently deploying Revenir™ in indications with high unmet medical need. At the same time, the Company is building a proprietary database containing the biophysical properties of mutant proteins, which can be used to develop stabilizers for diseases caused by protein families outside of the Company’s current focus.

Congruence Therapeutics was founded in 2021 by Clarissa Desjardins, Ph.D., previously the founder and chief executive officer of Clementia Pharmaceuticals, a company developing therapies for rare pediatric bone diseases and sold to Ipsen in early 2019 for US$1.3 billion.  Clementia’s lead product, Sohonos™ (palovarotene), was recently approved in Canada and is under review at the FDA as the first treatment for Fibrodysplasia Ossificans Progressiva, a debilitating and progressive rare bone disorder.  At Congruence, Dr. Desjardins has put into place a team of ‘drug hunters’ with domain experience in rare disease strategy and clinical development, computational chemistry, medicinal chemistry, and business development.

Concurrent with the financing, Kenneth J. Valenzano, Ph.D. has joined the company as Chief Scientific Officer. Dr. Valenzano most recently served as Senior Vice President of Drug Discovery for Amicus Therapeutics, a leading company historically focused on pharmacological chaperones to treat human genetic diseases. Dr. Valenzano participated in the discovery and development of several small molecule chaperones including Galafold™ (migalastat), the first and only orally-available, small molecule treatment for Fabry’s Disease approved in Europe and in the US in 2018.

About Congruence Therapeutics
Congruence Therapeutics is a biotechnology company working at the interface of computational and experimental drug discovery to design novel small molecules for diseases of protein misfolding.  Using structural bioinformatics, computational chemistry, and machine learning, the Company is deploying an in silico platform that enables the design of new pharmacological stabilizers, a validated class of small molecules, at an unprecedented speed and scale.

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

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

Media Contact
David Rosen
Argot Partners
David.rosen@argotpartners.com 
212-600-1902

SOURCE Congruence Therapeutics

XyloCor Therapeutics, a clinical‑stage biopharmaceutical company developing novel gene therapies for cardiovascular disease, today announced that it has appointed Elizabeth Tarka, M.D.  as Chief Medical Officer and A. Brian Davis as Chief Financial Officer. These experienced pharmaceutical industry executives will enhance the company’s clinical development, operational, and financial capabilities and drive its ongoing growth. “I am delighted to welcome Liz and Brian to the leadership team at XyloCor,” said Al Gianchetti, President and Chief Executive Officer. “Liz is a proven R&D leader who brings a strong track record managing late-stage clinical development programs with particular expertise in cardiovascular medicine. Brian will leverage his extensive management and finance experience in both private and public biotech companies to accelerate XyloCor’s strategic and corporate objectives. Their collective experience will drive XyloCor toward our objectives as we focus on advancing our pipeline of transformative gene therapies intended to improve the lives of people with cardiovascular disease.”

Dr. Tarka is a cardiologist with over 20 years of experience in the pharmaceutical and biotechnology industry.  She has dedicated her career to the development of innovative therapies that improve human health.  Dr. Tarka’s experience includes leadership roles across all phases of late-stage clinical development and a track record of effectively partnering with stakeholders to enable the successful execution of clinical trials. She joins the company from Idera Pharmaceuticals where she served as CMO. Before that, Dr. Tarka was Vice President, Clinical Development at Complexa, Inc., a clinical stage biopharmaceutical company focused on life-threatening fibrosis and inflammatory diseases. Earlier in her career, she served as Clinical Program Leader for Xarelto^® (rivaroxaban) at Janssen Pharmaceuticals, where she was responsible for the design, implementation, and medical oversight for large multinational trials. Prior to her tenure at Janssen, Dr. Tarka worked at GlaxoSmithKline in the Metabolic Pathways and Cardiovascular Therapeutic Area. She has been on the faculty and had numerous major teaching and clinical responsibilities at the University of Pennsylvania and affiliated hospitals. She is trained in Cardiology and Internal Medicine and has published in a number of peer-reviewed journals. Dr. Tarka earned a BA in Biochemistry and an MD from the University of Pennsylvania where she also completed her residency and fellowship training.

Mr. Davis joins XyloCor with a proven background as a seasoned financial executive, including over 15 years of experience as a CFO for publicly traded, commercial- and clinical-stage biopharmaceutical companies, and nearly 30 years as a financial professional in the life sciences industry. He has extensive expertise in fundraising, financial strategy, negotiating strategic transactions involving acquisition and disposition of commercial and clinical-stage assets, shareholder relations, and SEC accounting, reporting, and compliance.  Mr. Davis has raised over $600 million in public and private equity financings, including leading an initial public offering, and nearly $200 million in debt financings. Most recently, he was CFO at Verrica Pharmaceuticals, where he held managerial responsibility for executing equity and debt financings, analyst and shareholder relations, financial aspects of commercial launch preparation, business development, finance, accounting, tax, and treasury. Before that, Mr. Davis had similar duties as CFO at Strongbridge Biopharma plc, Tengion, Inc., and Neose Technologies, Inc. Mr. Davis is a Certified Public Accountant. He earned an MBA from The Wharton School, University of Pennsylvania and an undergraduate degree in Accounting from Trenton State College.

About XyloCor

XyloCor Therapeutics 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 currently being investigated in a Phase 2 clinical trial for patients with refractory angina for which 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, MD, and Todd Rosengart, MD, has an exclusive license from Cornell University. For more information, visit www.xylocor.com.

Edesa Biotech, Inc. (NASDAQ:EDSA), a clinical-stage biopharmaceutical company focused on inflammatory and immune-related diseases, today reported that the company has received approval from Health Canada to test its monoclonal antibody candidate, designated EB05, as a rescue therapy for critically ill patients in the Phase 3 part of a Phase 2/3 clinical study.

Edesa believes that EB05 regulates the overactive and dysfunctional immune response associated with Acute Respiratory Distress Syndrome (ARDS), a life-threatening form of respiratory failure that accounts for ~10% of all ICU admissions (pre-pandemic) and is the leading cause of death among COVID-19 patients. Approval of the company’s Phase 3 study design follows favorable Phase 2 results, which demonstrated compelling preliminary evidence of EB05’s ability to reduce mortality in the sickest patients. Among the results, critically ill hospitalized COVID-19 patients given EB05 plus standard of care treatment had a 68.5% reduction in the risk of dying when compared to placebo plus standard of care at 28 days.

“Health Canada’s approval represents a significant milestone in our goal of demonstrating the broad potential utility of EB05 in a critical care setting,” said Par Nijhawan, MD, Chief Executive Officer of Edesa. He noted that there’s an urgent need for therapeutics that are agnostic to SARS-CoV2 variants.

“The growing number of COVID variants – and the inherent limits of vaccines and anti-viral drugs – have highlighted the importance of agnostic therapies, like EB05, that target the patient’s immune response rather than the virus. While the Phase 3 study is designed to confirm the Phase 2 results, the preliminary data shows that EB05 has already saved lives. Perhaps just as important, this innovative technology is providing greater confidence that COVID can be well managed one day, like other endemic diseases,” said Dr. Nijhawan.

Edesa reported that the Phase 3 double-blind study is designed to assess the efficacy and safety of EB05 among critically ill COVID-19 patients receiving extracorporeal membrane oxygenation (ECMO) and/or invasive mechanical ventilation plus organ support (IMV+), defined as Level 7 on the World Health Organization’s COVID-19 Severity Scale. The primary endpoint for the Level 7 patients will be 28-day mortality. Ventilator free days and 60-day mortality will also be measured among other secondary endpoints. The amended trial protocol design calls for approximately 315 evaluable subjects. The company has opened patient enrollment under the amended protocol.

Health Canada reviewed Edesa’s study design amendment and approved it by issuing a “Notice of Authorization,” which allows the company to proceed with its planned study design. Under the amended protocol, Edesa also has the option to complete a separate study group of less severe patients receiving only invasive mechanical ventilation (WHO COVID-19 Severity Scale Level 6), which would be evaluated independently.

Edesa has filed similar protocol amendments with the U.S. Food and Drug Administration (FDA) as well as other jurisdictions. In the U.S., the company is currently in discussions with the FDA on the design of the final Phase 3 protocol.

About EB05
EB05 is a monoclonal antibody designed to inhibit toll-like receptor 4 (TLR4) signaling – an important mediator of inflammation responsible for acute lung injury that has been shown to be activated by SARS-CoV2, SARS-CoV1 and Influenza viruses. In September 2021, the company reported that an independent monitoring board for the Phase 2/3 study concluded that “a clinically important efficacy signal” was detected and that the Phase 2 study “met its objective.” The monitoring board further recommended continuation of the study into a Phase 3 confirmatory trial. Edesa’s Phase 2 study of EB05 in hospitalized COVID-19 patients was funded in part by a C$14 million grant from the Canadian Government’s Strategic Innovation Fund.

About ARDS
Acute Respiratory Distress Syndrome is the leading cause of death in COVID-19 patients. The U.S. Centers for Disease Control (CDC) reports that 20% to 42% of hospitalized COVID-19 patients develop ARDS, which increases to 67% to 85% for patients admitted to the ICU. Mortality among patients admitted to the ICU ranges from 39% to 72% depending on the study and characteristics of patient population, according to the CDC. ARDS involves an exaggerated immune response leading to inflammation and injury to the lungs that prevents the lungs from oxygenating blood and ultimately deprives the body of oxygen. For moderate to severe cases, there are currently few meaningful treatments, other than supplemental oxygen and mechanical ventilation, and patients suffer high mortality rates. In addition to virus-induced pneumonia, ARDS can be caused by smoke/chemical inhalation, sepsis, chest injury and other causes. Prior to COVID-19, ARDS accounted for 10% of intensive care unit admissions, representing more than 3 million patients globally each year.

About Edesa Biotech, Inc.
Edesa Biotech, Inc. (NASDAQ:EDSA) is a clinical-stage biopharmaceutical company focused on developing innovative treatments for inflammatory and immune-related diseases with clear unmet medical needs. The company’s two lead product candidates, EB05 and EB01, are in later stage clinical studies. EB05 is a monoclonal antibody therapy that we are developing as a treatment for Acute Respiratory Distress Syndrome (ARDS). ARDS is a life-threatening form of respiratory failure, and the leading cause of death among COVID-19 patients. Edesa is also developing an sPLA2 inhibitor, designated as EB01, as a topical treatment for chronic allergic contact dermatitis (ACD), a common, potentially debilitating condition and occupational illness. By targeting sPLA2 with enzyme inhibitors – at the inception of inflammation rather than after inflammation has occurred – Edesa believes that drugs based on this technology could provide a powerful anti-inflammatory therapeutic strategy for treating diverse inflammatory/allergic conditions. The company is based in Markham, Ontario, Canada, with a U.S. subsidiary located in Southern California. Sign up for news alerts. Connect with us on Twitter 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 EB05 could regulate the overactive and dysfunctional immune response associated with ARDS; the company’s belief in the broad potential life-saving impact of its EB05 monoclonal antibody candidate; the company’s belief that there’s a critical need for therapeutics that are agnostic to SARS-CoV2 variants; 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, such as COVID-19. 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.
(805) 488-2800 ext. 150
investors@edesabiotech.com

SOURCE: Edesa Biotech

LQT Therapeutics, Inc. (“LQTT”) is pleased to announce a collaboration with the laboratory of Dr. Donald McDonnell of Duke University School of Medicine to evaluate LQTT SGK1 inhibitors as potential treatment for patients with resistant prostate and breast cancer. Despite the approval of recent treatments for patients with these cancer types, resistance often emerges, and new treatments are needed. Recently, numerous scientists have produced evidence of SGK1’s role in the resistance of various cancers to existing therapies, and Dr. McDonnell’s laboratory has extensive experience in understanding this specific role. “The research in our group is focused on the development and application of mechanism-based approaches to identify novel therapeutics for use in the treatment and prevention of hormonally responsive cancers. We are interested in estrogen and androgen receptors (AR) as therapeutic targets in breast and prostate cancers and in defining how these receptors influence the pathogenesis of these diseases. The utility of targeting processes downstream of AR, such as SGK1 inhibition, could provide an alternate approach to therapy. In our laboratories we have demonstrated SGK1 inhibition blocks the effect of androgens in known cancer cell proliferation assays, and we believe SGK1 inhibition is a promising target in treatment resistant cancer. We are excited to evaluate LQTT’s platform of SGK1 inhibitors in our models of treatment resistant cancers,” said Dr. Donald McDonnell. Research at LQTT has validated the effects of SGK1 inhibition on cell proliferation pathways in prostate and colorectal cancers. Often difficult to treat cancers may be resistant from the start, or develop resistance during treatment with existing therapies through a variety of mechanisms. The recent approval of novel targeted therapies against cancer also generated additional pathways of resistance, and new opportunities to develop precision treatments to address this resistance. Additional roles for the activation of SGK1 in various cancer pathways continue to be discovered, and LQTT intends to explore these pathways and continues to advance a series of compounds with promise to address the treatment challenges of these resistant cancers. “We are looking forward to working with Dr. McDonnell and his team to further develop SGK1 inhibitors in different models of resistant cancers” said Dr. Eric Campeau, Vice President of Translational Research at LQT Therapeutics, Inc. “Dr. McDonnell and his scientific team have demonstrated the role of SGK1 in promoting tumor growth and overcoming resistance to anticancer treatments. We are hopeful SGK1 inhibition will prevent cancer progression in animal models of these resistant cancers. Our collaboration will aim to identify the optimal SGK1 inhibition profile required for anti-tumor activity, as well as the patient population that could most benefit from such therapy.”

About LQT Therapeutics, Inc.
LQT Therapeutics, Inc. is pioneering a precision medicine approach to treat patients with Long QT Syndrome and potentially other arrhythmias based on research from Beth Israel Deacons Medical Center, Massachusetts General Hospital, and Sanofi, S.A. By combining leading-edge cardiovascular genetics and diagnostics with recent advances in the understanding of the role of SGK1, LQT Therapeutics seeks to make a meaningful difference in the lives of people suffering from Long QT Syndrome and resistant cancers. Launched in 2019 by the Fonds de Solidarité FTQ. LQT Therapeutics, Inc. was founded by world-class experts in cardiovascular disease, cardiac muscle biology and drug development. For more information, please visit www.lqttrx.com

Media Inquiries:
Daphne Doucet | daphne@lqttrx.com | +1 (514) 973-0915

Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, today announced nomination of the first targeted alpha therapy (TAT) candidate under the Company’s collaboration agreement with AstraZeneca (LSE/STO/Nasdaq: AZN). Both companies will jointly develop through a Phase 1 study the novel TAT which utilizes Fusion’s Fast-Clear™ linker technology to radiolabel an AstraZeneca-owned bispecific antibody with the alpha-emitting isotope, actinium-225.

“Fusion is at the forefront developing a new wave of cancer therapies using alpha-emitting medical isotopes and various targeting vehicles,” said John Valliant, Ph.D. “One of the pillars of our platform is to create next-generation immunoconjugates, and we are excited to be working with AstraZeneca to pursue a novel radioimmunoconjugate for oncology. We had strong alignment among our joint development committee to move this novel TAT forward into a planned Phase 1 study and we look forward to sharing more information as we approach the investigational new drug application (IND) filing.”

Under the terms of the previously announced collaboration agreement, Fusion will be operationally responsible for preclinical development through first-in-human studies, while AstraZeneca will be responsible for subsequent clinical development. The companies will share costs equally through clinical development. IND enabling studies are ongoing.

About Fusion

Fusion Pharmaceuticals is a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines. Employing a proprietary Fast-Clear™ linker technology, Fusion connects alpha particle emitting isotopes to various targeting molecules in order to selectively deliver the alpha emitting payloads to tumors.

Fusion’s lead program, FPI-1434 targeting insulin-like growth factor 1 receptor, is currently in a Phase 1 clinical trial. The pipeline includes FPI-1966 targeting the fibroblast growth factor receptor 3 (FGFR3) and FPI-2059, a small molecule recently acquired from Ipsen, targeting neurotensin receptor 1 (NTSR1).

In addition to a robust proprietary pipeline, Fusion has a collaboration with AstraZeneca to jointly develop up to three novel targeted alpha therapies (TATs) and explore up to five combination programs between Fusion’s TATs and AstraZeneca’s DNA Damage Response Inhibitors (DDRis) and Immuno-Oncology (IO) agents.

About AstraZeneca

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines, primarily for the treatment of diseases in three therapy areas – Oncology, Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com.

Forward Looking Statements

This press release contains “forward-looking statements” for purposes of the safe harbor provisions of The Private Securities Litigation Reform Act of 1995, including but not limited to the statements regarding Fusion Pharmaceuticals Inc.’s (the “Company”) future business and financial performance. For this purpose, any statements contained herein that are not statements of historical fact may be deemed forward-looking statements. Without limiting the foregoing, the words “expect,” “plans,” “anticipates,” “intends,” “will,” and similar expressions are also intended to identify forward-looking statements, as are expressed or implied statements with respect to the Company’s potential drug candidates, including any expressed or implied statements regarding the successful development of the novel TAT to be developed with AstraZeneca and the potential for it to move into Phase 1 clinical studies. Actual results may differ materially from those indicated by such forward-looking statements as a result of risks and uncertainties, including but not limited to the following: there can be no guarantees that the Company will advance any clinical product candidate or other component of its potential pipeline to the clinic, to the regulatory process or to commercialization; management’s expectations could be affected by unexpected regulatory actions or delays; uncertainties relating to, or unsuccessful results of, research, development or clinical activities; the Company’s ability to obtain additional funding required to conduct its research, development and commercialization activities; changes in the Company’s business plan or objectives; competition in general; and the Company’s ability to obtain, maintain and enforce patent and other intellectual property protection for its product candidates and its discoveries. Such forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. These and other risks which may impact management’s expectations are described in greater detail under the heading “Risk Factors” in the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2021 as filed with the SEC and in any subsequent periodic or current report that the Company files with the SEC. All forward-looking statements reflect the Company’s estimates only as of the date of this release (unless another date is indicated) and should not be relied upon as reflecting the Company’s views, expectations or beliefs at any date subsequent to the date of this release. While Fusion may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so, even if the Company’s estimates change.

SOURCE Fusion Pharmaceuticals Inc.

Honor Recognizes Companies Advancing Innovative Solutions to Improve Women’s Health and Safety in Emerging Markets

Antiva Biosciences, a biopharmaceutical company developing novel, topical therapeutics for the treatment of pre-cancerous lesions caused by human papilloma virus (HPV) infection, today announced that the World Bank Group and the Consumer Technology Association (CTA) have named it one of the winners of The Global Women’s HealthTech Awards. These awards recognize innovative companies that leverage technology to improve women’s health and safety in emerging markets. Antiva was one of the winners selected by a panel of industry experts from entries submitted by over 70 companies from 35 countries. Antiva’s lead development candidate, ABI-2280, is being developed as a potential treatment for high-grade cervical intraepithelial neoplasias (HSIL, CIN 2,3). ABI-2280 is a prodrug of an acyclic nucleoside phosphonate with known potent antiviral activity which works by directly blocking HPV replication and inducing apoptosis in HPV-infected lesions, while sparing normal cells. Antiva has leveraged its development expertise to create a topical formulation of the compound that is rapidly taken up into epithelial cells, avoiding the potential systemic toxicity often seen when potent antiviral drugs are delivered systemically. The company recently dosed the first subject in its Phase 1 clinical trial of ABI-2280 and expects data from the study to be available in mid-2022. “We are honored to be recognized by the World Bank Group and CTA and offer our congratulations to all of the other winners who are working to improve women’s health around the world. At Antiva, we have made women’s and global health a central tenet of our mission and our work with ABI-2280 is a perfect example of our efforts in this area,” said Gail Maderis, president and chief executive officer of Antiva. “To this end, we are excited to have recently advanced ABI-2280 into clinical development. We believe that this topical therapy, together with innovative screening and diagnostic tools, can offer a ‘see and treat’ solution to prevent cervical cancer, a disease which kills more than 300,000 women annually.”

About HPV-Related Diseases and Cervical Cancer
Human Papilloma Virus (HPV) is so common that nearly all sexually active men and women are infected with the virus at some point in their lives. While many of these are transient infections which the body is capable of fighting off, the infections that persist are known to drive the formation of malignancies, including cervical, anal, vulvar, penile, and head and neck cancers. The introduction of prophylactic vaccines for HPV was a major step forward in the fight against HPV-associated cancers by preventing infection by certain high-risk HPV subtypes. However, due to low adoption rates in the US, EU, and Japan, and limited access to the vaccines in developing countries, HPV infections and the disease states driven by such infections remain a major unmet clinical need. Globally, cervical cancer is the fourth most common cancer in women and as such represents a major public health problem. According to the World Health Organization, an estimated 570,000 women were diagnosed with cervical cancer worldwide and approximately 311,000 women died from the disease in 2018.

About Antiva Biosciences
Antiva Biosciences, Inc. is a clinical-stage biopharmaceutical company developing novel, topical therapeutics for the treatment of diseases caused by HPV infection. The company, based in South San Francisco, was founded in 2012 by Dr. Karl Hostetler of The University of California San Diego. The company’s lead drug candidate, ABI-2280 is initially being developed as a topical treatment for high-grade cervical intraepithelial neoplasias (HSIL, CIN 2,3). Antiva believes that development of ABI-2280 may also be expanded to include other pre-cancers attributed to HPV, such as vulvar (VIN 2,3) and anal (AIN 2,3) neoplasias.

For more information, please visit: www.antivabio.com.

Contact Information:

Gail Maderis
Antiva Biosciences, Inc.
650-822-1401
gmaderis@antivabio.com
Tim Brons
Vida Strategic Partners (media)
646-319-8981
tbrons@vidasp.com

Closing the gap between industry and academia to place patients directly at the center of research efforts. 

LQT Therapeutics, a privately-held biopharmaceutical company advancing novel SGK1 kinase therapeutics, today announced that the project, “SILENCE-LQTS: SGK1 inhibition as a novel therapeutic approach in Long QT Syndrome” was selected for funding by the European Joint Program on Rare Diseases (EJP RD). The EJP RD is a collaboration of over 130 institutions from 35 countries, working together to create a comprehensive, sustainable ecosystem between research, care, and medical innovation. 

Congenital Long QT Syndrome (LQTS) is a rare inherited disorder associated with life- threatening arrhythmias and sudden cardiac death (SCD) in relatively young and otherwise healthy individuals. LQTS has a heterogeneous genetic basis, usually impacting one of 3 key cardiac ion channels caused by mutations in distinct genes. Current symptom-directed therapies aimed at reducing arrhythmia triggering events, including lifestyle changes, beta blockade, and left cardiac sympathetic denervation, only partly prevent arrhythmic events, and syncope and SCD still occurs in a substantial number of LQTS patients. 

Within SILENCE-LQTS, the EJP RD consortium will investigate a novel, mechanism- targeted therapy, comprising pharmacological inhibition of the serum and glucocorticoid regulated kinase-1 (SGK1). In contrast to current symptom-directed therapies, this novel approach is designed to correct the pro-arrhythmic alterations in ion channel homeostasis caused by the underlying genetic defect. Efficacy of a SGK1 inhibitor, developed by LQT Therapeutics, will be systematically tested to establish the anti-arrhythmic potential of SGK1 inhibition, paving the way for future clinical application aimed at preventing symptoms and SCD in LQTS. 

“Because current symptom-directed therapies aimed at reducing arrhythmia triggering events, including lifestyle changes, beta blockade, and left cardiac sympathetic denervation, only partly prevent (lethal) arrhythmic events, there remains a crucial unmet need for novel, more efficacious, mechanism-driven therapies,” said Arthur Wilde, MD, PhD, Amsterdam University Medical Centers and EJP RD Consortium Member. 

LQT Therapeutics has shown that SGK1 inhibition in cardiomyocytes from patients with different LQT genetic mutations leads to shortening of the action potential duration. If this behavior translates in vivo, there is the potential to change the lives of patients with Congenital Long QT Syndrome, many of whom rely on medications that are not fully effective and have unpleasant side effects or implanted cardiac defibrillators. 

“This award represents a significant milestone for LQT Therapeutics as we advance our platform of SGK1 inhibitors,” said Debra Odink, PhD, Chief Development Officer of LQT Therapeutics. “We are grateful to the EJP RD for this award and the opportunity of working with the consortium. Advancing therapeutics for people with Long QT Syndrome is a focus of LQT Therapeutics and we aim to restore normal life to those that live in fear of developing a potentially life-threatening arrhythmia.” 

About LQT Therapeutics, Inc. 

LQT Therapeutics, Inc. is pioneering a precision medicine approach to treat patients with Long QT Syndrome and other arrhythmias based on research from Beth Israel Deacons Medical Center, Massachusetts General Hospital, and Sanofi, S.A. By combining leading- edge cardiovascular genetics and diagnostics with recent advances in the understanding of the role of SGK1, LQT Therapeutics seeks to make a meaningful difference in the lives of people suffering from Long QT Syndrome and resistant cancers. Launched in 2019 by the Fonds de Solidarité FTQ. LQT Therapeutics, Inc. was founded by world-class experts in cardiovascular disease, cardiac muscle biology and drug development. For more information, please visit www.lqttrx.com 

About the EJP RD 

The European Joint Program on Rare Diseases (EJP RD) is a program aiming to create an effective rare diseases research ecosystem for progress, innovation and for the benefit of everyone with a rare disease. We support rare diseases stakeholders by funding research, bringing together data resources and tools, providing dedicated training courses, and translating high quality research into effective treatments. Consortium members include Arthur Wilde (Amsterdam University Medical Center), Lia Crotti (Istituto Auxologico Italiano), Katja Odening (University of Bern) and Lior Gepstein (Technion – Israel Institute of Technology), along with the patient advocacy organization, Hart4Onderzoek (Hart4Research) of the Netherlands. 

—

LQT Therapeutics Inc.

500, boulevard Cartier Ouest

Laval (Québec)  H7V 5B7

Endotronix, Inc., a digital health and medical technology company dedicated to advancing the treatment of heart failure (HF), today announced the U.S. Food and Drug Administration (FDA) has granted approval for an amendment to the company’s PROACTIVE-HF study, a pivotal Investigational Device Exemption (IDE) trial investigating the Cordella Pulmonary Artery (PA) Pressure Sensor¹, shifting the design from a randomized control study to a single-arm study. Following the 2021 update to the 2017 ACC Expert Consensus Decision Pathway for Optimization of Heart Failure Treatment and the recently presented GUIDE-HF data, the FDA’s decision underscores the clinical benefits of PA pressure-guided HF management for New York Heart Association (NYHA) class III heart failure patients^2,3.  The new PROACTIVE-HF trial design ensures the over 100 previously enrolled patients have immediate access to the proven clinical benefits of PA pressure-guided therapy and expands the study to include clinical sites across Europe, as well as the U.S. The company is currently implementing the updated PROACTIVE-HF study design at trial sites worldwide.

“The FDA’s decision to approve the amended study design further validates what we are seeing in clinical practice. PA pressure-guided heart failure management is quickly becoming standard of care for NYHA class III patients based on the growing body of evidence supporting this approach,” commented Dr. Liviu Klein, Section Chief, Advanced Heart Failure, Mechanical Circulatory Support, Pulmonary Hypertension and Heart Transplant at UCSF and National Principal Investigator of the PROACTIVE-HF trial. “Understanding changes in PA pressure data is key to properly implementing guideline directed medical therapy and keeping these patients out of the hospital. The Cordella HF System and Sensor offers the next gen technology in PA pressure sensing and will play a critical part in how heart failure patients are managed moving forward.” 

The single-arm study design maintains similar safety and efficacy endpoints as the original study design, with the primary efficacy endpoint designed to demonstrate low rates of all-cause mortality and HF hospitalizations in the high-risk NYHA class III patient cohort. Under the updated study design, the PROACTIVE-HF trial will enroll over 400 NYHA class III patients at more than 100 sites worldwide. The trial is expected to complete enrollment in the second half of this year.

“The FDA’s decision is great news for our PROACTIVE-HF study patients – they now have immediate access to technology that is designed to keep them healthier and out of the hospital. Furthermore, it is an important acknowledgement for the therapeutic category and the benefits that PA pressure-guided heart failure management enables,” stated Harry Rowland, CEO of Endotronix. “Our team and clinical partners have been energized by the announcement and it obviously has a significant impact on our ability to bring the Cordella Sensor to market sooner in the U.S. and Europe.”

About Endotronix
Endotronix, Inc., a medical technology company, delivers an integrated platform that provides comprehensive, reimbursable health management innovations for patients suffering from advanced heart failure. Their solution, the Cordella™ Heart Failure System, includes a cloud-based disease management data system and at home hemodynamic management with a breakthrough implantable wireless pulmonary artery pressure sensor for early detection of worsening heart failure.  Learn more at www.endotronix.com.

The Cordella System, without the sensor, is available for commercial use in the U.S. and E.U. and is currently in cardiology centers across the U.S. The Cordella PA Sensor is currently under clinical investigation in Europe (SIRONA II CE Mark Trial) and the U.S. (PROACTIVE-HF IDE Trial) and is not currently available for commercial use in any geography.

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.

1. CAUTION: Investigational device. Limited by Federal law to investigational use.
2. Maddox TM, Januzzi JL et al. 2021 J Am Coll Cardiol. 2021 Feb, 77 (6) 772-810.
3. Lindenfeld J, Zile MR, et al. Lancet. 2021 Sept 11; 398 (10304):991-1001.

MEDIA CONTACT:
Carla Benigni
SPRIG Consulting, LLC
+1 (847) 951-7430
carla@sprigconsulting.com 

SOURCE Endotronix, Inc.

Specific Biologics Inc. (“Specific”) today announced that it has closed its first institutional financing, led by Lumira Ventures and adMare BioInnovations which have recently established an innovative partnership to build Canadian life sciences companies. Gene editing holds the potential to cure a wide range of genetic diseases by altering DNA in the genome inside cells – but today’s gene editors have limitations. To better realize the significant opportunity of this therapeutic approach, and allow for its application to new therapeutic areas, Specific Biologics has developed the unique next-generation Dualase™ gene editing platform which can more accurately alter DNA sequences by cutting DNA in two spots as a single molecule. Specific has demonstrated the benefit of this two-site mechanism in proof-of-concept studies in disease-relevant models.

“Based on our early work with Dualase™, we believe it will have broad utility across a number of diseases with high unmet need,” said Dr. Brent Stead, CEO of Specific Biologics Inc. “This investment by Lumira and adMare, two recognized leaders in life sciences, will help accelerate the advancement of our promising platform, and a pipeline of differentiated Dualase™-based gene editing therapeutics in a diverse range of indications.”

“Current gene editing technologies are constrained by low on-target editing efficiency and high unintended off-target effects. Data suggest that Dualase™ has the potential to overcome these well-known challenges in relevant therapeutic indications,” noted Dr. Daniel Hetu, Managing Director at Lumira Ventures. “Built on the work of Dr. David Edgell at Western University’s Schulich School of Medicine & Dentistry, Specific Biologics represents another example of how Canadian health science innovators are at the leading edge of treatment modalities,” added Nikhil Thatte, Principal at Lumira Ventures. “We are excited to work alongside adMare and the Specific team to grow the company and bring transformative therapies to patients.”

“The investment in Specific Biologics stems from a new partnership between adMare and Lumira Ventures,” commented Dr. Frédéric Lemaître Auger, Managing Partner, Investments with adMare. “adMare builds life sciences companies by partnering with and investing in Canadian innovators and entrepreneurs. The partnership with Lumira recognizes the complementary nature of our organizations’ resources, expertise, and roles in the ecosystem, and has resulted in our firms aligning to co-identify, support, and advance opportunities of mutual interest in a range of development stages. By working together in this way, adMare and Lumira are committed to expanding our contribution to the growth of the industry, and to making Canada a global life sciences leader. The investment in Specific Biologics is a great example of how we are effectively doing that – helping scale a highly-promising young company founded around world-leading Canadian research.”

About Specific Biologics Inc.

Specific Biologics Inc. (“Specific”) is a venture-backed early-stage biotechnology company on a mission to develop novel gene editing technologies to treat diseases through precision gene editing. Our two-site Dualase™ platform gene editors cut DNA in a way that optimally exploits the cell’s naturally occurring DNA repair pathways. This enables two gene editing outcomes, precise DNA deletions to disrupt genes or increased repair to correct genes. Specific also develops lipid nanoparticles to deliver the gene editor to target cells and is developing a pipeline of Dualase™-based therapeutics in areas of high unmet medical need. To learn more, visit www.specificbiologics.com.

About Lumira Ventures

Lumira Ventures is a North American healthcare venture capital firm with decades of experience of investing in and helping to build transformative biomedical companies. We are a multi-stage investor that partners with mission-driven entrepreneurs and like-minded investors to build innovative healthcare companies. These companies are harnessing rapidly evolving innovations in genomics, cell therapy, gene therapy, bioengineering, robotics and artificial intelligence to develop high impact, often transformative products for patients while generating exceptional returns for their investors and meaningful economic value to society. To date, Lumira’s companies have brought dozens of biomedical innovations to the market, impacting the lives of patients worldwide. Lumira Ventures manages its activities from offices in Toronto, Montréal, Vancouver and Boston. For more information, please visit www.lumiraventures.com

About adMare BioInnovations

With a wealth of scientific discovery, Canada is primed to be a global leader in life sciences. To realize this potential, adMare uses its scientific and commercial expertise, specialized R&D infrastructure, and investment capital to build investable companies, robust ecosystems and industry-ready talent – and re-invests its returns back into the Canadian industry to ensure it is sustained for the long-term. adMare’s 25+ portfolio companies have attracted more than $1.4billion of investment, have a combined value of over $3 billion, and employ more than 900 Canadians. Its Innovation Centres are home to 35+ life sciences companies, contract research organizations, and specialized commercialization support organizations; and the adMare Academy has trained 500+ alumni – 95% of whom are now working to build the Canadian life sciences industry. For more information, please visit www.admarebio.com

Media Inquiries:
info@specificbiologics.com
Ph: 647-773-4591

SOURCE Specific Biologics Inc.

www.specificbiologics.com

Antios Therapeutics, Inc. (“Antios”) and Arbutus Biopharma Corporation (Nasdaq: ABUS) today announced that the first patient has been dosed in a triple combination treatment in patients with chronic hepatitis B virus (HBV) infection. A single cohort in the ongoing Antios Phase 2a SAVE-1 (Sustained Anti-Viral Efficacy) clinical trial will evaluate a triple combination of Antios’ proprietary active site polymerase inhibitor nucleotide (ASPIN), ATI-2173, Arbutus’ proprietary GalNAc delivered RNAi therapeutic, AB-729, and tenofovir disoproxil fumarate (TDF), a nucleotide reverse transcriptase inhibitor.

The multi-center, double-blind, combination clinical trial plans to enroll 40 patients including a cohort of 10 patients with chronic HBV infection assigned 8:2 to active drugs (ATI-2173+AB-729) or matching placebos. The active drugs (ATI-2173+AB-729) or placebos will be administered in combination with 300 mg of tenofovir disoproxil fumarate. ATI-2173 and tenofovir disoproxil fumarate will be administered orally and by injections once daily for 90 days. AB-729 will be administered by subcutaneous injection at Day 28 and Day 90. Following this 90-day treatment period, patients will be followed-up for safety and sustained antiviral responses for six additional months.

“The need for a functional cure for HBV is clear. Current therapies only partially suppress HBV replication and require ongoing treatment, adding to patient burden,” said Douglas Mayers, M.D., Chief Medical Officer and Co-Founder of Antios. “By combining AB-729 with ATI-2173 and tenofovir disoproxil fumarate, we hope to reduce hepatitis B surface antigens and sustain HBV DNA suppression while off treatment.”

ATI-2173 is the only ASPIN in clinical development and pre-clinical data to date for ATI-2173, alone or combined with TDF, indicate the potential for sustained HBV DNA suppression off treatment, unique among approved nucleos(t)ides and investigational anti-HBV therapies.

Dr. Gaston Picchio, Chief Development Officer of Arbutus, commented, “In our common endeavor to find a cure for chronic HBV, we are eager to evaluate the combination of these two unique assets which we expect will allow us to further understand the potential benefits of such combination therapy for patients with chronic HBV.”

AB-729 is an RNAi therapeutic that inhibits viral replication and reduces all HBV antigens. In clinical trials to date, AB-729 has demonstrated positive safety and tolerability data as well as meaningful reductions in HBV surface antigen.

About ATI-2173

ATI-2173, Antios Therapeutics’ lead once-daily, oral drug candidate for treating HBV, is an investigational phosphoramidate prodrug of clevudine monophosphate. ATI-2173 has the potential, if approved, to become the cornerstone of a curative HBV regimen. It is the only Active Site Polymerase Inhibitor Nucleotide (ASPIN) in clinical development and its mechanism of action is designed to be complementary to other approaches that also seek to achieve a functional cure. ATI-2173 is currently in Phase 2a clinical development. The SAVE-1 (Sustained Anti-Viral Efficacy) trial is an ongoing, double-blind, randomized, placebo-controlled study of 30 patients designed to assess the safety and efficacy of 25 and 50 mg doses of ATI-2173 daily for 90 days in combination with tenofovir disoproxil fumarate (TDF) compared with TDF plus ATI-placebo (control) in chronic HBV-infected subjects.

About AB-729

AB-729 is an RNA interference (RNAi) therapeutic specifically designed to reduce all HBV viral proteins and antigens, including hepatitis B surface antigen, which is thought to be a key prerequisite to enable reawakening of a patient’s immune system to respond to the virus. AB-729 targets hepatocytes using Arbutus’ novel covalently conjugated N-acetylgalactosamine (GalNAc) delivery technology that enables subcutaneous delivery. Clinical data generated thus far has shown single- and multi-doses of AB-729 to be generally safe and well-tolerated while providing meaningful reductions in hepatitis B surface antigen and hepatitis B DNA. AB-729 is currently in a Phase 2a clinical trial in combination with Peg-IFNα-2a and nucleos(t)ide analog (“NA”) therapy.

About HBV

Hepatitis B is a potentially life-threatening liver infection caused by HBV. HBV can cause chronic infection which leads to a higher risk of death from cirrhosis and liver cancer. Chronic HBV infection (CHB) represents a significant unmet medical need. The World Health Organization estimates that up to 300 million people worldwide suffer from chronic HBV infection, while other estimates indicate that approximately 2 million people in the United States suffer from chronic HBV infection. Approximately 900,000 people die every year from complications related to chronic HBV infection despite the availability of effective vaccines and current treatment options.

About Antios Therapeutics, Inc.

Antios Therapeutics is a clinical-stage biopharmaceutical company focused on the development of innovative therapies to treat and cure viral diseases. Its lead drug candidate ATI-2173 – the only Active Site Polymerase Inhibitor Nucleotide (ASPIN) in clinical development – has the potential, if approved, to become the cornerstone of a curative therapeutic regimen for chronic HBV. Antios recently entered into an agreement with IRBM to acquire a novel series of fourth-generation capsid assembly modulators (CAMs) to further expand Antios’ portfolio of differentiated molecules in the HBV space. HBV is a major unmet global health problem affecting up to 300 million people worldwide, more than hepatitis C and HIV combined. For more information, please visit www.antiostherapeutics.com.

About Arbutus

Arbutus Biopharma Corporation (Nasdaq: ABUS) is a clinical-stage biopharmaceutical company primarily focused on discovering, developing and commercializing a broad portfolio of assets with different modes of action to provide a cure for people with chronic hepatitis B virus (HBV) infection. The Company is advancing multiple product candidates with distinct mechanisms of action that suppress viral replication, reduce surface antigen and reawaken the immune system. Arbutus believes this three-prong approach is key to transforming the treatment and developing a potential cure for chronic HBV infection. Arbutus’ HBV product pipeline includes RNA interference (RNAi) therapeutics, oral capsid inhibitors, oral compounds that inhibit PD-L1 and oral HBV RNA destabilizers. In addition, Arbutus has an ongoing drug discovery and development program directed to identifying orally active agents for treating coronaviruses (including COVID-19). For more information, visit www.arbutusbio.com.

Forward-Looking Statements and Information

This press release contains forward-looking statements within the meaning of the Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, and forward-looking information within the meaning of Canadian securities laws (collectively, “forward-looking statements”). Forward-looking statements in this press release include statements about our future development plans for our product candidates, including the expected trial design of the triple combination trial with Antios; our expectations regarding the results of the triple combination trial with Antios; and the potential for our product candidates to achieve success in clinical trials.

With respect to the forward-looking statements contained in this press release, Arbutus has made numerous assumptions regarding, among other things: the effectiveness and timeliness of preclinical studies and clinical trials, and the usefulness of the data; the timeliness of regulatory approvals; the continued demand for Arbutus’ assets; and the stability of economic and market conditions. While Arbutus considers these assumptions to be reasonable, these assumptions are inherently subject to significant business, economic, competitive, market and social uncertainties and contingencies, including uncertainties and contingencies related to the ongoing COVID-19 pandemic.

Additionally, there are known and unknown risk factors which could cause Arbutus’ actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements contained herein. Known risk factors include, among others: anticipated pre-clinical studies and clinical trials may be more costly or take longer to complete than anticipated, may never be initiated or completed, or may not generate results that warrant future development of the tested product candidate; Arbutus or Antios may elect to change its strategy regarding its product candidates and clinical development activities; Arbutus may not receive the necessary regulatory approvals for the clinical development of Arbutus’ products; economic and market conditions may worsen; market shifts may require a change in strategic focus; and the ongoing COVID-19 pandemic could significantly disrupt Arbutus’ or its partners’ clinical development programs.

A more complete discussion of the risks and uncertainties facing Arbutus appears in Arbutus’ Annual Report on Form 10-K, Arbutus’ Quarterly Reports on Form 10-Q and Arbutus’ continuous and periodic disclosure filings, which are available at www.sedar.com and at www.sec.gov. All forward-looking statements herein are qualified in their entirety by this cautionary statement, and Arbutus 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.

For More Information:

Jenny Hooper
JPA Health
jhooper@jpa.com
+1 (202) 899-5379 / +1 (956) 638-5095

OpSens Inc. (“OpSens” or the “Company”) (TSX: OPS) (OTCQX: OPSSF), a medical device cardiology-focused company delivering innovative solutions based on its proprietary optical technology, today announced that it has filed a 510(k) submission with the U.S. Food & Drug Administration (“FDA”) for regulatory clearance of its new guidewire (“SavvyWire”) for transcatheter aortic valve replacement, or TAVR procedures. OpSens has also filed for approval with Health Canada.

“The FDA submission is a key milestone for OpSens, in introducing our innovative product to the cardiovascular market. The SavvyWire has been designed and developed to improve the workflow in transcatheter aortic valve replacement,” commented Louis Laflamme, President and Chief Executive Officer of OpSens. “The introduction of a novel and advanced guidewire that has the ability to both deliver a valvular prosthesis while allowing continuous hemodynamic pressure measurement during the procedure is considered to be a significant benefit to the medical community, especially given the rapid growth in TAVR procedures. We look forward to the agencies review of our application and will continue to prepare our organization for an anticipated approval in late summer or fall of 2022.”

The SavvyWire, a new intelligent, pre-shaped, structural guidewire with integrated pressure monitoring, aims at improving procedural efficiency and clinical outcomes by allowing multiple steps over the same device without exchange. This device has been designed to support the minimalist TAVR approach which has been growing among structural heart physicians. With the SavvyWire, physicians can expect to diagnose and   implant the percutaneous valve over the same device while getting continuous and accurate hemodynamic measurements.

About OpSens Inc. (www.OpSens.com or www.OpSensmedical.com)

OpSens focuses mainly in interventional cardiology. The Company offers an advanced optical-based pressure guidewire that aims at improving the clinical outcome of patients with coronary artery disease. Its flagship product, the OptoWire, is a second-generation fiber optic pressure guidewire designed to provide the lowest drift in the industry and excellent lesions access. The OptoWire has been used in the diagnosis and treatment of over 150,000 patients in more than 30 countries. It is approved for sale in the United States, European Union, Japan, and Canada.

OpSens is also involved in industrial activities in developing, manufacturing, and installing innovative fiber optic sensing solutions for critical applications.

Forward-looking statements contained in this press release involve known and unknown risks, uncertainties and other factors that may cause actual results, performance, and achievements of OpSens to be materially different from any future results, performance or achievements expressed or implied by the said forward-looking statements.

Neither TSX nor its Regulation Services Provider (as that term is defined in the policies of the TSX) accepts responsibility for the adequacy or accuracy of this release.

SOURCE OPSENS Inc.

For further information:

Louis Laflamme, CPA, CA, Chief Executive Officer, 418.781.0333

Robin Villeneuve, CPA, CA Chief Financial Officer, 418.781.0333

OncoMyx Therapeutics, a privately-held immuno-oncology platform company, today announced the closing of a $50 million Series B financing, co-led by Lumira Ventures and B Capital Group with participation from LYZZ Capital and all Series A investors: Boehringer Ingelheim Venture Fund, Delos Capital, Xeraya Capital, Korea Investment Partners, City Hill Ventures, and Madison Partners. In conjunction with the financing, Benjamin (Beni) Rovinski, Ph.D., Managing Director of Lumira Ventures and Widya Mulyasasmita, Ph.D., Senior Principal, Healthcare at B Capital Group will join the company’s Board of Directors. The proceeds of the financing will support the further development of OncoMyx’s pipeline of multi-armed myxoma immunotherapies for the treatment of solid tumors and hematological malignances and the advancement of the company’s lead candidate into clinical trials.

OncoMyx has leveraged myxoma’s unique qualities to build an immunotherapy platform to deliver multiple cancer killing payloads in one therapeutic. Myxoma is a natural oncolytic virus, selectively infecting and killing a wide range of cancer cell types. It is also inherently highly immuno-interactive, and as a large dsDNA poxvirus, is engineerable to express multiple payloads. OncoMyx’s multi-armed myxoma virus delivers different antitumor immunomodulatory proteins that hit critical points in the cancer immunity cycle to modulate the tumor microenvironment and stimulate a robust anti-tumor response. Because myxoma is not pathogenic to humans, it does not have to overcome pre-existing immunity and is highly amenable to IV and repeat dosing with a longer dosing window.

“OncoMyx builds on more than three decades of research into the myxoma virus, brings together the top team with experience developing targeted cancer treatments, immunotherapies, and virotherapies, and is fueled by a commitment to addressing unmet medical needs in patients with cancer,” said Steve Potts, Ph.D., MBA, cofounder and CEO of OncoMyx. “The power of our myxoma platform is the potential to systemically deliver broad and selective targeting of cancer cells combined with robust activation of the cancer immunity cycle in a single therapeutic. With this financing, we plan to advance our lead multi-armed myxoma immunotherapy into clinical trials to demonstrate initial safety and efficacy of intravenous dosing.”

“I have long been interested and was previously involved in the development and use of modified, recombinant viruses as immunotherapeutic agents. Oncolytic viruses in particular have shown clinical promise but a significant challenge has been the inability to engineer a safe and versatile oncolytic virus platform that is not restricted to local intratumoral delivery,“ said Dr. Rovinski. “The use of myxoma virus, which is not pathogenic to humans and can accommodate several insertions of heterologous therapeutic agents, as the backbone of OncoMyx’s immunotherapy platform, represents a uniquely differentiated feature that has the potential to overcome the well-known historical challenge of effectively delivering oncolytic viruses systemically for optimal dosing. We believe that OncoMyx’s myxoma-based oncolytic viruses may become best-in-class components of novel immunotherapeutic regimens, and we look forward to supporting the world class team at OncoMyx to bring these therapies to patients in need.”

“We always seek to invest in companies with visionary founders and world-changing technology, and we saw the unmatched potential of OncoMyx’s myxoma platform to make a significant breakthrough in cancer immunotherapy by delivering multiple immune-activating and anti-tumor agents in one off-the-shelf package. We are impressed with the extensive preclinical data OncoMyx has generated demonstrating efficacy and safety of the myxoma platform across a broad range of cancers, from hematological malignancies to solid tumors,” said Dr. Mulyasasmita. “OncoMyx has attracted an exceptionally experienced and talented team who are as committed as the founders about building the next great immuno-oncology company that delivers lifesaving medicines for many difficult to treat cancers. We couldn’t be more thrilled and honored to support their mission.”

OncoMyx has presented preclinical safety and efficacy data at major scientific conferences demonstrating their myxoma immunotherapies stimulate anti-tumor immunity and produce anti-tumor efficacy in a wide range of models following IV or intratumoral administration. The data, along with previously published studies, support the advancement of these therapies into clinical development as a monotherapy and in combination with many cancer therapeutics and immunotherapies, including checkpoint inhibitors and chemotherapies. The company plans to advance its lead candidate into clinical trials next year to generate safety and efficacy data for IV dosing.

About OncoMyx Therapeutics
OncoMyx Therapeutics is advancing multi-armed, systemic immunotherapies with the potential to be broadly effective in treating solid tumors and heme cancers. Successful immuno-oncology cancer treatment generally requires combination therapy, and OncoMyx’s myxoma platform is designed to deliver multiple cancer-killing payloads in one therapeutic and orchestrate an immune response capable of treating a wide range of cancers. OncoMyx has assembled the top immuno-oncology team to develop next generation immunotherapies that can have the greatest therapeutic benefit for more cancer patients. The company’s myxoma virus platform is poised to be a best-in-class oncolytic virus approach and was developed based on breakthrough research from Dr. Grant McFadden’s lab that was exclusively licensed from Arizona State University. For more information, please visit www.oncomyx.com and connect with the company on LinkedIn and Twitter.

www.oncomyx.com

Maryland-based biotech company Deka Biosciences (“Deka”) today announced that it has successfully closed a USD 20 Million Series A financing with a syndicate of life science investors led by Leaps by Bayer, the impact investment arm of Bayer AG, and new investor Lumira Ventures. Additional investors include O-Bio (Echo Investment Capital), Viva BioInnovator, and Alexandria Venture Investments.

Novel cytokine-based therapies have the potential to provide patients with innovative curative treatment options for cancer, autoimmune diseases, and many types of infectious diseases. Understanding the known function of each cytokine, Deka has developed Diakines™ – intentionally engineered therapeutic proteins that are designed to deliver clinically validated cytokines, coupled in combination in the Diakine™ structure, to diseased tissue. Deka has also combined this therapeutic platform with companion diagnostic assays that ensure delivery of each Diakine™ to patients that will benefit the most.

In Deka’s Diakines™, the scaffold platform is derived from a human antibody fragment, called a single-chain variable fragment, that uniquely functions both as a stabilizing, half-life extension technology and a targeting vector to deliver the cytokine(s) function to specific cell types or the microenvironment of affected tissues. Through stabilization and improved manufacturing techniques, the Diakine™ scaffold increases production yields and reduces manufacturing costs, and the unique structure enhances the specific functions of each cytokine, unlike most other half-life extending technologies. The company has demonstrated positive responses in preclinical studies, in both cancer and inflammatory disease models. Through extensive investigation, Deka has found that not all people respond to the same cytokine in the same way. Deka has therefore developed assays that evaluate each patients’ response to each cytokine pair and found genetic signatures that are uniquely associated with response to each Diakine™. Deka will evaluate this genetic signature in future clinical trials to ensure that each patient is matched with their best Diakine™. The team at Deka is committed to developing Diakines™ that can treat every patient.

“This investment by our multi-national, top-tier syndicate enables our first step in developing the Diakine™ platform,” said John Mumm, CEO and founder of Deka Biosciences. “We are proud to join forces with our board of directors to bring these life-changing medicines to patients as quickly and effectively as possible. We share the vision to fundamentally change the nature of drug development and change the standard of healthcare through coupling our platform technology with predictive precision medicine. We are honored to have Leaps by Bayer as our lead investor as we share the goals to develop cures for patients through innovative science and precision medicine. We dare to leap as they do.”

“Leaps by Bayer aims to achieve life transforming breakthroughs for patients, this is why we invest in technologies of tomorrow already today,” said Juergen Eckhardt, MD, Head of Leaps by Bayer. “One of humankind’s biggest challenges and one of the big goals Leaps by Bayer is trying to solve is to prevent and cure cancer. We believe next-generation immunotherapies will play a pivotal role in addressing this challenge and Deka Biosciences’ cytokine therapy approach has the potential to change treatment-paradigms for cancer patients and for those suffering from auto-immune diseases.”

“Deka Biosciences has developed a unique and strongly differentiated platform to produce cytokines with therapeutically complementary functions that circumvent several challenges associated with naturally occurring and modified cytokines,” said Benjamin (Beni) Rovinski, PhD, Managing Director of Lumira Ventures. “At Lumira, our mission is to invest in companies at the forefront of biomedical innovation whose products have the potential to transform patient outcomes. It is gratifying to support Deka in pursuit of such a goal.”

“Deka has made impressive progress since its inception. We are excited to partner with Deka, and its Diakine™ platform, to provide best-in-class therapies to patients,” said Yi-Yen Chen, managing director at Echo Investment Capital’s O-Bio fund, a life science focused fund headquartered in Oklahoma City.

The investment will enable Deka to advance its research and talent acquisition and further expand development of its platform, in particular to file the Investigation New Drug (IND) application for the lead oncology program and advance the lead compound into Phase I clinical trials.

About Deka Biosciences
Deka is an early-stage biotech company focused on the development of novel cytokine therapies to treat cancer and inflammatory diseases such as Crohn’s, psoriasis, rheumatoid arthritis and sepsis. The company is led by serial entrepreneur John Mumm, who is backed by a team of experienced biopharma and CDMO innovators with expertise in drug discovery, product development, characterization and testing. Deka has developed disease specific Diakines™ that maximize patient benefits through improved pharmacokinetics / pharmacodynamics (PK/PD) function via targeted delivery of dual and complimentary cytokines to affected tissues or cells. Through the use of precision medicine, Deka’s vision is to maximize the impact of its Diakine™ therapeutic proteins by ensuring treatment of patients who will best benefit from and respond to our cytokine therapies. To learn more, visit www.dekabiosciences.com.

SOURCE Deka Biosciences

http://www.dekabiosciences.com

Antios Therapeutics, Inc. (“Antios”), a clinical-stage biopharmaceutical company developing innovative therapies to treat and cure chronic hepatitis B virus (HBV), and IRBM, a drug discovery and early development research institute and global contract research organization, announced today they entered into an agreement for Antios to purchase the IP rights to a novel series of fourth-generation capsid assembly modulators (CAMs) being developed for the treatment of HBV. The CAMs were originated and developed at IRBM in conjunction with Promidis/CNCCS (a subsidiary of IRBM), Istituto Nazionale Genetica Molecolare (INGM) and San Raffaele Hospital (OSR). These CAMs enable the selection of a new pipeline candidate, which will be the second molecule in Antios’ HBV portfolio. The total potential value of the deal to the Italian sellers/parties is up to $50 million, in addition to royalties.

Derived from a novel chemical scaffold, the CAMs have shown a strong in vitro and in vivo activity in a transgenic mouse model of HBV infection. Early in vivo data point to a differentiated mechanism of action which prevents accumulation of empty capsids, unlike most CAMs, and potentially provides for a more targeted, productive clearance by the immune system. Pre-clinical candidate selection is anticipated in the first half of 2022 with clinical development beginning in mid-2023.

“Unlike traditional CAMs, which target capsid assembly and disassembly, we believe these molecules may have an additional mechanism of action. Their distinct potential in vivo, along with their pre-clinical activity profile to date, position our candidate selection with best-in-class potential among the field,” said Katherine Squires, PhD, Head of Research and Development at Antios. “These particular CAMs will expand the Antios portfolio of differentiated molecules and offer a unique addition to the development of a potentially curative regimen.”

“We hope that the development of these compounds can bring significant benefit to the 296 million people living with this devastating disease,” said Romano Di Fabio, PhD, Corporate Director of Small Molecule R&D at IRBM, whose team designed and then optimized this novel CAM series. Dr. Di Fabio holds more than 70 international patents.

“This agreement is a tribute to the potential of partnerships between Italian academia and industry experts to develop novel therapies to address unmet clinical need,” said Carlo Toniatti, Chief Scientific Officer of IRBM.

“We are pleased to find a home for this program with an experienced team that recognizes the unique potential of these compounds that, in pre-clinical research, showed differentiation from others in its class,” said Luca Guidotti, MD, PhD, a co-inventor and a pioneering HBV researcher who serves as Deputy Scientific Director at OSR.

“Antios, because of its focus on HBV, is the ideal company to guide this program through clinical trials and advance its journey toward potential commercialization,” said Raffaele De Francesco, PhD, Co-inventor of the novel CAM series and Head of the Virology Laboratory at INGM.

Antios’ lead Phase 2b clinical candidate, ATI-2173, is the only Active Site Polymerase Inhibitor Nucleotide (ASPIN) in clinical development, and has potential, if approved, to be the cornerstone of curative, once-daily HBV therapy. Pre-clinical data to date for ATI-2173, alone or combined with tenofovir disoproxil fumarate (TDF), indicate the potential for sustained HBV DNA suppression off treatment, unique among approved nucleosides and investigational anti-HBV therapies.

Destum Partners acted as business development advisor to Antios for this transaction. Closing of the transaction is subject to customary closing conditions, including clearance under the Italian Golden Power legislation.

About ATI-2173
ATI-2173, Antios Therapeutics’ lead once-daily, oral drug candidate for treating HBV, is an investigational phosphoramidate prodrug of clevudine monophosphate. ATI-2173 has the potential, if approved, to become the cornerstone of a curative HBV regimen. It is the only Active Site Polymerase Inhibitor Nucleotide (ASPIN) in clinical development and its mechanism of action is complementary to other approaches that also seek to achieve a functional cure. ATI-2173 is currently in Phase 2b clinical development. The SAVE-1 (Sustained Anti-Viral Efficacy) trial is an ongoing, double-blind, randomized, placebo-controlled study of 30 patients designed to assess the safety and efficacy of 25 and 50 mg doses of ATI-2173 daily for 90 days in combination with tenofovir disoproxil fumarate (TDF) compared with TDF plus ATI-placebo (control) in chronic HBV-infected subjects.

About Antios Therapeutics, Inc.
Antios Therapeutics is a clinical-stage biopharmaceutical company focused on the development of innovative therapies to treat and cure viral diseases. Its lead drug candidate ATI-2173 – the only Active Site Polymerase Inhibitor Nucleotide (ASPIN) in clinical development – has the potential, if approved, to become the cornerstone of a curative therapeutic regimen for chronic HBV, a major unmet global health problem affecting up to 300 million people worldwide, more than hepatitis C and HIV combined. For more information, please visit www.antiostherapeutics.com.

About IRBM
IRBM is a drug hunting company working primarily as an innovative contract research organization across all aspects of drug discovery and early development for different modalities – small molecule, peptides, and antibodies. IRBM fosters collaborations with organizations from the pharmaceutical, biotech and academic sectors to accelerate drug discovery from target validation and hit identification to candidate nomination. More than 200 scientists work at IRBM’s state-of-the-art R&D facility near Rome where projects are carried out “under one roof” enabling rapid cycle times and close integration of the scientific teams. IRBM’s scientists have discovered several drugs that are on the market, and more than 25 compounds have gone into clinical testing. The company was founded in 2010, as a spin-off from MSD. Now in its 11th year as an independent research organization, IRBM has laid the foundations to become a global Centre for Excellence in all aspects of drug discovery and early development. IRBM’s affiliate, Promidis, is based at the San Raffaele Hospital and conducts research with the objective to discover innovative drugs in areas of unmet need. IRBM is also part of The National Consortium and Collection of Chemical Compounds (CNCCS), a private-public consortium with a mission to identify compounds acting on innovative biological targets. It also acts as a center for translational research in rare, neglected and poverty-related diseases. Both Promidis and CNCCS together with IRBM were involved in the discovery and development of the fourth-generation CAM inhibitors. For more information visit www.irbm.com.  

About INGM
INGM is a biomedical research centre aimed at Preventive Precision Medicine with a special focus on genetics and epigenetics of immunological aspects of chronic diseases (autoimmune, infectious and tumoral). INGM develops translational research projects aimed at the identification of new biomarkers and novel therapeutic targets which could improve secondary and tertiary prevention of human chronic diseases. INGM projects exploit the most advance single cell omics technologies combined with the most advanced big data interrogation tools. For more information visit www.ingm.org.

About OSR
San Raffaele Hospital (OSR) is a large clinical-research-university hospital with more than 60 clinical specialties that provides advanced care for the most complex and difficult health conditions. OSR is part of Gruppo San Donato, the leading hospital group in Italy. Research at OSR involves more than 1,200 scientists and covers all fields of biomedicine. The overall biomedical innovation process is operative at OSR: from basic discoveries made in the labs to therapy design and test in pre-clinical models, up to first phase and pilot clinical trials in humans. The institute is recognized as a global authority in molecular medicine and gene therapy and is at the forefront of research in many other fields, including infectious diseases. OSR currently holds a portfolio of over 600 patents, most of which are licensed to industrial partners for further development. For more information, please visit: www.hsr.it.

SOURCE Antios Therapeutics