Thryv Therapeutics Inc., a clinical-stage biotechnology company, received positive top-line results from the Wave I Part 2 proof-of-concept clinical study evaluating LQT-1213 for the treatment of congenital Long QT Syndrome (cLQTS) Types 2 and 3. LQT-1213, a potent and selective serum glucocorticoid inducible kinase 1 (SGK1) inhibitor, demonstrated clinically meaningful and statistically significant reductions in the QTcF interval (a measure of how long it takes the heart to “reset” between beats) after only two days of dosing in patients with cLQTS Types 2 and 3. LQT-1213 appeared to be generally well-tolerated in the study with no drug-related adverse events (AEs) reported.
Congenital LQTS is a rare genetic condition that affects the heart’s electrical system, leading to an increased risk of life-threatening arrhythmias in children and adults. While Wave I Part 1 evaluated the effect of LQT-1213 in a model of LQTS induced by the drug dofetilide, Wave 1 Part 2 included, for the first time, patients with genetically confirmed congenital LQTS Types 2 and 3, providing direct clinical insights into the acute efficacy of LQT-1213 in the targeted patient population. It is believed that reductions in QTcF will reduce arrhythmic risk in pediatric and adult patients with prolonged QTc intervals.
“We are excited about the positive top-line results from the Wave I Part 2 study in cLQTS Types 2 and 3 patients. Two days of treatment with LQT-1213 demonstrated rapid and clinically meaningful reductions in QTcF. As well, a clear dose level effect was seen with the highest dose of LQT-1213 reducing QTcF for all key endpoints of the study,” said Philip Sager, MD, FAHA, Chief Medical Officer of Thryv Therapeutics. “The totality of LQT-1213 treatment effects, which are on top of beta blocker therapy, has allowed us to engage regulatory agencies about our next steps towards a pivotal program in patients with congenital Long QT Syndrome.”
Study Highlights
Wave I is a multi-part, proof-of-concept clinical study designed to evaluate the safety and efficacy of LQT-1213 in reducing QT in patients with both acquired and congenital forms of Long QT Syndrome. Further details about the study can be found at clinicaltrials.gov (NCT05906732).
Full data from the Wave I Part 2 study in congenital LQTS Types 2 and 3 will be presented at an upcoming scientific conference in 2025. Part 3 of the Wave I study will enroll patients with cLQTS Type 1 who demonstrate continued prolongation of their QT interval despite standard of care.
About LQT-1213
LQT-1213 is a potent and selective serum glucocorticoid regulated kinase 1 (SGK1) inhibitor being developed as an investigational therapeutic to treat Long QT Syndrome Types 1, 2, and 3. By targeting SGK1, LQT-1213 aims to reduce the prolonged QTc interval in children and adults with LQTS, thereby decreasing the risk of life-threatening arrhythmias. Currently, LQT-1213 is being evaluated in clinical studies for its efficacy and safety in treating both congenital and acquired LQTS. LQT-1213 has been granted Orphan Drug Designation by the US FDA for the treatment of congenital Long QT Syndrome.
About Long QT Syndrome
LQTS, is a disorder of the electrical system that results in the heartbeat taking longer than usual to “reset” between beats, measured on the ECG as the QTc interval. LQTS can be either congenital or acquired due to certain drugs. Congenital LQTS is a set of rare orphan diseases in which people are genetically predisposed to chronic prolongation of their QTc interval (commonly more than 480 milliseconds), leading to increased risk of torsades de pointes, a lethal cardiac arrhythmia that can cause sudden cardiac death. Acquired Long QT may develop from the administration of therapies which block electrical pathways in the heart, leading to a similar mechanistic prolongation of QTc and risk of sudden cardiac death.
About Thryv Therapeutics
Thryv Therapeutics Inc. is a privately owned company based in Montreal, Quebec, Canada. Thryv Therapeutics is pioneering a precision medicine approach to develop highly selective SGK1 inhibitors to treat Long QT Syndrome, atrial fibrillation and heart failure, with potent and selective inhibitors of serum glucocorticoid inducible kinase. For more information, please visit www.thryvtrx.com.
SOURCE Thryv Therapeutics Inc.