Phase II Gene Therapy Trial for CHF Initiates with Patient Randomization

A clinical trial known as GenePHIT is underway to test a novel gene therapy called AB-1002, developed by Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG. The therapy is designed to treat adults with non-ischemic cardiomyopathy and NYHA Class III heart failure symptoms. The GenePHIT trial marks a significant advancement in the field of gene therapy for congestive heart failure (CHF), a condition where the heart struggles to pump blood effectively, leading to a buildup of blood and fluids in the body.

The trial is a Phase II, adaptive, double-blind, placebo-controlled, and randomized multicenter study. It will involve between 90 to 150 participants who have a left ventricular ejection fraction between 15 and 35 percent and continue to experience heart failure symptoms despite standard medical treatment. The primary goal of the study is to assess the safety and efficacy of a single intracoronary infusion of AB-1002 after the patients have been medically stable for at least four weeks.

The study's primary efficacy endpoint will be evaluated at 52 weeks, focusing on a composite measure of several clinically meaningful assessments. The trial's duration from dosing to the primary analysis is expected to be around 37 months, including a recruitment period of 25 months and a 52-week observation period post-dosing. Following the completion of the observation period, the treatment groups will be revealed, and the primary analysis will be conducted. Participants will be followed up for an additional four years after the initial 52-week study period for long-term safety, efficacy assessments, and survival data.

The trial is a collaborative effort between Bayer and AskBio, reflecting a commitment to developing innovative treatments for heart failure, a condition that affects millions of people worldwide and presents a growing medical need, particularly in aging populations. The potential of gene therapy to address the root cause of CHF is considered to be substantial, and the GenePHIT trial represents a significant step towards potentially transforming the treatment landscape for this devastating disease.

AB-1002 is still in the investigational stage and has not yet received marketing authorization. Its safety and efficacy profiles are still under evaluation. The manufacturing of AB-1002 is carried out by Viralgen Vector Core, another subsidiary of AskBio, known for its cGMP certified AAV manufacturing capabilities and the Pro10™ platform, which enhances the scalability and performance of AAV therapies.

AskBio is a fully integrated gene therapy company with a diverse clinical program targeting various diseases, including neuromuscular, CNS, cardiovascular, and metabolic disorders. The company has a robust pipeline and a strong intellectual property portfolio with over 800 patents and patent applications related to gene therapy.

Bayer, a global life science company, is dedicated to driving sustainable development and aims to create innovative solutions to address major challenges faced by a growing and aging global population. The company's commitment to research and development is evident in its substantial R&D expenses and its focus on delivering impactful treatments.