💉 Bayer’s subsidiary, AskBio, has started Phase II testing of gene therapy AB-1002 for heart failure patients.
The trial, called GenePHIT, is the largest of its kind and will evaluate the safety and efficacy of intracoronary infusion of AB-1002.
AB-1002 improves intracellular calcium cycling, decreases fibrosis, and reverses remodeling.
The therapy has already shown promising results, improving left ventricle ejection fraction in heart failure patients.
The trial, called GenePHIT, is the largest of its kind and will evaluate the safety and efficacy of intracoronary infusion of AB-1002.
AB-1002 improves intracellular calcium cycling, decreases fibrosis, and reverses remodeling.
The therapy has already shown promising results, improving left ventricle ejection fraction in heart failure patients.
📢 Revolutionary Heart Failure Gene Therapy Enters Phase II Trials
Introduction:
Bayer SE subsidiary Asklepios BioPharmaceutical Inc. (AskBio) has started Phase II testing of AB-1002 (NAN-101) in patients with congestive heart failure.
Main points:
- The Phase II trial called GenePHIT is the largest adaptive, double-blind, placebo-controlled, randomised, multicentre trial ever to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in stable 90 to 150 patients with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms. AB1002 is an AAV vector that encodes an protein phosphatase I blocker.
- Patients will be randomized into one of three treatment groups in a 1:1:1 fashion to either 3.25E13vg (n=30-50), 6.5E13 (n=30-50) or placebo group (n=30-50). Primary outcome measures include cardiovascular related death and change from baseline in NYHA classification, left ventricular ejection fraction (LVEF), peak oxygen uptake (pVO2), and Six Minute Walk Test. According to headline results reported in November 2023, the higher dose of AB-1002 improved left ventricle ejection fraction in four out of five heart failure patients at 12 months. One of this five patients died not due to the medication.
- AB-1002 is manufactured by the CDMO Viralgen Vector Core, S.L., a wholly owned and independently operated subsidiary of AskBio.
Conclusion:
The Phase II trial of AB-1002 in patients with congestive heart failure shows promising results in improving left ventricle ejection fraction and may offer a potential gene therapy option for heart failure treatment. Further studies and evaluations are needed to determine the long-term safety and efficacy of AB-1002 in larger patient populations.
