First Patient Randomized in AskBio Phase II Gene Therapy Trial for Congestive Heart Failure Treatment

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, have announced that the first patient has been randomized in GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002, also known as NAN-101, for the treatment of congestive heart failure (CHF).

Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, have announced that the first patient has been randomized in GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase II trial of AB-1002, also known as NAN-101, for the treatment of congestive heart failure (CHF). Image courtesy: AskBio

February 15, 2024 — Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, have announced that the first patient has been randomized in the GenePHIT (Gene PHosphatase Inhibition Therapy) trial, a Phase II trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). According to a written statement, the GenePHIT trial aims to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with New York Heart Association (NYHA) Class III Heart Failure and non-ischemic cardiomyopathy who have been medically stable for at least 4 weeks.

The AskBio statement detailing the update also reported that GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date. It added that this is a significant milestone in the development of AB-1002 for the treatment of CHF, potentially bringing this important investigational therapy one step closer to treating patients with high unmet medical need. It is estimated that 26 million worldwide are living with congestive heart failure. Heart failure is a leading cause of morbidity and mortality worldwide despite available therapies, and quality of life is poor.

GenePHIT is an adaptive, double-blind, placebo-controlled, randomized, multicenter trial to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms who have been medically stable for at least four weeks. This milestone in the development of AB-1002 for the treatment of CHF potentially brings this investigational therapy one step closer to treating patients with high unmet medical need.^[2]

GenePHIT will include between 90 and 150 adults with left ventricular ejection fraction between 15 and 35 percent, who continue to suffer from heart failure symptoms despite guideline recommended therapy. The primary efficacy endpoint at 52 weeks is a modified win ratio of several clinically meaningful assessments.^[1]

“The randomization of the first patient as a part of the Phase II GenePHIT trial is an important moment for the heart failure community,” said Timothy D. Henry, MD, MSCAI, Principal Investigator and Steering Committee Member. He added, “GenePHIT will evaluate the safety and efficacy of AB-1002 in the largest number of patients to date and improve our understanding of gene therapy overall for the treatment of congestive heart failure. The initiation of this trial brings us a step closer to potentially changing the course of this deadly and devastating disease.”

“Being able to announce this important GenePHIT trial update during Heart Failure Awareness Week adds special significance to this milestone,” said Roger J. Hajjar, MD, Scientific Chair CHF, AskBio. “The enrollment of this first patient in the Phase II trial represents the culmination of many years of dedicated research and development in all aspects of cardiac gene therapy for congestive heart failure. Although there is still much to learn about this early-stage investigational gene therapy, we hope today’s announcement, which highlights AskBio’s ability to advance AB-1002 gene therapy for the treatment of congestive heart failure, is encouraging news for everyone hoping to see new treatment options.”

“Heart failure is a devastating disease with increasing unmet medical need, especially in a progressively aging population,” said Christian Rommel, PhD, Member of the Executive Committee of Bayer’s Pharmaceuticals Division and Head of Research and Development. He added, “The potential impact of gene therapy to address this disease at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients.”

AB-1002 is an investigational gene therapy that has not received marketing authorization, and its efficacy and safety have not been established or fully evaluated. AB-1002 is manufactured by Viralgen Vector Core, S.L., a wholly owned and independently operated subsidiary of AskBio.

GenePHIT, the statement further noted, is a Phase II adaptive, double-blinded, placebo-controlled, randomized, multi-center trial to evaluate the safety and efficacy of the one-time administration of AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and NYHA Class III heart failure symptoms.^[1] Subjects are randomized into one of three treatment groups in a 1:1:1 fashion to either low dose, high dose, or placebo. 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 (6MWT).^[1]

Study Background

On January 5, 2023, the company, announced that it is initiating GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase 2 trial of investigational gene therapy AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). It stated that the GenePHIT trial aims to evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischemic cardiomyopathy classified as New York Heart Association (NYHA) Class III Heart Failure who have been medically stable for at least 4 weeks.

On November 13, 2023, during the American Heart Association Scientific Sessions, AHA2023, Asklepios BioPharmaceutical, Inc. (AskBio), AskBio presented first-in-human data from a Phase 1 trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF). At the time, a written statement reported that the trial was designed to establish the safety and preliminary efficacy of gene therapy AB-1002 in patients with NYHA Class III Heart Failure, also known as advanced heart failure.

For more information, www.askbio.com