AskBio Announces Initiation of Phase 2 GenePHIT Trial in Congestive Heart Failure

Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that it is initiating GenePHIT (Gene PHosphatase Inhibition Therapy), a Phase 2 trial of AB-1002 (also known as NAN-101) for the treatment of congestive heart failure (CHF). 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 classified as New York Heart Association (NYHA) Class III Heart Failure who have been medically stable for at least 4 weeks.1 The advancement of AB-1002 into Phase 2 marks a significant milestone for this novel gene therapy for CHF and, if successful, could bring this important investigational therapy one step closer to treating patients with high unmet medical need.

GenePHIT will include between 90 and 150 adults with left ventricle ejection fraction between 15 and 35%, 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

“AskBio is excited to initiate GenePHIT under the leadership of Roger Hajjar, MD, Scientific Chair CHF, and Lothar Roessig, MD, Integrated Product Team Lead CHF,” said Jude Samulski, PhD, Co-Founder and Chief Scientific Officer, AskBio. “We believe this trial will help determine the potential of AB-1002 as a treatment for one of the world’s most devastating diseases, and we look forward to learning more about this important investigational cardiac gene therapy. Our hope is that one day AB-1002 will potentially help patients suffering from congestive heart failure.”

The GenePHIT trial, which includes 52-week safety and primary efficacy and four-year long-term follow-up periods, is currently recruiting. AskBio plans to conduct the trial in the U.S. and multiple countries in Europe.1 For more information, please visit (NCT#05598333) or visit

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

About Congestive Heart Failure
Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.2 Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up.3 This causes congestion in the body’s tissues. Symptoms include swelling in the legs and ankles. Sometimes, fluid collects in the lungs and interferes with breathing. Approximately 26 million people worldwide are living with congestive heart failure.4

About AskBio
Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications with a clinical-stage pipeline that includes therapeutics for congestive heart failure, Huntington’s disease, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library. With global headquarters in Research Triangle Park, North Carolina, and European headquarters in Edinburgh, Scotland, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field, with over 900 employees in five countries, the company holds more than 800 patents and patent applications in areas such as AAV production and chimeric capsids.

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