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Bayer's AskBio Enrolls First Patient in Phase II Gene Therapy Trial for Heart Failure

NEW YORK – Bayer subsidiary Asklepios BioPharmaceutical (AskBio) on Tuesday said it has randomized the first patient in a Phase II trial of an investigational gene therapy for congestive heart failure, AB-1002 (NAN-101).

AB-1002 is a gene therapy administered as a one-time intracoronary infusion and designed to deliver an active I-1 transgene to promote production of protein inhibitor 1, which can block the activity of the congestive heart failure-linked protein phosphatase 1. The gene therapy is designed as a treatment for patients with non-ischemic cardiomyopathy and class III heart failure symptoms, according to the New York Heart Association (NYHA).

Research Triangle Park, North Carolina-based AskBio plans to enroll 90 to 150 adults with left ventricular ejection fraction between 15 percent and 35 percent into the randomized-controlled Phase II trial, called Gene PHosphatase Inhibition Therapy, or GenePHIT. To be eligible for enrollment, a participant must have continued to experience heart failure symptoms despite being on guideline-recommended therapy.

Participants in the trial will receive either an infusion of a low or high dose of AB-1002 or a placebo. Investigators will assess the safety and efficacy of the experimental gene therapy by monitoring cardiovascular-related death, change from baseline NYHA classification, left ventricular ejection fraction, peak oxygen uptake, and a six-minute walk test.

"The potential impact of gene therapy to address [heart failure] at its root cause is immense, and we are thrilled about this step in our path to deliver truly innovative treatment options for patients," Christian Rommel, a member of the executive committee of Bayer's pharmaceuticals division and head of R&D, said in a statement.