One-time gene therapy for heart failure moves to Phase II trial


Gene therapy company Asklepios BioPharmaceutical (AskBio, a subsidiary of Bayer) has announced plans to initiate a Phase II trial of gene therapy AB-1002 for the treatment of congestive heart failure (CHF).

GenePHIT (Gene PHosphatase Inhibition Therapy) will evaluate the safety and efficacy of a single intracoronary infusion of AB-1002 in adults with non-ischaemic cardiomyopathy classified as New York Heart Association (NYHA) Class III Heart Failure who have been medically stable for at least four weeks.

The trial 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.

Jude Samulski, Co-Founder and Chief Scientific Officer, AskBio, said: “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.”

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.

In Phase I trials, the therapy was well tolerated and patients showed clinically meaningful improvements following treatment.

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