AskBio, a Bayer subsidiary, has initiated the European arm of its Phase 2 GenePHIT trial for AB-1002, a gene therapy for congestive heart failure (CHF). This follows the commencement of the US arm in 2024 and marks a significant step towards potentially offering this one-time treatment to European patients. The therapy targets non-ischemic cardiomyopathy with NYHA Class III heart failure symptoms.
This advancement is crucial given the high prevalence and mortality rate of cardiovascular disease in Europe, representing a substantial unmet medical need. A successful outcome for this trial could offer a disease-modifying approach, impacting both individual patients and strained healthcare systems grappling with the growing burden of heart failure, especially as the population ages. The potential for a one-time treatment represents a significant shift from current ongoing management strategies, promising improved quality of life and potentially reduced long-term healthcare costs.
The GenePHIT trial, enrolling 90-150 adults with reduced ejection fraction (15-35%), is a double-blind, placebo-controlled study evaluating the safety and efficacy of a single intracoronary infusion of AB-1002. It is the largest study to date for AB-1002 and spans clinical centers across the US and several European countries, including Austria, Germany, Hungary, Netherlands, Spain, and the UK. The first European participant was enrolled in Spain. AB-1002 aims to address intracellular abnormalities contributing to heart failure.
The initiation of this European arm represents substantial progress in the development of AB-1002. Positive results could lead to regulatory approval and ultimately provide a novel therapeutic option for a large patient population severely affected by this debilitating condition. It also strengthens AskBio’s position in the gene therapy field and highlights the potential of this approach to transform the treatment of heart failure.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
