AskBio Presents Baseline Characteristics from Phase 2 GenePHIT Trial of Umiposgene Parvec in Heart Failure
核心洞察
AskBio presented baseline characteristics from the Phase 2 GenePHIT trial of the investigational gene therapy umiposgene parvec (搜索) (AB-1002) at the European Society of Cardiology Congress in Munich, Germany.
GenePHIT randomized more than 170 participants across 64 sites in 12 countries, making it one of the largest gene therapy trials conducted in heart failure (搜索) to date.
The enrolled population reflects a representative non-ischemic heart failure with reduced ejection fraction (搜索) (HFrEF) cohort receiving guideline-directed medical therapy.
AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced the presentation of baseline participant characteristics from GenePHIT, one of the largest randomized gene therapy trials conducted in heart failure (搜索) to date. The Phase 2 clinical trial is evaluating the investigational one-time gene therapy umiposgene parvec (搜索) (AB-1002) for its potential to improve cardiac function. Baseline characteristics were presented at the European Society of Cardiology (ESC) Congress, held in Munich, Germany, August 28–31, 2026.
The presentation highlights the trial's successful enrollment and further validates that multinational gene therapy trials of this size can be conducted. GenePHIT successfully randomized and completed enrollment of more than 170 participants across 64 sites in 12 countries.
Trial Design and Patient Population
GenePHIT is a randomized, double-blind, placebo-controlled, multicenter Phase 2 adaptive trial designed to evaluate the efficacy, safety, and tolerability of umiposgene parvec (搜索) following a direct infusion to the heart, with no immune suppression, via a standard catheter. The therapy is administered via antegrade intracoronary artery infusion in males and females over age 18 with non-ischemic cardiomyopathy, New York Heart Association (NYHA) Class III heart failure (搜索) symptoms, and reduced ejection fraction.
The baseline characteristics demonstrate the successful recruitment of a representative non-ischemic heart failure with reduced ejection fraction (搜索) (HFrEF) population receiving guideline-directed medical therapy. GenePHIT participants were treated with established heart failure (搜索) therapies, including beta-blockers, angiotensin receptor-neprilysin inhibitors, sodium-glucose cotransporter 2 inhibitors, and mineralocorticoid receptor antagonists.
The enrolled population included participants with significant disease burden, with nearly half having a history of atrial fibrillation and almost 45% having an implantable cardioverter defibrillator. The clinical trial is among the largest to evaluate intracoronary administration of gene therapy in patients with heart failure (搜索).
Endpoints and Geographic Scope
The trial was designed to evaluate cardiovascular-related deaths and change from baseline in NYHA classification, left ventricular ejection fraction, and six-minute walking distance. GenePHIT participants were randomized at 64 locations across the United States, Canada, Austria, Germany, the Netherlands, Spain, Belgium, Hungary, Poland, Bulgaria, Romania, and the United Kingdom.
Expert Perspectives
"Despite remarkable advances in heart failure (搜索) therapy, many patients continue to experience progressive ventricular dysfunction, recurrent hospitalization, and premature death," said Timothy D. Henry, MD, MSCAI, GenePHIT Principal Investigator and Steering Committee Member. "GenePHIT represents an important milestone for the field because it demonstrates that successful enrollment of large, multinational randomized gene therapy trials in heart failure is feasible. The enrolled population closely reflects contemporary patients receiving current standard of care, providing an excellent foundation to evaluate whether a one-time investigational gene therapy can meaningfully improve outcomes."
"We are pleased to share these baseline characteristics from GenePHIT, which represents an important milestone in the advancement of our umiposgene parvec (搜索) program for heart failure (搜索)," said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. "Having 64 sites in 12 countries demonstrates the feasibility of evaluating this investigative approach in a representative population. These baseline characteristics establish a strong foundation for interpreting the initial efficacy and safety outcomes, which are expected in the first half of 2027 and will further inform the continued development of umiposgene parvec as a potential treatment for patients with heart failure."
Mechanism of Action
Umiposgene parvec (搜索) is an investigational one-time gene therapy administered directly to the heart to promote production of a modified version (I-1c) of the naturally occurring protein inhibitor-1 (搜索). It is designed to block the action of protein phosphatase 1 (搜索), which is linked to heart failure (搜索), with the goal of improving cardiac contractility by restoring intracellular calcium signaling.
Disease Burden
Heart failure (搜索) occurs when the heart cannot pump blood efficiently enough to meet the body's needs, including providing sufficient oxygen to the organs. This causes congestion in the body's tissues, with symptoms that may include shortness of breath, swelling in the legs and ankles caused by fluid retention, and fatigue. More than 64 million people worldwide are estimated to be living with heart failure.
Umiposgene parvec (搜索) has not been approved by any regulatory authority, and its safety and efficacy have not been established. Initial efficacy and safety outcomes from GenePHIT are expected in the first half of 2027.