Long-Term Data Demonstrate Safety and Preliminary Efficacy of Investigational One-Time Gene Therapy in Patients with Non-Ischemic Heart Failure
AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the publication in peer-reviewed journal Nature Medicine of 12-month data from its Phase 1 trial of AB-1002 investigational gene therapy in participants with congestive heart failure (CHF).1
This non-randomized, sequential dose-escalation trial (NCT04179643) includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).1 It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity remain very high.2,3
The publication, which is available online, confirms that no adverse events were deemed related to AB-1002 in this trial and that clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF.1 The data further support that the AB-1002 capsid may be highly cardiotrophic when administered as a single intracoronary injection.
AskBio thanks the participants who volunteered for this important clinical trial, the sites that made this effort possible, and the skilled investigators who conducted this invaluable research and contributed to the scientific body of knowledge related to AB-1002.
We believe there is a critical need to progress innovative therapies that target the root causes of congestive heart failure, so we’re pleased to see these data for AB-1002 published and shared with the scientific community via Nature Medicine, a high-impact peer-reviewed journal,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “We’re eager to further assess the safety and efficacy of AB-1002 in our ongoing Phase 2 trial, GenePHIT, which is currently enrolling in Canada, Europe, the United Kingdom, and the United States, and look forward to sharing those results once available.
GenePHIT is a Phase 2, adaptive, randomized, double-blind, placebo-controlled trial investigating the safety and efficacy of AB-1002 in non-ischemic heart failure.
The Global Burden of Heart Failure
Heart failure is a major public health challenge, affecting an estimated 64 million individuals worldwide. Despite significant advances in pharmacologic therapy, device therapy, and lifestyle interventions, many patients continue to experience high rates of hospitalization, morbidity, and mortality.
Non-ischemic HFrEF, in particular, represents a subset of patients whose heart failure arises without prior myocardial infarction or ischemic injury, making targeted therapies that modulate cardiac molecular pathways particularly appealing. Current therapies can improve symptoms and survival but do not address the underlying molecular drivers of disease progression.
Gene therapy approaches such as AB-1002 are uniquely positioned to provide a one-time treatment that directly targets disease mechanisms, potentially reducing the need for chronic polypharmacy and repeated hospitalizations, and improving both longevity and quality of life.
The publication of the 12-month Phase 1 AB-1002 data represents an important milestone in the development of cardiac gene therapies. It highlights the feasibility, tolerability, and early signs of efficacy of this approach and lays the groundwork for larger, controlled trials designed to definitively assess clinical benefit.
AskBio’s continued commitment to innovation in heart failure therapy underscores the potential of precision gene therapy to transform outcomes for patients who currently have limited treatment options. The company remains focused on advancing AB-1002 through its clinical development program and providing the scientific community with ongoing updates as new data become available.
About the GenePHIT Phase 2 Trial
GenePHIT is designed to build upon the promising findings of the Phase 1 study by evaluating AB-1002 in a larger, more diverse cohort of patients with non-ischemic HFrEF. The study’s adaptive design allows for modifications based on interim analyses, ensuring both patient safety and efficient assessment of efficacy endpoints.
Key features of the GenePHIT trial include:
- Randomization and blinding to minimize bias and allow rigorous AskBio comparison between AB-1002 and placebo
- Primary endpoints focused on safety and measures of cardiac function
- Secondary endpoints including patient-reported outcomes, functional capacity, and biomarker changes
- Global enrollment, facilitating diverse patient representation and generalizable findings
By leveraging a randomized, placebo-controlled approach, GenePHIT will provide AskBio more robust data on the clinical potential of AB-1002 and inform future regulatory submissions and clinical development strategies.
About AB-1002
AB-1002 is an investigational gene therapy AskBio administered directly to the heart with the goal of enhancing production of a modified version of therapeutic inhibitor 1 (I-1c) protein. I-1c is designed to inhibit protein phosphatase 1, a key enzyme linked to impaired cardiac function in congestive heart failure. By modulating this pathway, AB-1002 aims to improve cardiac contractility and overall heart performance, addressing one of the root molecular contributors to HFrEF progression.
This investigational therapy has not been AskBio approved by any regulatory authority, and its efficacy and safety have not yet been fully established. The ongoing clinical development program, including the Phase 2 GenePHIT trial, will be essential to determining the potential role of AB-1002 in standard CHF management.
