AskBio Announces Completion of Enrollment in Phase 2 Clinical Trial of AB-1002 Investigational Gene Therapy for Heart Failure

Durham, N.C., April 01, 2026 (GLOBE NEWSWIRE) --

• Final participant randomized in the GenePHIT Phase 2 clinical trial assessing safety and efficacy of AB-1002 for adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms
• Initial results are expected in the first half of 2027
• Completion of enrollment marks significant milestone in progression of heart failure clinical program and included the largest number of participants to date to receive AB-1002

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the randomization of the last participant was completed earlier this year for GenePHIT, its Phase 2 clinical trial of AB-1002, an investigational gene therapy being developed as a potential treatment for heart failure with reduced ejection fraction (HFrEF).

“Heart failure is a major public health challenge and places a massive strain on healthcare systems around the world,” said Timothy D. Henry, MD, MSCAI, GenePHIT Principal Investigator and Steering Committee Member. “Prevalence is increasing, and the need for innovative therapies has never been greater. Completing enrollment in this trial brings us another step closer to evaluating a potential treatment strategy for heart failure with reduced ejection fraction.”

GenePHIT includes 173 participants, and the completion of enrollment marks a significant milestone in the development of AB-1002, an investigational gene therapy administered via a single intracoronary infusion on top of the standard of care in adults with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms.¹ Initial trial results are expected in the first half of 2027.

“We are pleased to have randomized the last participant in our GenePHIT trial,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. “AskBio’s Phase 2 heart failure program investigates the efficacy and safety of AB-1002, which is designed to potentially mitigate the symptoms of heart failure with reduced ejection fraction and improve survival rates and quality of life. The data we receive from the participants will help us better understand the potential of our investigational gene therapy in an area of significant medical need.”

An estimated 64 million people worldwide are living with heart failure, and despite advances in treatment, mortality and morbidity remain very high.^2,3 The completion of enrollment represents a significant step in potentially bringing a new treatment to those who need it most.

AskBio explored AB-1002 in a Phase 1 non-randomized, sequential dose escalation trial for participants with NYHA Class III non-ischemic HFrEF.⁴ Twelve-month data were published online in Nature Medicine in October 2025 and in print in November 2025.⁵ These data had previously been presented in May 2025 as a late-breaker presentation at the European Society of Cardiology Heart Failure Meeting.⁶

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. 

About AB-1002

AB-1002 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, designed to block the action of protein phosphatase 1, which is linked to heart failure.^7,8

About Heart Failure

Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.⁹ This causes congestion in the body’s tissues.¹⁰ Symptoms may include shortness of breath, swelling in the legs and ankles caused by fluid retention, and fatigue.¹⁰ More than 64 million people worldwide are estimated to be living with heart failure.²

About GenePHIT

GenePHIT is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial conducted to evaluate the safety and efficacy of the one-time administration of investigational gene therapy AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy, New York Heart Association (NYHA) Class III heart failure symptoms and reduced ejection fraction.¹ The trial was designed to evaluate cardiovascular-related deaths and change in baseline from NYHA classification, left ventricular ejection fraction, and six-minute walking distance.¹ GenePHIT enrolled participants at 46 locations across the United States, Canada, Austria, Germany, the Netherlands, Spain, Belgium, Hungary, Poland, Bulgaria, Romania, and the United Kingdom. For more information, please visit euclinicaltrials.eu (EUCT#2024-510581-17-00), clinicaltrials.gov (NCT#05598333), or askbio.com.

About AskBio

AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to steering gene therapy into a new era where it can transform the lives of a wider range of people living with rare and more common diseases. The company maintains a portfolio of clinical programs across a range of disease indications related to a single gene or multiple factors across cardiovascular, central nervous system, and neuromuscular conditions, with a clinical-stage pipeline that includes investigational therapeutics for heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease. AskBio’s end-to-end gene therapy platform includes our Pro10™ technology and Aava™ manufacturing platform, which make gene therapies more accessible by making research and commercial grade manufacturing more affordable. With global headquarters in Durham, North Carolina, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing. An early innovator in the gene therapy field with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at http://www.askbio.com/ or follow us on LinkedIn.

About Bayer

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2025, the Group employed around 88,000 people and had sales of 45.6 billion euros. R&D expenses amounted to 5.8 billion euros. For more information, go to www.bayer.com.

AskBio Forward-Looking Statements

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding AskBio’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office. Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

References

[1] Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (GenePHIT). Available at: https://clinicaltrials.gov/study/NCT05598333. Last accessed: April 2026.

[2] Savarese G, Becher PM, Lund LH, et al. Global burden of heart failure: a comprehensive and updated review of epidemiology. Cardiovasc Res. 2023 Jan 18;118(17):3272-3287. 

[3] Myhre P, Tromp J, Ouwerkerk W, et al. Digital tools in heart failure: addressing unmet needs. Lancet Digit Health. 2024 Oct;6(10):e755-e766.

[4] Clinical trials.gov. AB-1002 in Patients With Class III Heart Failure (NAN-CS101). Available at: https://clinicaltrials.gov/study/NCT04179643. Last accessed: April 2026.

[5] Henry TD, Chung, E.S, Alvisi M, et al. Cardiotropic AAV gene therapy for heart failure: a phase 1 trial. Nat Med31, 3845–3852 (2025). https://doi.org/10.1038/s41591-025-04011-z

[6] Hajjar R. NAN-CS101: A first-in-human Phase 1 open-label dose-escalation study of AB-1002 gene therapy for the treatment of NYHA class III HF. Oral presentation. ESC Heart Failure Meeting 2025.

[7] Henry T, Chung E, Alvisiet M, et al. Preliminary safety and efficacy of a Phase 1 clinical gene therapy trial in patients with advanced heart failure using a rationally designed cardiotropic AAV vector targeting Protein Phosphatase Inhibitor-1. Presented at American Heart Association Scientific Sessions, November 2023.

[8] Nicolaou P & Kranias E. Role of PP1 in the regulation of Ca cycling in cardiac physiology and pathophysiology. Front Biosci (Landmark Ed). 2009 Jan 1;14(9):3571-85.

[9] Centers for Disease Control and Prevention. Heart failure. Published 2022. Available at: https://www.cdc.gov/heart-disease/about/heart-failure.html. Last accessed: April 2026.

[10] American Heart Association. Heart Failure Signs and Symptoms. Available at: https://www.heart.org/en/health-topics/heart-failure/warning-signs-of-heart-failure. Last accessed: April 2026.

Phil McNamara AskBio Inc. (AskBio) +1 (984) 5207211 pmcnamara@askbio.com