AB-1002 Demonstrates Improvements for Congestive Heart Failure in First-in-Human Study


Improvements were seen in left ventricular ejection fraction, NYHA Functional Class, and Minnesota Living with Heart Failure Questionnaire at 1-year post-dose.

First-in-human phase 1 study data revealed AB-1002 (Asklepios BioPharmaceuticals Inc), also known as NAN-101, demonstrated clinically meaningful improvements in left ventricular ejection fraction (LVEF) in individuals with congestive heart failure (CHF). The data were presented at the American Heart Association Scientific Session from November 11 to 13, 2023, in Philadelphia, Pennsylvania.1

Female with chest pain | Image Credit: Dragana Gordic - stock.adobe.com

Dragana Gordic - stock.adobe.com

“We believe these encouraging early results in patients with advanced [HF] are important for the [CHF] community, as they bring hope to a sub-population where treatment options are needed,” Litsa G. Kranias, PhD, Hanna Chair of Cardiology at the University of Cincinnati, said in a press release. “Seeing the potential of gene therapy being explored in [HF] is a key step forward in one day potentially changing the direction of this devastating disease, which is a leading cause of morbidity and mortality in westernized countries.”1

According to the National Library of Medicine, approximately 6.2 million individuals in the United States had HF from 2013 to 2016, with the current worldwide prevalence of CHF being approximately 64.34 million cases worldwide.2 HF is also the primary cause of hospitalization in elderly individuals in the United States and accounts for approximately 8.5% of cardiovascular-related deaths. The report also stated that HF increases with age and metabolic risk factors, combined with sedentary lifestyles, are major risk factors. In men, the incidence of HF doubles with each 10-year age increase after age 65 and women’s risk triples for the same age cohort, according to the report.2

The study’s aim was to establish the safety and preliminary efficacy of AB-1002, a gene therapy that targets protein phosphatase inhibitor-1, in individuals with advanced HF. Protein phosphatase inhibitor-1 has been linked with HF.1

Among the 6 individuals in cohort 1, 3 individuals who received a single-dose of AB-1002 and completed a 12-month follow-up demonstrated improvements in LVEF, NYHA Functional Class (NYHA FC), Minnesota Living with Heart Failure Questionnaire (MLHFQ), cardiopulmonary exercise test, and 6-minute walk test at 1-year post-dose. The other 3 individuals are within the first 3 months post-dose, according to the press release.1

Among 5 individuals in Cohort 2, only 4 could be evaluated due to a fatal serious adverse event (SAE) in 1 individual, which was determined to not be related to the study treatment. Investigators found that 2 individuals had clinically meaningful improvements in MLHFQ and NYHA FC, and 4 showed clinically meaningful improvements in LVEF at 1 year.1

There were no treatment emergent adverse events or SAEs related to the study treatment, according to the press release.1

“Although current management has improved survival in CHF patients, most therapies do not treat the underlying causes, consequently the current standard of care does not reverse the trajectory of the disease to ultimate end-stage heart failure and death. These results show delivery of AB-1002 was well tolerated and resulted in positive efficacy outcomes in some patients with non-ischemic congestive heart failure,” Timothy D. Henry, MD, MSCAI, the principal investigators and Steering Committee member, said in the press release.1


  1. AskBio presents preliminary data from phase 1 trial of gene therapy for congestive heart failure at the 2023 American Heart Association Scientific Session. News release. Bayer Global. November 13, 2023. Accessed November 14, 2023. https://www.bayer.com/media/en-us/askbio-presents-preliminary-data-from-phase-1-trial-of-gene-therapy-for-congestive-heart-failure-chf-at-the-2023-american-heart-association-scientific-sessions/
  2. Malik A, Brito D, Vaqar S, Chhabra L. Congestive Heart Failure. Treasure Island (FL): StatPearls Publishing; November 7, 2022. Accessed November 13, 2023. https://www.ncbi.nlm.nih.gov/books/NBK430873/
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