One-time gene therapy could change treatment of heart failure

Heart monitor

Asklepios BioPharmaceutical (AskBio), a gene therapy subsidiary of Bayer, has presented first-in-human data from a Phase I trial investigating AB-1002 (also known as NAN-101) for the treatment of patients with congestive heart failure (CHF).

The data were shared at the American Heart Association Scientific Sessions, which were held in Philadelphia, US, 11-13 November 2023.

The trial was designed to establish the safety and preliminary efficacy of gene therapy AB-1002 in patients with NYHA Class III heart failure, also known as advanced heart failure.

AB-1002 is a rationally designed cardiotropic AAV vector targeting protein phosphatase inhibitor-1, which has been linked to heart failure.

“We believe these encouraging early results in patients with advanced heart failure are important for the congestive heart failure community, as they bring hope to a sub-population where treatment options are needed,” said Litsa Kranias, Hanna Chair of Cardiology at the University of Cincinnati and US Coordinator, Cure-PLaN. “Seeing the potential of gene therapy being explored in heart failure 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 westernised countries.”

Treating the underlying cause of heart failure

In the trial, single-dose administration of AB-1002 resulted in clinically meaningful improvements in key efficacy parameters.

Among the six patients in Cohort 1, three who completed 12-month follow-up showed clinically meaningful improvements in left ventricular ejection fraction (LVEF), NYHA Functional Class (NYHA FC), Minnesota Living with Heart Failure Questionnaire (MLHFQ), cardiopulmonary exercise test (VO2 max), and six-minute walk test (6MWT) at 12 months post-dose.

Among the four patients evaluated in Cohort 2, two showed clinically meaningful improvements in MLHFQ and NYHA FC, and four (100%) showed clinically meaningful improvements in LVEF at 12 months, compared with baseline.

No treatment emergent adverse events (TEAEs) or serious adverse events (SAEs) were deemed related to study treatment.

Timothy Henry, Principal Investigator and Steering Committee Member, commented: “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-ischaemic congestive heart failure.”

Related Articles

Join FREE today and become a member
of Drug Discovery World

Membership includes:

  • Full access to the website including free and gated premium content in news, articles, business, regulatory, cancer research, intelligence and more.
  • Unlimited App access: current and archived digital issues of DDW magazine with search functionality, special in App only content and links to the latest industry news and information.
  • Weekly e-newsletter, a round-up of the most interesting and pertinent industry news and developments.
  • Whitepapers, eBooks and information from trusted third parties.
Join For Free