Affinia Therapeutics Announces FDA Acceptance of IND Application to Advance AFTX-201 to a Phase 1/2 Trial for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM)

AFTX-201 uses Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than doses of gene therapies using conventional capsids

Company on track to initiate the UPBEAT^© clinical trial to investigate safety, pharmacodynamics, and preliminary efficacy of AFTX-201 in BAG3 DCM in the first half of 2026

WALTHAM, Mass.–(BUSINESS WIRE)–Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating cardiovascular diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AFTX-201, an investigational genetic medicine for the treatment of BAG3 DCM.

“We are grateful to the FDA for the timely review of our IND application for AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy and to the patient and clinician community who have provided valuable input and support for our investigational program to-date,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “We look forward to initiating the UPBEAT clinical trial at multiple trial sites in the coming weeks and bringing a much-needed treatment option for patients and families affected by this devastating disease.”

The UPBEAT^© clinical trial is a multicenter, open-label Phase 1/2 clinical trial designed to evaluate the safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201 in adults with genetically confirmed BAG3-associated dilated cardiomyopathy.

“BAG3 DCM is a genetic heart disease with significant medical need despite current standard of care,” said Matthew Wheeler, M.D., Ph.D., a physician scientist in genetic cardiomyopathies and Associate Professor in Cardiovascular Medicine at Stanford Medicine. “A gene therapy approach could make a real difference to patients living with BAG3 DCM.”

AFTX-201 is designed to deliver a fully human, full-length functional BAG3 transgene using Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than those associated with gene therapy products using conventional capsids such as AAV9 or AAVrh74. AFTX-201 is intended to be given as a simple one-time intravenous administration. Preclinical studies in an animal disease model demonstrated that AFTX-201 increased BAG3 protein levels in the heart and completely restored cardiac function.

About the UPBEAT^© clinical trial

The UPBEAT^© clinical trial is a multicenter, single-arm open-label Phase 1/2 clinical trial designed to evaluate the safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201 in adults with genetically confirmed BAG3-associated dilated cardiomyopathy. The trial includes a dose-exploration phase followed by a dose-expansion phase. All participants will receive a single intravenous infusion of AFTX-201 at a dose that has been deemed safe and efficacious based on preclinical studies. The primary objective of the trial is to evaluate safety and tolerability through 52 weeks following administration. Secondary and exploratory objectives include pharmacodynamic and preliminary efficacy assessments, which will be evaluated as changes from baseline. Study design, dose selection, and monitoring plans are informed by input from patients, clinicians, and regulators and by completed nonclinical proof-of-concept, biodistribution, and safety studies, which demonstrated complete correction of heart ejection fraction to normal (wild type) level and adequate safety margin for the doses being explored in the clinical trial. Protocol-defined stopping rules, centralized safety data review, and oversight by an independent Data Safety Monitoring Board are implemented to ensure the safety of participants. Interested participants are encouraged to reach out via clinicaltrials@affiniatx.com.

About BAG3 DCM

BAG3 dilated cardiomyopathy (DCM) is a serious, inherited heart condition with a high mortality rate and a significant unmet medical need. The disease affects more than 70,000 patients in the Canada, E.U., U.S., and U.K. regions. The BAG3, or Bcl2-associated athanogene 3, gene encodes for a protein that is critical to the normal structure and function of heart cells. Patients with BAG3 DCM have a mutation in the BAG3 gene and a deficiency in functional BAG3 protein, resulting in early onset heart failure that progresses rapidly. Despite current standard of care, almost 25% of patients require a heart transplant.

About Affinia Therapeutics

Affinia Therapeutics is a clinical-stage company pioneering a shift to a new class of rationally designed gene therapies that treat rare and prevalent diseases. Affinia Therapeutics’ pipeline of first-in-class or best-in-class product candidates, initially in cardiovascular diseases, leverages its proprietary next-generation capsids, payloads, or manufacturing approaches and have shown efficacy, safety, and differentiation in relevant animal models. For more information, visit https://www.affiniatx.com.

Contacts

Media contact:
Kathy Vincent
kathy@kathyvincent.com
310-403-8951