An investigational medicine that addresses the genetic root cause of BAG3 DCM, AFTX-201 is being evaluated in the UPBEAT© clinical trial
AFTX-201 is designed using 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
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 granted Fast Track designation (FTD) for AFTX-201, a potential best-in-class investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM).
AFTX-201 is designed to deliver a fully human, full-length 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 therapies using conventional capsids such as AAV9 or AAVrh74. The safety and efficacy of AFTX-201 as a treatment for people living with BAG3 DCM is being investigated in the UPBEAT© clinical trial. AFTX-201 is 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.
“We are grateful to the FDA for granting the Fast Track designation to AFTX-201 as a treatment for BAG3 DCM and to the patient and physician communities for their support of our UPBEAT clinical trial,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “This Fast Track designation, together with the FDA’s recent approval of our IND application for AFTX-201 and the European Medicines Agency’s designation of AFTX-201 as an Orphan Drug, strengthens our conviction that AFTX-201 has the potential to significantly improve the lives of those affected by BAG3 DCM.”
“DCM is the leading cause of heart failure in young people and the most frequent indication for heart transplantation despite current treatments,” said Greg Ruf, Founder and Executive Director of the DCM Foundation. “People whose DCM is caused by a genetic mutation such as BAG3 may benefit from a gene therapy approach.”
Fast Track designation is designed to help drugs reach patients faster based on the drug’s potential to fill an unmet medical need and treat serious conditions. Programs receiving FTD benefit from early and frequent interactions with the FDA during the clinical development process and review of the marketing application on a rolling basis before the sponsor submits the complete application. If relevant criteria are met, the FDA may also consider an accelerated approval and priority review of the marketing application. These benefits could decrease the development time of the drug to approval.
About the UPBEAT© clinical trial
The UPBEAT clinical trial (NCT07426419 on clinicaltrials.gov) 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 to Affinia via [email protected] and to their medical care provider.
About Affinia Therapeutics
Affinia Therapeutics is a clinical-stage biotech 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
[email protected]
310-403-8951
