FDA Grants Orphan Drug Designation to Affinia Therapeutics’ AFTX-201 for the Treatment of BAG3-Associated Dilated Cardiomyopathy (DCM)

AFTX-201 is a potential best-in-class investigational genetic medicine given as a simple one-time IV infusion designed to address the underlying mutation responsible for BAG3 DCM

AFTX-201 is being evaluated as a treatment for BAG3-associated DCM in the UPBEAT^© clinical trial actively recruiting at multiple sites in the U.S. and Canada

BAG3 DCM leads to early onset progressive heart failure, impaired quality of life, and reduced survival, despite current treatments

WALTHAM, Mass.--(BUSINESS WIRE)--Affinia Therapeutics (“Affinia”), an innovative clinical-stage 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 Orphan Drug designation for AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy (DCM).

A potential best-in-class investigational genetic medicine given as a simple one-time intravenous (IV) infusion, AFTX-201 is designed to address the underlying mutation responsible for BAG3-associated DCM. AFTX-201 is designed to deliver a functional BAG3 transgene utilizing Affinia’s proprietary rationally designed capsid, which is engineered for efficient cardiac transduction at doses that are 5-10-fold lower than those used in traditional gene therapies employing conventional capsids like AAV9 or AAVrh74. Preclinical studies in an animal model of BAG3 DCM demonstrated that AFTX-201 increased BAG3 protein levels in the heart, completely restored cardiac function, and reversed the structural abnormalities of the heart that are characteristic of the disease. A survival benefit was also seen.

AFTX-201 is being evaluated as a treatment for BAG3 DCM in the UPBEAT^© clinical trial (NCT07426419; www.clinicaltrials.gov) in the U.S. and Canada. The UPBEAT clinical trial is a Phase 1/2 study to evaluate the safety and efficacy of AFTX-201 in adults diagnosed with BAG3 DCM who are 18 to 55 years of age who experience challenges in performing everyday physical activities due to heart failure.

“Receiving Orphan Drug designation, in addition to the recent Fast Track designation, from the FDA marks a significant achievement for the AFTX-201 program and we thank the FDA for their decision,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “AFTX-201 is designed to address the underlying root cause of BAG3 DCM. The UPBEAT clinical trial is actively recruiting at multiple institutions and we look forward to continue progressing the AFTX-201 clinical program so that we can bring this potentially transformative treatment to people living with BAG3 DCM.”

“BAG3 DCM is a devastating heart disease with a known genetic cause. It is a condition that leads to early onset, progressive heart failure, and premature death, with no approved treatment that addresses the underlying mechanism. The DCM Foundation and Genetic Cardiomyopathy Awareness Consortium commend the FDA for awarding Orphan Drug designation to AFTX-201 for the treatment of BAG3-associated DCM, acknowledging the pressing demand for innovative treatment alternatives for rare diseases like BAG3 DCM for which there remains significant unmet medical need,” said Greg Ruf, Founder and Executive Director, the DCM Foundation.

BAG3-associated DCM is a disease in which a genetic mutation reduces production of BAG3 protein in cardiomyocytes, leading to early onset progressive heart failure, impaired quality of life, and reduced survival, despite current treatments. Based on current published literature, the estimated prevalence of BAG3 mutation-associated DCM is estimated to account for 2.3-3.6% of DCM worldwide. There is no approved treatment that addresses the underlying mechanism of BAG3 DCM and new treatment options are urgently needed.

The FDA’s Orphan Drug designation is intended to encourage the development of treatments for rare diseases, defined as those affecting fewer than 200,000 people in the United States. Orphan Drug designation provides certain development and commercial incentives, including tax credits for qualified clinical trial costs, exemption from FDA user fees for marketing applications, and the potential for seven years of market exclusivity upon FDA approval. The designation can also facilitate interactions with the FDA and support eligibility for additional expedited development programs.

About Affinia Therapeutics

Affinia Therapeutics is a clinical-stage biotech company pioneering a transformational treatment paradigm 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.