WALTHAM, Mass. — Affinia Therapeutics has received approval from Health Canada to begin a Phase 1/2 clinical trial evaluating AFTX-201, an investigational gene therapy targeting BAG3-associated dilated cardiomyopathy, a rare and often fatal inherited heart condition.
The therapy, designed to address the genetic root cause of the disease, will be studied in the UPBEAT clinical trial across sites in the U.S. and Canada. AFTX-201 is administered as a one-time intravenous infusion and uses a proprietary engineered capsid intended to enhance delivery to heart tissue at doses significantly lower than those used in conventional gene therapies.
Preclinical studies demonstrated that the therapy increased BAG3 protein levels in the heart and restored cardiac function in animal models, supporting its advancement into human trials.
“BAG3 DCM is a serious, inherited heart condition with a high mortality and a large unmet medical need. It leads to early onset, progressive heart failure, and premature death and there is no treatment that exists which targets the underlying mechanism of disease. We applaud Affinia for developing promising therapies such as AFTX-201 that address the genetic root cause of BAG3 DCM,” said Rafik Tadros, M.D., Ph.D., Canadian Research Chair in Translational Cardiovascular Genetics and Director of the Cardiovascular Genetics Centre at the Montréal Heart Institute.
The Canadian clearance follows recent progress in the U.S., where the Food and Drug Administration accepted Affinia’s Investigational New Drug application and granted AFTX-201 Fast Track designation. The European Medicines Agency has also granted Orphan Drug designation for the therapy.
“The approval of our CTA in Canada for AFTX-201 is another significant clinical milestone, following the FDA’s recent acceptance of our IND application and granting of the Fast Track designation for AFTX-201,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “We are working diligently with multiple trial sites and investigators across the U.S. and Canada to begin patient enrollment and dosing in the next few weeks in the UPBEAT clinical trial so that we can bring this potentially transformative treatment to patients living with BAG3 DCM and make a meaningful impact in their lives.”
The UPBEAT trial is a multicenter, open-label study designed to evaluate safety, tolerability, pharmacodynamics, and early signs of efficacy in adults with genetically confirmed BAG3-associated dilated cardiomyopathy. Participants will receive a single infusion of AFTX-201 and be monitored for 52 weeks, with additional assessments tracking biological activity and clinical outcomes.
BAG3-associated dilated cardiomyopathy affects more than 70,000 people across major global markets and is caused by mutations that impair a protein essential to maintaining heart muscle structure and function. The disease often leads to early-onset heart failure, and nearly a quarter of patients ultimately require a heart transplant despite existing treatments.
