<div id="readability-page-1"><div>Affinia Therapeutics Receives Approval from Health Canada to Initiate the UPBEAT© Trial, a Phase 1/2 Clinical Trial to Investigate AFTX-201 as a Treatment for BAG3-Associated Dilated Cardiomyopathy (DCM) An investigational medicine that addresses the genetic root cause of BAG3 DCM, AFTX-201 is being evaluated as a treatment for BAG3 DCM in the UPBEAT trial in the U.S. and Canada 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 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 the approval of its clinical trial application (CTA) by Health Canada 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. “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, Director of the Cardiovascular Genetics Centre at the Montréal Heart Institute, and the Philippa and Marvin Carsley Chair in Cardiovascular Genetics and Associate Professor at the University of Montréal. This approval from Health Canada follows recent advances in the clinical development of AFTX-201. Affinia’s Investigational New Drug (IND) application for AFTX-201 has been accepted by the U.S. Food and Drug Administration (FDA) and the FDA granted AFTX-201 Fast Track designation. The European Medicines Agency (EMA) has granted Orphan Drug designation to Affinia for AFTX-201. “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.” 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. Multiple trial sites across the U.S. and Canada are planned to be available. Travel arrangements can be made for interested participants not living near a trial site. Interested participants who have a BAG3 mutation, are 18 – 55 years old, and not able to perform normal activities 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 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. <img src="https://imageproxy.pbkrs.com/https://cts.businesswire.com/ct/CT/query-ZGlzdHJvPW54JmlkPWJ3bmV3cyZsYW5nPWVuJnNpZD1tc3RyMyZzdHk9MjAyNjA0MDY5MTAxNzZyMQ?x-oss-process=image/auto-orient,1/interlace,1/resize,w_1440,h_1440/quality,q_95/format,jpg" original-src="https://imageproxy.pbkrs.com/https://cts.businesswire.com/ct/CT/query-ZGlzdHJvPW54JmlkPWJ3bmV3cyZsYW5nPWVuJnNpZD1tc3RyMyZzdHk9MjAyNjA0MDY5MTAxNzZyMQ"/> Media contact: Kathy Vincent kathy@kathyvincent.com 310-403-8951 View source version on businesswire.com: https://www.businesswire.com/news/home/20260406910176/en/</div></div>

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Affinia Therapeutics 已获得加拿大卫生部的批准，启动 UPBEAT© 试验，这是针对 AFTX-201 的 1/2 期临床试验，旨在治疗与 BAG3 相关的扩张型心肌病（DCM）。AFTX-201 旨在通过一次性静脉注射解决 BAG3 DCM 的遗传原因。该试验将评估 AFTX-201 的安全性和有效性，此前已获得 FDA 的接受并获得快速通道认证。UPBEAT 试验将在美国和加拿大的多个地点进行，重点关注经过基因确认的 BAG3 DCM 患者

Affinia Therapeutics Receives Approval from Health Canada to Initiate the UPBEAT© Trial, a Phase 1/2 Clinical Trial to Investigate AFTX-201 as a Treatment for BAG3-Associated Dilated Cardiomyopathy (DCM)

Affinia Therapeutics 获得加拿大卫生部的批准，启动 UPBEAT©试验，这是一项 1/2 期临床试验，旨在研究 AFTX-201 用于治疗 BAG3 相关扩张型心肌病（DCM）的效果