Announcement • Sep 15

Tenaya Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Designation For TN-401 For Treatment Of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy

Tenaya Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TN-401, Tenaya’s gene therapy candidate for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC). RMAT designation is supported by positive interim data from RIDGE-1 Phase 1b/2 clinical trial of TN-401 which demonstrated reduced arrhythmia burden. Additional data and updates on regulatory discussions are anticipated in the Fourth Quarter 2026. Tenaya’s RMAT application included data presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in May 2026. Interim results from the RIDGE-1 clinical trial of TN-401 showed improvements in multiple electrical stability measures, including reduced rates of premature ventricular contractions (PVCs) and non-sustained ventricular tachycardias (NSVTs), two of the most frequently occurring forms of ventricular arrhythmias that characterize ARVC. RMAT designation is an FDA expedited program intended to facilitate the development and review of regenerative medicine therapies for serious conditions where preliminary clinical evidence indicates the potential to address unmet medical needs. RMAT designation provides enhanced opportunities for interaction with the FDA, including early and ongoing guidance regarding clinical development, manufacturing and potential regulatory pathways, and it may provide eligibility for accelerated approval, priority review and rolling review. Plakophilin-2 (PKP2) mutations are the most common genetic cause of arrhythmogenic right ventricular cardiomyopathy (ARVC, also known as arrhythmogenic cardiomyopathy or ACM), occurring in approximately 40% of the overall ARVC population. The prevalence of PKP2-associated ARVC is estimated at more than 70,000 people in the U.S. In PKP2-associated ARVC, mutations of the PKP2 gene results in insufficient expression of a protein needed for the proper functioning of the desmosomal complex that maintains physical connections and electrical signaling between heart muscle cells. As the desmosome structure degrades, cardiac muscle cells are replaced by fibrofatty tissue and electrical pulses in the heart become unstable, resulting in potentially life threatening heart rhythms. ARVC symptoms include arrhythmias, palpitations, lightheadedness, dizziness and fainting. It is typically diagnosed before age 40, and sudden cardiac arrest due to ventricular arrhythmia is frequently the first manifestation of disease. Current treatments include anti-arrhythmic medications, implantable cardioverter-defibrillators (ICDs) and ablation procedures, which do not address the underlying genetic cause of disease. TN-401 is an investigational AAV9-based gene therapy being developed for the treatment of ARVC due to mutations in the PKP2 gene. AAV9 was selected as the vector for delivery of Tenaya’s PKP2 gene therapy based on its extensive clinical and commercial safety record and demonstrated ability to target heart muscle cells. TN-401 has received Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration, as well as PRIME designation from the European Medicines Agency. Tenaya’s development of TN-401 is supported in part by a grant from the California Institute for Regenerative Medicine (CIRM). The RIDGE-1 Phase 1b/2 clinical trial ClinicalTrials.gov (NCT06228924) of TN-401 in patients with PKP2-associated ARVC is a multi-center, open-label, dose escalation study being conducted in the U.S. and UK. RIDGE-1 is intended to assess the safety, tolerability and preliminary clinical efficacy of a one-time intravenous infusion of TN-401. RIDGE-1 seeks to enroll up to fifteen adults who have been diagnosed with PKP2-associated ARVC, have an ICD and are at increased risk for arrhythmias as determined by premature ventricular count (PVC) during screening. As part of its TN-401 development program, Tenaya is also conducting RIDGE ClinicalTrials.gov (NCT06311708), believed to be the largest natural history study of adults with PKP2-associated ARVC.

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