Tenaya Therapeutics(US:TNYA) Globenewswire·2025-03-31 14:00Core Insights - Tenaya Therapeutics announced promising interim data from the MyPEAK-1 Phase 1b/2 clinical trial of TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), presented at the 2025 American College of Cardiology Scientific Sessions [1][2][3] Group 1: Clinical Trial Data - TN-201 was well tolerated at a dose of 3E13 vg/kg, with treatment-emergent adverse events primarily mild and manageable [2][3] - All three patients in Cohort 1, who had severe disease at baseline, achieved NYHA Class I, indicating no limitations on physical activity [3][4] - Serial biopsies showed sustained presence of TN-201 DNA in the heart and robust RNA expression, increasing up to 13-fold from Week 8 to Week 52 post-dose [2][3] - MyBP-C protein levels increased from 56% to 59% and from 62% to 64% of normal between Week 8 and Week 52 for Patients 1 and 2, respectively [2][3] - Cardiac troponin levels decreased by more than 60% in two patients, returning to normal or near normal [2][3] Group 2: Future Expectations - Enrollment for Cohort 2 is expected to be completed in the first half of 2025, with initial data to be reported in the second half of 2025 [1][2][8] - The company anticipates sharing additional data from Cohort 1 and initial safety and biopsy data from Cohort 2 later this year [3][8] - Tenaya has updated its cash guidance into the second half of 2026, positioning itself to achieve important clinical data milestones for both TN-201 and TN-401 gene therapy programs [3][4] Group 3: Background on MYBPC3-Associated HCM - MYBPC3 mutations are the most common genetic cause of HCM, affecting approximately 120,000 patients in the U.S. alone [12][13] - Patients with MYBPC3-associated HCM are at higher risk for serious outcomes, including heart failure and sudden cardiac death, with younger patients experiencing more rapid disease progression [11][12]