Tenaya Therapeutics(TNYA) Newsfilter·2024-12-17 12:00Core Insights - Tenaya Therapeutics reported early data from the MyPEAK-1 clinical trial of TN-201 gene therapy, showing it was well tolerated and demonstrated promising early efficacy in treating MYBPC3-associated hypertrophic cardiomyopathy (HCM) [2][3][4] Group 1: Clinical Trial Results - Preliminary data from three patients in the first dose cohort (3E13 vg/kg) indicated that TN-201 was generally well tolerated, with detectable vector DNA in the heart and increasing levels of TN-201 mRNA and MyBP-C protein over time [3][6] - Clinical measures of HCM mostly remained stable or showed improvement from baseline, with some patients experiencing better NYHA classification scores [10][11] - The trial is ongoing, with additional data readouts expected from Cohort 1 and a higher dose cohort anticipated in 2025 [3][4] Group 2: Safety Profile - TN-201 exhibited a manageable safety profile, with no observed cardiac toxicities or serious adverse events related to the treatment [6][7] - Isolated elevations in liver enzymes were noted, which were well managed and consistent with known side effects of AAV-based gene therapies [6][7] Group 3: Mechanism and Efficacy - TN-201 achieved robust transduction into cardiomyocytes, with measurable transgene RNA expression and a 50% increase in TN-201 mRNA levels observed at Week 52 for one patient [8][9] - Total levels of MyBP-C protein showed a 3% increase from Weeks 8 to 52, indicating successful transcription and expression of the gene therapy [9] Group 4: Future Outlook - The company expressed confidence in TN-201's potential based on the early data, with plans to continue monitoring patients and gathering more data [12] - The MyPEAK-1 trial is designed to assess the safety and efficacy of TN-201, with a focus on symptomatic adults diagnosed with MYBPC3-associated HCM [14][15]