Tenaya Therapeutics Reports Promising Early Data from MyPEAK™-1 Phase 1b/2 Clinical Trial of TN-201 for Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy

Core Insights - Tenaya Therapeutics reported early data from the MyPEAK-1 clinical trial of TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), indicating that TN-201 was well tolerated and showed promising early efficacy [2][3][4] Group 1: Clinical Trial Results - Preliminary data from three patients in the first dose cohort (3E13 vg/kg) showed 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] - Circulating biomarkers of cardiac muscle strain and injury remained stable, with some clinical measures showing improvement from baseline, particularly in Patients 1 and 2 [9][10] - The MyPEAK-1 trial is designed to assess the safety, tolerability, and clinical efficacy of TN-201, with a focus on symptomatic adults diagnosed with MYBPC3-associated HCM [12] 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 but were well managed and not indicative of liver damage [7] - Most adverse events were mild and transient, typical of AAV-based gene therapies [7] Group 3: Future Outlook - Tenaya plans to continue monitoring the initial patients and anticipates additional data readouts from Cohort 1 and a higher dose cohort in 2025 [3][4] - The company expressed confidence in TN-201's potential based on early evidence of transgene expression and cardiac transduction [10][11] - The MyPEAK-1 trial may expand to enroll up to 24 patients with MYBPC3-associated HCM in planned dose expansion cohorts [12]