SOUTH SAN FRANCISCO, Calif., June 3, 2026
Tenaya Therapeutics announced new interim data from its ongoing MyPEAK™-1 Phase 1b/2 clinical trial evaluating TN-201 gene therapy in adults with MYBPC3-associated hypertrophic cardiomyopathy (HCM). Results from six evaluable patients showed improvements across multiple hallmarks of disease, including reductions in cardiac hypertrophy, improvements in heart failure symptoms, enhanced quality of life, and gains in functional capacity. The data included follow-up of up to two years for patients in the low-dose cohort and up to one year for patients in the higher-dose cohort.
TN-201 Demonstrated Evidence of Cardiac Remodeling and Symptom Relief
Tenaya reported that all six evaluable patients experienced reductions in left ventricular mass index (LVMI), a key measure of cardiac hypertrophy and disease severity in HCM. Five of six patients also demonstrated reductions in one or more measures of heart wall thickness. Improvements were durable through two years in the first cohort, while patients receiving the higher dose appeared to achieve benefits earlier. In addition, five of six patients improved by at least one New York Heart Association (NYHA) functional class, with all five reaching Class I status, indicating no significant limitations from heart failure symptoms during daily activities.
Functional Capacity and Quality of Life Improved Following Treatment
The study also demonstrated clinically meaningful improvements in symptom burden and quality of life. Four patients achieved substantial gains in Kansas City Cardiomyopathy Questionnaire (KCCQ) scores, ranging from 12 to 56 points above baseline. All three evaluable patients in the higher-dose cohort reported improved KCCQ scores, with an average increase of 36 points. Three patients also experienced meaningful improvements in exercise capacity as measured by six-minute walk testing, with increases ranging from 50 to 255 meters. One higher-dose patient additionally demonstrated significant improvement in peak oxygen consumption, suggesting enhanced cardiovascular performance.
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Gene Therapy Remained Well Tolerated as Development Advances
TN-201 continued to demonstrate a favorable safety profile, with no new treatment-related safety signals reported since the previous data update. No dose-limiting toxicities were observed, and all treated patients successfully discontinued immunosuppressive medications. Biopsy analyses confirmed successful gene delivery and increased expression of MyBP-C protein, the deficiency underlying MYBPC3-associated HCM. Alongside the clinical update, Tenaya announced that TN-201 received PRIME designation from the European Medicines Agency and was accepted into the FDA’s Rare Disease Evidence Principles (RDEP) program for pediatric patients. The company plans to provide additional long-term follow-up data and regulatory updates during the second half of 2026.
Source: Tenaya Therapeutics press release
