Clinical TrialJun 3, 2026, 07:01 AM
Tenaya TN-201 Phase 1b/2 Trial Shows Positive HCM Data
AI Summary
Tenaya Therapeutics reported positive interim safety and efficacy data from its MyPEAK-1 Phase 1b/2 clinical trial of TN-201 for MYBPC3-associated hypertrophic cardiomyopathy (HCM). All evaluable patients demonstrated improvements in multiple clinical parameters, including echocardiographic measures of hypertrophy and symptom burden, with higher doses showing earlier and more substantial changes. Additionally, TN-201 received PRIME designation from the EMA and was accepted into the FDA's Rare Disease Evidence Principles process for pediatric HCM.
Key Highlights
- All 6 evaluable patients achieved improvements in multiple clinical parameters of MYBPC3-associated HCM.
- Cohort 1 (3E13 vg/kg) showed sustained benefits up to two years (78-104 weeks follow-up).
- Cohort 2 (6E13 vg/kg) showed greater symptom relief and improved cardiac function at earlier timepoints.
- All 6 evaluable patients achieved reductions in left ventricular mass index (LVMI).
- 5 of 6 patients improved NYHA classification by at least one class, now Class I.
- 4 of 6 patients had KCCQ-CSS improvements of 12-56 points; Cohort 2 mean increase of 36 points.
- TN-201 granted PRIority MEdicine (PRIME) designation by the European Medicines Agency (EMA).
- TN-201 accepted into FDA's Rare Disease Evidence Principles (RDEP) process for pediatric HCM.
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