Tenaya’s TN-201 Gene Therapy Reduces LV Mass Index Over Two Years and Improves Walk Tests by Up to 255m
TNYA•Tenaya’s TN-201 gene therapy drove sustained left ventricular mass index decreases in six MYBPC3-HCM patients, with Cohort 1 maintaining reductions through 104 weeks and Cohort 2 achieving earlier remodeling at a 6E13 vg/kg dose. Patients also recorded 50–255m six-minute walk test improvements and 12–56 point KCCQ gains.
1. MyPEAK-1 Phase 1b/2 Trial Overview
The MyPEAK-1 trial enrolled seven patients with severe nonobstructive MYBPC3-associated hypertrophic cardiomyopathy, dosing three at 3E13 vg/kg (Cohort 1) and four at 6E13 vg/kg (Cohort 2). Follow-up ranged from 78–104 weeks for Cohort 1 and 26–52 weeks for Cohort 2, with six patients evaluable for efficacy endpoints.
2. Left Ventricular Remodeling Results
All six evaluable patients achieved reductions in left ventricular mass index and wall thickness measures. Cohort 1 maintained LVMI decreases through 104 weeks, while Cohort 2 showed comparable hypertrophy reversal at an earlier timepoint post 6E13 vg/kg infusion.
3. Functional and Symptom Improvements
Five of six patients improved New York Heart Association classification by at least one class, with all reaching Class I. Four patients saw 12–56 point increases in Kansas City Cardiomyopathy Questionnaire scores, and three achieved 50–255m gains on six-minute walk tests, indicating enhanced exercise capacity.
4. Biomarker Changes and Regulatory Status
Cardiac troponin remained stable or improved in five of six patients, and NT-proBNP levels dropped in three. MyBP-C protein expression rose by an average 4% on serial biopsies. TN-201 has earned EMA PRIME designation and entered FDA’s Rare Disease Evidence Principles process for a pediatric indication, with a webcast scheduled to review these data.
