Tenaya TN-201 MyPEAK-1 Interim HCM Data

Key Takeaways

• MyPEAK-1 (NCT05836259) May 2026 cutoff covers Cohort 1 at 3E13 vg/kg (78–104 weeks) and Cohort 2 at 6E13 vg/kg (26–52 weeks).
• Six evaluable patients reduced left ventricular mass index; five of six improved to NYHA Class I.
• TN-201 was generally well tolerated at both doses, with no new TN-201-linked safety events since the prior update.
• EMA granted PRIME designation; Tenaya plans continued 6E13 vg/kg enrollment and additional H2 2026 follow-up.

What did the June 3, 2026 TN-201 readout show?

Tenaya’s June 3, 2026 GlobeNewswire release said all six evaluable patients improved on multiple clinical parameters, including echocardiographic hypertrophy measures and at least one symptom-burden measure.

Five of six patients improved by at least one NYHA class and were Class I at the latest visit. Four of six had KCCQ Clinical Summary Score gains of 12 to 56 points. A change of ≥5 points on KCCQ-CSS is considered clinically meaningful.

Cardiac troponin improved or stayed stable in five of six patients. NT-proBNP, a marker of myocardial strain, improved in three patients. These biomarker shifts sit alongside imaging changes rather than replacing them.

How do Cohort 1 and Cohort 2 doses compare?

Cohort 1 used 3E13 vg/kg with longer follow-up of 78 to 104 weeks for three patients. Cohort 2 used 6E13 vg/kg with 26 to 52 weeks of follow-up for four patients. Left ventricular mass index declines appeared earlier after the higher dose.

Three evaluable Cohort 2 patients had a mean KCCQ-CSS increase of 36 points. Functional capacity improved on six-minute walk test or cardiopulmonary exercise testing in three patients overall, including 6MWT gains of 50 to 255 meters.

Patient 4 in Cohort 2 also showed a clinically meaningful rise in peak oxygen consumption. One-year CPET data for the remaining Cohort 2 patients were not available at the May 2026 cutoff, so dose-response on objective exercise capacity is still incomplete.

What is TN-201 designed to do in MYBPC3-HCM?

TN-201 is an AAV9 gene therapy delivering a working MYBPC3 gene intended to raise insufficient MyBP-C protein after a single intravenous infusion. Company materials place MYBPC3 variants among the most common genetic causes of HCM, about 20% of cases or roughly 120,000 U.S. patients.

The open-label MyPEAK-1 trial is registered as NCT05836259 on ClinicalTrials.gov. It enrolls symptomatic adults with MYBPC3-associated nonobstructive HCM and escalates across 3E13 and 6E13 vg/kg dose levels.

What safety findings were reported?

Across seven patients dosed to date, TN-201 was generally well tolerated at both dose levels. No new treatment-related safety events were identified since the prior update, and no dose-limiting toxicities were observed.

Immunosuppression monitoring changes after Cohort 1 allowed faster corticosteroid tapers and lower cumulative steroid exposure in Cohort 2 despite the higher vector dose, according to Tenaya. All patients had tapered off immunosuppression protocols as described in the release.

What regulatory designations does TN-201 hold?

The FDA has granted TN-201 Fast Track, Orphan Drug, and Rare Pediatric Drug designations. The European Commission has granted orphan medicinal product status. On June 3, 2026, Tenaya also reported EMA PRIME designation.

PRIME is a development-support pathway, not an approval. Marketing authorization still requires a complete dossier and CHMP plus European Commission decisions. U.S. designations likewise do not change the need for adequate and well-controlled evidence.

What remains unproven after this interim cut?

MyPEAK-1 remains a small, open-label Phase 1b/2 study. Durability beyond about two years is not established. Controlled comparisons against myosin inhibitors or standard care are not part of this interim package.

Remodeling and NYHA gains are encouraging signals. They are not yet confirmatory evidence for approval. Investors should wait for larger cohorts, fuller CPET completion, and a registrational design before treating TN-201 as de-risked.

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Frequently Asked Questions

Primary Sources

1. GlobeNewswire: Tenaya MyPEAK-1 interim data (June 3, 2026)
2. ClinicalTrials.gov: NCT05836259 MyPEAK-1
3. SEC exhibit: Tenaya TN-201 update