Tenaya to present two-year TN-201 gene therapy data in HCM in H2 2026
MyPEAK-1's planned late-2026 update will extend safety and MyBP-C biomarker signals already reported from a 7-patient dose-finding cohort in a trial with no direct clinical competitor.
Executive Summary
- Tenaya Therapeutics intends to show longer-term follow-up on both dose cohorts of its MYBPC3 gene therapy for hypertrophic cardiomyopathy later this year, building directly on safety and biomarker data it has already disclosed.
- The therapy is being tested in a trial with no direct clinical comparator, so the update functions as the primary evidence base for whether a single-dose gene therapy can durably alter the disease biology behind this inherited cardiomyopathy.
- The study remains actively recruiting toward a 30-patient enrollment target, and its primary completion timeline has already been pushed out substantially since the trial began, a fact that shapes how much this interim update should be read as a bridge, not a final answer.
- Tolerability across both dose levels and a dose-dependent biomarker signal reported earlier put the coming data update in a position to extend, not introduce, the clinical story.
The catalyst
Tenaya said in its March 11, 2026 fourth-quarter update that it expects to share longer-term follow-up data from MyPEAK-1 (TN-201) for MYBPC3-associated hypertrophic cardiomyopathy (HCM) during 2026, alongside a separate update on its TN-401 program for a different cardiomyopathy. The trial, registered as NCT05836259, is an open-label, single-ascending-dose Phase 1/2 study testing TN-201, an AAV9-delivered gene therapy meant to restore MyBP-C protein production in patients with MYBPC3 gene mutations that cause HCM. The registered primary endpoints are the number and severity of adverse events and the number of drug-related serious adverse events, making safety, not efficacy, the formal bar this trial is designed to clear. Tenaya+1Tenaya Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Provides ...Mar 11, 2026Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCMNCT05836259
Probability of SuccessBased on the AppliedXL Probability of Success model. For more information about the methodology, read the research here.
What's already known
Tenaya disclosed interim results from this trial in November 2025 covering seven patients dosed to date: three at a 3E13 vg/kg dose (Cohort 1, followed 52 to 78 weeks) and three at a higher 6E13 vg/kg dose (Cohort 2, as of a July 2025 data cutoff). No dose-limiting toxicities were observed at either dose level, all patients tapered off immunosuppressive medicine, and MyBP-C protein levels rose over time in both cohorts, with a larger increase seen in the first evaluable Cohort 2 patient at the higher dose. Multiple patients followed more than 26 weeks showed improvement in circulating injury biomarkers and hypertrophy measures, and all patients with efficacy assessments improved to New York Heart Association Class I on functional status. A data safety monitoring board recommended advancing to dose expansion based on this tolerability picture. TenayaTenaya Therapeutics Reports Fourth Quarter and Full Year 2025 Financial Results and Provides ...Mar 11, 2026
Trial mechanics
The trial's primary completion date has moved from December 2025 to August 2027, a shift recorded alongside an enrollment target increase from 15 to 30 patients in November 2024. That enrollment increase, in an early-phase dose-escalation trial expanding to accommodate additional cohorts, sits within the range the operational model treats as routine rather than a departure from plan. The trial remains in Recruiting status across ten U.S. sites, with completion of the full study not expected until August 2032. NCT05836259Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCMNCT05836259
The competitive frame
No other clinical-stage program targets MYBPC3 with a gene-therapy approach in hypertrophic cardiomyopathy, and the broader HCM field is dominated by small-molecule mechanisms: cardiac myosin inhibitors such as aficamten and sodium-channel or SGLT modulators such as ranolazine and sotagliflozin, none of which shares TN-201's mechanism of correcting the underlying protein deficit. That leaves the H2 2026 update as the only source of clinical evidence on whether a genetic-correction strategy can produce durable biomarker and functional change in this population, with no resolved same-mechanism trial to benchmark it against.
This analysis was produced using AI-assisted reporting systems, AppliedXL data, and official public records. These systems undergo editorial review, quality checks, and regular audits by human experts. Errors may still occur, as with any automated system. Always consult the linked primary sources. Read our AI Editorial Policy.