Tenaya Therapeutics presented interim safety and efficacy data from its MyPEAK‑1 Phase 1b/2a trial of the gene therapy TN‑201 at the American Heart Association’s Scientific Sessions 2025. The data were delivered in the Late‑Breaking Science: Main Event session and were simultaneously published in the journal Cardiovascular Research.
The presentation focused on two small cohorts. Cohort 1 included three patients who received 3 × 10¹³ vg/kg; follow‑up ranged from week 52 to week 78. Cohort 2 also had three patients, each given 6 × 10¹³ vg/kg. For Cohort 2, a week‑12 biopsy was obtained for Patient 6 and week‑26 assessments for Patient 4; Patient 5 was lost to follow‑up after week 12. Across both cohorts, the data showed robust viral transduction, a dose‑dependent increase in MyBP‑C protein expression, and reductions in circulating biomarkers and left‑ventricular hypertrophy.
The trial enrolls adults aged 18 to 65 years, not “up to 24 years” as previously reported. In addition, the U.S. Food and Drug Administration has placed the MyPEAK‑1 study on a clinical hold to standardize patient monitoring and immunosuppression management. Tenaya has stated that the hold is unlikely to delay data milestones or the overall development timeline.
These early signals reinforce the safety profile and potential efficacy of TN‑201, informing the design of expansion cohorts and future development plans. The FDA hold introduces regulatory uncertainty, while the loss of a patient to follow‑up may affect the completeness of the data set. Together, these factors temper the optimism that the data alone might generate.
Tenaya will host a conference call on Monday, November 10 at 8:00 a.m. ET to discuss the data and the status of the MyPEAK‑1 trial. The independent Data Safety Monitoring Board has approved the expansion cohorts, and the company’s pipeline remains a key driver of investor interest despite its ongoing net losses.
In summary, the interim data represent a significant milestone for TN‑201, but the FDA hold and patient attrition underscore the need for continued monitoring of regulatory developments and forthcoming data releases.
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