Tenaya Therapeutics Announces Late Breaker Presentation of New Data from MyPEAK™-1 Phase 1b/2 Clinical Trial of TN-201 at American College of Cardiology Annual Meeting

TN-201 Has Been Well Tolerated at 3E13 vg/kg Dose

New Biopsy Data Reaffirm Robust Transduction and RNA Expression with TN-201; RNA and Protein Levels Increase Over Time

All Cohort 1 Patients with Severe Disease at Baseline Achieved NYHA Class I

Two of Three Patients Experienced Improvements in One or More Measures of Hypertrophy

Expects to Complete Enrollment of Cohort 2 in 1H25 and to Report Initial Data in 2H25

Excerpt from the Press Release:

SOUTH SAN FRANCISCO, Calif., March 31, 2025 (GLOBE NEWSWIRE) — Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, announced that interim data from the first three patients enrolled in the company’s MyPEAK-1 Phase 1b/2 clinical trial of TN-201 were highlighted in a Late-Breaker presentation at the 2025 American College of Cardiology Scientific Sessions (ACC.25). TN-201 is being developed for the potential treatment of Myosin Binding Protein C3 MYBPC3-associated hypertrophic cardiomyopathy (HCM), a condition caused by insufficient levels of myosin-binding protein C (MyBP-C).

“TN-201 is the first gene therapy to be tested in HCM patients whose disease is caused by mutations to the MYBPC3 gene. For patients with this mutation, their disease is often more aggressive in its progression and they are at higher risk of serious – sometimes fatal – complications,” said Milind Desai, M.D., M.B.A, Haslam Family Endowed Chair in Cardiovascular Medicine, Vice Chair of Education in the Heart Vascular Thoracic Institute, Director of the Hypertrophic Cardiomyopathy Center at Cleveland Clinic, and an investigator for the MyPEAK-1 Phase 1b/2 clinical trial. “I look forward to continuing to study and evaluate this to see if this patient population is one that could be treated with gene therapy, as we have seen with other diseases.” 

“TN-201’s emerging safety profile at the 3E13 vg/kg dose, combined with biopsy results showing robust transduction, encouraging expression in the heart and suggestions of clinical improvement or stability are positive early signals of TN-201’s clinical impact,” said Whit Tingley, M.D., PhD., Tenaya’s Chief Medical Officer. “These data reinforce our optimism about TN-201’s potential to transform the treatment landscape for MYBPC3-associated HCM patients by addressing the underlying cause of their disease.

Click the button below to read the entire Press Release:

Continue Reading The Press Release