Tenaya Therapeutics to Present New TN-201 Clinical Trial Data at ACC Meeting

Tenaya Therapeutics has announced promising interim results from their MyPEAK-1 Phase 1b/2 clinical trial, revealing initial data on TN-201, a gene therapy aimed at addressing MYBPC3-associated hypertrophic cardiomyopathy (HCM). The trial, conducted on three patients, was highlighted at the 2025 American College of Cardiology Scientific Sessions.

TN-201, which targets the Myosin Binding Protein C3 gene mutation, represents the first gene therapy for this aggressive form of HCM. Dr. Milind Desai, a leading investigator of the trial, expressed optimism about the potential of gene therapy to offer substantial benefits to patients dealing with this condition. He emphasized the importance of continuing research to determine the therapy's suitability for a broader patient group.

The initial results show that TN-201 has been largely well-tolerated at a dosage of 3E13 vg/kg. Tenaya's Chief Medical Officer, Dr. Whit Tingley, indicated that the safety profile, along with positive biopsy results demonstrating effective transduction and RNA expression, are encouraging signs of TN-201's potential impact. The biopsy data affirmed that TN-201 increased MyBP-C protein and RNA levels in the heart over time, indicating successful gene transduction.

All three patients in the trial exhibited severe HCM at the start, with symptoms impacting daily activities. Following treatment with TN-201, all achieved NYHA Class I status, experiencing no physical activity limitations. Improvements were noted in cardiac biomarkers: cardiac troponin levels decreased substantially, and key hypertrophy measures, like left ventricular wall thickness, showed significant reduction in two patients.

Tenaya is preparing to complete enrollment for Cohort 2 by mid-2025, with initial data expected later that year. Patients in Cohort 2 will receive a higher dose of 6E13 vg/kg. The company has also secured funding extending into the latter half of 2026, allowing continued milestone achievements in both the TN-201 and TN-401 programs.

The trial also provided insights into the broader implications of MYBPC3-associated HCM. Findings indicated that patients with these mutations face significant risks, including heart failure and arrhythmias, with younger patients progressing more rapidly. Approximately half of the adult patients diagnosed before age 40 experience severe cardiac events by age 50.

The MyPEAK-1 trial, still ongoing, examines the safety and efficacy of TN-201 in different dosing cohorts. Initial results have been promising, with TN-201 demonstrating potential to transform the treatment landscape for MYBPC3-associated HCM by addressing its root genetic cause. The U.S. FDA has recognized TN-201 with several designations reflecting its potential to address this unmet medical need.

Tenaya Therapeutics continues to focus on developing innovative therapies for heart disease, leveraging their integrated research capabilities. Their pipeline includes several gene therapies and small molecules, aiming to tackle both rare genetic conditions and more common heart issues.