Tenaya Therapeutics Lifts Clinical Hold on MyPEAK-1™ Clinical Trial for TN-201

SOUTH SAN FRANCISCO, Calif., Dec. 11, 2025 (GLOBE NEWSWIRE) – Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a pioneering clinical-stage biotechnology company, has announced the removal of the clinical hold on its MyPEAK-1™ Phase 1b/2a clinical trial for the innovative gene therapy, TN-201. This decision follows a comprehensive review by the U.S. Food and Drug Administration (FDA), which confirms that all previously raised concerns have been satisfactorily addressed.

Overview of TN-201 and MyPEAK-1 Clinical Trial

TN-201 is specifically developed to treat Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM), a genetic condition characterized by insufficient myosin-binding protein C levels. Following successful amendments to the study protocol, Tenaya is collaborating with MyPEAK-1 clinical sites to resume patient dosing, ensuring enhanced patient monitoring and management during treatment.

Key Protocol Amendments

The protocol amendments aim to standardize trial practices while maintaining the current immunosuppression regimen that includes prophylactic prednisone and sirolimus. Adjustments made are expected to optimize the dosing strategies and durations of immunosuppressants, despite the higher doses of TN-201 being administered.

• Immunosuppression Regimen: Unchanged and includes prednisone and sirolimus.
• Study Design: Multi-center, open-label, dose-escalating trial.
• Patient Population: Up to 24 symptomatic adults diagnosed with MYBPC3-associated HCM.

Recent Developments and Data Highlights

Recent findings regarding the safety and tolerability of TN-201 have been positively received, with the Data Safety Monitoring Board (DSMB) supporting ongoing patient enrollment. These findings were highlighted in a late-breaker presentation at the American Heart Association Scientific Sessions and were simultaneously published in Cardiovascular Research. The company remains optimistic that the clinical hold will have no adverse effects on data milestones or overall development timelines.

Understanding MYBPC3-Associated HCM

MYBPC3-associated hypertrophic cardiomyopathy is the most prevalent genetic cause of HCM, affecting approximately 120,000 patients in the United States alone. This severe condition may lead to adverse symptoms such as:

• Chest pain
• Shortness of breath
• Palpitations
• Fainting

Patients with MYBPC3 mutations often experience earlier disease onset and a higher risk of serious outcomes, including heart failure and sudden cardiac events. Currently, there are no approved treatments that address the genetic root of HCM.

About TN-201 and Its Development

TN-201 employs an adeno-associated virus serotype 9 (AAV9) vector to deliver a functional MYBPC3 gene directly to heart muscle cells via a single intravenous infusion. The treatment aims to mitigate disease progression by boosting MyBP-C protein levels. The FDA has awarded TN-201 with Fast Track, Orphan Drug, and Rare Pediatric Disease designations, demonstrating recognition of its potential therapeutic benefit in this niche.

About Tenaya Therapeutics

Tenaya Therapeutics is dedicated to revolutionizing heart disease treatment through innovative therapies. The company’s pipeline includes:

• TN-201: Gene therapy for MYBPC3-associated HCM
• TN-401: Gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy
• TN-301: Small molecule HDAC6 inhibitor aimed at treating heart failure

For additional information about Tenaya Therapeutics and its ongoing clinical trials, including the MyPEAK-1 study, please visit tenayatherapeutics.com.

Forward-Looking Statements

This article contains forward-looking statements, as defined by the Securities Act of 1933 and the Securities Exchange Act of 1934. The anticipated clinical advancements, including those related to MyPEAK-1, are subject to various risks that may impact Tenaya’s ability to achieve its development goals.