Tenaya Therapeutics Sets Strategic Vision for Future Advances

Tenaya Therapeutics Sets Strategic Vision for Future Advances

Continued Focus on Driving Advancement of TN-201 and TN-401 Gene Therapies for Cardiomyopathies

Dosing Initiated in Cohort 2 of the MyPEAK™-1 Phase 1b/2 Clinical Trial of TN-201 for Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy

Additional Cohort 1 Data to be Reported in 1H25 Building on Promising Initial Data

Initial Data from RIDGE™-1 Phase 1b Clinical Trial of TN-401 for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy Expected in 2H25

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a forward-thinking clinical-stage biotechnology company, shares recent updates on its clinical development initiatives, emphasizing its strategic focus for the coming year.

“The last year was tremendously significant for Tenaya, showcasing our commitment to operational excellence across our clinical gene therapy portfolio, which aims to address the root causes of heart diseases,” stated Faraz Ali, Chief Executive Officer of Tenaya. “As we step into 2025, we are excited to leverage this progress to ramp up patient enrollment while delivering crucial insights on safety and initial clinical outcomes from our TN-201 and TN-401 programs.”

Program Updates and 2025 Milestones

Updates on TN-201 – A Gene Therapy for MYBPC3-Associated Hypertrophic Cardiomyopathy (HCM)

• Tenaya is poised to unveil additional findings from Cohort 1 of the ongoing MyPEAK-1 Phase 1b/2 clinical trial in the first half of 2025. This data will include safety updates and assessments from the first three patients dosed, along with 52-week biopsy data from the second patient, and baseline and post-dose biopsy data from the third patient.
• In late 2024, preliminary interim data was shared, indicating that TN-201, delivered at a starting dose of 3E13 vg/kg, was well-tolerated among the initial patients, demonstrating encouraging signs of efficacy.
• Enrollment in Cohort 2 of the MyPEAK-1 trial is already underway, with plans to conclude in the early months of 2025. This expanded cohort allows eligibility for adults with obstructive disease and patients who do not have implantable cardioverter defibrillators (ICDs).

Additionally, Tenaya plans to publish findings from its pediatric non-interventional natural history study called MyClimb, which focuses on MYBPC3-associated HCM in young patients.

Exciting Developments for TN-401 – A Gene Therapy for PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)

• Enrollment of Cohort 1 in the RIDGE-1 Phase 1b clinical trial is expected to wrap up in the first half of 2025, paving the way for initial clinical data release.
• The first set of safety and post-dose biopsy insights from patients receiving TN-401 will arrive in the latter part of 2025. This potential first-in-class gene therapy utilizes an AAV9-based delivery method to introduce a functional PKP2 gene into cardiac muscle cells, which is vital for maintaining healthy cardiac structure.
• Additionally, Tenaya anticipates initiating its first international RIDGE-1 clinical site in early 2025, marking a significant step in expanding its research.
• Throughout 2025, Tenaya will be sharing findings from RIDGE, a study that currently involves over 100 participants with PKP2-associated ARVC.

Tenaya's commitment to establishing a robust portfolio of genetic medicines and enhancing its platform capabilities will continue to steer the company towards its mission of addressing heart disease.

Understanding the Impacts of MYBPC3 and PKP2 Gene Therapy

MYBPC3-Associated Hypertrophic Cardiomyopathy and TN-201 Gene Therapy

Variants in the Myosin Binding Protein C3 (MYBPC3) gene are recognized as the primary genetic contributors to hypertrophic cardiomyopathy, affecting a substantial number of individuals in the U.S. With TN-201, designed as an AAV9-based therapy, the goal is to deliver a functional MYBPC3 gene directly to heart muscle cells. This innovative approach aims to enhance the production of MyBP-C protein, potentially halting disease progression with just a single dose.

PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy and TN-401 Gene Therapy

TN-401 is targeting arrhythmogenic right ventricular cardiomyopathy, which arises from mutations in the PKP2 gene. By administering TN-401, research shows potential benefits including the induction of a new PKP2 gene in heart cells, which is instrumental in rectifying structural anomalies and stabilizing heart rhythms.

About Tenaya Therapeutics

Tenaya Therapeutics is dedicated to forging pathways towards revolutionary treatments for heart disease. With an adept internal framework that encompasses stringent target validation and advanced manufacturing methodologies, the company aims to deliver impactful gene therapies like TN-201 and TN-401, alongside other therapeutic strategies in its development pipeline.

Frequently Asked Questions

What is the focus of Tenaya Therapeutics' 2025 strategy?

Tenaya aims to enhance its gene therapy candidates, particularly TN-201 and TN-401, with a focus on operational excellence and advancing clinical trials.

What therapies does Tenaya Therapeutics offer?

Tenaya's portfolio includes TN-201 for MYBPC3-associated HCM and TN-401 for PKP2-associated ARVC, both aimed at addressing critical heart diseases.

When is the data from the MyPEAK-1 trial expected?

Additional data from Cohort 1 will be reported in the first half of 2025, while insights from Cohort 2 will follow in the latter half of the year.

What safety measures are in place for the trials?

The trials include rigorous monitoring of patient safety, with data from initial cohorts showing positive outcomes thus far.

Who should be contacted for further inquiries about Tenaya Therapeutics?

Contact Michelle Corral, VP of Corporate Communications and Investor Relations, for more information about Tenaya and its initiatives.

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