Tenaya Therapeutics (TNYA) TD Cowen 45th Annual Healthcare Conference summary

Corporate strategy and pipeline overview

• Focused exclusively on heart disease, with three clinical-stage programs and a modality-agnostic approach targeting both rare and prevalent forms of cardiomyopathy.

• Gene therapies TN-201 and TN-401 are the primary focus, with 45 sites activated in eight countries and hundreds of patients enrolled in natural history studies.

• Regulatory environment is favorable for AAV gene therapies, with precedents for early pivotal study transitions based on limited patient data.

• Internalized manufacturing at 1,000-liter scale de-risks execution and supports rapid clinical advancement.

• Deep pipeline includes gene therapy, gene editing, and gene silencing, with extensive capsid and promoter screening.

TN-201 gene therapy program (MYBPC3-associated HCM)

• Targets hypertrophic cardiomyopathy caused by MYBPC3 mutations, affecting 120,000 patients in the U.S.

• Preclinical models show single-dose therapy restores protein, reduces heart mass, improves function, and reverses disease.

• MyPEAK-1 phase 1b/2 study is open-label, multi-center, with up to 24 patients; high-dose cohort dosing underway.

• Early data show strong cardiac transduction and RNA expression at half the dose of comparable programs, with promising protein expression and safety profile.

• Initial clinical results indicate improvement in New York Heart Class and cardiac biomarkers in severely affected patients.

TN-401 gene therapy program (PKP2-associated ARVC)

• Addresses arrhythmogenic right ventricular cardiomyopathy, a severe disease with high risk of sudden cardiac death and no approved therapies.

• Lock-and-key gene therapy approach aims to restore PKP2 protein and reverse arrhythmia and heart enlargement.

• Uses AAV9 capsid, shown to outperform other capsids in cardiac expression; first two patients in low-dose cohort dosed.

• Data-rich study design captures biopsy, pharmacodynamic, and efficacy endpoints to support potential accelerated approval.

• Ex-U.S. expansion progressing, with U.K. sites activated and initial data expected in the second half of the year.