Tenaya Therapeutics (TNYA) investor relations material

Strategic vision and pipeline overview

• Focus on developing precision medicines for heart disease using both gene therapy and small molecules, aiming to address underlying disease causes over the next 5–10 years.

• Strong internal data and an energized team drive confidence in advancing programs, with a significant year ahead and key regulatory updates expected in Q4.

TN-201 gene therapy for MYBPC3-associated HCM

• Targets approximately 120,000 U.S. patients, addressing a significant unmet need, especially in severe cases.

• Key efficacy endpoint is reduction in left ventricular mass index (LVMI), with meaningful improvements in both LVMI and patient symptoms observed.

• Safety profile remains favorable at both tested doses, with no major adverse events reported.

• Ongoing expansion cohort uses a higher dose, showing strong efficacy and safety.

• Natural history study in pediatrics underway, with plans to engage regulators for both adult and pediatric development paths.

TN-401 gene therapy for PKP2-associated ARVC

• Addresses a population of 70,000–75,000 U.S. patients with high PVC burden and risk of sudden cardiac death.

• Clinical data show a 60–67% reduction in PVCs over 52 weeks, exceeding the 50% reduction considered clinically meaningful.

• Maintains a clean safety profile and demonstrates competitive advantages over other programs.

• Additional durability and regulatory updates expected in Q4.