Tenaya Therapeutics (TNYA) Corporate Presentation summary

Strategic focus and capabilities

• Dedicated to developing gene therapies and small molecules for serious heart diseases, with three clinical-stage programs targeting large patient populations.

• Deep expertise in cardiology, genetics, and rare disease drug development supports innovation and first-in-class potential.

• Foundational capabilities and a track record of execution position the pipeline for significant progress.

Clinical pipeline and target indications

• TN-201 targets MYBPC3+ hypertrophic cardiomyopathy (HCM), affecting over 120,000 in the U.S., with Orphan Drug and Fast Track designations.

• TN-401 addresses PKP2+ arrhythmogenic right ventricular cardiomyopathy (ARVC), impacting over 70,000 in the U.S., also with Orphan Drug and Fast Track designations.

• TN-301, an HDAC6 inhibitor, is in early clinical development for heart failure with preserved ejection fraction (HFPEF) and other conditions.

• Patient populations for these programs are significantly larger than those targeted by peer gene therapy companies.

TN-201 for MYBPC3+ HCM: Clinical data and impact

• MYBPC3 mutations account for 57% of familial HCM and 30% of childhood-onset HCM; disease is severe, progressive, and affects all ages.

• Phase 1b/2a MyPEAK-1 trial shows TN-201 is well tolerated, with most adverse events mild and reversible; no cardiotoxicity observed.

• Higher TN-201 doses led to greater increases in MyBP-C protein, with DNA and RNA levels exceeding efficacy thresholds.

• Cardiac biomarkers (troponin, NT-proBNP) improved or stabilized, and hypertrophy measures (LV wall thickness, mass index) declined by 21–39%.

• All patients improved by at least one NYHA class, with some becoming asymptomatic at 52 weeks.