Tenaya Therapeutics (TNYA) FY Conference Transcript

Summary of Tenaya Therapeutics (TNYA) FY Conference Call Company Overview - Company: Tenaya Therapeutics (TNYA) - Founded: 2016 - Mission: Focus on therapies for heart disease, including both rare genetic forms and prevalent forms [3][4] Pipeline and Clinical Development - Clinical Stage Assets: Three clinical stage assets currently in development [4] - Gene Therapies: Focus on gene therapies for genetic causes of cardiomyopathy and a small molecule for HFpEF [4] - Clinical Sites: Over 40 clinical sites active in seven countries for patient recruitment [4] Gene Therapy Programs TN-201 for MYBPC3 Positive Hypertrophic Cardiomyopathy - Target Disease: Leading genetic cause of hypertrophic cardiomyopathy, affecting approximately 120,000 patients in the US [15] - Mechanism: Addresses deficiency of myBPC3 protein, leading to heart thickening and potential heart failure [16][17] - Natural History Study: MyCLIMB study with over 220 children enrolled to characterize disease progression [20][21] - Phase 1b Study: Completed dosing of high dose cohort; initial results show improvement in heart function [25][27] - Upcoming Data: Full cohort data expected in Q4 2025, with focus on safety and efficacy [37] TN-401 for PKP2-Related Arrhythmogenic Right Ventricular Cardiomyopathy - Target Disease: Accounts for about 40% of arrhythmogenic right ventricular cardiomyopathy cases, approximately 70,000 patients in the US [44] - Mechanism: Aims to add a copy of the human gene to improve desmosome function and reduce arrhythmia burden [46] - Biopsy Data: Expected in Q4 2025, focusing on vector copy number, RNA, and protein levels [50][51] Safety and Efficacy - AAV9 Vector: Selected for its extensive safety database and effectiveness in cardiac applications [5][6] - Safety Record: No significant safety issues reported in ongoing trials; adverse events consistent with other gene therapies [13][14] - Patient Outcomes: Initial data shows symptomatic improvement in patients treated with TN-201 [25][27] Small Molecule Program TN-301 - Status: Completed first-in-human study with 72 patients; well tolerated with no dose-limiting toxicities [55] - Future Plans: Exploring opportunities to advance the program, focusing on HFpEF and potentially severe rare diseases [56] Key Takeaways - Market Opportunity: Both TN-201 and TN-401 target large orphan conditions with significant unmet medical needs [44][46] - Data Releases: Important data updates expected in Q4 2025 for both gene therapy programs, which could influence future clinical development strategies [37][50]