Tenaya Therapeutics (TNYA) investor relations material

Strategic Focus and Innovation

• Dedicated to developing curative therapies for heart disease, targeting both rare and prevalent forms using gene therapy, small molecules, and gene editing, with a singular focus on cardiology and rare genetic conditions.

• Modality-agnostic approach enables pursuit of multiple therapeutic strategies based on scientific merit, supported by deep expertise in cardiology, genetics, and rare disease drug development.

• Integrated internal capabilities in target discovery, validation, manufacturing, and drug delivery power a modality-agnostic discovery engine, accelerated by AI-enabled phenotypic screening and proprietary disease models.

• Strategic collaboration with Alnylam targets multiple undisclosed genetic targets in cardiovascular indications, with $10M upfront and over $1B in milestones.

Clinical Program Highlights

• Three clinical-stage programs: TN-201 and TN-401 (gene therapies) and TN-301 (small molecule), with near-term data readouts and regulatory milestones anticipated in 2026.

• TN-201 targets MYBPC3-associated HCM, showing significant cardiac remodeling, symptom improvement, and functional gains in early cohorts, with durable responses up to two years and most patients achieving ≥10% reduction in LVMI.

• TN-401 addresses PKP2-associated ARVC, demonstrating marked reductions in arrhythmia burden (PVCs, NSVTs) and strong safety profile, with effects sustained up to one year.

• TN-301, a highly selective HDAC6 inhibitor, shows broad preclinical efficacy across multiple cardiac indications, including HFPEF, genetic DCM, PAH, and DMD, and is advancing toward phase II studies.

Recent Data and Safety Outcomes

• TN-201: Durable improvements in cardiac structure and function, with most patients moving to NYHA Class 1, ≥10% LVMI reduction, and strong safety at both dose levels; most adverse events mild, transient, and reversible.

• TN-401: All treated patients achieved sustained reductions in PVCs and NSVTs, with dose-dependent effects and minimal adverse events; both doses well tolerated with no new safety signals.

• TN-301: Phase 1 trial in healthy volunteers completed; well tolerated with predictable pharmacokinetics and no significant safety concerns.

• Both gene therapies use relatively low vector doses, minimizing toxicity risks.