Tenaya Therapeutics (TNYA) Status Update summary

Program Overview and Clinical Context

• TN-201 is a first-in-class AAV9-based gene therapy targeting MYBPC3-associated hypertrophic cardiomyopathy (HCM), a severe, progressive genetic heart condition affecting over 120,000 people in the US, with no approved therapies for most patients.

• The therapy delivers a functional MYBPC3 gene to heart muscle cells, aiming to restore MyBP-C protein levels, improve cardiac function, and halt or reverse disease progression.

• TN-201 has received Fast Track, Orphan Drug, and Rare Pediatric Drug designations from the FDA and orphan medicinal product designation from the European Commission.

Study Design and Regulatory Status

• MyPEAK-1 is an open-label, multi-center, dose-escalation Phase 1b/2 trial evaluating safety, pharmacodynamics, and efficacy in symptomatic adults with severe non-obstructive MYBPC3-associated HCM.

• The FDA placed a temporary clinical hold on MyPEAK-1, requesting protocol amendments to standardize patient monitoring and immunosuppression; no recent safety concerns triggered the hold, and timelines are not expected to be impacted.

• The revised protocol has been submitted, with collaborative discussions ongoing with the FDA and optimism for a quick resolution.

• The Data Safety Monitoring Board allowed expansion at both dose levels after reviewing safety data.

• Cash and equivalents are sufficient to fund operations into the second half of 2026, supporting ongoing and planned clinical milestones.

Interim Clinical Data and Safety

• TN-201 was well tolerated at both 3E13 and 6E13 vg/kg doses, with no dose-limiting toxicities; most adverse events were mild, transient, or reversible, and all patients tapered off immunosuppression.

• Two treatment-related serious adverse events occurred but resolved without long-term effects; reversible, asymptomatic liver enzyme elevations were the most common adverse events.

• Immunosuppression regimens have been optimized to reduce steroid exposure while maintaining safety, with increased monitoring frequency and individualized tapering.

• Complement activation was observed in some patients but resolved without intervention; protocols now include readiness to use complement inhibitors if needed.

• The sponsor is aligned with evolving FDA expectations for consistent safety monitoring across gene therapy programs.