Tenaya Therapeutics (TNYA) Morgan Stanley 22nd Annual Global Healthcare Conference summary

Program updates and clinical milestones

• TN-201 gene therapy program for hypertrophic cardiomyopathy (HCM) is on track for an initial clinical data update in the second half of the year, with TN-401 also set to begin dosing patients in the same timeframe.

• The next 6–18 months are expected to yield the first clinical data for both gene therapy programs, marking a pivotal period.

• The myPEAK-1 study dosed its first patient in October and is focused initially on non-obstructive HCM patients with MYBPC3 mutations.

• Initial data release will include safety, biopsy, and circulating biomarker results from the first dose cohort, with more comprehensive data expected in 2025.

• TN-401 is also progressing, with data anticipated in the coming year.

Scientific and clinical rationale

• TN-201 uses an AAV9 capsid, chosen for its strong safety record and validated biodistribution in the human heart.

• Preclinical studies in human cells and mice showed reversal of disease symptoms, improved heart function, and survival with robust protein expression.

• The program features a novel promoter for targeted expression in cardiomyocytes and is protected by multiple IP filings.

• The therapy aims to address the underlying genetic cause of HCM by delivering a functional MYBPC3 gene to restore protein levels.

Disease landscape and unmet need

• HCM affects about 600,000 people in the US, with MYBPC3 mutations accounting for roughly 120,000 cases.

• Current treatments, including beta blockers and myosin inhibitors like Camzyos, provide symptomatic relief but do not address the genetic root cause.

• There is significant unmet need, especially for non-obstructive HCM patients, who lack effective pharmacological or surgical options.