Tenaya Therapeutics (TNYA) TD Cowen Genetic Medicines & RNA Summit summary

Market opportunity and genetic testing trends

• MYBPC3 mutation is the leading genetic cause of hypertrophic cardiomyopathy, with an estimated 120,000 patients in the U.S., representing a large orphan therapy market.

• Genetic testing rates for HCM are increasing, driven by medical societies and the availability of new therapies, aiding in family risk identification and patient management.

• Patients with sarcomeric mutations like MYBPC3 present earlier and with more severe symptoms, making genetic diagnosis crucial for care.

• Similar trends are seen in PKP2 arrhythmogenic cardiomyopathy, with an estimated 70,000 U.S. patients and growing genetic testing.

Unmet needs and therapeutic approaches

• Current therapies like Camzyos are approved only for obstructive HCM, leaving a significant unmet need for non-obstructive MYBPC3 patients, who make up about 70% of the target group.

• Gene therapy aims to address the underlying genetic cause, potentially offering greater and more durable benefits than small molecules.

• Both TN-201 and TN-401 target large patient populations with significant unmet needs, and gene therapy may be the only hope for severe pediatric cases.

Clinical development and data expectations

• Initial phase 1b data for TN-201 is expected in the second half of the year, focusing on safety, biopsy results, and biomarkers from the first dose cohort.

• Biopsies will assess vector delivery, RNA expression, and protein production, with multiple time points to track efficacy correlations.

• Circulating biomarkers and echo-based parameters will be measured, but early echo data may be noisy and require larger patient numbers for reliable interpretation.

• For TN-401, screening has begun at three sites, with dosing and data timelines expected to follow a similar conservative approach as TN-201.