Tenaya Therapeutics (TNYA) H.C. Wainwright 28th Annual Global Investment Conference summary

Clinical data and program updates

• Recent data from six evaluable patients for TN-201 show meaningful improvements across circulating biomarkers, hypertrophy, and functional endpoints, with durable effects observed up to two years after dosing.

• Four out of six patients had double-digit decreases in hypertrophy, and five out of six improved in New York Heart Association Class, indicating a consistent clinical effect.

• TN-401, targeting arrhythmogenic right ventricular cardiomyopathy, demonstrated a 64% average reduction in PVC burden among the first six patients, with all showing meaningful improvements in electrical instability.

• Both TN-201 and TN-401 are positioned as differentiated gene therapies addressing underlying genetic causes, with TN-201 showing promise especially in non-obstructive and pediatric populations.

• Additional data and regulatory updates for both programs are expected in the second half of the year, including dose selection and pivotal study planning.

Regulatory strategy and endpoints

• Regulatory engagement is ongoing, with alignment on endpoints expected to vary by patient subpopulation and disease form.

• For TN-201, endpoints may differ between obstructive, non-obstructive, and pediatric patients, with a focus on mapping the best population for pivotal studies.

• For TN-401, PVCs are considered a logical and clinically meaningful endpoint, supported by KOL feedback and recent literature.

• The company leverages a large natural history study (RIDGE) to inform endpoint selection and support regulatory discussions.

• Updates on regulatory alignment and study design for both assets are planned by year-end.

Competitive landscape and market positioning

• TN-201 is the first gene therapy targeting the MYBPC3 mutation, with a distinct profile compared to chronic therapies like cardiac myosin inhibitors.

• Market research and physician feedback indicate high interest and confidence in the data for both TN-201 and TN-401.

• TN-401's efficacy and safety data are viewed as the most consistent and deep among competitors, with differentiation in capsid choice, promoter, and manufacturing platform.

• The competitive landscape includes Rocket and Lexeo, but differences in product design and early data may offer opportunities for further differentiation.

• There is potential for multiple products to coexist in these large orphan indications, with unmet needs remaining in several patient segments.