Tenaya Therapeutics Eyes Q4 Updates for Cardiac Gene Therapies, TN-301

Tenaya Therapeutics expects fourth-quarter updates on its TN-201 and TN-401 cardiac gene therapy programs, including regulatory plans and additional clinical data. TN-201 has shown meaningful reductions in left ventricular mass and symptom improvements in MYBPC3-associated hypertrophic cardiomyopathy, while TN-401 has demonstrated approximately 60%–67% reductions in PVCs through as long as 52 weeks with a clean safety profile. Tenaya is restarting development of TN-301, an HDAC6 inhibitor, and plans to detail Phase 2 plans in the fourth quarter. The company reported $78 million in cash, expected to fund operations through the third quarter of next year, excluding potential pivotal or Phase 2 trial costs.

Tenaya Therapeutics NASDAQ: TNYA expects to provide regulatory and development updates in the fourth quarter for its two cardiac gene therapy programs and its small-molecule candidate TN-301, Chief Financial Officer Eric Hyllengren said during a Canaccord discussion. Hyllengren, who said he had been in the CFO role for nearly a month, described the company’s strategy as developing precision medicines for heart disease using multiple therapeutic approaches, including gene therapy and small molecules. He said the company is focused on addressing the underlying causes of disease rather than solely treating symptoms.

Tenaya’s TN-201 is a gene therapy candidate for MYBPC3-associated hypertrophic cardiomyopathy, or HCM. Hyllengren said the condition affects approximately 120,000 people in the United States, representing about 20% of the overall HCM population. In these patients, the left ventricle can thicken, reducing the heart’s ability to pump blood effectively, he said. Tenaya is evaluating reductions in left ventricular mass index, or LVMI, as an important outcome measure, while discussing appropriate endpoints with regulators. The ongoing expansion cohort is evaluating the higher 6E13 dose of TN-201, compared with a dose half that level in the first cohort. Hyllengren said the treatment has been well tolerated at both doses and that prior data showed meaningful LVMI reductions in most patients. He also said many patients experienced improvements in symptoms and function. The company is conducting a pediatric natural history study, citing limited existing data in younger patients. Hyllengren said earlier symptom onset is associated with greater risk of later complications, and Tenaya sees a potential future opportunity to study TN-201 in pediatric patients. Discussions with U.S. and European regulators are ongoing for both adult and pediatric development paths, with an update expected in the fourth quarter. While Hyllengren said the pediatric development strategy remains to be determined, he noted that the unmet need may be particularly high because there are no effective treatment options for pediatric patients in this setting.

Tenaya’s second gene therapy candidate, TN-401, is being developed for PKP2-associated arrhythmogenic right ventricular cardiomyopathy, or ARVC. Hyllengren estimated that roughly 70,000 to 75,000 patients in the U.S. have the condition. The program is focused on reducing electrical instability in the heart, including premature ventricular contractions, or PVCs. Hyllengren said about 90% of patients have more than 500 PVCs per day. He said reducing PVCs could potentially improve outcomes and help prevent serious events, including sudden cardiac death. According to Hyllengren, TN-401 has maintained a clean safety profile in the clinical data presented so far. He said the company has observed PVC redu...