- Tenaya Therapeutics (TNYA, Financial) shares interim results from its extensive RIDGE study for PKP2-associated ARVC.
- 83% of participants exhibit high-risk PVC counts despite standard treatments.
- 93% of patients qualify for TN-401 gene therapy due to sufficient AAV9 antibody levels.
Tenaya Therapeutics, Inc. (TNYA), a clinical-stage biotech firm, disclosed interim data from its RIDGE™ natural history study at Heart Rhythm 2025. This extensive study involved over 175 participants, focusing on arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by PKP2 gene mutations, marking it the largest of its kind.
The study's findings from 144 patients highlighted significant challenges in managing ARVC with current treatments. Despite treatments like beta blockers and anti-arrhythmic medications, 83% of participants continued to show high-risk premature ventricular contraction (PVC) counts. Additionally, 60% of the patients demonstrated progression in heart function, a critical indicator of the disease's severity.
Moreover, 93% of the patients exhibited AAV9 neutralizing antibody titers of ?1:40, making them eligible for Tenaya’s innovative TN-401 gene therapy, which utilizes an AAV9 capsid to deliver functional PKP2 genes to heart muscle cells. This discovery underscores the potential for TN-401 to address unmet medical needs in ARVC treatment.
Tenaya is advancing the development of TN-401, with plans to report safety and biopsy data from the first cohort of the RIDGE-1 clinical trial in the second half of 2025. The company's stock, TNYA, reflects its commitment to pioneering curative therapies by tackling the root causes of heart diseases.