Tenaya unveils promising data in pediatric heart study at ESC Congress
MyClimb study findings highlight significant unmet need and key risk predictors for rare genetic heart condition, bolstering its TN-201 gene therapy program.
Tenaya Therapeutics (NASDAQ: TNYA) took a significant step forward in its mission to treat rare genetic heart diseases, presenting encouraging interim data from its MyClimb natural history study at the European Society of Cardiology (ESC) Congress 2025. The results shed new light on the rapid progression of a pediatric heart condition, MYBPC3-associated hypertrophic cardiomyopathy (HCM), and strengthen the case for the company's lead gene therapy candidate, TN-201.
The study, one of the largest of its kind with over 200 participants under 18, revealed a critical unmet need: , for which there are currently no approved therapies. This finding underscores the urgency for a disease-modifying treatment for this vulnerable population.
A key insight from the research was the identification of Left Ventricular Mass Index (LVMI) as a significant predictor of risk. According to the data, every 10-unit increase in LVMI was associated with a 10% higher hazard of a serious event, such as heart failure or transplant. This suggests LVMI could serve as a crucial surrogate marker for evaluating the effectiveness of future treatments in clinical trials. 'These new insights into predictive risk factors can inform clinicians’ thinking on risk stratification and interventions,' said Whit Tingley, M.D., Ph.D., Tenaya’s Chief Medical Officer.
The provides a foundational dataset that helps de-risk and guide the clinical development of TN-201, an AAV9-based gene therapy designed to deliver a working copy of the MYBPC3 gene. By understanding the natural progression of the disease and identifying high-risk genetic profiles, Tenaya can better design trials for its potentially curative therapy.
Current options for children with severe MYBPC3-associated HCM are limited to highly invasive procedures like implantable defibrillators and heart transplants, which carry significant risks and do not address the underlying genetic cause. The data presented at the provides a clearer roadmap for developing TN-201 as a targeted intervention for patients who currently have none.