Insights from Tenaya Therapeutics on HCM in Pediatric Patients

Tenaya Therapeutics Highlights Interim Findings from MyClimb

Tenaya Therapeutics, Inc. (NASDAQ: TNYA), based in South San Francisco, has recently disclosed significant interim data from its MyClimb™ natural history study focused on pediatric patients diagnosed with MYBPC3-associated hypertrophic cardiomyopathy (HCM). This study represents one of the largest noninterventional studies of HCM in individuals under the age of 18, with over 200 participants involved. The findings were shared during a prominent cardiology congress, highlighting essential insights into this challenging condition.

Key Outcomes from the MyClimb Study

Among the notable findings from this interim data release were critical insights regarding the pediatric population. An impressive 93% of participants were found to exhibit the nonobstructive form of HCM, a variant for which no approved treatment options are currently available. These results underscore the urgent need for tailored therapies in this vulnerable demographic and illustrate the complexity of managing HCM in children.

Genotypic Status and Risk Prediction

The study revealed that genetic profiling serves as a significant predictor of risk in young patients with MYBPC3-associated HCM. Researchers pointed out that higher left ventricular mass index (LVMI) may act as an effective surrogate marker for assessing long-term outcomes. This discovery emphasizes the importance of comprehensive genetic counseling and monitoring in this population.

Clinical Implications and Future Directions

As the MyClimb study unfolds, clinicians are gaining valuable insights into distinguishing patients at a higher risk of developing severe complications such as life-threatening arrhythmias and potential heart failure. Dr. Whit Tingley, Tenaya's Chief Medical Officer, stated that understanding different genetic profiles can help streamline risk stratification and intervention strategies, paving the way for the development of innovative gene therapies aimed at treating this condition.

Details on Participant Data

By July 2025, the study had analyzed data from 213 participants diagnosed before the age of 18, gathering both retrospective and prospective information on their health outcomes. This multifaceted approach, involving 27 medical centers across various countries, reflects a significant collaborative effort to address pediatric HCM on a larger scale.

Important Findings Shared at ESC 2025

During the congress presentation, key findings indicated distinct patterns in disease progression among patients based on their genetic inheritance. The observations identified three main genetic groups: the homozygous group, compound heterozygous, and heterozygous children, each demonstrating different risk profiles and medical outcomes. These findings are critical as they emphasize the need for a targeted therapeutic approach for each patient group.

Impact of LVMI on Patient Risk Factors

Further modeling conducted during the study concluded that increments in LVMI correlate with an increasing hazard of serious events. Every 10-unit rise in LVMI was associated with a 10% increase in the risk of severe complications, leading researchers to regard LVMI as a pivotal measure for evaluating the potential effectiveness of future therapies.

The Future of Gene Therapy and HCM

Tenaya is dedicated to advancing gene therapy as a potential solution for pediatric patients with MYBPC3-associated HCM. The company’s TN-201 gene therapy aims to deliver a functional MYBPC3 gene to patients, substantially enhancing the MyBP-C protein levels in heart muscle cells. This approach targets the root causes of HCM to provide a more effective and less invasive treatment option.

Why There Is a Need for New Treatments

Currently, treatment options for children with this form of HCM are extremely limited, often resorting to invasive procedures such as heart transplants, which carry significant risks. Understanding the genetic underpinnings of the disease offers a glimmer of hope in developing less invasive, more effective therapies.

About Tenaya Therapeutics

Tenaya Therapeutics is a pioneering biotechnology company focused on discovering and developing potentially curative therapies that tackle the underlying causes of heart diseases. The company is keen on translating detailed genetic insights into clinical applications, building a robust pipeline that includes TN-201, aimed specifically at treating MYBPC3-associated hypertrophic cardiomyopathy.

Frequently Asked Questions

What is MyClimb?

MyClimb is a natural history study focusing on pediatric patients diagnosed with MYBPC3-associated hypertrophic cardiomyopathy (HCM) to understand the disease's progression and genetic influences.

What were the key findings from the MyClimb study?

The study found that 93% of participants had the nonobstructive form of HCM, with genetic status serving as a predictor of risk and outcomes.

How many participants were involved in the study?

Over 200 participants under the age of 18 were analyzed in the MyClimb study, contributing to its status as one of the largest studies on this condition.

What is the significance of LVMI in the study?

Left Ventricular Mass Index (LVMI) was identified as a significant marker for predicting serious complications and health outcomes in pediatric patients.

What is TN-201?

TN-201 is an innovative gene therapy from Tenaya Therapeutics designed to address the underlying genetic causes of MYBPC3-associated HCM through the delivery of a functional MYBPC3 gene to heart cells.

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