Milind Desai, MD, MBA, on Clinically Evaluating MYBPC3–Associated Hypertrophic Cardiomyopathy Gene Therapy

This is the second part of an interview with Milind Desai, MD, MBA. For the first part, click here.

“The science is there. The process has been operationalized. Now we have to wait and see the downstream ramifications of it. Based on animal data, the expectation is that this technology works, but we still have to prove it in humans.”

Earlier this month, Tenaya Therapeutics announced that it had dosed the first participant in the multicenter MyPeak-1 phase 1b clinical trial (NCT05836259) evaluating TN-201, an investigational adeno-associated virus (AAV)-vector based gene therapy intended to treat MYBPC3-associated hypertrophic cardiomyopathy (HCM). TN-201 is designed to provide a functional copy of MYBPC3 with the intention of halting or reversing disease progression via restoration of MYBPC3 protein production in heart muscle cells. The ongoing development of TN-201 is also being informed by the active MyCLIMB natural history study (NCT05112237) for pediatric patients with MYBPC3-associated HCM and a separate noninterventional study investigating seroprevalence to AAV9 antibodies in adults with MYBPC3-associated HCM.

Following the news of the first dosing, CGTLive™ interviewed Milind Desai, MD, MBA, an investigator on the MyPeak-1trial, the director of the Hypertrophic Cardiomyopathy Center, and the vice chair of the Heart Vascular Thoracic Institute at the Cleveland Clinic. Desai discussed the design of the MyPeak-1 trial, noting that the study utilizes a dose-escalation format. Highlighting that the primary focus of the study is on the safety of TN-201, Desai emphasized that gene therapy is still very new territory in cardiovascular medicine, especially in the treatment of HCM, and therefore demonstrating safety in the clinical setting is of very high importance. Desai also spoke about the rationale behind the MyCLIMB natural history study, pointing out that it will help to inform which patient demographics, such as younger or older patients, are most likely to benefit from a gene therapy like TN-201. He also touched on future plans for MyPeak-1.

REFERENCE
Tenaya Therapeutics doses first patient in the MyPeak-1 phase 1b clinical trial of TN-201 for the treatment of MYBPC3-associated hypertrophic cardiomyopathy. News release. Tenaya Therapeutics, Inc. October 5, 2023. Accessed October 20, 2023. https://investors.tenayatherapeutics.com/news-releases/news-release-details/tenaya-therapeutics-doses-first-patient-mypeak-1tm-phase-1b