NEW YORK – Tenaya Therapeutics remains on track to begin treating hypertrophic cardiomyopathy patients with TN-201 in the third quarter of this year, the company said Tuesday in announcing that its investigational gene therapy had garnered fast-track status from the US Food and Drug Administration.
The adeno-associated virus-based treatment, which is on an expedited development and regulatory review track, is designed to deliver a functional MYBPC3 gene and restore normal protein levels. Tenaya is hoping that TN-201 will halt and reverse HCM caused by MYBPC3 gene mutations, which occur in 20 percent of individuals with the heart condition, or approximately 150,000 patients in the US. Current treatments don't specifically address the underlying cause of the disease but may mitigate symptoms.
The FDA earlier this year cleared Tenaya's investigational new drug application for TN-201, allowing the firm to begin clinical trials. In the Phase Ib trial Tenaya expects to launch later this year, the company plans to enroll six symptomatic adults with MYBPC3-associated nonobstructive HCM who have an implantable cardioverter defibrillator.
Researchers will give patients a one-time intravenous infusion of TN-201 and follow them for safety, tolerability, and clinical efficacy. Tenaya will also explore markers of cardiac transduction and transgene expression in right ventricular biopsy samples, as well as cardiac and imaging biomarkers relevant to HCM.
The South San Francisco, California-based firm is expecting results from this Phase Ib study in 2024.