FDA Grants Fast Track to Tenaya for Rare Heart Disease Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Tenaya Therapeutics Fast Track designation for its experimental gene therapy in development for the treatment of a rare form of heart disease.

Tenaya’s experimental gene therapy is in development to treat myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM).

HCM is the most common inherited cardiac disorder and variants in the MYBPC3 gene are the most common genetic cause of HCM. MYBPC3-associated HCM is estimated to account for approximately 20 percent of the overall HCM population and to affect approximately 115,000 patients in the United States alone. MYBPC3-associated HCM is a chronic, progressive condition characterized by left ventricular thickening, hypercontractility, fibrosis, abnormal heart rhythms, cardiac dysfunction and impaired diastolic relaxation. This in turn leads to serious complications including symptoms such as shortness of breath, fainting and palpitations; heart failure; significant impairment in overall quality of life; and sudden cardiac death in some adults and children. There are currently no approved therapeutics addressing the underlying genetic cause of HCM.

TN-201 is Tenaya’s potential first-in-class adeno-associated virus (AAV)-based investigational gene therapy for the treatment of HCM caused by mutations in the MYBPC3 gene. TN-201 is designed to deliver a fully functional MYBPC3 gene to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies of MYBPC3 knock-out models, TN-201 has been shown to halt disease progression and demonstrated significant and durable disease reversal and survival benefit after a single dose.

“Receipt of Fast Track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations,” said Whit Tingley, chief medical officer of Tenaya.

The FDA Fast Track program is designed to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical data demonstrates the potential to address unmet medical need. Companies with therapies that receive the Fast Track designation from the FDA are eligible for increased communication with the agency and may qualify for accelerated approval and priority review if relevant criteria are met. The goal of the program is to deliver approved treatments to patients with a serious or life-threatening condition as quickly as possible. This designation is not an assurance that regulatory approval will be received.

TN-201 also has received Orphan Drug designation from the FDA and Orphan Medicinal Product designation from the European Commission for the treatment of HCM due to mutations in the MYBPC3 gene.

In January 2023, Tenaya announced that the FDA cleared its Investigational New Drug application (IND) for TN-201. Tenaya is initiating the MyPeak-1 Phase 1b clinical trial to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-201. The multi-center, open-label study will enroll adults diagnosed with MYBPC3-associated nonobstructive HCM. Tenaya anticipates the first patient will be dosed in this clinical trial during the third quarter of 2023. Tenaya is also conducting two non-interventional studies to support the development of TN-201: a study evaluating seroprevalence to AAV9 antibodies among adults with MYBPC3-associated HCM, and MyClimb, a natural history study of pediatric patients with MYBPC3-associated HCM.

Photo: Whit Tingley, chief medical officer of Tenaya