Tenaya Therapeutics Secures US FDA Nod to Initiate Clinical Testing

Tenaya Therapeutics, Inc., a clinical-stage biotechnology firm, announced that the FDA has approved the company's Investigational New Drug (IND) application to begin clinical testing of TN-401. TN-401 is Tenaya’s adeno-associated virus serotype 9 (AAV9)-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the plakophilin-2 (PKP2) gene. Based on this IND clearance, the company plans to initiate the RIDGE-1 phase 1b clinical trial of TN-401, a multi-centre, open-label study to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-401, as per pharmabiz. ARVC (arrhythmogenic cardiomyopathy) is a chronic, progressive, hereditary condition that normally manifests before the age of 40. ARVC patients have signs of ventricular arrhythmias such as palpitations, lightheadedness, and fainting, and they are at an elevated risk of sudden cardiac death.

The most prevalent genetic cause of ARVC is PKP2 mutations, which result in the loss of essential proteins required to maintain the structural integrity and cell-to-cell signaling of heart muscle cells. “People with arrhythmogenic cardiomyopathy report high levels of fear and stress and must withstand burdensome physical and lifestyle restrictions in an effort to manage the frequent abnormal heart rhythms and constant risk of sudden cardiac arrest associated with their disease,” said Whit Tingley, M.D., Ph.D., Tenaya’s chief medical officer. “TN-401 is intended to address the genetic mutation most frequently underlying ARVC. The initial dose for TN-401 in the RIDGE-1 study was associated with near maximal efficacy in our preclinical studies. With clinical site and patient community engagement well underway, we look forward to rapidly advancing TN-401 into the clinic.”