Tenaya Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for Gene Therapy for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy

Tenaya Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for Gene Therapy for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy

Tenaya Therapeutics, Inc.

Tenaya Therapeutics, Inc.

TN-401 is being developed for the treatment of genetic arrhythmogenic right ventricular cardiomyopathy (RAHC) caused by mutations in the PKP2 gene

Orphan Drug Designation for TN-401 is the first for a gene therapy treatment for ARVCs

Expect to Submit TN-401 IND Application to FDA in 2023

SOUTH SAN FRANCISCO, Calif., Nov. 28, 2022 (GLOBE NEWSWIRE) — Tenaya Therapeutics, a clinical-stage biotechnology company whose mission is to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease , today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to its second gene therapy product candidate, TN-401, for the treatment of arrhythmogenic right ventricular cardiomyopathy ( RVDA).

TN-401 is an adeno-associated virus (AAV)-based gene therapy in development for the treatment of genetic ARVC caused by plakophilin-2 (PKP2) genetic mutations. mutation of the PKP2 can cause serious illness, including right ventricular enlargement in affected individuals, cardiac dysfunction, significant arrhythmia, and sudden cardiac death in adults and children. PKP2 mutations are the most common genetic cause of ARVC, estimated to affect approximately 40% of the global ARVC population and affecting more than 70,000 people in the United States alone. TN-401 is designed to use an AAV9 vector to provide a healthy copy of the PKP2 gene to the heart muscle of affected patients via a single intravenous dose. Current treatments do not address the underlying genetic cause of the disease and do not appear to affect disease progression.

The FDA’s Orphan Drug Program assigns orphan designation to drugs and biologics intended for the treatment of rare diseases (those that affect fewer than 200,000 people in the United States). The orphan designation allows Tenaya to benefit from various development incentives under the Orphan Drug Act, including tax credits for certain clinical trial expenses. This designation does not guarantee that regulatory approval will be received, but if approved, it would allow TN-401 to become eligible for seven years of market exclusivity in the United States.

Tenaya intends to launch a global, non-interventional study on ARVC PKP2 carriers of genetic mutations by the end of this year to collect treatment history and anti-AAV antibody seroprevalence data. Tenaya expects to submit an Investigational New Drug (IND) application for the program to the FDA in 2023.

Tenaya presented preclinical data supporting the activity of TN-401 in a Pkp2-deficient mouse model of ARVC earlier this year at the annual meeting of the Heart Rhythm Society (HRS), as well as at the annual meeting of the American Society of Gene and Cell Therapy (ASGCT). These data demonstrated significant improvement in right ventricular dilation, cardiac dysfunction, and electrophysiological deficits, including the characteristic arrhythmia associated with PKP2 mutations. The effects were dose-dependent and stable after a single infusion, and a significant survival benefit was observed compared to untreated controls. No safety signal has been observed to date.

About Tenaya Therapeutics
Tenaya Therapeutics is a clinical-stage biotechnology company committed to a bold mission: to discover, develop and deliver curative therapies that address the underlying drivers of heart disease. Founded by leading cardiovascular scientists from the Gladstone Institutes and the University of Texas Southwestern Medical Center, Tenaya develops therapies for rare genetic cardiovascular disorders, as well as more prevalent heart diseases, through three platforms: distinct but interrelated product forms: gene therapy, cell regeneration and precision medicine. For more information, visit www.tenayatherapeutics.com.

Forward-looking statements
This press release contains forward-looking statements as that term is defined in Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. Statements in this press release that are not purely statements are forward-looking statements. Words such as “expects”, “would”, “intends” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include, among other things, the expected timeline for submitting an IND application for TN-401; the therapeutic and commercial potential of TN-401 as a treatment for patients with ARVC; the potential benefits of obtaining orphan drug designation from the FDA for TN-401; and plans to launch a global, non-interventional study of patients with ARVC. The forward-looking statements contained herein are based on Tenaya’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. These forward-looking statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, including, but not limited to: the timing, scope and likelihood of filings and regulatory approvals for the TN-401 and Tenaya’s other product candidates; Tenaya’s ability to develop, initiate or complete preclinical studies and clinical trials for TN-401 and its other product candidates; the possibility that results of a clinical trial will differ from preclinical, interim, preliminary, primary or expected results; risks associated with the process of discovering, developing and commercializing safe and effective drugs for human therapeutic use; Tenaya’s ability to raise any additional financing it needs to pursue its business and product development plans; adverse impacts of the COVID-19 pandemic on Tenaya’s operations, including preclinical studies and planned clinical trials; Tenaya’s dependence on third parties; Tenaya’s sales and marketing capabilities and strategy; loss of key scientific or management personnel; competition in the industry in which Tenaya operates; Tenaya’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks can be found in the section titled “Risk Factors” in documents Tenaya files from time to time with the Securities and Exchange Commission. These forward-looking statements are made as of the date of this press release, and Tenaya undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except if required by law.

michelle corral
Vice President, Investor Relations and Corporate Communications
Therapeutic Tenaya

wendy ryan
Ten-bridge communications

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