Tenaya Therapeutics Receives Orphan Drug Designation from the U.S. Food and Drug Administration for its Gene Therapy for Genetic Arrhythmogenic Right Ventricular Cardiomyopathy
November 28 2022 - 8:30AM
Tenaya Therapeutics, a clinical-stage biotechnology company with a
mission to discover, develop and deliver potentially curative
therapies that address the underlying causes of heart disease,
today announced that the U.S. Food and Drug Administration (FDA)
has granted orphan drug designation for its second gene therapy
product candidate, TN-401, for the treatment of arrhythmogenic
right ventricular cardiomyopathy (ARVC).
TN-401 is an adeno-associated virus (AAV)-based gene therapy
being developed for the treatment of genetic ARVC caused by
Plakophilin-2 (PKP2) gene mutations. Mutations of
the PKP2 gene can cause severe disease, including
enlargement of the right ventricle 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 represent approximately 40 percent of
the overall ARVC population and to effect more than 70,000 people
in the U.S. alone. TN-401 is designed to use an AAV9 vector to
deliver 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 disease
and do not appear to affect disease progression.
The FDA Orphan Drug program provides orphan designation to drugs
and biologics that are intended for the treatment of rare diseases
(those affecting fewer than 200,000 people in the United States).
Orphan designation qualifies Tenaya for various development
incentives as part of the Orphan Drug Act, including tax credits
for certain clinical trial expenses. This designation is not an
assurance that regulatory approval will be received, but if
approved, would allow TN-401 to become eligible for seven years of
market exclusivity in the United States.
Tenaya intends to initiate a global non-interventional study of
ARVC PKP2 gene mutation carriers by the end of this year in order
to collect treatment history and data on seroprevalence to AAV
antibodies. Tenaya expects to submit an Investigational New Drug
(IND) application for the program to the FDA in 2023.
Tenaya presented preclinical data supporting TN-401’s activity
in a Pkp2-deficient murine 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 of right
ventricular dilation, cardiac dysfunction, and electrophysiological
deficits, including the hallmark arrythmia associated with PKP2
mutations. The effects were dose-dependent and stable following a
single infusion, and a significant survival benefit was observed
compared to untreated controls. No safety signals have been
observed to date.
About Tenaya TherapeuticsTenaya 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 Gladstone Institutes and the
University of Texas Southwestern Medical Center, Tenaya is
developing therapies for rare genetic cardiovascular
disorders, as well as for more prevalent heart
conditions, through three distinct but interrelated product
platforms: Gene Therapy, Cellular Regeneration and Precision
Medicine. For more information,
visit www.tenayatherapeutics.com.
Forward-Looking StatementsThis 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 historical are forward-looking statements.
Words such as “expect,” “would,” “intends,” and similar expressions
are intended to identify forward-looking statements. Such
forward-looking statements include, among other things, the
expected timing for submission of 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 receipt of orphan
drug designation by the FDA for TN-401; and plans to initiate a
global non-interventional study of patients with ARVC. The
forward-looking statements contained herein are based upon 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 regulatory filings and
approvals for 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 potential for any clinical trial results to differ
from preclinical, interim, preliminary, topline or expected
results; risks associated with the process of discovering,
developing and commercializing drugs that are safe and effective
for use as human therapeutics; Tenaya’s ability to raise any
additional funding it will need to continue to pursue its business
and product development plans; negative impacts of the COVID-19
pandemic on Tenaya’s operations, including planned preclinical
studies and clinical trials; Tenaya’s reliance on third parties;
Tenaya’s commercialization and marketing capabilities and strategy;
the 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 may
be found in the section entitled “Risk Factors” in documents that
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 assumes no obligation to
update or revise any forward-looking statements, whether as a
result of new information, future events or otherwise, except as
required by law.
ContactsInvestorsMichelle
CorralVice President, Investor Relations and Corporate
CommunicationsTenaya TherapeuticsIR@tenayathera.com
MediaWendy RyanTen Bridge
Communicationswendy@tenbridgecommunications.com
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