Tenaya Therapeutics Receives FDA Fast Track Designation for TN-201
May 02 2023 - 7:50AM
Tenaya Therapeutics, Inc. (NASDAQ: TNYA), 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 Fast Track designation for
its gene therapy product candidate, TN-201, being developed for the
treatment of Myosin Binding Protein C3 (MYBPC3)-associated
hypertrophic cardiomyopathy (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, the most common genetic cause of HCM.
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, M.D.,
Ph.D., Chief Medical Officer of Tenaya. “As we prepare to begin
dosing patients later this year, we look forward to continued close
collaboration with the FDA under this designation in support of
TN-201’s development.”
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.
About
MYBPC3-Associated Hypertrophic
Cardiomyopathy Hypertrophic cardiomyopathy (HCM) is the
most common inherited cardiac disorder and variants in the Myosin
Binding Protein C3 (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 debilitating 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
first-in-class adeno-associated virus (AAV)-based gene therapy
candidate being developed to treat hypertrophic cardiomyopathy
(HCM) due to disease-causing variants in the MYBPC3 gene.
About Tenaya Therapeutics Tenaya
Therapeutics is a clinical-stage biotechnology company committed to
a bold mission: to discover, develop and deliver potentially
curative therapies that address the underlying drivers of heart
disease. Leveraging its integrated and interrelated Gene Therapy,
Cellular Regeneration and Precision Medicine platforms and
proprietary core capabilities, the company is advancing a pipeline
of novel therapies with diverse treatment modalities for rare
genetic cardiovascular disorders and more prevalent heart
conditions. Tenaya’s most advanced candidates include TN-201, a
gene therapy for MYBPC3-associated hypertrophic cardiomyopathy
(HCM), TN-401, a gene therapy for PKP2-associated
arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301,
a small molecule HDAC6 inhibitor being initially developed for
heart failure with preserved ejection fraction (HFpEF). Tenaya also
has multiple early-stage programs progressing through preclinical
development. 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 historical are forward-looking
statements. Words such as “potential,” “look forward,” “eligible,”
“may,” “anticipates,” and similar expressions are intended to
identify forward-looking statements. Such forward-looking
statements include, among other things, statements regarding the
therapeutic potential of TN-201 for MYBPC3-associated HCM
patients; the timing for dosing of patients in the Phase 1b
clinical trial evaluating TN-201; eligibility for increased
communication with the FDA and potential qualification for
accelerated approval and priority review for TN-201; and statements
by Tenaya’s Chief Medical Officer. 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; Tenaya’s ability to
initiate and complete clinical trials for its product candidates;
risks associated with the process of discovering, developing and
commercializing drugs that are safe and effective for use as human
therapeutics and operating as an early stage company; the potential
for any clinical trial results to differ from preclinical, interim,
preliminary, topline or expected results; the level of costs
associated with Tenaya’s research, development, manufacturing and
other activities and the company’s ability to raise any additional
funding it will need to continue to pursue its business and product
development plans; Tenaya’s ability to successfully operate a
manufacturing facility for clinical or commercial supply; 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 reliance on third
parties; 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.
Contacts
Investors Michelle Corral Vice
President, Investor Relations and Corporate Communications Tenaya
Therapeutics IR@tenayathera.com
Media Wendy Ryan Ten Bridge
Communications wendy@tenbridgecommunications.com
Tenaya Therapeutics (NASDAQ:TNYA)
Historical Stock Chart
From Jun 2024 to Jul 2024
Tenaya Therapeutics (NASDAQ:TNYA)
Historical Stock Chart
From Jul 2023 to Jul 2024