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 first patient has
been dosed with TN-201 gene therapy for the treatment of Myosin
Binding Protein C3 (MYBPC3)-associated HCM in the MyPeak-1
Phase 1b clinical trial at the Cleveland Clinic, Cleveland, Ohio.
Tenaya anticipates sharing initial data from the MyPeak-1 trial in
2024.
TN-201 is Tenaya’s potential first-in-class adeno-associated
virus (AAV)-based gene therapy designed to deliver a working,
full-length copy of the human MYBPC3 gene to heart muscle cells.
The working MYBPC3 gene is intended to restore normal levels of
myosin-binding protein, which regulates the contraction and
relaxation of the heart muscle. In preclinical studies, TN-201
halted disease progression and demonstrated significant and durable
disease reversal and survival benefit following a single dose.
“MYBPC3 gene mutations are the most common genetic cause of HCM
and people with MYBPC3-associated HCM are at increased risk for
accelerated decline and serious complications associated with their
condition,” said Milind Desai, M.D., MBA, Director at the Cleveland
Clinic Hypertrophic Cardiomyopathy Center and Vice Chair, Heart
Vascular Thoracic Institute, Cleveland Clinic, and an investigator
for the MyPeak-1 Phase 1b clinical trial. “TN-201, a gene therapy
for MYBPC3-associated HCM, offers the potential of a one-time
treatment to correct the underlying genetic cause of disease and
improve patient outcomes. We are pleased to participate in the
first-in-human clinical trial of TN-201 to explore this new use of
gene therapy treatment.”
The MyPeak-1 Phase 1b clinical trial is a multi-center,
open-label, dose-escalating study designed to assess the safety,
tolerability and clinical efficacy of a one-time intravenous
infusion of TN-201. The trial will initially seek to enroll at
least six symptomatic (New York Heart Association Class II or III)
adults who have been diagnosed with MYBPC3-associated
nonobstructive HCM and have an implantable cardioverter
defibrillator, and potentially treat up to 15 subjects in total
from the U.S. and outside the U.S.
“The initiation of the MyPeak-1 clinical trial of TN-201 – the
first gene therapy for MYBPC3-associated HCM to be studied in
humans and the first of Tenaya’s gene therapy candidates to reach
clinical stage – is a significant milestone in our efforts to
improve patients’ lives through the development of new treatments
that target the genetic underpinnings of heart disease,” said Whit
Tingley, M.D., Ph.D., Tenaya’s Chief Medical Officer. “We are
grateful for the support of study sites, referral centers, patient
advocacy organizations, and patients and families who are actively
engaged with Tenaya in our efforts to explore the potential of
TN-201 as a novel treatment for MYBPC3-associated HCM. We look
forward to continuing this close partnership as we enroll
additional patients in the MyPeak-1 study and in subsequent
studies.”
The first dose of TN-201 being assessed in the MyPeak-1 clinical
trial is 3E13 vg/kg, a dose associated with near-maximal efficacy
in preclinical studies. Once three patients have been dosed at the
3E13 vg/kg level, a data safety and monitoring board (DSMB) of
external advisors will review safety data and advise Tenaya on
plans to enroll patients at the dose level of 6E13vg/kg and enroll
additional patients in the initial dose cohort.
The MyPeak-1 clinical trial will be conducted at up to twelve
leading U.S. centers specializing in HCM care. The first site that
is actively recruiting patients is the Hypertrophic Cardiomyopathy
Center at the Cleveland Clinic, Cleveland, Ohio. To learn more
about gene therapy for HCM and participation in the MyPeak-1 study,
please visit HCMStudies.com or ClinicalTrials.gov
(NCT05836259).
About MYBPC3-Associated
Hypertrophic CardiomyopathyHypertrophic 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. In MYBPC3-associated HCM, mutations of the
MYBPC3 gene result in insufficient expression of a protein needed
to regulate heart contraction. It 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 that address the underlying genetic cause of
HCM.
About TN-201TN-201 is an investigational
first-in-class adeno-associated virus (AAV)-based gene therapy
being developed to treat HCM due to disease-causing variants in the
MYBPC3 gene. TN-201 gene therapy is intended to deliver a working
MYBPC3 gene to specific cells of the heart via a single infusion to
address the underlying cause of MYBPC3-associated HCM. The U.S.
Food and Drug Administration has granted TN-201 Fast Track and
Orphan Drug Designation. TN-201 has also received orphan medicinal
product designation from the European Commission.
Tenaya is conducting the Phase 1b MyPeak-1 clinical trial in
symptomatic adults diagnosed with MYBPC3-associated nonobstructive
HCM. As safety and dosing are established, Tenaya plans to develop
TN-201 for any patient with a pathogenic MYBPC3 mutation, including
adults with obstructive disease and infants, children and teens
with heterozygous or homozygous mutations.
A non-interventional natural history study designed to
understand MYBPC3-associated HCM in infants, children and teens
known as the MyClimb Natural History study is also ongoing at
twenty-four sites in the U.S., Canada, and Europe. More information
can be found HCMStudies.com or ClinicalTrials.gov
NCT05112237.
About Tenaya TherapeuticsTenaya 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 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 “anticipates,” “potential,” “seek,” “look forward,”
“will,” “plan,” and similar expressions are intended to identify
forward-looking statements. Such forward-looking statements
include, among other things, the expected timing for sharing
initial clinical data from MyPeak-1; the clinical and therapeutic
potential of TN-201 as a one-time treatment to correct the
underlying genetic cause of MYBPC3-associated HCM and improve
patient outcomes; enrollment targets for MyPeak-1; and clinical
development plans for TN-201. 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 and
progress of MyPeak-1; unexpected concerns that may arise as a
result of the occurrence of adverse safety events in MyPeak-1; the
potential failure of TN-201 to demonstrate safety and/or efficacy
in clinical testing; the potential for MyPeak-1 initial clinical
trial results to differ from preclinical or expected results; the
timing, scope and likelihood of regulatory filings and approvals
for TN-201; Tenaya’s ability to successfully operate a
manufacturing facility for clinical supply for TN-201; 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; Tenaya’s
ability to develop, initiate or complete preclinical studies and
clinical trials, and obtain approvals, for any of its product
candidates; Tenaya’s continuing compliance with applicable legal
and regulatory requirements; Tenaya’s ability to raise any
additional funding it will need to continue to pursue its business
and product development plans; 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.
Contacts
Michelle CorralVice President, Investor Relations and Corporate
CommunicationsTenaya TherapeuticsIR@TenayaThera.com
InvestorsJulie SeidelStern
IRJulie.Seidel@SternIR.com
MediaWendy RyanTen Bridge
Communicationswendy@tenbridgecommunications.com
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