Type B meeting scheduled with FDA Division of
Cardiology and Nephrology
Company plans to discuss new clinical data
that may inform a potential NDA submission
BOSTON, June 14,
2022 /PRNewswire/ -- Stealth BioTherapeutics
Corp (Nasdaq: MITO), a clinical-stage biotechnology company focused
on the discovery, development and commercialization of novel
therapies for diseases involving mitochondrial dysfunction, today
announced its plan to meet with the U.S. Food & Drug
Administration (FDA) Division of Cardiology and Nephrology (DCN) to
discuss new clinical data that may inform a potential new drug
application (NDA) submission for the treatment of Barth
Syndrome.
In October 2021, the FDA refused
to file the Company's NDA for the treatment of Barth Syndrome. The
Company recently requested a Type B pre-NDA meeting to discuss new
clinical data on patients treated with elamipretide for over 3.5
years in the open-label extension period of the Phase 2 TAZPOWER
study. The Company believes that the new data may support an NDA
filing and approval of elamipretide as a treatment for Barth
Syndrome. The FDA has granted the meeting request and the Company
expects this meeting to occur during the third quarter.
"We welcome the opportunity to meet with FDA to discuss our
promising new clinical data and a potential NDA submission," said
Reenie McCarthy, Chief Executive
Officer of Stealth BioTherapeutics. "We look forward to working
with the Agency toward our goal of securing the regulatory review
and approval necessary to make elamipretide available to patients
with Barth Syndrome. We are eager to present our new clinical data
at an upcoming scientific meeting."
About Barth Syndrome
Barth Syndrome is an ultra-rare genetic condition characterized
by cardiac abnormalities often leading to heart failure and reduced
life expectancy, recurrent infections, muscle weakness and delayed
growth. Barth Syndrome occurs almost exclusively in males and is
estimated to affect one in 200,000 to 400,000 individuals
worldwide. There are currently no FDA- or EMA-approved therapies
for patients with Barth Syndrome. Elamipretide has Orphan
Drug, Fast Track and Rare Pediatric Designation from the FDA and
Orphan Drug Designation from the EMA for the treatment of Barth
Syndrome.
About Stealth BioTherapeutics
We are a clinical-stage biotechnology company focused on the
discovery, development, and commercialization of novel therapies
for diseases involving mitochondrial dysfunction. Mitochondria,
found in nearly every cell in the body, are the body's main source
of energy production and are critical for normal organ function.
Dysfunctional mitochondria characterize a number of rare genetic
diseases and are involved in many common age-related diseases,
typically involving organ systems with high energy demands such as
the heart, the eye, and the brain. We believe our lead product
candidate, elamipretide, has the potential to treat rare diseases
including primary mitochondrial myopathy due to nuclear DNA
mutations, rare cardiomyopathies such as Barth Syndrome, Duchenne
muscular dystrophy and Friedreich's ataxia, as well as ophthalmic
diseases entailing mitochondrial dysfunction, such as dry
age-related macular degeneration. We are evaluating our
second-generation clinical-stage candidate, SBT-272, and our new
series of small molecules, SBT-550, for rare neurological disease
indications following promising preclinical data. We have optimized
our discovery platform to identify novel mitochondria-targeted
compounds which may be nominated as therapeutic product candidates
or utilized as mitochondria-targeted vectors to deliver other
compounds to mitochondria.
Forward-looking Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. Such forward-looking statements include those regarding
Stealth BioTherapeutics' plans, strategies and expectations for its
clinical advancement of its drug development programs, including
its plans for the potential submission of an NDA and the potential
benefits of Stealth BioTherapeutics' product candidates.
Statements that are not historical facts, including
statements about Stealth BioTherapeutics' beliefs, plans and
expectations, are forward-looking statements. The words
"anticipate," "expect," "hope," "plan," "potential," "possible,"
"will," "believe," "estimate," "intend," "may," "predict,"
"project," "would" and similar expressions are intended to identify
forward-looking statements, although not all forward-looking
statements contain these identifying words. Stealth
BioTherapeutics may not actually achieve the plans, intentions or
expectations disclosed in these forward-looking statements, and you
should not place undue reliance on these forward-looking
statements. Actual results or events could differ materially
from the plans, intentions and expectations disclosed in the
forward-looking statements as a result of known and unknown risks,
uncertainties and other important factors, including: Stealth
BioTherapeutics' ability to obtain additional funding and to
continue as a going concern; the impact of the COVID-19 pandemic;
the ability to successfully demonstrate the efficacy and safety of
Stealth BioTherapeutics' product candidates and future product
candidates; the preclinical and clinical results for Stealth
BioTherapeutics' product candidates, which may not support further
development and marketing approval; the potential advantages of
Stealth BioTherapeutics' product candidates; the content and timing
of decisions made by the U.S. FDA, the EMA or other regulatory
authorities, investigational review boards at clinical trial sites
and publication review bodies, which may affect the initiation,
timing and progress of preclinical studies and clinical trials of
Stealth BioTherapeutics product candidates; Stealth
BioTherapeutics' ability to obtain and maintain requisite
regulatory approvals and to enroll patients in its planned clinical
trials; unplanned cash requirements and expenditures; competitive
factors; Stealth BioTherapeutics' ability to obtain, maintain and
enforce patent and other intellectual property protection for any
product candidates it is developing; and general economic and
market conditions. These and other risks are described in
greater detail under the caption "Risk Factors" included in Stealth
BioTherapeutics' most recent Annual Report on Form 20-F filed with
the Securities and Exchange Commission ("SEC"), as well as in any
future filings with the SEC. Forward-looking statements
represent management's current expectations and are inherently
uncertain. Except as required by law, Stealth BioTherapeutics does
not undertake any obligation to update forward-looking statements
made by us to reflect subsequent events or circumstances.
Investor Relations
Kendall Investor Relations
Adam Bero, Ph.D.
abero@kendallir.com
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SOURCE Stealth BioTherapeutics Inc.