BOSTON, June 1, 2021 /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 that the European
Medicines Agency (EMA) has granted orphan drug designation (ODD)
for elamipretide for the treatment of Barth syndrome, an ultra-rare
genetic condition.
"We are pleased that EMA has recognized, by granting ODD for
elamipretide for Barth syndrome, the high unmet need for innovative
treatments for this ultra-rare genetic condition," said Chief
Executive Officer Reenie McCarthy. "This marks an important
step toward advancing our regulatory initiatives in Europe as we work towards potential ex-U.S.
partnerships and approvals. We are committed to increasing
medical and regulatory awareness of this devastating condition,
which is believed to contribute to often fatal, infant-onset
idiopathic cardiomyopathy globally."
ODD is granted to investigational therapies intended for the
safe and effective treatment of rare diseases with an unmet medical
need that affect fewer than 5 in 10,000 people in the European
Union. This designation provides companies with certain benefits
and incentives including clinical protocol assistance,
differentiated evaluation procedures for Health Technology
Assessments in certain countries, access to a centralized
marketing authorization procedure valid in all EU Member States,
and if approved, marketing exclusivity in the EU for 10 years.
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.
About Stealth
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 both rare
metabolic cardiomyopathies, such as Barth, Duchenne muscular
dystrophy and Friedreich's ataxia, rare mitochondrial diseases
entailing nuclear DNA mutations, as well as ophthalmic diseases
entailing mitochondrial dysfunction, such as dry age-related
macular degeneration and Leber's hereditary optic neuropathy. 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, but are not limited
to, statements relating to preliminary clinical data. 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: those regarding Stealth BioTherapeutics' plans,
strategies and expectations for its preclinical and clinical
advancement of its drug development programs, including its ongoing
clinical trials of elamipretide; whether results obtained in
preclinical studies and clinical trials will be indicative of
results that will be generated in future clinical trials;
whether elamipretide will successfully advance through
the clinical trial process on a timely basis, or at all; and
whether the results of the company's clinical trials will warrant
regulatory submissions and whether elamipretide will
receive approval from the FDA or equivalent foreign regulatory
agencies for GA, AMD, Barth syndrome or any other indication when
expected or at all. These and other risks are described in greater
detail under the caption "Risk Factors" included in the Stealth
BioTherapeutics' most recent Annual Report on Form 20-F filed with
the Securities and Exchange Commission ("SEC") on April 6, 2021, 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
Stern Investor Relations
Janhavi Mohite, 212-362-1200
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.