BOSTON, Sept. 15,
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 that the company is participating in upcoming events to
discuss challenges and opportunities in ultra-rare disease drug
development.
Reenie McCarthy, Stealth's CEO,
will be participating in "Rare Disease Research: A Prescription"
hosted by STAT which will take place on Thursday, September 15th from 5:30 p.m.–9:00 p.m.
ET at Convene located at 600 14th Street NW, Washington, DC.
Ms. McCarthy will also participate in a discussion on
"Incentives, Investments & the Way Forward for Ultra Rare
Disease Drug Development" at The Business of RARE Biotech Summit on
Tuesday, September 20 from 9:00
a.m.–4:30 p.m. at the Hyatt Regency Capitol Hill located at 400 New
Jersey Ave NW, Washington,
DC. A copy of the presentation will be available on the
Investors & News section of Stealth's website
at https://investor.stealthbt.com/.
These discussions are expected to include Stealth's clinical and
regulatory development experience with Barth syndrome, an
ultra-rare disease of cardiolipin deficiency. Stealth has recently
met with the Division of Cardiology and Nephrology at the FDA to
discuss data from Week 168 of its SPIBA-201 Part 2 open-label
extension, which was the last visit completed by all SPIBA-201 Part
2 trial participants. At that time point, after over 3.5
years of chronic elamipretide therapy, a >40% mean improvement
from baseline in left ventricular stroke volume (p=0.007) and end
diastolic volume (p=0.003) was observed. Also, subjects continued
to show improved exercise tolerance on the six-minute walk test
(>90-meter mean improvement from baseline; p=0.003), muscle
strength (>60 newton mean improvement from baseline;
p<0.0001) and other functional endpoints. The company has
requested an additional meeting with the FDA to gain further
clarity on a regulatory path forward.
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 eye, the neuromuscular system, the heart and the brain. We
believe our lead product candidate, elamipretide, has the potential
to treat ophthalmic diseases entailing mitochondrial dysfunction,
such as dry age-related macular degeneration, rare neuromuscular
disorders, such as primary mitochondrial myopathy and Duchenne
muscular dystrophy, and rare cardiomyopathies, such as Barth
syndrome. We are evaluating our second-generation clinical-stage
candidate, SBT-272, for rare neurological disease indications, such
as amyotrophic lateral sclerosis and frontotemporal dementia,
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' expectations for
elamipretide clinical data and development efforts. 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 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
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.