BOSTON, May 17, 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 company is
presenting a poster at the 2021 American College of Cardiology
(ACC) meeting being held virtually from May
15-17, 2021.
The poster entitled "Elamipretide Improves Functional
Assessments when Compared to the Natural History Progression of
Cardiomyopathy-related Disease Symptomatology in Patients with
Barth Syndrome: A TAZPOWER Analysis" presents data from a Phase 3
retrospective natural history control trial comparing the cardiac
and functional effects of elamipretide on Barth syndrome patients
at week 72 of the open label extension portion to a natural history
control (NHC) cohort derived from a 9-year long NHC study and
matched using propensity scoring. The cardiac findings show that
long-term treatment with elamipretide results in significant
improvements in left ventricular stroke volume, which continue to
strengthen over time with elamipretide, and which would otherwise
be expected to decline during the natural course of the
disease. Significant improvements were also observed in
distance walked on the six-minute walk test and other functional
endpoints, which also strengthen over time with elamipretide and
would not be expected to improve during the natural course of the
disease.
About the Phase 3 Natural History Control Study
The Phase 3 retrospective natural history control study compared
findings on functional assessments collected through weeks 36 and
48 and, as an FDA-requested post hoc analysis, week 72 of the
open-label extension portion of the TAZPOWER trial with findings on
the same assessments for up to 19 prognostically matched natural
history control subjects, for whom data were collected
longitudinally between 2012 and 2019 by investigators at Johns
Hopkins Kennedy-Krieger Institute. The primary endpoint was
the change in distance walked during the 6MWT. Secondary
endpoints included all additional functional assessments for which
there were natural history data, including measurements of muscle
strength, balance, and the time to complete an assessment requiring
patients to sit and stand five times in succession. A
multi-domain responder analysis was also conducted to assess the
percentage of patients experiencing at least a 10% clinically
meaningful gain (or loss) on these endpoints. Additionally,
this study compared changes in left ventricular stroke
volume observed during the open-label extension portions of
the TAZPOWER trial with changes observed in the natural history
dataset. All types of heart failure are associated with a
deterioration of stroke volume, which is a key component of both
cardiac output and ejection fraction.
About the TAZPOWER Study
TAZPOWER is a Phase 2/3 crossover study evaluating the effects
of daily subcutaneous (SC) treatment with elamipretide in 12
patients with genetically confirmed Barth syndrome followed by an
open-label treatment extension. Part 1 was a 28-week crossover
trial of patients randomized to elamipretide 40 mg SC daily for 12
weeks, followed by SC placebo daily for 12 weeks, or vice versa,
separated by a 4-week washout. Part 2 is an open-label assessment
for up to 192 weeks of functional and cardiac assessments,
patient-reported outcomes and safety/tolerability.
The primary endpoints included change in distance walked during
the 6MWT and change in patient-reported outcomes. Secondary
endpoints included additional functional assessments,
patient-reported outcomes, echocardiographic assessments of cardiac
structure and function, and safety/tolerability.
About Barth Syndrome
Barth syndrome is an ultra-rare genetic condition characterized
by cardiac abnormalities often leading to heart failure, muscle
weakness, recurrent infections, delayed growth and reduced life
expectancy, typically due to premature cardiac-related death. Barth
syndrome occurs almost exclusively in males and is estimated to
affect one in 300,000 to 400,000 individuals worldwide at birth.
There are currently no FDA-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 the company's clinical
trials will be fully enrolled and completed when anticipated;
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; 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; whether,
if Stealth BioTherapeutics' products receive approval, they will be
successfully distributed and marketed; its expectations
regarding regulatory interactions; the potential benefits of
Stealth BioTherapeutics' product candidates; its key milestones for
2021 and 2022; and its plans regarding future data presentations;
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 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 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.