BOSTON, Oct. 19, 2020 /PRNewswire/ -- Stealth
BioTherapeutics (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 will have a poster at
the upcoming 2020 American Society of Human Genetics (ASHG)
Meeting, which is being held online from October 27-30, 2020. Stealth's poster has
received a "Reviewers' Choice" award, granted to the top 10% of all
poster abstracts, and will be available on-demand to conference
attendees via the conference website.
The poster entitled "TAZPOWER Analysis: Elamipretide
Significantly Improves Disease Symptomatology versus Natural
History Controls in Barth Syndrome," demonstrates the importance of
longitudinal natural history data in understanding and validating
clinical trial outcomes for ultra-rare diseases like Barth. The
natural history control study compared changes over time on
functional assessments of exercise endurance and strength for Barth
patients receiving elamipretide during the TAZPOWER interventional
study and long-term natural history control patients, concluding
that patients receiving elamipretide experienced statistically
significant functional improvements that are not anticipated by the
natural history. In addition, Barth patients receiving elamipretide
during the TAZPOWER interventional study experienced statistically
significant improvements over time in their left ventricular stroke
volume, compared to a decline in stroke volume observed in
long-term natural history control patients. Stroke volume is
an important parameter of cardiac function, which is known to be
impaired in Barth syndrome.
About the Natural History Control Study
This observational study compared findings on functional
assessments collected through weeks 36 and 48 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 was 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 was natural history data, which were measurements of muscle
strength, balance, and the time to complete an assessment requiring
patients to sit and stand five times in succession (5XSST). 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 168 weeks of functional assessments, patient-reported
outcomes and safety/tolerability.
The primary endpoints included change in distance walked during
the 6MWT and change in the BTHS-SA Total Fatigue score.
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 muscle weakness, cardiac abnormalities often leading to heart
failure, 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 and Becker
muscular dystrophies and Friedreich's ataxia, rare mitochondrial
diseases entailing nuclear DNA mutations, such as POLG-related
disorders, 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, for rare
neurodegenerative disease indications following promising
preclinical data in amyotrophic lateral sclerosis, or ALS. We have
optimized our discovery platform to identify novel
mitochondria-targeted compounds, including SBT-259, the SBT-550
series of compounds, and other compounds which may be nominated as
therapeutic product candidates or utilized as scaffolds 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 preclinical and clinical advancement of its
drug development programs, including its ongoing clinical trials of
elamipretide. 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
the 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
Stern Investor Relations
Janhavi Mohite, 212-362-1200
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.