Pre-IND meeting request granted by FDA's
Division of Neurology I
BOSTON, May 12, 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, announced today that the US
Food and Drug Administration (FDA) Office of Orphan Products
Development has granted Orphan Drug Designation to elamipretide for
the treatment of patients with Duchenne muscular dystrophy
(DMD).
The company also announced that the FDA's Division of Neurology
I has granted its request for a pre-IND meeting to discuss a
development path for elamipretide in combination with products
within the approved therapeutic class of exon-skipping
phosphorodiamidate morpholino oligomers (PMO). The company
has previously presented data demonstrating that administration of
elamipretide in combination with a PMO significantly improves
dystrophin expression levels in the X-linked muscular dystrophy
(mdx) mouse model. The company also previously conducted a
pre-IND meeting with the FDA's Division of Cardiology and
Nephrology during which it aligned on a potential development path
for elamipretide for cardiomyopathy associated with DMD.
"We are pleased that the FDA has recognized the high unmet need
for innovative treatments for DMD," said Chief Executive Officer
Reenie McCarthy. "We look forward to
further discussions with the FDA regarding our development
initiatives, which we hope will bring new options to patients
suffering from this devastating disease."
DMD is an X chromosome-linked genetic disorder caused by
mutations in the DMD gene which encodes the dystrophin protein. DMD
is a fatal disease of muscle membrane instability in which the loss
of dystrophin and ensuing mitochondrial dysfunction results in a
cascade of events leading to progressive loss of muscle
function. The disease, which is typically diagnosed in
childhood, is associated with progressive skeletal muscle
dysfunction typically starting in early childhood, progressing to
loss of ambulation in early adolescence, loss of upper limb
function and assisted ventilation during late adolescence into
early adulthood. Despite recent advances in treatment options
for DMD, including the conditional approvals of several PMOs, most
patients die in early adulthood from respiratory or, more commonly,
cardiac failure. Cellular uptake and retention of PMOs and
PMO-mediated dystrophin is thought to be limited by mitochondrial
dysfunction associated with DMD.
The Orphan Drug Act was enacted in 1983 to encourage development
of drugs for rare diseases, which are diseases that affect fewer
than 200,000 persons in the United
States. Once granted, Orphan Drug Designation provides
various development benefits for an investigational drug, including
seven-year exclusivity after marketing approval is received.
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 AMD, 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 lobar 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 preclinical data and development efforts for Duchenne
muscular dystrophy. 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 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
Kendall Investor Relations
Adam Bero, 617-914-0008
IR@StealthBT.com
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SOURCE Stealth BioTherapeutics Inc.