Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today announced that the U.S.
Food & Drug Administration (FDA) has granted EDG-5506 Orphan
Drug Designation (ODD) for the treatment of Duchenne muscular
dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and
Rare Pediatric Disease Designation (RPDD) for the treatment of
Duchenne. EDG-5506 is an investigational orally administered small
molecule designed to prevent contraction-induced muscle damage in
dystrophinopathies, including Duchenne and Becker. EDG-5506 is
currently advancing in multiple Phase 2 trials for individuals with
Duchenne, Becker and other dystrophinopathies. The FDA previously
granted Fast Track designation for the investigation and
development of EDG-5506 for the treatment of Becker.
“Receiving orphan drug and rare pediatric disease designations
are important milestones in advancing our novel small molecule
therapeutic approach to treating individuals with Duchenne and
Becker,” said Kevin Koch, Ph.D., President and Chief Executive
Officer of Edgewise. “These regulatory designations highlight the
urgent and critical need for new and better therapeutic options for
people living with these rare, serious or life-threatening
disorders.”
Benefits of ODD and RPDD
The FDA grants ODD to support development of medicines for rare
diseases or conditions that affect fewer than 200,000 people in the
U.S. Potential benefits of the ODD include market exclusivity for
the first ODD drug for an approved indication within the ODD for a
seven-year period upon FDA approval, federal tax credit for
qualified clinical research expenses incurred in the U.S., and a
waiver of Prescription Drug User Fee Act (PDUFA) fees (currently
worth over $4 million).
RPDD acknowledges therapies under investigation for rare
pediatric diseases affecting less than 200,000 people in the U.S.
with serious or life-threatening manifestations primarily affecting
individuals up to 18 years of age. RPDD provides priority review of
the marketing application, and, if approved for marketing, grants
that sponsor a priority review voucher which can be transferred or
sold to another sponsor.
About Duchenne Muscular Dystrophy
Duchenne is a severe, degenerative muscle disorder with a median
life expectancy of around 30 years old. People living with Duchenne
begin to lose their ability to walk without assistance by their
early teens and nearly all will require the use of a wheelchair by
the time they are in their mid-teens. Duchenne is the most common
type of muscular dystrophy, and genetic mutations in the dystrophin
gene result in contraction-induced muscle damage, which is the
primary driver of irreversible muscle loss and impaired motor
function. Currently, there is no cure for Duchenne; early, active
multidisciplinary care from neuromuscular specialists,
cardiologists, physical therapists, and other specialists is
critical for optimized disease management. Current therapeutic
options for Duchenne are inadequate to prevent significant
morbidity and mortality; novel therapies in development for
Duchenne, including muscle targeted interventions, aim to
positively impact disease trajectory.
About Becker Muscular Dystrophy
Becker is a genetic, progressive neuromuscular disorder that
imposes significant physical, emotional, financial, and social
impacts predominantly on males and their caregivers. Genetic
mutations in the dystrophin gene resulting in Becker lead to
contraction-induced muscle damage, which is the primary driver of
muscle loss and impaired motor function in muscular dystrophies.
Functional decline can begin at any age, and once that muscle loss
occurs, the decline in function is irreversible and continues
throughout the individual’s life. Some individuals living with
Becker experience heart failure from cardiomyopathy, which may
result in heart transplantation or early death. Currently, there is
no cure for Becker; early and long-term multidisciplinary care from
neuromuscular specialists, cardiologists, physical therapists, and
other specialists is critical for optimized disease management.
Novel therapies are in development for Becker, including muscle
targeted interventions, aimed at positively impacting disease
trajectory.
About EDG-5506 for Duchenne and Becker Muscular
Dystrophies
EDG-5506 is an orally administered small molecule designed to
prevent contraction-induced muscle damage in dystrophinopathies
including Duchenne and Becker. EDG-5506 presents a novel mechanism
of action designed to selectively limit the exaggerated muscle
damage caused by the absence or loss of functional dystrophin. By
minimizing the progressive muscle damage that leads to functional
impairment, EDG-5506 has the potential to benefit a broad range of
patients suffering from debilitating neuromuscular disorders. Its
unique mechanism of action provides the potential to establish
EDG-5506 as a foundational therapy in dystrophinopathies, either as
a single agent therapy or in combination with available therapies
and those in development. The Company is advancing EDG-5506 through
the clinic including completing enrollment of a Phase 2 study
cohort, called CANYON, evaluating safety and effects on function
and biomarkers of muscle damage in adult males with Becker, which
has been expanded to include an additional 120 adult participants
in a pivotal cohort called GRAND CANYON, which is currently
enrolling. In Duchenne, the Company is advancing its Phase 2
clinical trials, LYNX, assessing safety, pharmacokinetics and
biomarkers of muscle damage, and FOX, which includes children and
adolescents previously treated with gene therapy. For more
information on Edgewise’s clinical trials
www.edgewisetx.com/clinical-trials.
About Edgewise Therapeutics
Edgewise Therapeutics is a leading muscle disease
biopharmaceutical company developing novel therapeutics for
muscular dystrophies and serious cardiac conditions. The Company’s
deep expertise in muscle physiology is driving a new generation of
first-in-class therapeutics. EDG-5506 is an orally administered
skeletal myosin inhibitor in clinical trials in patients with
Becker, Duchenne, and Limb-Girdle muscular dystrophies as well as
McArdle Disease. EDG-7500, currently in a Phase 1 trial, is a novel
cardiac sarcomere modulator for the treatment of HCM and other
disorders of cardiac diastolic dysfunction. The entire team at
Edgewise is dedicated to our mission: changing the lives of
patients and families affected by serious muscle diseases. To learn
more, go to: www.edgewisetx.com or follow us on LinkedIn, X
(formerly Twitter), Facebook, Instagram and Threads.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as that
term is defined in Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. Statements in
this press release that are not purely historical are
forward-looking statements. Such forward-looking statements
include, among other things, statements regarding the potential of,
and expectations regarding EDG-5506; and statements regarding
Edgewise’s expectations relating to its clinical trials. Words such
as “believes,” “anticipates,” “plans,” “expects,” “intends,”
“will,” “goal,” “potential” and similar expressions are intended to
identify forward-looking statements. The forward-looking statements
contained herein are based upon Edgewise’s current expectations and
involve assumptions that may never materialize or may prove to be
incorrect. Actual results could differ materially from those
projected in any forward-looking statements due to numerous risks
and uncertainties, including but not limited to: risks associated
with the process of discovering, developing and commercializing
drugs that are safe and effective for use as human therapeutics and
operating as an early clinical stage company including the
potential for Edgewise’s product candidates to cause serious
adverse events; Edgewise’s ability to develop, initiate or complete
clinical trials for, obtain approvals for and commercialize any of
its product candidates; the timing, progress and results of
clinical trials for EDG-5506 and EDG-7500; Edgewise’s ability to
enroll and maintain patients in clinical trials; Edgewise’s ability
to raise any additional funding it will need to continue to pursue
its business and product development plans; the timing, scope and
likelihood of regulatory filings and approvals; the potential for
any clinical trial results to differ from preclinical, interim,
preliminary, topline or expected results; Edgewise’s ability to
develop a proprietary drug discovery platform to build a pipeline
of product candidates; Edgewise’s manufacturing, commercialization
and marketing capabilities and strategy; the size of the market
opportunity for Edgewise’s product candidates; the loss of key
scientific or management personnel; competition in the industry in
which Edgewise operates; Edgewise’s reliance on third parties;
Edgewise’s ability to obtain and maintain intellectual property
protection for its product candidates; general economic and market
conditions; and other risks. Information regarding the foregoing
and additional risks may be found in the section entitled “Risk
Factors” in documents that Edgewise files from time to time with
the U.S. Securities and Exchange Commission. These forward-looking
statements are made as of the date of this press release, and
Edgewise assumes no obligation to update the forward-looking
statements, or to update the reasons why actual results could
differ from those projected in the forward-looking statements,
except as required by law.
This press release contains hyperlinks to information that is
not deemed to be incorporated by reference into this press
release.
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version on businesswire.com: https://www.businesswire.com/news/home/20231130724778/en/
Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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