– Initiating new Phase 2 FOX trial in boys with
Duchenne who have been previously treated with gene therapy –
– Continuing dose escalation of Phase 2 LYNX
trial and initiating a cohort in boys with Duchenne not currently
treated with corticosteroids –
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today announced an expansion of
their clinical development program of EDG-5506, an investigational
orally administered small molecule designed to prevent
contraction-induced muscle damage in dystrophinopathies, including
Duchenne. The Company is initiating FOX, a new Phase 2
placebo-controlled trial in children and adolescent boys with
Duchenne who have been previously treated with gene therapy.
Further, the Company is continuing dose escalation and expanding
enrollment in their Phase 2 placebo-controlled LYNX trial; one of
the new LYNX cohorts will study EDG-5506 in boys with Duchenne not
currently treated with corticosteroids.
With strong interest from the Duchenne community and supportive
initial safety data from LYNX, the Company initiated the FOX trial,
a new Phase 2 placebo-controlled trial in children and adolescents
with Duchenne who have been previously treated with gene therapy.
The FOX trial will assess the effect of EDG-5506 over 12 weeks on
safety, pharmacokinetics (PK) and biomarkers of muscle damage. The
study will also explore changes in functional measures, such as the
North Star Ambulatory Assessment (NSAA) and
self-reported/caregiver-reported outcomes. Approximately 24
participants, aged 6 to 14 years, are expected to be enrolled in
the trial at multiple sites across the United States. Participants
will then continue in an open-label extension portion of the trial
for a total of 12 months to gain further insights into safety, PK,
function and biomarker measures.
The LYNX trial in children with Duchenne has rapidly enrolled at
14 sites across the United States, with the first three cohorts
over-enrolled. Based on the safety profile observed to date, the
Company added an additional cohort to continue dose escalation of
EDG-5506. LYNX is designed to identify a dose of EDG-5506 that will
reduce biomarkers of muscle damage and has the potential to provide
functional benefit to patients in a Phase 3 trial. The Company
expects to report Phase 2 interim data in the first half of 2024,
with the Phase 3 dose identified. Additionally, the Company is
adding a new cohort to LYNX to include children aged 4 to 9 years
with Duchenne who are not currently treated with corticosteroids.
The Company also extended the open label portion of the trial for a
total of 24 months.
“Having provided care for individuals with Duchenne for over 20
years, I’m excited to see Edgewise expand its clinical studies with
EDG-5506 to include boys with Duchenne who are not currently
treated with steroids as well as those who have received gene
therapy and remain symptomatic,” said Barry J. Byrne, M.D., Ph.D.,
Director, Powell Gene Therapy Center, University of Florida and
Chief Medical Adviser for MDA. “The patient community is desperate
to find additional treatment options to curb the effects of this
devastating disease.”
“We have listened to the Duchenne community and thoughtfully
initiated FOX and expanded LYNX, both important advances in our
EDG-5506 clinical development program. We anticipate moving towards
a Phase 3 trial start in Duchenne in the second half of 2024,” said
Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “We
are thrilled to open our studies of EDG-5506 to broader groups of
individuals with Duchenne exploring the potential of a
muscle-targeted therapeutic against the evolving treatment
landscape.”
About FOX Phase 2 Trial in Children with Duchenne
FOX is a Phase 2 placebo-controlled trial to assess the effect
of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle
damage in children and adolescents with Duchenne who have been
previously treated with gene therapy. Approximately 24
participants, aged 6 to 14 years, are expected to be enrolled in
the trial at multiple sites across the United States, which is
expected to start in early 2024. Participants will then continue in
an open-label extension portion of the trial for a total of 12
months to gain further insights into safety, PK, function and
biomarker measures. Go to clinicaltrials.gov to learn more about
this trial (NCT06100887).
About LYNX Phase 2 Trial in Children with Duchenne
LYNX is a Phase 2 placebo-controlled trial to assess the effect
of multiple doses of EDG-5506 over 12 weeks on safety, PK and
biomarkers of muscle damage. The study will also explore changes in
functional measures, such as the North Star Ambulatory Assessment
(NSAA) and self-reported/caregiver-reported outcomes. Approximately
45 children with Duchenne aged 4 to 9 years are expected to be
enrolled in this trial. Participants will then continue in an
open-label extension portion of the trial for a total of 24 months
to gain further insights into safety and functional measures.
Importantly, this trial is designed to identify a dose of EDG-5506
that will reduce biomarkers of muscle damage and has the potential
to provide functional benefit to patients in a Phase 3 trial. Go to
clinicaltrials.gov to learn more about this trial
(NCT05540860).
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 EDG-5506 for Becker and Duchenne 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. In August 2021, the U.S. Food and Drug
Administration (FDA) granted Fast Track designation to EDG-5506 for
the treatment of individuals with Becker.
The Company has advanced EDG-5506 through the clinic including
completed a Phase 1 trial evaluating safety, tolerability, PK and
pharmacodynamics of EDG-5506 in adult healthy volunteers (Phase 1a)
and in adults with Becker (Phase 1b) (NCT04585464). In ARCH, an
open-label, single-center trial (NCT05160415) assessing long-term
safety and PK, decreases in biomarkers of muscle damage and trends
toward improvement in NSAA have been observed following 12 months
of treatment with EDG-5506. A Phase 2 cohort of EDG-5506 in Becker
(CANYON) is fully enrolled and has now been expanded to include an
additional 120 adult participants in a pivotal cohort called GRAND
CANYON, which is currently enrolling (NCT05291091). In Duchenne,
the Company is advancing its Phase 2 studies, LYNX (NCT05540860)
and FOX (NCT06100887), both assessing safety, PK and biomarkers of
muscle damage in individuals with Duchenne. The Company is also
continuing to advance the DUNE Phase 2 exercise challenge study, to
evaluate the effect of EDG-5506 on biomarkers of muscle damage
following exercise in adults with LGMD2I, Becker or McArdle disease
at a single site in Denmark.
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; statements regarding
Edgewise’s expectations relating to its clinical trials, including
the timing of reporting data (including the Phase 2 interim data
from the LYNX clinical trial of EDG-5506) and commencement of
trials (including the FOX clinical trial and Phase 3 clinical trial
of EDG-5506 Duchenne); statements regarding Edgewise’s pipeline of
product candidates and programs; and statements by Edgewise’s Chief
Medical Officer. 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; 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/20231026677052/en/
Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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