– The North Star Ambulatory Assessment (NSAA)
remained stable relative to declines reported in Becker natural
history studies –
– Significant decreases were observed in
circulating levels of creatine kinase (CK) and fast skeletal muscle
troponin I (TNNI2), biomarkers associated with skeletal muscle
damage –
– Sevasemten was well-tolerated –
– Edgewise leadership to discuss ARCH findings
on Tuesday, April 16 at 8:30 a.m. Eastern Time at virtual investor
event –
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today announced positive
two-year topline results from the ARCH trial. ARCH is an open
label, single-center study assessing safety, tolerability, impact
on muscle damage biomarkers, pharmacokinetics (PK) and functional
measures with sevasemten (EDG-5506) in adults with Becker.
Sevasemten is an orally administered small molecule designed to
prevent contraction-induced muscle damage in dystrophinopathies
including Becker and Duchenne muscular dystrophy (Duchenne).
This press release features multimedia. View
the full release here:
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Figure 1: NSAA stabilized with sevasemten
treatment and continues to diverge from natural history studies at
2 years (Photo: Business Wire)
The ARCH trial evaluated sevasemten administered daily over two
years in adults with Becker. Sevasemten was well-tolerated in all
12 participants with no discontinuations or dose reductions due to
adverse events.
Preserving NSAA functions that correlate to activities of daily
living are important to individuals living with Becker. As seen in
Figure 1, during two years of sevasemten treatment, participants’
NSAA scores stabilized and continued to diverge relative to
functional declines reported across multiple Becker natural history
studies, in which two-year mean decreases of 2.4 NSAA points were
reported1, 2,3.
In addition, significant decreases in key biomarkers of muscle
damage including CK and TNNI2 were observed in participants treated
with sevasemten, which are consistent with prior observations.
“We are pleased by the promising and consistent functional
results observed over two years of treatment with sevasemten,
together with the favorable safety and tolerability profile,” said
Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “We
thank the Becker community for engaging with us on this promising
therapy.”
“Becker is a devastating neuromuscular disease that currently
has no treatment,” said Barry J. Byrne, M.D., Ph.D., Director,
Powell Gene Therapy Center, University of Florida and Chief Medical
Adviser for MDA. “Once symptoms of muscle weakness occur,
individuals with Becker have a relentless course of disease
progression. In my experience, each one-point decrease in NSAA
represents a loss of function important to daily activities. I have
been closely following the progress of sevasemten in the clinic and
am encouraged by the two-year results and the potential of this
novel muscle-targeted therapy for individuals with Becker.”
The positive results from the two-year ARCH trial further
support the hypothesis that a reduction in contraction-induced
muscle damage in muscular dystrophies has the potential to preserve
function and halt disease progression in Becker.
Upcoming ARCH 24-Month Data Presentations with Investor,
Medical and Patient Communities:
Virtual Investor Event
Members of the Edgewise management team will hold a live webcast
on Tuesday, April 16, at 8:30 a.m. ET to discuss the ARCH two-year
data, and will be joined by Dr. Byrne, who will share his
perspective of sevasemten and Becker. An accompanying slide
presentation will also be available. To register for the live
webcast and replay, please visit the Edgewise events page.
American Academy of Neurology 2024
Annual Meeting Podium Presentation
Session: Inherited Myopathies and Neuropathies: New
Therapeutic Approaches and Observations
Title: Effects of EDG-5506, a Fast Myosin Modulator, on
Function and Biomarkers of Muscle Damage in Adults with Becker
Muscular Dystrophy
Presenter: Joanne Donovan, M.D., Ph.D., Chief Medical
Officer, Edgewise Therapeutics
Date: Tuesday, April 16, 2024, 1:12 p.m. ET
The presentation will be available on the Edgewise website when
it’s presented.
Patient Community
Webinar
Members of Edgewise management will hold a community webinar on
Monday, May 13 at 1 p.m. ET to discuss these data and the GRAND
CANYON pivotal study. Further event details will be shared when
they are available.
About the ARCH Open-Label Trial
ARCH, an open-label, single-center trial, assessed sevasemten in
12 adult males with Becker. The trial evaluated sevasemten
administered daily over two years. Safety, tolerability, PK,
changes in biomarkers of muscle damage such as CK and fast skeletal
muscle troponin I, measures of function and patient-reported
outcomes were evaluated. Go to clinicaltrials.gov to learn more
about this trial (NCT05160415).
About GRAND CANYON, a Global Pivotal Study in Becker
The Company is advancing GRAND CANYON, a global pivotal study of
EDG-5506 in individuals with Becker. GRAND CANYON is an expansion
of the CANYON placebo-controlled trial. CANYON, which was
over-enrolled, includes cohorts of 40 adults and 29 adolescents and
a treatment period of 12 months. The Company expects to report
CANYON data in the fourth quarter of 2024. GRAND CANYON is a
multicenter, randomized, double-blind, placebo-controlled study to
evaluate the safety and efficacy of EDG-5506 in adults with Becker.
Data from GRAND CANYON, if positive, could support a marketing
application. The primary endpoint of GRAND CANYON is NSAA. In
addition, other functional assessments, biomarkers of muscle damage
and safety will be assessed. GRAND CANYON is anticipated to recruit
approximately 120 individuals with Becker, aged between 18 and 50
years old, at up to 50 sites in 10 countries. The treatment period
for participants will be 18 months. To learn more, go to
clinicaltrials.gov (NCT05291091) or the GRAND CANYON microsite:
https://www.beckergcstudy.com.
About Becker Muscular Dystrophy
Becker is a rare, genetic, life-shortening, debilitating and
degenerative neuromuscular disorder. The disease predominantly
affects males and imposes significant physical, emotional,
financial, and social impacts on the individual and their
caregivers. Individuals with Becker experience 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 is critical for
optimized disease management. There is a great need for more
Becker-specific scientific research, clinical programs, and
treatment guidelines to improve management of this disease. To
learn more about Becker, go to
https://beckermusculardystrophy.com/
About Sevasemten (EDG-5506) for Becker and Duchenne Muscular
Dystrophies
Sevasemten is an orally administered small molecule designed to
prevent contraction-induced muscle damage in dystrophinopathies
including Becker and Duchenne. Sevasemten 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, sevasemten 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 sevasemten as a foundational therapy in
dystrophinopathies, either as a single agent therapy or in
combination with available therapies and those in development.
In Becker, Edgewise is advancing sevasemten in a Phase 2 trial,
called CANYON, evaluating safety and effects on function and
biomarkers of muscle damage in adult males with Becker. The CANYON
trial, which is fully enrolled, has been expanded to include an
additional 120 adult participants in a pivotal cohort called GRAND
CANYON. This study is currently enrolling at sites in the United
States and Europe.
In Duchenne, Edgewise is advancing its LYNX Phase 2 clinical
trial, assessing safety, PK, biomarkers of muscle damage and
functional measures in boys with Duchenne. It also is advancing a
second Phase 2 trial, called FOX, assessing safety, PK, biomarkers
of muscle damage and functional measures in children and
adolescents previously treated with gene therapy.
For more information on Edgewise’s clinical trials
https://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. Sevasemten is an orally administered
skeletal myosin inhibitor in late-stage clinical trials in Becker
and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac
sarcomere modulator for the treatment of hypertrophic
cardiomyopathy and other diseases of diastolic dysfunction,
currently in clinical development. 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.
References
[1] Bello L, et al. Functional changes in Becker muscular
dystrophy: implications for clinical trials in dystrophinopathies.
Sci Rep. 2016;6:32439. doi:10.1038/srep32439
[2] van de Velde NM, et al. Selection approach to identify the
optimal biomarker using quantitative muscle MRI and functional
assessments in Becker muscular dystrophy. Neurology.
2021;97(5):e513-e522. doi: 10.1212/WNL.0000000000012233.
[3] De Wel B, et al. Lessons for future clinical trials in
adults with Becker muscular dystrophy: disease progression detected
by muscle magnetic resonance imaging, clinical and patient-reported
outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282.
Online ahead of print.
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 Edgewise’s expectations relating to its
clinical trials and clinical development of sevasemten; statements
regarding the potential of, and expectations regarding, Edgewise’s
product candidates and programs, including EDG-5506 and EDG-7500;
statements regarding Edgewise’s milestones, including timing of
data from its CANYON trial; statements regarding whether data from
GRAND CANYON could support a marketing application; and statements
by Edgewise’s chief medical officer and Barry J. Byrne, M.D.,
Ph.D.. 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; Edgewise’s
ability to take advantage of potential benefits associated with
designations granted by FDA and/or to maintain qualifications for
applicable designations over time; 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; the potential that the
outcome of preclinical testing and early clinical trials may not be
predictive of the success of later clinical trials; 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240415221564/en/
Edgewise Contacts Investors: Michael Carruthers,
Chief Financial Officer ir@edgewisetx.com
Media: Maureen Franco, VP Corporate Communications
media@edgewisetx.com
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