– GRAND CANYON is the first pivotal study of an
investigational therapy for Becker – – CANYON, the initial Phase 2
cohorts, is fully enrolled –
Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today announced the start of
enrollment of GRAND CANYON, a global pivotal study of EDG-5506 in
individuals with Becker. GRAND CANYON is an expansion of the CANYON
study. CANYON, which was over-enrolled, includes 39 adults and 24
adolescents. EDG-5506 is an orally administered small molecule
designed to prevent contraction-induced muscle damage in
dystrophinopathies including Becker and Duchenne muscular
dystrophy. There are currently no approved therapies for
individuals with Becker, a serious genetic, progressive
neuromuscular disorder with significant unmet need.
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 North Star Ambulatory Assessment
(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.
“Based on the strength of safety, functional and biomarker
results from our ARCH open label study, we have rapidly initiated
this ground-breaking clinical study,” said Joanne Donovan, M.D.,
Ph.D., Chief Medical Officer of Edgewise. “This study is critical
for the Becker community, as there currently are no approved
treatments for Becker, a condition that for far too long has been
neglected.”
“It is exciting to move to the next stage of testing of the
clinical hypothesis that a reduction in contraction-induced muscle
damage has the potential to benefit people living with muscular
dystrophies,” said Alan Russell, Ph.D., Co-Founder and Chief
Scientific Officer of Edgewise. “Having seen promising preclinical
results with EDG-5506 translate clinically, I am thrilled we are
entering into a pivotal study.”
Edgewise will host a community webinar with Parent Project
Muscular Dystrophy on October 18, 2023, at 1:00 pm ET to share
details about the trial and answer questions from the community.
The registration page will be available on the PPMD website in
early October.
Positive 12-Month Results from the ARCH Open Label Study of
EDG-5506 in Adults with Becker
The ongoing ARCH study is an open label, single-center study
assessing the safety, tolerability, impact on muscle damage
biomarkers, function and pharmacokinetics (PK) of EDG-5506 in
adults with Becker. The ARCH study is evaluating varying doses of
EDG-5506 administered daily over 24 months in 12 adults with
Becker. The Company reported data at the end of 12 months of
treatment with EDG-5506. EDG-5506 was well-tolerated in all
participants with no dose reductions or discontinuations due to
adverse events.
Consistent with prior observations, significant decreases in key
biomarkers of muscle damage were seen with treatment with EDG-5506.
Importantly, creatine kinase (CK) and fast skeletal muscle troponin
I were reduced by an average of 37% (p=0.001) and 79% (p<0.0001)
from baseline, respectively, at the participants’ 12-month visit.
After 12 months of EDG-5506 dosing, North Star Ambulatory
Assessment (NSAA) scores continued to trend in a positive
direction. Nine of the twelve participants showed either a
functional improvement (n=6) or exhibited stability (n=3) on NSAA
scores relative to their baselines. NSAA scores showed a consistent
positive trend that diverges from trajectories observed in the
natural history studies reported by Bello et al. (2016)1 and van de
Velde et al. (2021)2 in which the yearly decline was -1.2 NSAA
points. Overall, after one-year, there was a +0.4-point trend
toward improvement on the NSAA compared to the -1.2-point decline
observed in natural history studies in Becker patients. 1, 2
The encouraging results from the 12-month ARCH study support the
hypothesis that a reduction in contraction-induced muscle damage in
muscular dystrophies has the potential to preserve and improve
muscle function while preventing disease progression in
dystrophinopathies. Observations from ARCH identified key factors,
including the dose of EDG-5506, for the design of a potentially
registrational trial. Go to clinicaltrials.gov to learn more about
this study (NCT05160415).
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 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 genetic neuromuscular
disorders. It is anticipated to be used as a single agent therapy,
but it may also provide an additional benefit in combination with
available therapies and therapies currently 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 completed a Phase 1 clinical trial of EDG-5506
designed to evaluate 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. The Phase 2 trial of EDG-5506 in Becker
(CANYON) has been expanded to include an additional 120 adult
participants in a pivotal cohort called GRAND CANYON. CANYON is
fully enrolled; GRAND CANYON is currently enrolling. LYNX, an
ongoing Phase 2 trial (NCT05540860), is assessing safety, PK and
biomarkers of muscle damage in participants with Duchenne. The
Company is also continuing to recruit 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 advanced 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.
References
[1] Bello L, et al., Functional Changes in Becker Muscular
Dystrophy: Implications for Clinical Trials in Dystrophinopathies,
Scientific Reports, 2016.
[2] van de Velde et al., Selection Approach to Identify the
Optimal Biomarker Using Quantitative Muscle MRI and Functional
Assessments in Becker Muscular Dystrophy, Neurology, 2021.
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, including
the GRAND CANYON study and enrollment of such study. 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
preclinical studies and clinical trials for, obtain approvals for
and commercialize any of its product candidates; the timing,
progress and results of preclinical studies and 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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Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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