– Sustained positive trend in North Star
Ambulatory Assessment (NSAA) scores relative to BMD natural history
–
– Significant decreases in levels of serum
creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2),
enzyme biomarkers associated with skeletal muscle damage–
– EDG-5506 was well-tolerated at all doses
–
– Company continuing to enroll patients with
BMD in the CANYON Phase 2 trial which now includes a pivotal cohort
called GRAND CANYON –
– Edgewise leadership to discuss ARCH findings
on Tuesday, June 27 at 8:30 a.m. Eastern Time at virtual investor
event –
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a clinical-stage
biopharmaceutical company focused on developing orally
bioavailable, targeted, small molecule therapies for the treatment
of devastating muscle disorders, announced today positive 12-month
topline results from the ongoing ARCH study, an open label,
single-center study assessing the safety, tolerability, impact on
muscle damage biomarkers, and pharmacokinetics (PK) of EDG-5506 in
adults with BMD. EDG-5506 is an orally administered small molecule
designed to prevent contraction-induced muscle damage in
dystrophinopathies including BMD and Duchenne muscular dystrophy
(DMD).
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Edgewise Therapeutics Figure 1. NSAA
shows stabilization and trend toward improvement after 12 months of
dosing with EDG-5506 (Graphic: Business Wire)
The ARCH study is evaluating varying doses of EDG-5506
administered daily over 24 months in 12 adults with BMD. The
Company is reporting data at the end of 12 months of treatment with
EDG-5506. EDG-5506 was well-tolerated in all participants with no
discontinuations or dose reductions due to adverse events.
Consistent with prior observations, treatment with EDG-5506 led
to significant decreases in key biomarkers of muscle damage.
Importantly, CK and TNNI2 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, NSAA scores continued to
trend in a positive direction. Nine of the twelve participants
showed either a functional improvement (n=6) or exhibited stable
disease (n=3) on NSAA relative to their baselines. As seen in
Figure 1, NSAA scores show 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, one-year
functional results were observed to have a +0.4-point improvement
on the NSAA compared to the -1.2-point anticipated natural history
decline in a population of BMD patients predicted to have a
relentless course of disease progression.
The positive results from the 12-month ARCH study support the
hypothesis that a reduction in contraction-induced muscle damage in
muscular dystrophies, associated with EDG-5506 administration, has
the potential to preserve and improve muscle function while
preventing disease progression in dystrophinopathies. Observations
from ARCH identified key factors, including the optimal dosing
strategy of EDG-5506, for the design of a potentially
registrational trial. A pivotal cohort, GRAND CANYON, has been
added to the CANYON study and is anticipated to begin enrollment in
the third quarter of 2023.
“We are quite pleased by the robust and consistent results
observed at 12 months with EDG 5506 treatment, which, together with
the favorable safety/tolerability profile, are highly encouraging.
Moreover, these data have allowed us to quickly pivot our CANYON
study to include a potentially registration-enabling cohort,” said
Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise. “We
thank the patients, their families, caregivers, and the broader
Becker community, who are currently underserved with no available
treatment options, for engaging with us on this promising
therapy.”
“Becker is a devastating, neuromuscular disease which currently
has no approved therapies. In my experience, once symptoms of
muscle weakness occur, muscle has already been lost. Once
declining, individuals with Becker have a relentless course of
disease progression,” said Craig McDonald, M.D., Professor and
Chair, Department of Physical Medicine & Rehabilitation,
University of California Davis. “I have been closely following the
progress of EDG-5506 in the clinic and am excited by the 12-month
results and EDG-5506’s potential as a novel therapy for individuals
with Becker.”
ARCH Data Review Virtual Investor Event
Members of the Edgewise management team will hold a live webcast
on Tuesday, June 27, at 8:30 a.m. ET to discuss the ARCH 12-month
data, and will be joined by Dr. Craig McDonald, who will share his
perspective of EDG-5506 and BMD. An accompanying slide presentation
will also be available. To register for the live webcast and
replay, please visit the Edgewise events page.
Further, Edgewise plans to discuss these data at the Parent
Project Muscular Dystrophy Annual Conference, June 29 – July 1,
2023.
Edgewise Therapeutics Presentations at Parent Project
Muscular Dystrophy Conference
Edgewise Therapeutics’ Chief Medical Officer, Joanne Donovan,
M.D., Ph.D., will present at the Parent Project Muscular Dystrophy
Annual Conference on July 29, during the “Emerging Therapies in
Becker” session at 5:00 pm CT / 6:00 pm ET and on June 30, during
the “Research Row: Impacting Downstream” session at 10:45 am CT /
11:45 am ET. Only registered conference attendees can attend the
presentations. The Impacting Downstream session will be streamed to
the virtual element of the event and both will be available for
replay via the conference platform. The Edgewise presentations will
be available on the Edgewise website when they are presented.
About the ARCH Open-Label Study
The ARCH open-label study is evaluating EDG-5506 in 12 adult
males with BMD. The study is evaluating varying doses of EDG-5506
administered daily over 24 months. Safety, PK, changes in
biomarkers of muscle damage such as CK and fast skeletal muscle
troponin I, measures of function with NSAA and NSAD, time function
tests and patient-reported outcomes are being evaluated. Go to
clinicaltrials.gov to learn more about this study
(NCT05160415).
About the CANYON Clinical Trial
The CANYON clinical trial is evaluating EDG-5506 in individuals
with BMD. CANYON is assessing the effect of EDG-5506 over a
12-month period on safety, PK, biomarkers of muscle damage, such as
CK and fast skeletal muscle troponin I, fat fraction as measured by
muscle MRI and function in individuals with BMD aged 12 to 50
years. This placebo-controlled trial is anticipated to recruit
approximately 32 adults and 18 adolescents at up to 14 sites in the
United States, United Kingdom and the Netherlands. The GRAND CANYON
cohort has been designed as a potentially registrational cohort
with NSAA as the primary endpoint. GRAND CANYON will be a global
placebo-controlled study and is anticipated to recruit
approximately 120 BMD patients who will receive study drug for 18
months. Go to clinicaltrials.gov to learn more about this trial
(NCT05291091).
About Becker Muscular Dystrophy
BMD is a genetic, progressively debilitating, and potentially
fatal inherited X-linked neuromuscular disorder. BMD results from
mutation of the dystrophin gene yielding unstable and/or
dysfunctional dystrophin expression in muscles. Individuals with
BMD, typically males, have ongoing muscle fiber (myofiber)
degeneration that eventually leads to fibrosis, progressive loss of
skeletal muscle function, and that can lead to severe disability
and early death. BMD typically presents with juvenile onset of
muscle wasting and progressive symmetrical, proximal muscle
weakness, calf hypertrophy, activity-induced muscle cramping and
elevated CK activity. While the course of BMD is variable, it is
unidirectional in terms of the inevitable progressive limb weakness
resulting in severe disability. BMD is also associated with early
mortality from cardiac disease. The incidence of BMD is
approximately 1 in every 18,450 live male births. It is estimated
that there are approximately 5,000 individuals with BMD in the
U.S., with similar numbers of individuals living with BMD in the EU
and UK. Despite the seriousness of the disease, for many with BMD,
the disease remains one of considerable unmet medical need as there
are no approved therapies.
About EDG-5506
EDG-5506 is an orally administered small molecule designed to
prevent contraction-induced muscle damage in dystrophinopathies
including DMD and BMD. 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 rare 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
BMD.
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 BMD (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. CANYON, an ongoing Phase 2 trial
(NCT05291091), is assessing safety, PK, biomarkers of muscle
damage, fat fraction as measured by muscle MRI and function in
participants with BMD aged 12 to 50 years. CANYON now also includes
the GRAND CANYON cohort, designed as a potentially registrational
cohort with NSAA as the primary endpoint. LYNX, an ongoing Phase 2
trial (NCT05540860), is assessing safety, PK and biomarkers of
muscle damage in participants with DMD. 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 Limb Girdle muscular dystrophy 2i
(LGMD2i), BMD or McArdle disease at a single site in Denmark.
About Edgewise Therapeutics
Edgewise Therapeutics is a clinical-stage biopharmaceutical
company focused on the discovery, development, and
commercialization of innovative treatments for severe, rare
neuromuscular and cardiac disorders for which there is significant
unmet medical need. Guided by its holistic drug discovery approach
to targeting the muscle as an organ, Edgewise has combined its
foundational expertise in muscle biology and small molecule
engineering to build its proprietary, muscle-focused drug discovery
platform. Edgewise’s platform utilizes custom-built high throughput
and translatable systems that measure integrated muscle function in
whole organ extracts to identify small molecule precision medicines
regulating key proteins in muscle tissue. The Company’s lead
candidate, EDG-5506, an investigational orally administered small
molecule designed to protect injury-susceptible fast skeletal
muscle fibers in dystrophinopathies, is advancing in multiple
clinical trials in individuals with Duchenne, Becker and Limb
Girdle 2I/R9 muscular dystrophies, and McArdle disease. The Company
is also advancing EDG-7500, a novel sarcomere modulator for
hypertrophic cardiomyopathy, through IND-enabling preclinical
development. To learn more, go to: www.edgewisetx.com or follow us
on LinkedIn, Twitter and Facebook.
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 Edgewise’s expectations relating to its
clinical trials and clinical development of EDG-5506; statements
regarding a potentially registrational cohort in the CANYON study
and its ability to recruit patients for such study; statements
regarding Edgewise’s pipeline of product candidates and programs;
and statements by Edgewise’s president and chief medical officer
and the Chair, Department of Physical Medicine &
Rehabilitation, University of California Davis. 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 for muscular
dystrophy patients or other patient populations; the timing,
progress and results of clinical trials for EDG-5506; 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20230626317214/en/
Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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