– Advancing Phase 2 trials of EDG-5506 in
Becker muscular dystrophy (BMD, CANYON) and Duchenne muscular
dystrophy (DMD, LYNX) –
– Advancing exercise challenge study of
EDG-5506 in Limb girdle muscular dystrophy 2I, BMD and McArdle
Disease (LGMD2I/R9, BMD, McArdle, DUNE) –
– Advancing IND-enabling studies of EDG-7500, a
novel sarcomere modulator for hypertrophic cardiomyopathy (HCM);
Phase 1 start expected in 2H2023 –
– Cash, cash equivalents and marketable
securities of $328 million as of March 31, 2023 –
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, today reported financial results
for the first quarter of 2023 and recent business highlights.
“We’re off to a great start in 2023! The team has focused on
advancing our LYNX and CANYON clinical trials of EDG-5506 in
addition to completing IND-enabling studies of EDG-7500, our novel
sarcomere modulator for HCM,” said Kevin Koch, Ph.D., President and
Chief Executive Officer of Edgewise. “During the remainder of 2023,
we expect to share 12-month ARCH open label data and interim
Duchenne data from our Phase 2 LYNX trial. We also plan to initiate
a potentially registration-enabling cohort in CANYON and a Phase 1
trial for EDG-7500 in healthy volunteers.”
Recent Highlights
Advancing CANYON Clinical Trial of
EDG-5506 in Individuals with BMD
The Company is continuing to recruit the CANYON clinical trial
evaluating EDG-5506 in individuals with BMD. CANYON is assessing
the effect of EDG-5506 over a 12-month period on safety,
pharmacokinetics (PK), biomarkers of muscle damage, such as
creatine kinase (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 Company plans to amend the CANYON study to include
a potentially registration-enabling cohort in the second half of
2023. Go to clinicaltrials.gov to learn more about this trial
(NCT05291091).
Advancing LYNX Phase 2 Clinical Trial of
EDG-5506 in Children with DMD
The Company is recruiting the LYNX Phase 2 clinical trial of
EDG-5506 in children with DMD. LYNX is a placebo-controlled trial
to assess the effect of three doses of EDG-5506 over 12 weeks on
safety, PK and biomarkers of muscle damage. Approximately 27
children with DMD aged 4 to 9 years on stable corticosteroids
and/or exon skippers are expected to be enrolled at up to 14 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 and functional measures.
Importantly, this trial is designed to identify the doses of
EDG-5506 that have the potential to reduce biomarkers of muscle
damage and provide functional benefit to patients in a Phase 3
trial. The Company expects to report Phase 2 interim data in the
fourth quarter of 2023. Go to clinicaltrials.gov to learn more
about this trial (NCT05540860).
Advancing ARCH Open Label Study of
EDG-5506 in Adults with BMD
The Company is continuing to advance the ARCH open-label study
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. The Company expects to report 12-month results
in the second quarter of 2023. Go to clinicaltrials.gov to learn
more about this study (NCT05160415).
Advancing DUNE Phase 2 Trial of EDG-5506
in Adults with LGMD2I/R9, BMD and McArdle Disease
The Company is 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/R9, BMD
or McArdle disease at a single site in Denmark. The
placebo-controlled study is expected to enroll 36 participants for
16 weeks, then continue to an open label extension through 52
weeks. The goal of these studies is to assess safety and efficacy
in individuals with myopathy distinct from DMD/BMD where muscle
contraction is associated with exaggerated injury. LGMD2I/R9 is a
myopathy caused by a dysfunctional dystroglycan complex while
McArdle is caused by deficiencies in glycogen mobilization leading
to metabolic crisis and injury of skeletal muscle. In addition to
biomarkers of muscle damage, secondary measures will include
measures of strength and exercise capacity.
Journal of Clinical Investigation
Published Key Preclinical Data Linking Modulation of Fast Skeletal
Muscle Contraction to Protection of Skeletal Muscle in Models of
DMD
In March 2023, the Journal of Clinical Investigation published
the article, “Modulating fast skeletal muscle contraction protects
skeletal muscle in animal models of Duchenne muscular dystrophy.”
This article provides the preclinical rationale and
proof-of-concept behind EDG-5506, demonstrating that modulation of
fast skeletal muscle contraction protects against muscle injury,
degeneration and fibrosis in models of DMD. Importantly, the study
found that modest inhibition of fast skeletal muscle myosin
provides maximum and robust protection of skeletal muscles and was
associated with increases in strength and physical activity in
mouse and dog models of DMD. To view article, click here.
Advancing IND-enabling studies of
EDG-7500, a First-In-Class Sarcomere Modulator for HCM
The Company is continuing to advance EDG-7500, a first-in-class
oral, selective, sarcomere modulator for diseases of diastolic
dysfunction, through IND-enabling studies with plans to initiate a
Phase 1 trial in the second half of 2023. EDG-7500 is a result of
Edgewise’s robust discovery platform that is yielding novel
compounds targeting important unmet needs of patients suffering
from disorders of cardiac and skeletal muscle. The compound is
designed to improve impaired cardiac relaxation and slow
contraction velocity, hallmarks of HCM. This novel mechanism is
anticipated to have a broader therapeutic index relative to cardiac
myosin inhibition for treatment of both obstructive and
non-obstructive HCM. Preclinical data of EDG-7500 support activity
in both obstructive HCM and non-obstructive HCM with minimal
changes in left ventricle contractility.
Strengthened Engagement with Muscular
Dystrophy Patient and Medical Communities
The Company hosted an industry forum at the Muscular Dystrophy
Association Annual Clinical and Scientific Conference in March
2023. A replay of the forum can be viewed here. Further, leadership
discussed and answered questions about the Company’s clinical
trials in DMD and BMD during a patient community webinar hosted by
CureDuchenne in March 2023. A replay of the webinar can be viewed
here. The Company continues to sponsor and participate in numerous
patient-focused events hosted by patient advocacy
organizations.
First Quarter Financial Results
Cash, cash equivalents and marketable securities were
$328.0 million as of March 31, 2023.
Research and development (R&D) expenses were $19.9
million for the first quarter of 2023, compared to $16.6 million
for the immediately preceding quarter. The increase of $3.3 million
was primarily driven by an increase of $1.6 million related to
preclinical development of EDG-7500 and the research efforts of our
cardiovascular discovery program, $0.9 million of higher expenses
related to our EDG-5506 clinical program such as clinical site and
CRO costs to support ongoing Phase 2 trials, an increase of $0.5
million in employee-related costs and an increase of $0.3 million
in facilities and other costs that support the growth of our
research and development programs.
General and Administrative (G&A) expenses were $5.8
million for the first quarter of 2023, compared to $5.5 million for
the immediately preceding quarter. The increase of $0.3 million was
primarily driven by increased professional and consulting and other
administrative costs.
Net loss and net loss per share for the first quarter of
2023 was $22.8 million or $0.36 per share, compared to $19.4
million or $0.31 per share for the immediately preceding
quarter.
About EDG-5506
EDG-5506 is an orally administered small molecule designed to
prevent muscle damage induced by mechanical stress 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 6 months of
treatment with EDG-5506. CANYON, an ongoing Phase 2 trial
(NCT05291091), is assessing safety, PK, biomarkers and functional
measures in participants with BMD. LYNX, an ongoing Phase 2 trial
(NCT05540860), is assessing safety, PK and biomarkers of muscle
damage in participants with DMD.
About EDG-7500
EDG-7500 is a first-in-class oral, selective, sarcomere
modulator for HCM, advancing through IND-enabling studies with
plans to initiate a Phase 1 trial in the second half of 2023. The
compound is designed to improve impaired cardiac relaxation and
slow contraction velocity, hallmarks of HCM. This novel mechanism
is anticipated to have a broader therapeutic index relative to
cardiac myosin inhibition for treatment of both obstructive and
non-obstructive HCM. Preclinical data of EDG-7500 support activity
in both obstructive HCM and non-obstructive HCM with minimal
changes in left ventricle contractility.
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, into IND-enabling preclinical
development. To learn more, go to: www.edgewisetx.com or follow us
on LinkedIn, Twitter and Facebook.
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 drug discovery platform,
product candidates and programs, including EDG-5506 and EDG-7500;
statements regarding Edgewise’s expectations relating to its
preclinical studies and clinical trials, including timing of
reporting data (including the 12-month ARCH open label data and
interim Duchenne data from our Phase 2 LYNX trial) and commencing
studies and trials; statements regarding Edgewise’s plans to amend
the CANYON study, including expected timing; statements about the
expected timing of Edgewise’s initiation of a Phase 1 clinical
trial for EDG-7500; statements regarding Edgewise’s pipeline of
product candidates and programs; and statements by Edgewise’s
president and chief executive 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
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 preclinical studies and clinical trials for
EDG-5506 and the EDG-7500; Edgewise’s ability to obtain IND
clearance for EDG-7500; 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.
Edgewise Therapeutics,
Inc.
Condensed Statement of
Operations
(in thousands except share and
per share amounts, unaudited)
Three months ended
March 31, 2023
December 31, 2022
Operating expenses: Research and development
$
19,876
$
16,612
General and administrative
5,828
5,467
Total operating expenses
25,704
22,079
Loss from operations
(25,704
)
(22,079
)
Interest income
2,866
2,664
Net loss
$
(22,838
)
$
(19,415
)
Net loss per share - basic and diluted
$
(0.36
)
$
(0.31
)
Weighted-average shares outstanding, basic and diluted
63,265,800
63,231,580
Edgewise Therapeutics,
Inc.
Condensed Balance Sheet
Data
(in thousands,
unaudited)
March 31,
December 31,
2023
2022
Assets Cash, cash equivalents and marketable securities
$
327,952
$
351,947
Other assets
18,448
15,154
Total assets
$
346,400
$
367,101
Liabilities and stockholders' equity Liabilities
17,560
20,385
Stockholders' equity
328,840
346,716
Total liabilities and stockholders' equity
$
346,400
$
367,101
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230511005233/en/
Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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