– Announced positive 12-month topline results
from the ARCH open label study of EDG-5506 in adults with Becker
muscular dystrophy (Becker) –
– Advancing Phase 2 trial of EDG-5506 in Becker
muscular dystrophy (Becker, CANYON), which now includes a pivotal
cohort called GRAND CANYON –
– Advancing Phase 2 trial of EDG-5506 in
Duchenne muscular dystrophy (Duchenne, LYNX) –
– Advancing EDG-7500, a novel sarcomere
modulator for hypertrophic cardiomyopathy (HCM); Phase 1 start
expected in 2H2023 –
– Cash, cash equivalents and marketable
securities of $310 million as of June 30, 2023 –
Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a clinical-stage
biopharmaceutical company focused on the discovery, development,
and commercialization of innovative treatments for severe, genetic
neuromuscular and cardiac disorders for which there is significant
unmet medical need, today reported financial results for the second
quarter of 2023 and recent business highlights.
“Edgewise has made significant progress in the first half of the
year most notably with the positive ARCH data that have allowed us
to quickly pivot our CANYON study to include a potentially
registration-enabling cohort in individuals with Becker, a disorder
for which there are no approved treatments,” said Kevin Koch,
Ph.D., President and Chief Executive Officer of Edgewise. “The team
continues to advance our LYNX, CANYON and DUNE clinical trials of
EDG-5506 in addition to advancing EDG-7500, our novel sarcomere
modulator for HCM, towards the clinic. Over the coming months, we
expect to start enrolling GRAND CANYON and a Phase 1 trial of
EDG-7500 and share interim Duchenne data from our Phase 2 LYNX
trial.”
Recent Highlights
Announced Positive 12-Month Topline
Results from the ARCH Open Label Study of EDG-5506 in Adults with
Becker
The Company announced 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,
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, treatment with EDG-5506 led
to significant decreases in key biomarkers of muscle damage.
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, 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 Becker patients.
The positive 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).
Announced GRAND CANYON Pivotal Cohort of
EDG-5506 in Individuals with Becker
The Company announced plans to expand the placebo-controlled
Phase 2 CANYON trial of EDG-5506 to include a new global pivotal
cohort, called GRAND CANYON, that is a potentially registrational
cohort with NSAA as the primary endpoint. GRAND CANYON is
anticipated to recruit approximately 120 Becker patients who will
receive study drug for 18 months. GRAND CANYON is anticipated to
begin enrollment in the second half of 2023. Go to
clinicaltrials.gov to learn more (NCT05291091).
Advancing LYNX Phase 2 Clinical Trial of
EDG-5506 in Children with Duchenne
The Company is recruiting the LYNX Phase 2 clinical trial of
EDG-5506 in children with Duchenne. LYNX is a placebo-controlled
trial to assess the effect of multiple doses of EDG-5506 over 12
weeks on safety, PK and biomarkers of muscle damage. Approximately
27 children with Duchenne 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 optimal dose of EDG-5506 that reduces 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 fourth quarter of 2023. Go to
clinicaltrials.gov to learn more about this trial
(NCT05540860).
Advancing DUNE Phase 2 Trial of EDG-5506
in Adults with LGMD2I/R9, Becker 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,
Becker or McArdle disease at a single site in Denmark. Participants
in the placebo-controlled study are enrolled for 16 weeks, then
continue to an open label extension through 52 weeks. The goal of
this trial is to assess safety and efficacy in individuals with
myopathy distinct from Duchenne/Becker where muscle contraction is
associated with exaggerated injury. LGMD2I/R9 is a muscular
dystrophy 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.
Advancing 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, with plans to initiate a Phase 1 trial in the second
half of 2023, subject to obtaining IND clearance. 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
beneficial activity in both obstructive HCM and non-obstructive HCM
with minimal changes in left ventricle contractility.
Strengthened Engagement with Muscular
Dystrophy Patient Communities
Leadership presented at the Parent Project Muscular Dystrophy
(PPMD) Annual Conference in June 2023, during the “Emerging
Therapies in Becker” session and the “Research Row” session. The
Company’s presentations are available on the Edgewise website.
Additionally, leadership discussed and answered questions about the
Company’s clinical trials in Becker during a patient community
webinar hosted by PPMD in May 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.
Second Quarter Financial Results
Cash, cash equivalents and marketable securities were
approximately $310 million as of June 30, 2023.
Research and development (R&D) expenses were $19.6
million for the second quarter of 2023, compared to $19.9 million
for the immediately preceding quarter. The decrease of $0.3 million
was primarily driven by a decrease of $1.5 million in preclinical
development expense based on higher costs incurred in the first
quarter related to IND-enabling studies and API manufactured for
EDG-7500. This decrease was partially offset by an increase of $0.8
million in personnel-related costs and facilities and other costs
that support the growth of the company’s R&D programs, and $0.3
million of higher expenses related to the EDG-5506 clinical program
such as clinical site and contract research organization costs to
support ongoing Phase 2 trials.
General and Administrative (G&A) expenses were $5.8
million for the second quarter of 2023, compared to $5.8 million
for the immediately preceding quarter.
Net loss and net loss per share for the second quarter of
2023 was $21.5 million or $0.34 per share, compared to $22.9
million or $0.36 per share for the immediately preceding
quarter.
About EDG-5506
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. CANYON, an ongoing Phase 2 trial
(NCT05291091), is assessing safety, PK, biomarkers of muscle
damage, function and fat fraction as measured by muscle MRI in
participants with Becker 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 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 EDG-7500
EDG-7500 is a first-in-class oral, selective, sarcomere
modulator for HCM, and the Company 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 beneficial 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, genetic
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, with plans to initiate a Phase 1 trial
in the second half of 2023. 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 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 Phase 2 interim data for the LYNX
Phase 2 clinical trial of EDG-5506) and commencement of trials
(including patient enrollment for GRAND CANYON and a Phase 1 trial
of 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.
This press release contains hyperlinks to information that is
not deemed to be incorporated by reference into this press
release.
Edgewise Therapeutics, Inc. Condensed Statement of
Operations (in thousands except share and per share amounts,
unaudited)
Three months ended
June 30, 2023
March 31, 2023
Operating expenses: Research and development
$
19,559
$
19,876
General and administrative
5,780
5,828
Total operating expenses
25,339
25,704
Loss from operations
(25,339
)
(25,704
)
Interest income
3,870
2,866
Net loss
$
(21,469
)
$
(22,838
)
Net loss per share - basic and diluted
$
(0.34
)
$
(0.36
)
Weighted-average shares outstanding, basic and diluted
63,380,430
63,265,800
Edgewise Therapeutics, Inc. Condensed
Balance Sheet Data (in thousands, unaudited)
June 30,
December 31,
2023
2022
Assets Cash, cash equivalents and marketable securities
$
309,928
$
351,947
Other assets
20,697
15,154
Total assets
$
330,625
$
367,101
Liabilities and stockholders' equity
Liabilities
19,170
20,385
Stockholders' equity
311,455
346,716
Total liabilities and stockholders' equity
$
330,625
$
367,101
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230810644738/en/
Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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