– Fully enrolled CANYON and now enrolling the
global pivotal cohort, GRAND CANYON, of EDG-5506 in Becker muscular
dystrophy (Becker) – – Expanding Phase 2 LYNX trial of EDG-5506
including new cohort for boys with Duchenne not currently treated
with corticosteroids – – Initiated new Phase 2 FOX trial in boys
with Duchenne muscular dystrophy (Duchenne) who have been
previously treated with gene therapy – – Advancing Phase 1 trial of
EDG-7500, a novel sarcomere modulator for hypertrophic
cardiomyopathy (HCM), and other serious diseases of cardiac
diastolic dysfunction –
Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle
disease biopharmaceutical company, today reported financial results
for the third quarter of 2023 and recent business highlights.
“I’m thrilled with the progress we’ve made in 2023 across our
skeletal and cardiovascular programs,” said Kevin Koch, Ph.D.,
President and Chief Executive Officer of Edgewise. “Most recently,
we started enrolling individuals in two important studies: GRAND
CANYON, a global pivotal study of EDG-5506 in Becker, a disorder
with no approved treatments and a Phase 1 trial of EDG-7500, our
novel sarcomere modulator for HCM. The team also initiated FOX, the
Phase 2 trial of EDG-5506 in boys with Duchenne who have received
gene therapy and continues to rapidly enroll the Phase 2 LYNX trial
in Duchenne.”
Recent Highlights
Musculoskeletal Program /
EDG-5506
Initiated GRAND CANYON,
a Global Pivotal Study in Becker
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. The Company
initiated enrollment of GRAND CANYON, a global pivotal study of
EDG-5506 in individuals with Becker. GRAND CANYON is an expansion
of the CANYON placebo-controlled study. CANYON, which was
over-enrolled, includes 40 adults and 29 adolescents and a
treatment period of 12 months.
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.
Rapidly enrolling and
continuing dose escalation of Phase 2 LYNX trial
Based on the safety profile observed to date,
the Company is continuing dose escalation and expanding enrollment
in their Phase 2 placebo-controlled LYNX trial of EDG-5506 in
children aged 4 to 9 years with Duchenne; one of the new LYNX
cohorts will include boys with Duchenne not currently treated with
corticosteroids. The LYNX trial is enrolling at 14 sites across the
United States, with the first three cohorts having over-enrolled.
LYNX is a Phase 2 placebo-controlled trial to assess the effect of
multiple doses of EDG-5506 over 12 weeks on safety, PK and
biomarkers of muscle damage. The trial will also explore changes in
functional measures, such as the North Star Ambulatory Assessment
(NSAA) and self-reported/caregiver-reported outcomes. Approximately
45 children with Duchenne aged 4 to 9 years are expected to be
enrolled in this trial. Participants will then continue in an
open-label extension portion of the trial for a total of 24 months
to gain further insights into safety and functional measures.
Importantly, this trial is designed to identify a dose of EDG-5506
that will reduce 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 first half of
2024, once the Phase 3 dose is identified. Go to clinicaltrials.gov
to learn more about this trial (NCT05540860).
Initiated new Phase 2
FOX trial in boys with Duchenne who have been previously treated
with gene therapy
FOX is a Phase 2 placebo-controlled trial to
assess the effect of EDG-5506 over 12 weeks on safety, PK and
biomarkers of muscle damage in children and adolescents with
Duchenne who have been previously treated with gene therapy.
Approximately 24 participants, aged 6 to 14 years, are expected to
be enrolled in the trial at multiple sites across the United
States, which is expected to begin dosing in early 2024.
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, PK, function and biomarker measures. Go to
clinicaltrials.gov to learn more about this trial
(NCT06100887).
Advancing Phase 2 DUNE
Trial in Adults with LGMD2I/R9, Becker and McArdle
Disease
The Company is advancing the DUNE Phase 2
exercise challenge trial to evaluate the effect of EDG-5506 on
biomarkers of muscle damage following controlled exercise in adults
with LGMD2I/R9, Becker or McArdle disease at a single site in
Denmark. Participants in the placebo-controlled 16-week trial 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. The Company
expects to announce placebo-controlled data in the first half of
2024.
Initiated Phase 2
Open-Label Extension MESA Trial in Adults and Adolescents with
Becker
The Company initiated MESA, an open-label
treatment extension trial to assess the long-term effect of
EDG-5506 on safety, biomarkers, and functional measures. MESA will
provide continued access to EDG-5506 treatment to study
participants who were previously enrolled in Edgewise studies. Go
to clinicaltrials.gov to learn more about this trial
(NCT06066580).
Cardiovascular Program /
EDG-7500
Advancing Phase 1 Trial
of EDG-7500, a First-In-Class Sarcomere Modulator
EDG-7500 is a first-in-class oral, selective,
cardiac sarcomere modulator, specifically designed to slow early
contraction velocity and address impaired cardiac relaxation
associated with HCM and other diseases of diastolic dysfunction.
The Company is enrolling a randomized, double-blind,
placebo-controlled, single and multiple ascending dose Phase 1
trial evaluating safety, tolerability, pharmacokinetics and
pharmacodynamics in healthy adults. To learn more about this trial
(NCT06011317), go to clinicaltrials.gov. The Company is also
planning to begin a Phase 1b trial of EDG-7500 in individuals with
obstructive HCM in the first half of 2024.
Strengthened Engagement with the
Scientific and Patient Communities
Musculoskeletal Program /
EDG-5506
The Company continued its education and outreach on its Becker
program with the medical and patient communities. In October 2023,
the Company held an Industry Symposium at the Annual Congress of
the World Muscle Society (WMS). Additionally, the Company held
webinars with clinicians hosted by Muscular Dystrophy Association
in August 2023 and the patient community hosted by Parent Project
Muscular Dystrophy in October 2023. Replays of the webinars can be
viewed here. The Company continues to sponsor and participate in
numerous other clinician and patient-focused events.
Cardiovascular Program /
EDG-7500
The Company sponsored and attended several
cardiovascular-focused conferences, building relationships with the
medical and patient communities. In October 2023, the Company
shared information on its Phase 1 trial of EDG-7500, at the Heart
Failure Society of America Annual Scientific Meeting, participated
in a panel discussion on emerging pharmacologic therapies for HCM
at the HCM Society Annual Scientific Sessions and attended the HCM
Association Annual Patient Meeting. The Company is also presenting
on EDG-7500 at the American Heart Association Scientific Sessions
on November 13.
The presentations are available on the Edgewise website.
Third Quarter Financial Results
Cash, cash equivalents and marketable securities were
approximately $290 million as of September 30, 2023.
Research and development (R&D) expenses were $23.8
million for the third quarter of 2023, compared to $19.6 million
for the immediately preceding quarter. The increase of $4.2 million
was primarily driven by an increase of $3.3 million in clinical
trial expenses for both the EDG-5506 and EDG-7500 clinical programs
in addition to $0.6 million of higher personnel-related costs and
$0.3 million of other R&D expenses.
General and Administrative (G&A) expenses were $5.7
million for the third quarter of 2023, compared to $5.8 million for
the immediately preceding quarter.
Net loss and net loss per share for the third quarter of
2023 was $25.7 million or $0.41 per share, compared to $21.5
million or $0.34 per share for the immediately preceding
quarter.
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 neuromuscular disorders Its
unique mechanism of action provides the potential to establish
EDG-5506 as a foundational therapy in dystrophinopathies, either as
a single agent therapy or in combination with available therapies
and those 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 advanced EDG-5506 through the clinic including
completing a Phase 1 trial evaluating 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. A Phase 2 cohort of EDG-5506 in Becker
(CANYON) is fully enrolled and has now been expanded to include an
additional 120 adult participants in a pivotal cohort called GRAND
CANYON, which is currently enrolling (NCT05291091). In Duchenne,
the Company is advancing its Phase 2 studies, LYNX (NCT05540860)
and FOX (NCT06100887), both assessing safety, PK and biomarkers of
muscle damage. The Company is also advancing the DUNE Phase 2
exercise challenge trial, 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 for Hypertrophic Cardiomyopathy
EDG-7500 is a first-in-class oral, selective, cardiac sarcomere
modulator that is advancing in a Phase 1 trial (NCT06011317). The
compound is designed to slow early contraction velocity and improve
impaired cardiac relaxation. This novel mechanism is anticipated to
have a broad therapeutic index that may facilitate fixed patient
dosing and minimize monitoring during therapy. Preclinical data of
EDG-7500 support beneficial activity in animal models of both
obstructive HCM and cardiac diastolic dysfunction with minimal
changes in left ventricular systolic performance.
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 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.
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 product candidates and
programs, including EDG-5506 and EDG-7500; statements regarding
Edgewise’s expectations relating to its clinical trials, including
timing of reporting data (including the Phase 2 interim data for
the LYNX Phase 2 clinical trial of EDG-5506 and Phase 2 DUNE Trial
of EDG-5506) and commencement of trials (including patient
enrollment for GRAND CANYON, certain milestones of the Phase 2 FOX
trial in boys with Duchenne and a Phase 1b trial of EDG-7500); and
statements regarding Edgewise’s pipeline of product candidates and
programs. 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; the timing,
progress and results of clinical trials for EDG-5506 and the
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; 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
September 30, 2023
June 30, 2023
Operating expenses: Research and development
$
23,786
$
19,559
General and administrative
5,666
5,780
Total operating expenses
29,452
25,339
Loss from operations
(29,452
)
(25,339
)
Interest income
3,739
3,870
Net loss
$
(25,713
)
$
(21,469
)
Net loss per share - basic and diluted
$
(0.41
)
$
(0.34
)
Weighted-average shares outstanding, basic and diluted
63,459,560
63,380,430
Edgewise Therapeutics, Inc. Condensed
Balance Sheet Data (in thousands, unaudited)
September 30,
December 31,
2023
2022
Assets Cash, cash equivalents and marketable securities
$
289,697
$
351,947
Other assets
21,667
15,154
Total assets
$
311,364
$
367,101
Liabilities and stockholders' equity
Liabilities
20,937
20,385
Stockholders' equity
290,427
346,716
Total liabilities and stockholders' equity
$
311,364
$
367,101
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231109421952/en/
Investors & Media Michael Carruthers Chief Financial
Officer ir@edgewisetx.com
Edgewise Therapeutics (NASDAQ:EWTX)
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