Full Press Release Details
Edgewise Therapeutics Reports Fourth Quarter
and Full Year 2023 Financial Results and Recent Business Highlights
CANYON and advanced the global pivotal cohort, GRAND CANYON, of EDG-5506 in Becker muscular dystrophy (Becker) -
2 LYNX trial of EDG-5506 including new cohort for boys with Duchenne muscular dystrophy (Duchenne) not currently treated with corticosteroids
2 FOX trial in boys with Duchenne who have been previously treated with gene therapy -
1 trial of EDG-7500, a novel cardiac sarcomere modulator for hypertrophic cardiomyopathy (HCM), and other serious diseases of cardiac
diastolic dysfunction -
balance sheet with net proceeds of approximately $232M from January 2024 public offering; pro-forma cash balance exceeds $550M -
Boulder, Colo., (February 22, 2024)
- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial
results for the fourth quarter and full year of 2023 and recent business highlights.
"In 2023, we made tremendous progress advancing
our novel muscle-targeted therapeutics in the clinic," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
"Most recently, we completed a successful financing enabling us to fully execute on our near and long-term goals including potential
U.S. commercial launch of EDG-5506 in Becker, completion of a Phase 3 trial with EDG-5506 in Duchenne, completion of Phase 2 trials of
EDG-7500 in obstructive and non-obstructive HCM and the advancement of our ongoing research and development programs."
Strengthened Financial Position
Raised $240 million in gross proceeds in
an underwritten offering. In January 2024, Edgewise completed the underwritten offering of 21,818,182 shares of its common stock at a
price of $11.00 per share. The total net proceeds from the offering, after deducting underwriters' discounts and commissions and
estimated offering costs were $231.8 million.
Musculoskeletal Program / EDG-5506
BECKER MUSCULAR DYSTROPHY
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
GRAND CANYON, a global pivotal study in Becker:
The Company is advancing GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker. GRAND CANYON is an expansion
of the CANYON placebo-controlled trial. CANYON, which was over-enrolled, includes cohorts of 40 adults and 29 adolescents and a treatment
period of 12 months. The Company expects to report CANYON data in the fourth quarter of 2024. 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:
ARCH open-label trial in adults with Becker:
ARCH is an open-label, single-center trial assessing the safety, tolerability, impact on muscle damage biomarkers, function and pharmacokinetics
(PK) of EDG-5506 in adults with Becker. In June 2023, the Company announced positive 12-month ARCH data. The Company expects to report
24-month data from the open label ARCH trial in the second quarter of 2024.
Phase 2 DUNE trial in adults with Becker,
LGMD2I/R9 or 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 Becker, LGMD2I/R9 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 pharmacodynamic markers of muscle damage 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 second quarter of 2024.
Phase 2 open-label extension MESA trial in
adults and adolescents with Becker: The Company is advancing 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 trials. Go to clinicaltrials.gov to learn more about this trial (NCT06066580).
DUCHENNE MUSCULAR DYSTROPHY
Phase 2 LYNX trial in boys with Duchenne:
Based on the safety profile observed to date, the Company is continuing dose escalation and expanding enrollment in the 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 all 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. Over 60 children with Duchenne 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 3-month dose-ranging data in the
second quarter of 2024, once the Phase 3 dose is identified. Go to clinicaltrials.gov to learn more about this trial (NCT05540860).
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).
Received Fast Track, Orphan Drug and Rare
Pediatric Disease Designations from the U.S. Food & Drug Administration (FDA): The FDA granted EDG-5506 Fast Track designation
for the treatment of Duchenne in February 2024, and Orphan Drug Designation (ODD) for the treatment of Duchenne and Becker and Rare Pediatric
Disease Designation (RPDD) for the treatment of Duchenne in November 2023. The FDA previously granted Fast Track designation for EDG-5506
for the treatment of Becker.
Cardiovascular Program / EDG-7500
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. Preclinical data in models of both obstructed and non-obstructed HCM suggest the
ability to drive a broadly effective clinical response at a low risk of decreasing left ventricular ejection fraction below normal at
all doses tested. Due to EDG-7500's self-limiting mechanism on systolic contraction, the Company plans to investigate fixed-dose
regimens of EDG-7500 thus potentially eliminating the echo-mediated dose titration and intense follow-up requirements of current therapies.
Phase 1 Trial of EDG-7500, a first-in-class
cardiac sarcomere modulator: The Company is enrolling a randomized, placebo-controlled, single and multiple ascending dose Phase
1 trial evaluating safety, tolerability, PK and pharmacodynamics in healthy adults. To learn more about this trial (NCT06011317),
go to clinicaltrials.gov. The Company expects to report Phase 1 data in healthy volunteers and Phase 2 data in individuals with obstructive
HCM in the third quarter of 2024. Further, the Company is planning to begin a 28-day Phase 2 trial of EDG-7500 in individuals with HCM
and initiate an open-label extension trial of EDG-7500 in the fourth quarter 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 December 2023, the Company launched an educational website dedicated
to the Becker community: www.beckermusculardystrophy.com. This is the first website solely focused on providing Becker-specific resources
to help individuals and caregivers better understand the disease, learn different approaches to care and stay up to date on advocacy
partnerships and available services. Also in December 2023, the Company partnered on the inaugural Becker Education and Engagement Day
event for individuals with Becker and their families. In October 2023, the Company held an Industry Symposium at the Annual Congress
of the World Muscle Society (WMS) and held a webinar with the patient community hosted by Parent Project Muscular Dystrophy. The Company
continues to sponsor and participate in numerous other clinician and patient-focused events.