Full Press Release Details
Edgewise Therapeutics Reports First Quarter
2025 Financial Results and Recent Business Highlights
- Announced positive top-line results
from Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy (HCM) -
- Strengthened balance sheet with net
proceeds of approximately $188 million from the April 2025 public offering; pro-forma cash balance exceeds $624 million -
- On track to report data from the Phase
2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy (Duchenne) in the second quarter of 2025 -
- Expect to report data from Part D
of the Phase 2 CIRRUS-HCM trial in the second half of 2025 -
Boulder, Colo., (May 8, 2025) - Edgewise Therapeutics, Inc.,
(Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the first quarter of 2025 and
recent business highlights.
"We're seeing strong progress across our skeletal and cardiac
muscle programs," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "Most recently, we announced
positive top-line results from CIRRUS-HCM and completed a $200 million offering enabling the execution of our near and long-term goals.
Our goals include commercial readiness for a potential U.S. approval of sevasemten in Becker, advancement of a Phase 3 trial in Duchenne,
a Phase 3 program in HCM and our ongoing research and development activities. We anticipate several key milestones this year in both our
sevasemten and EDG-7500 programs."
Strengthened Financial Position
On April 3, 2025, the Company closed an underwritten registered
direct offering of 9,935,419 shares of common stock at an offering price of $20.13 per share. The aggregate gross proceeds from the offering
were $200 million. The net proceeds after deducting underwriting discounts and commissions but before offering expenses were $188 million.
Muscular Dystrophy Program / Sevasemten
Sevasemten and Becker
Sevasemten is an orally administered first-in-class fast skeletal myosin
inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne.
Becker is a rare, genetic, life-shortening, debilitating and degenerative
neuromuscular disorder. Genetic mutations in the dystrophin gene result in contraction-induced muscle damage, which is the primary driver
of irreversible muscle loss and impaired motor function. The disease predominantly affects males, with functional decline beginning at
any age. Once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual's life. Currently,
there are no approved therapies on the market to treat Becker.
CANYON Phase 2 placebo-controlled trial in adults with Becker:
In December 2024, the Company announced positive topline results from CANYON, the largest interventional Becker trial to date, which
evaluated 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. Data from CANYON was presented at the 2025 MDA
Clinical and Scientific Conference. The Company is engaging with the U.S. FDA to discuss potential marketing authorization filing strategies
for sevasemten in Becker.
GRAND CANYON, a global pivotal placebo-controlled cohort in Becker:
In February 2025, the Company completed enrollment in GRAND CANYON, an expansion of the CANYON placebo-controlled trial. The
18-month GRAND CANYON study is active in 12 countries, including the United States, United Kingdom, Netherlands, Denmark, Belgium, France,
Spain, Germany, Italy, Israel, New Zealand and Australia. GRAND CANYON enrolled 175 adults, reflective of the Becker community's
enthusiasm to have access to a therapy with the potential to stabilize their debilitating decline in function and ability to perform
everyday activities. Data from the study are expected in Q4 2026. To learn more, go to clinicaltrials.gov (NCT05291091).
MESA Phase 2 open label extension trial in adults with Becker:
The Company is advancing MESA, an open-label extension trial to assess the long-term effect of sevasemten in individuals with Becker.
MESA provides continued access to sevasemten to participants who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON,
or DUNE. MESA has continuing enrollment of 99% of eligible participants completing these prior trials.
Duchenne, a severe degenerative muscle disorder, is the most common
type of muscular dystrophy with a median life expectancy of around 30 years. Genetic mutations in the dystrophin gene result in contraction-induced
muscle damage, which is the primary driver of irreversible muscle loss and impaired motor function. While there are approved therapies
on the market aimed to treat the disease, there remains a high unmet need for additional therapies.
LYNX and FOX Phase 2 trials in boys with Duchenne: LYNX is an
ongoing multi-center, dose-finding Phase 2 trial to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function
in children with Duchenne treated with oral, once-daily sevasemten.
FOX is a Phase 2 ongoing placebo-controlled trial to assess the effect
of sevasemten on safety, biomarkers of muscle damage and function in children and adolescents with Duchenne who have been previously treated
Based on collective dose finding observations from both LYNX and FOX,
the Company will form its Phase 3 strategy for sevasemten in Duchenne including patient and dose selection. The Company expects to report
data from LYNX and FOX as well as its future clinical trial plans in the second quarter of 2025.
For more information, go to clinicaltrials.gov to learn more about
LYNX (NCT05540860) and FOX (NCT06100887).
Cardiovascular and Cardiometabolic Programs
EDG-7500 is a novel 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. HCM is the most common form of genetic heart disease, affecting approximately one in 500 people, and is associated with reduced
quality of life and an elevated risk of heart failure, abnormal heart rhythms, and sudden cardiac death. There are two major forms of
HCM: obstructive and nonobstructive. Despite advancements in treatment options for some HCM patients, there remains a significant unmet
need for additional therapeutic approaches for patients, including nonobstructive HCM patients, for which there are no approved treatment
CIRRUS-HCM Phase 2 trial in adults with HCM: The Company is
advancing CIRRUS-HCM, a multi-part, open-label trial, in participants with HCM at up to 20 clinical sites in the U.S. Part A of the
trial was designed to evaluate the safety and tolerability of a single oral dose of EDG-7500 in obstructive HCM. In September 2024,
the Company announced positive top-line data from Part A of the trial showing that a single oral dose of EDG-7500 in participants
with obstructive HCM demonstrated robust reductions in left ventricular outflow tract gradient (LVOT-G) without meaningful changes in
left ventricular ejection fraction (LVEF).
Part B and Part C of the trial were designed to evaluate
the safety and efficacy of once-daily doses of EDG-7500 for four weeks in participants with obstructive HCM (Part B) and in participants
with nonobstructive HCM (Part C). In April 2025, the Company announced positive top-line data from Parts B and C. EDG-7500 over
four weeks demonstrated rapid and sustained clinically meaningful reductions in LVOT-G. Further, four-week treatment with EDG-7500 demonstrated
substantial improvements in measures of feel and function (Kansas City Cardiomyopathy Questionnaire and/or New York Heart Association
functional class scores), reductions in NT-proBNP, a key biomarker of heart failure, and positive trends in measures of diastolic function.
EDG-7500 was generally well-tolerated; clinical activity was observed without meaningful changes in LVEF, including no participant with
a value below 50% at any measurement. The most frequently reported adverse events were dizziness, upper respiratory tract infection and
atrial fibrillation (AF); nearly all were considered mild to moderate in severity.
The Company expects to report data from the 12-week (Part D)
CIRRUS-HCM trial in participants with obstructive HCM and nonobstructive HCM in the second half of 2025. To learn more about CIRRUS-HCM,
visit clinicaltrials.gov, NCT06347159 (Phase 2).
Preclinical programs: During 2025, the Company expects to file
an investigational new drug application for a novel candidate for the treatment of heart failure and continue to advance a novel cardiometabolic
Strengthened Engagement with the Scientific and Patient Communities
The Company continued its education and outreach with the muscular
dystrophy and HCM medical and patient communities. The team participated in the Muscular Dystrophy Association annual conference and the
American College of Cardiology Scientific Sessions. The Company continues to sponsor and participate in numerous other clinician and patient-focused
First Quarter Financial Results
Cash, cash equivalents and marketable securities were approximately
$436.4 million as of March 31, 2025. Our cash and cash equivalents at March 31, 2025 do not include the $188 million in net
proceeds after deducting underwriting discounts and commissions but before offering expenses from our underwritten registered direct offering
that closed on April 3, 2025. Combining the actual balance at March 31, 2025 with the net proceeds of the offering provides
for a proforma balance of $624.4 million.
Research and development (R&D) expenses were $36.8
million for the first quarter of 2025, compared to $36.4 million for the preceding quarter. The increase of $0.4 million was primarily