Full Press Release Details
Edgewise Therapeutics Reports Second Quarter
2025 Financial Results and Recent Business Highlights
positive top-line data from the open-label extension MESA trial of sevasemten in participants with Becker muscular dystrophy
who previously completed ARCH and CANYON studies -
encouraging top-line data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy -
- Advanced the Phase 2 CIRRUS-HCM trial
of EDG-7500 in Hypertrophic Cardiomyopathy (HCM) -
Boulder, Colo., (August 7, 2025)
- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial
results for the second quarter of 2025 and recent business highlights.
"In the first half of 2025, we reached key milestones that bring
us closer to delivering on our mission," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "With
strong funding in place, we're advancing our skeletal and cardiac muscle programs and building the commercial infrastructure with
precision and purpose to support a potential U.S. launch of sevasemten in Becker. We're also actively exploring Phase 3 trial designs
in HCM and Duchenne, while continuing to advance our pipeline through bold, innovative R&D."
Muscular Dystrophy Program
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.
MESA open-label extension trial in
adults with Becker: In June 2025, the Company announced positive data from MESA, an open-label extension trial that provides
continued access to sevasemten to participants with Becker who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or
DUNE. As of the March 2025 data cut, 99% of eligible participants (n=85) are enrolled in MESA, which was designed to collect long-term
safety and efficacy data.
The MESA data demonstrated sustained disease stabilization in North
Star Ambulatory Assessment (NSAA), reinforcing prior ARCH and CANYON findings. Importantly, CANYON participants who rolled over to MESA
showed stable NSAA scores over 18 months with a trend toward improvement in placebo participants switching to sevasemten. During the 18
months of sevasemten treatment, participants' NSAA scores continued to diverge relative to the expected functional declines seen
in multiple Becker natural history studies. Further, NSAA scores for ARCH participants who rolled over to MESA remained stable after three
years of treatment. Sevasemten continues to demonstrate a favorable safety profile after up to three years of treatment.
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 cohort is active in 12 countries, 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
GRAND CANYON is highly powered to be
able to show a statistically significant difference in NSAA versus placebo over 18 months and is on track for topline data in the fourth
quarter of 2026. In the second quarter of 2025, the Company completed a successful Type C meeting with the FDA, which provided a clear
path to registration of sevasemten as the first ever therapy for Becker. To learn more, go to clinicaltrials.gov (NCT05291091).
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 at treating the disease, there remains a high unmet need for additional therapies.
LYNX Phase 2 placebo-controlled, dose-finding
trial in boys with Duchenne: LYNX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and
function in four- to nine-year-old participants with Duchenne treated with sevasemten in a three-month placebo-controlled dose ranging
study, followed by an open-label extension period. In June 2025, the Company announced encouraging observations across functional measures,
including Stride Velocity 95th Centile (SV95C), NSAA and 4 stair-climb, while identifying a dose of 10 mg to evaluate in Phase 3.
FOX Phase 2 placebo-controlled trial
in boys with Duchenne: FOX is designed to evaluate the effect of sevasemten on safety, biomarkers of muscle damage and function
in six- to 14-year-old participants with Duchenne who have been previously treated with gene therapy. FOX participants are on average
over 10 years old and four years out from receiving gene therapy. Despite the lack of extensive natural history in Duchenne gene therapy
treated boys, initial results from the FOX study support that sevasemten 10 mg has the potential to reduce the rate of functional decline.
The Company plans to meet with the FDA in the fourth quarter of 2025
to discuss a Phase 3 design including input on the patient population and endpoints, with plans to initiate a pivotal study in 2026. In
addition, the Company plans to continue to collect longer-term open label extension data, which will provide further access to the drug
to trial participants.
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 (nHCM)
patients, for which there are no approved treatment options.
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 over 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 (oHCM). 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 oHCM (Part B) and in participants with nHCM (Part C). Part D of the trial, a 12-week study in both oHCM and
nHCM, is actively recruiting participants. The Company expects to provide an update on the CIRRUS-HCM trial in participants with oHCM
and nHCM during the fourth quarter of 2025. To learn more about CIRRUS-HCM, visit clinicaltrials.gov, NCT06347159 (Phase 2).
Heart failure and preclinical programs:
During the third quarter of 2025, the Company expects to begin dosing in a first-in-human Phase 1 trial of EDG-15400, a novel drug candidate
intended for the treatment of heart failure. The Company continues to advance its preclinical cardiometabolic programs.
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 Parent Project Muscular Dystrophy annual conference, CureDuchenne
Futures conference and the Annual Congress of the Heart Failure Association of the ESC. The Company continues to sponsor and participate
in numerous other clinician and patient-focused events.
Second Quarter Financial Results
Cash, cash equivalents and marketable
securities were approximately $594.0 million as of June 30, 2025.
Research and development (R&D)
expenses were $33.6 million for the second quarter of 2025, compared to $36.8 million for the immediately preceding quarter.
The decrease of $3.2 million was primarily driven by a $2.6 million decrease in sevasemten clinical development activities, a $0.9 million
decrease in EDG-7500 clinical development activities, a $1.1 million decrease in personnel related costs due to higher stock-based compensation
in the prior quarter, and a $0.3 million decrease in professional fees and other research costs, offset by a $1.7 million increase in
manufacturing expenses to support clinical development across our programs. The decrease in R&D expense for sevasemten is related
to higher costs incurred in the first quarter related to a drug interaction study that was substantially executed in the prior quarter
and higher costs related to final enrollment activities of GRAND CANYON. The decrease in R&D expense for EDG-7500 related to the completion
of a human pharmacokinetic study which was substantially completed in the prior quarter.
General and Administrative (G&A)
expenses were $9.1 million for the second quarter of 2025, compared to $9.2 million for the immediately preceding