Full Press Release Details
Edgewise Therapeutics Reports Third Quarter
2025 Financial Results and Recent Business Highlights
- Pivotal GRAND CANYON cohort of sevasemten
in Becker expected to read out in Q4 2026; building infrastructure to support potential commercial launch -
- MESA open-label extension trial of sevasemten
in Becker muscular dystrophy continues to enroll nearly all eligible participants -
- Continuing to advance Phase 2 CIRRUS-HCM
trial of EDG-7500 in Hypertrophic Cardiomyopathy; on track to provide program update in Q4 2025 -
- Initiated Phase 1 healthy adult trial
of EDG-15400 for future studies in Heart Failure -
Boulder, Colo., (November 6, 2025) - Edgewise
Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the
third quarter of 2025 and recent business highlights.
"With a strong balance sheet, we continue to make great progress
on our cardiac and skeletal muscle programs," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. "Planning
for success in GRAND CANYON, we are building the commercial infrastructure to support a potential launch of sevasemten in Becker. We recently
initiated clinical development of EDG-15400, our new clinical candidate targeting heart failure and we're actively developing Phase
3 trial designs in HCM and Duchenne, while continuing to advance our pipeline through innovative R&D."
Muscular Dystrophy Program
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.
MESA open-label extension trial in adults with Becker: The Company
continues to advance MESA, an open-label extension trial that collects long term safety and efficacy data of sevasemten to participants
with Becker who were previously enrolled in ARCH, or completed CANYON, GRAND CANYON, or DUNE. As of September 2025, 99% of eligible
participants are enrolled in MESA.
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, and has 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. GRAND CANYON is highly powered to
be able to show a statistically significant difference in NSAA versus placebo over 18 months and the Company expects to report topline
data in the fourth quarter of 2026. To learn more, go to clinicaltrials.gov (NCT05291091).
LYNX and FOX Phase 2 placebo-controlled trials 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 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. 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 to discuss a Phase 3 study
design including input on the patient population and endpoints, with plans to initiate a pivotal study in 2026. The Company will continue
to collect longer-term safety and functional data in the open-label extensions of LYNX and FOX. 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
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 was designed to evaluate the safety and efficacy of
EDG-7500 for 12 weeks in participants with oHCM and nHCM and has achieved strong enrollment. The Company is on track to share a program
update in the fourth quarter of 2025, with more comprehensive data expected in the first half of 2026. To learn more about CIRRUS-HCM,
visit clinicaltrials.gov, NCT06347159 (Phase 2).
EDG-15400 and Heart Failure:
EDG-15400 is a novel oral, selective, cardiac sarcomere modulator,
being developed for the treatment of heart failure with preserved ejection fraction (HFpEF), a very common form of heart failure. EDG-15400
is currently being dosed in healthy adults in a Phase 1, randomized, double-blind, placebo-controlled, single and multiple ascending
dose study evaluating safety, tolerability, pharmacokinetics and pharmacodynamics. The Company expects topline results from this study
in the first half of 2026. To learn more about this study, go to clinicaltrials.gov (NCT07177066).
Cardiometabolic preclinical program:
The Company continues to advance its preclinical cardiometabolic program.
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 Annual International Congress of the World Muscle Society,
Becker Education and Engagement Day, European Society of Cardiology Congress, Heart Failure Society of America Annual Scientific Meeting,
and the International Workshop on Cardiomyopathies. The Company continues to sponsor and participate in numerous other clinician and patient-focused
Third Quarter Financial Results
Cash, cash equivalents and marketable securities were approximately
$563.3 million as of September 30, 2025.
Research and development (R&D) expenses were $37.5
million for the third quarter of 2025, compared to $33.6 million for the immediately preceding quarter. The increase of $3.9 million was
primarily driven by a $1.4 million increase in EDG-15400 clinical development activities related to initiation of a Phase 1 trial in the
third quarter 2025, a $0.9 million increase in EDG-7500 clinical development activities related to continued patient activity in our CIRRUS-HCM
trial and other pharmacokinetic studies, a $0.5 million increase in sevasemten clinical development activities related to additional costs
incurred to support continued patient activity in GRAND CANYON and roll over to the MESA open label extension, a $0.9 million increase
in professional fees and other research costs, and a $0.2 million increase in manufacturing expenses to support clinical development across
our cardiac programs.
General and administrative (G&A) expenses were $9.4
million for the third quarter of 2025, compared to $9.1 million for the immediately preceding quarter. The increase of $0.3 million was
primarily driven by additional personnel-related costs.
Net loss and net loss per share for the third quarter of
2025 was $40.7 million or $0.39 per share, compared to $36.1 million or $0.34 per share for the immediately preceding quarter.
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 novel therapeutics. Sevasemten is an orally administered first-in-class fast skeletal
myosin inhibitor in late-stage clinical trials in Becker and Duchenne muscular dystrophies. EDG-7500 is a novel cardiac sarcomere modulator
for the treatment of hypertrophic cardiomyopathy and other diseases of diastolic dysfunction, currently in Phase 2 clinical development.
EDG-15400 is a novel cardiac sarcomere modulator for the treatment of heart failure, currently in Phase 1 clinical development. The entire
team at Edgewise is dedicated to our mission: changing the lives of patients and families affected by serious muscle diseases. To learn
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
sevasemten, EDG-7500, EDG-15400 and its cardiovascular and cardiometabolic programs; statements regarding a potential U.S. launch of sevasemten
in Becker; statements regarding the market opportunity for Duchenne, Becker and HFpEF; statements regarding Edgewise's expectations
relating to its clinical trials, including timing of reporting data (including an update and comprehensive data on the CIRRUS-HCM trial,