Edgewise Therapeutics Reports Fourth Quarter and Full Year 2024 Financial Results and Recent Business Highlights

-- Completed enrollment of the GRAND CANYON global pivotal study of sevasemten in Becker muscular dystrophy (Becker) --

-- Announced positive top-line results from Phase 2 CANYON trial of sevasemten in Becker --

-- On track to report data from Phase 2 CIRRUS-HCM trial of EDG-7500 in Hypertrophic Cardiomyopathy $(HCM)$ in the first quarter of 2025 --

-- On track to report data from the Phase 2 LYNX and FOX trials of sevasemten in Duchenne muscular dystrophy (Duchenne) in the first half of 2025 --

BOULDER, Colo.--(BUSINESS WIRE)--March 03, 2025-- 

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2024 and recent business highlights.

"Closing out 2024 with positive Phase 2 CANYON results, completing enrollment in GRAND CANYON and advancing CIRRUS-HCM, we are well positioned in 2025 across our muscular dystrophy and cardiovascular portfolio," said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise Therapeutics. "In the first half of this year, we look forward to announcing additional data from EDG-7500 in HCM as well as regulatory and clinical program updates on sevasemten in Becker and Duchenne."

Recent Highlights

Muscular Dystrophy Program / Sevasemten

Sevasemten and Becker

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect muscle 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, and 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 that evaluated 40 adults and 29 adolescents with a sevasemten treatment period of 12 months. Data from CANYON will be presented at the 2025 MDA Clinical and Scientific Conference. The Company plans to engage the U.S. FDA in the first half of 2025 to discuss 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 at 51 sites 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 work toward a therapeutic option. 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. To date, MESA has enrolled 99% of eligible participants completing these prior trials.

Duchenne

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 with gene therapy.

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 first half of 2025.

For more information, go to clinicaltrials.gov to learn more about LYNX (NCT05540860) and FOX (NCT06100887).

Cardiovascular and Cardiometabolic Programs

EDG-7500 and HCM

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 non-obstructive. Despite advancements in treatment options for some HCM patients, there remains a significant unmet need for additional therapeutic approaches for patients.

CIRRUS-HCM Phase 2 trial in adults with HCM: The Company is advancing CIRRUS-HCM, a multi-part, open-label trial, in individuals 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 topline data from Part A showing that treatment with EDG-7500 led to robust left ventricular outflow tract gradient reductions without meaningful changes in left ventricle ejection fraction. Parts B and C of CIRRUS-HCM are evaluating safety and efficacy of multiple doses of EDG-7500 over 28 days in individuals with either obstructive or non-obstructive HCM. The Company expects to report topline CIRRUS-HCM 28-day data in the first quarter of 2025. Further, the Company expects to report data from the 12-week CIRRUS-HCM trial in individuals with obstructive HCM and non-obstructive 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 select a proprietary cardiometabolic drug candidate based on preclinical proof-of-concept data.

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. Presentations were made at the International HCM Summit 8, American Heart Association Scientific Sessions, Global CVCT/Cardiovascular Clinical Trialists Forum, and the 29th International Annual Congress of the World Muscle Society. The team also connected with the community at the HCMA annual patient meeting and held a Becker community webinar hosted by Parent Project Muscular Dystrophy. The Company continues to sponsor and participate in numerous other clinician and patient-focused events.

Fourth Quarter Financial Results

Cash, cash equivalents and marketable securities were approximately $470.2 million as of December 31, 2024.

Research and development (R&D) expenses were $36.4 million for the fourth quarter of 2024, compared to $32.2 million for the immediately preceding quarter. The increase of $4.2 million was primarily driven by an additional $2.5 million higher personnel related costs, $2.0 million increase in clinical development activities related to the CIRRUS-HCM trial and continued enrollment in the GRAND CANYON and MESA trials, and a $0.3 million increase in professional fees and other research costs, offset by a $0.6 million decrease in manufacturing expenses.

General and Administrative (G&A) expenses were $9.2 million for the fourth quarter of 2024, compared to $8.2 million for the immediately preceding quarter. The increase of $1.0 million was primarily driven by increased personnel-related costs.

Net loss and net loss per share for the fourth quarter of 2024 was $39.7 million or $0.42 per share, compared to $34.1 million or $0.36 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. 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 , Facebook and Instagram.

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