Edgewise Therapeutics Q1 2024 Financial Results and Business Highlights

Edgewise Therapeutics, Inc., a biopharmaceutical company specializing in muscle disease, reported its first-quarter financial results for 2024 and shared recent business updates. Kevin Koch, Ph.D., the company's President and CEO, highlighted the significant progress made in their skeletal and cardiac muscle programs.

Musculoskeletal Programs

Becker Muscular Dystrophy (Becker)

Edgewise is developing sevasemten, an oral small molecule aimed at preventing muscle damage in dystrophinopathies like Becker and Duchenne muscular dystrophies. In the ARCH open-label trial for Becker, positive two-year results were reported. The study assessed the safety, tolerability, and impact on muscle damage biomarkers and functional measures. Sevasemten was well-tolerated among all 12 participants, with no discontinuations or dose reductions due to adverse events. Key biomarkers of muscle damage, such as creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), showed significant decreases, supporting the hypothesis that reducing contraction-induced muscle damage can preserve muscle function.

The CANYON Phase 2 trial, the largest interventional Becker trial to date, is ongoing with 40 adults and 29 adolescents. The primary endpoint is the change in CK levels over a 12-month treatment period. Data from this trial are expected in the fourth quarter of 2024.

The GRAND CANYON pivotal cohort is an extension of the CANYON trial, evaluating safety and efficacy of sevasemten in adults with Becker. The 18-month study aims to recruit around 120 participants globally. Positive data from this study could support a marketing application.

The DUNE Phase 2 Exercise Challenge trial is also advancing. This 16-week randomized, double-blind, placebo-controlled study examines the safety and biomarker response to exercise in adults with Becker, LGMD2I, or McArdle disease. Preliminary results indicate that sevasemten significantly reduces biomarkers of muscle damage in Becker participants.

Duchenne Muscular Dystrophy

The LYNX Phase 2 trial is evaluating sevasemten in boys with Duchenne muscular dystrophy. This multi-center, dose-finding study aims to determine the effect of sevasemten on safety, pharmacokinetics (PK), and muscle damage biomarkers in over 60 children. Initial results have shown safety and biomarker reduction, and the company plans to report LYNX data in the fourth quarter of 2024.

The FOX Phase 2 trial is focused on boys with Duchenne who have previously received gene therapy. The study aims to assess the safety and biomarkers of muscle damage over 12 weeks. Exceptional enthusiasm from the Duchenne community has led to rapid enrollment completion.

Edgewise has also received Orphan Drug Designation from the European Medicines Agency (EMA) and Fast Track designation from the U.S. Food & Drug Administration (FDA) for sevasemten for the treatment of Becker and Duchenne.

Cardiovascular Program

EDG-7500

Edgewise is developing EDG-7500, an oral cardiac sarcomere modulator aimed at treating hypertrophic cardiomyopathy (HCM) and other diastolic dysfunction diseases. The Phase 2 CIRRUS-HCM trial was initiated in April 2024, with the first patient dosed. This trial will assess the safety, tolerability, PK, and pharmacodynamics of EDG-7500 in up to 30 adults with obstructive HCM.

Additionally, a Phase 1 trial of EDG-7500 is ongoing, evaluating safety and PK in healthy adults. Data from the single dose arm of CIRRUS-HCM and the Phase 1 trial are expected in the third quarter of 2024.

Financial Highlights

As of March 31, 2024, Edgewise reported cash, cash equivalents, and marketable securities totaling approximately $532.8 million. Research and development expenses were $27.7 million, while general and administrative expenses were $7.1 million for the first quarter of 2024. The net loss for the quarter was $28.5 million or $0.33 per share.

Conclusion

Edgewise Therapeutics is making significant strides in developing novel therapeutics for muscular dystrophies and serious cardiac conditions. With multiple clinical trials underway and promising preliminary results, the company is poised for a year of potential breakthroughs and advancements in muscle disease treatment.

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