Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a clinical-stage biopharmaceutical company focused on developing orally bioavailable, targeted, small molecule therapies for the treatment of devastating muscle disorders, today reported financial results for the first quarter of 2023 and recent business highlights.
“Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy.”
“We’re off to a great start in 2023! The team has focused on advancing our LYNX and CANYON clinical trials of EDG-5506 in addition to completing IND-enabling studies of EDG-7500, our novel sarcomere modulator for HCM,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise. “During the remainder of 2023, we expect to share 12-month ARCH open label data and interim Duchenne data from our Phase 2 LYNX trial. We also plan to initiate a potentially registration-enabling cohort in CANYON and a Phase 1 trial for EDG-7500 in healthy volunteers.”
Recent Highlights
Advancing CANYON Clinical Trial of EDG-5506 in Individuals with BMD
The Company is continuing to recruit the CANYON clinical trial evaluating EDG-5506 in individuals with BMD. CANYON is assessing the effect of EDG-5506 over a 12-month period on safety, pharmacokinetics (PK), biomarkers of muscle damage, such as creatine kinase (CK) and fast skeletal muscle troponin I, fat fraction as measured by muscle MRI and function in individuals with BMD aged 12 to 50 years. This placebo-controlled trial is anticipated to recruit approximately 32 adults and 18 adolescents at up to 14 sites in the United States, United Kingdom and the Netherlands. The Company plans to amend the CANYON study to include a potentially registration-enabling cohort in the second half of 2023. Go to clinicaltrials.gov to learn more about this trial (NCT05291091).
Advancing LYNX Phase 2 Clinical Trial of EDG-5506 in Children with DMD
The Company is recruiting the LYNX Phase 2 clinical trial of EDG-5506 in children with DMD. LYNX is a placebo-controlled trial to assess the effect of three doses of EDG-5506 over 12 weeks on safety, PK and biomarkers of muscle damage. Approximately 27 children with DMD aged 4 to 9 years on stable corticosteroids and/or exon skippers are expected to be enrolled at up to 14 sites across the United States. Participants will then continue in an open-label extension portion of the trial for a total of 12 months to gain further insights into safety and functional measures. Importantly, this trial is designed to identify the doses of EDG-5506 that have the potential to reduce biomarkers of muscle damage and provide functional benefit to patients in a Phase 3 trial. The Company expects to report Phase 2 interim data in the fourth quarter of 2023. Go to clinicaltrials.gov to learn more about this trial (NCT05540860).
Advancing ARCH Open Label Study of EDG-5506 in Adults with BMD
The Company is continuing to advance the ARCH open-label study evaluating EDG-5506 in 12 adult males with BMD. The study is evaluating varying doses of EDG-5506 administered daily over 24 months. Safety, PK, changes in biomarkers of muscle damage such as CK and fast skeletal muscle troponin I, measures of function with NSAA and NSAD, time function tests and patient-reported outcomes are being evaluated. The Company expects to report 12-month results in the second quarter of 2023. Go to clinicaltrials.gov to learn more about this study (NCT05160415).
Advancing DUNE Phase 2 Trial of EDG-5506 in Adults with LGMD2I/R9, BMD and McArdle Disease
The Company is continuing to recruit the DUNE Phase 2 exercise challenge study, to evaluate the effect of EDG-5506 on biomarkers of muscle damage following exercise in adults with LGMD2I/R9, BMD or McArdle disease at a single site in Denmark. The placebo-controlled study is expected to enroll 36 participants for 16 weeks, then continue to an open label extension through 52 weeks. The goal of these studies is to assess safety and efficacy in individuals with myopathy distinct from DMD/BMD where muscle contraction is associated with exaggerated injury. LGMD2I/R9 is a myopathy caused by a dysfunctional dystroglycan complex while McArdle is caused by deficiencies in glycogen mobilization leading to metabolic crisis and injury of skeletal muscle. In addition to biomarkers of muscle damage, secondary measures will include measures of strength and exercise capacity.
Journal of Clinical Investigation Published Key Preclinical Data Linking Modulation of Fast Skeletal Muscle Contraction to Protection of Skeletal Muscle in Models of DMD
In March 2023, the Journal of Clinical Investigation published the article, “Modulating fast skeletal muscle contraction protects skeletal muscle in animal models of Duchenne muscular dystrophy.” This article provides the preclinical rationale and proof-of-concept behind EDG-5506, demonstrating that modulation of fast skeletal muscle contraction protects against muscle injury, degeneration and fibrosis in models of DMD. Importantly, the study found that modest inhibition of fast skeletal muscle myosin provides maximum and robust protection of skeletal muscles and was associated with increases in strength and physical activity in mouse and dog models of DMD.
Advancing IND-enabling studies of EDG-7500, a First-In-Class Sarcomere Modulator for HCM
The Company is continuing to advance EDG-7500, a first-in-class oral, selective, sarcomere modulator for diseases of diastolic dysfunction, through IND-enabling studies with plans to initiate a Phase 1 trial in the second half of 2023. EDG-7500 is a result of Edgewise’s robust discovery platform that is yielding novel compounds targeting important unmet needs of patients suffering from disorders of cardiac and skeletal muscle. The compound is designed to improve impaired cardiac relaxation and slow contraction velocity, hallmarks of HCM. This novel mechanism is anticipated to have a broader therapeutic index relative to cardiac myosin inhibition for treatment of both obstructive and non-obstructive HCM. Preclinical data of EDG-7500 support activity in both obstructive HCM and non-obstructive HCM with minimal changes in left ventricle contractility.
Strengthened Engagement with Muscular Dystrophy Patient and Medical Communities
The Company hosted an industry forum at the Muscular Dystrophy Association Annual Clinical and Scientific Conference in March 2023. A replay of the forum can be viewed here. Further, leadership discussed and answered questions about the Company’s clinical trials in DMD and BMD during a patient community webinar hosted by CureDuchenne in March 2023. A replay of the webinar can be viewed here. The Company continues to sponsor and participate in numerous patient-focused events hosted by patient advocacy organizations.
First Quarter Financial Results
Cash, cash equivalents and marketable securities were $328.0 million as of March 31, 2023.
Research and development (R&D) expenses were $19.9 million for the first quarter of 2023, compared to $16.6 million for the immediately preceding quarter. The increase of $3.3 million was primarily driven by an increase of $1.6 million related to preclinical development of EDG-7500 and the research efforts of our cardiovascular discovery program, $0.9 million of higher expenses related to our EDG-5506 clinical program such as clinical site and CRO costs to support ongoing Phase 2 trials, an increase of $0.5 million in employee-related costs and an increase of $0.3 million in facilities and other costs that support the growth of our research and development programs.
General and Administrative (G&A) expenses were$5.8 million for the first quarter of 2023, compared to $5.5 million for the immediately preceding quarter. The increase of $0.3 million was primarily driven by increased professional and consulting and other administrative costs.
Net loss and net loss per share for the first quarter of 2023 was $22.8 million or $0.36 per share, compared to $19.4 million or $0.31 per share for the immediately preceding quarter.
Source: https://www.businesswire.com/
