Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)
– The North Star Ambulatory Assessment (NSAA) remained stable relative to declines reported in Becker natural history studies –
– Significant decreases were observed in circulating levels of creatine kinase (CK) and fast skeletal muscle troponin I (TNNI2), biomarkers associated with skeletal muscle damage –
– Sevasemten was well-tolerated –
– Edgewise leadership to discuss ARCH findings on
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Figure 1: NSAA stabilized with sevasemten treatment and continues to diverge from natural history studies at 2 years (Photo: Business Wire)
The ARCH trial evaluated sevasemten administered daily over two years in adults with Becker. Sevasemten was well-tolerated in all 12 participants with no discontinuations or dose reductions due to adverse events.
Preserving NSAA functions that correlate to activities of daily living are important to individuals living with Becker. As seen in Figure 1, during two years of sevasemten treatment, participants’ NSAA scores stabilized and continued to diverge relative to functional declines reported across multiple Becker natural history studies, in which two-year mean decreases of 2.4 NSAA points were reported1, 2,3.
In addition, significant decreases in key biomarkers of muscle damage including CK and TNNI2 were observed in participants treated with sevasemten, which are consistent with prior observations.
“We are pleased by the promising and consistent functional results observed over two years of treatment with sevasemten, together with the favorable safety and tolerability profile,” said
“Becker is a devastating neuromuscular disease that currently has no treatment,” said
The positive results from the two-year ARCH trial further support the hypothesis that a reduction in contraction-induced muscle damage in muscular dystrophies has the potential to preserve function and halt disease progression in Becker.
Upcoming ARCH 24-Month Data Presentations with Investor, Medical and
Virtual Investor Event
Members of the Edgewise management team will hold a live webcast on
Session: Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations
Title: Effects of EDG-5506, a Fast Myosin Modulator, on Function and Biomarkers of Muscle Damage in Adults with Becker Muscular Dystrophy
Presenter:
Date:
The presentation will be available on the Edgewise website when it’s presented.
Patient Community Webinar
Members of Edgewise management will hold a community webinar on
About the ARCH Open-Label Trial
ARCH, an open-label, single-center trial, assessed sevasemten in 12 adult males with Becker. The trial evaluated sevasemten administered daily over two years. Safety, tolerability, PK, changes in biomarkers of muscle damage such as CK and fast skeletal muscle troponin I, measures of function and patient-reported outcomes were evaluated. Go to clinicaltrials.gov to learn more about this trial (NCT05160415).
About GRAND CANYON, a Global Pivotal Study in Becker
The Company is advancing GRAND CANYON, a global pivotal study of EDG-5506 in individuals with Becker. GRAND CANYON is an expansion of the CANYON placebo-controlled trial. CANYON, which was over-enrolled, includes cohorts of 40 adults and 29 adolescents and a treatment period of 12 months. The Company expects to report CANYON data in the fourth quarter of 2024. GRAND CANYON is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of EDG-5506 in adults with Becker. Data from GRAND CANYON, if positive, could support a marketing application. The primary endpoint of GRAND CANYON is NSAA. In addition, other functional assessments, biomarkers of muscle damage and safety will be assessed. GRAND CANYON is anticipated to recruit approximately 120 individuals with Becker, aged between 18 and 50 years old, at up to 50 sites in 10 countries. The treatment period for participants will be 18 months. To learn more, go to clinicaltrials.gov (NCT05291091) or the GRAND CANYON microsite: https://www.beckergcstudy.com.
About Becker Muscular Dystrophy
Becker is a rare, genetic, life-shortening, debilitating and degenerative neuromuscular disorder. The disease predominantly affects males and imposes significant physical, emotional, financial, and social impacts on the individual and their caregivers. Individuals with Becker experience contraction-induced muscle damage, which is the primary driver of muscle loss and impaired motor function in muscular dystrophies. Functional decline can begin at any age, and once that muscle loss occurs, the decline in function is irreversible and continues throughout the individual’s life. Some individuals living with Becker experience heart failure from cardiomyopathy, which may result in heart transplantation or early death. Currently, there is no cure for Becker; early and long-term multidisciplinary care is critical for optimized disease management. There is a great need for more Becker-specific scientific research, clinical programs, and treatment guidelines to improve management of this disease. To learn more about Becker, go to https://beckermusculardystrophy.com/
About Sevasemten (EDG-5506) for Becker and Duchenne Muscular Dystrophies
Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne. Sevasemten presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. By minimizing the progressive muscle damage that leads to functional impairment, sevasemten has the potential to benefit a broad range of patients suffering from debilitating neuromuscular disorders. Its unique mechanism of action provides the potential to establish sevasemten as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development.
In Becker, Edgewise is advancing sevasemten in a Phase 2 trial, called CANYON, evaluating safety and effects on function and biomarkers of muscle damage in adult males with Becker. The CANYON trial, which is fully enrolled, has been expanded to include an additional 120 adult participants in a pivotal cohort called GRAND CANYON. This study is currently enrolling at sites in
In Duchenne, Edgewise is advancing its LYNX Phase 2 clinical trial, assessing safety, PK, biomarkers of muscle damage and functional measures in boys with Duchenne. It also is advancing a second Phase 2 trial, called
For more information on Edgewise’s clinical trials https://edgewisetx.com/clinical-trials.
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References
[1] Bello L, et al. Functional changes in Becker muscular dystrophy: implications for clinical trials in dystrophinopathies. Sci Rep. 2016;6:32439. doi:10.1038/srep32439
[2] van de Velde NM, et al. Selection approach to identify the optimal biomarker using quantitative muscle MRI and functional assessments in Becker muscular dystrophy. Neurology. 2021;97(5):e513-e522. doi: 10.1212/WNL.0000000000012233.
[3] De Wel B, et al. Lessons for future clinical trials in adults with Becker muscular dystrophy: disease progression detected by muscle magnetic resonance imaging, clinical and patient-reported outcome measures. Eur J Neurol. 2024:e16282. doi:10.1111/ene.16282. Online ahead of print.
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 expectations relating to its clinical trials and clinical development of sevasemten; statements regarding the potential of, and expectations regarding, Edgewise’s product candidates and programs, including EDG-5506 and EDG-7500; statements regarding Edgewise’s milestones, including timing of data from its CANYON trial; statements regarding whether data from GRAND CANYON could support a marketing application; and statements by Edgewise’s chief medical officer and
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