Edgewise Therapeutics Now Enrolling CANYON Phase 2 Clinical Trial of EDG-5506 in Individuals with Becker Muscular Dystrophy (BMD)
Edgewise Therapeutics, Inc. (EWTX) has initiated the CANYON Phase 2 clinical trial for EDG-5506, targeting Becker Muscular Dystrophy (BMD). This trial will observe 66 individuals over 12 months to assess safety, pharmacokinetics, biomarkers, and functional measures. There are no approved treatments for BMD, highlighting the unmet medical need. EDG-5506 is designed to protect muscle fibers in dystrophinopathies like BMD and Duchenne muscular dystrophy, following positive Phase 1 results. This trial marks a critical step in potential therapy development for individuals with BMD.
- Initiation of CANYON Phase 2 clinical trial for EDG-5506.
- EDG-5506 designed to address unmet medical needs in BMD treatment.
- Positive safety and biomarker results from previous Phase 1 studies.
- None.
CANYON will assess the effect of EDG-5506 over a 12-month period on safety, pharmacokinetics (PK), biomarkers such as creatine kinase (CK), and functional measures in individuals with BMD aged 12 years and above. This placebo-controlled trial is anticipated to recruit approximately 66 individuals at up to 14 sites in
“We are delighted to advance EDG-5506 into Phase 2, based on the safety and positive biomarker data observed in our Phase 1 and ARCH studies,” said
“I look forward to working with fellow investigators and the BMD community on this Phase 2 trial of EDG-5506,” said
About Becker Muscular Dystrophy
BMD is a serious, progressively debilitating, and potentially fatal inherited X-linked neuromuscular disorder. BMD results from mutation of the dystrophin gene yielding unstable and/or dysfunctional dystrophin expression in muscles. Individuals with BMD, typically males, have ongoing muscle fiber (myofiber) degeneration that eventually leads to fibrosis, progressive loss of skeletal muscle function, and that can lead to severe disability and early death. BMD typically presents with juvenile onset of muscle wasting and progressive symmetrical, proximal muscle weakness, calf hypertrophy, activity-induced muscle cramping and elevated creatine kinase activity. While the course of BMD is variable, it is unidirectional in terms of the inevitable progressive limb weakness resulting in severe disability. BMD is also associated with early mortality from cardiac disease. The incidence of BMD is approximately 1 in every 18,450 live male births. It is estimated that there are between 4,000 to 5,000 individuals with BMD in the
About EDG-5506 for DMD and BMD
EDG-5506 is an orally administered small molecule designed to address muscle damage induced by mechanical stress in dystrophinopathies including DMD and BMD. EDG-5506 presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development. In
The Company has completed a Phase 1 clinical trial of EDG-5506 designed to evaluate safety, tolerability, PK and PD of EDG-5506 in adult healthy volunteers (Phase 1a) and in adults with BMD (Phase 1b). Go to clinicaltrials.gov to learn more about this clinical trial (NCT04585464). A follow-on open-label, single-center trial is assessing the long-term safety and PK of EDG-5506 in adults with BMD (NCT05160415). We anticipate initiation of a Phase 2 trial in individuals with DMD in the second half of 2022.
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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 drug discovery platform, product candidates and programs, including EDG-5506; statements regarding the CANYON Phase 2 clinical trial, including the enrollment of individuals in such trial and the potential results from such trial; statements about the expected timing of Edgewise’s initiation of a Phase 2 clinical trial of EDG-5506 in DMD; statements by Edgewise’s Chief Medical Officer; and statements regarding Edgewise’s pipeline of product candidates and programs. Words such as “believes,” “anticipates,” “plans,” “expects,” “intends,” “will,” “goal,” “potential” and similar expressions are intended to identify forward-looking statements. The forward-looking statements contained herein are based upon Edgewise’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those projected in any forward-looking statements due to numerous risks and uncertainties, including but not limited to: risks associated with the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics and operating as an early clinical stage company including the potential for Edgewise’s product candidates to cause serious adverse events; Edgewise’s ability to develop, initiate or complete preclinical studies and clinical trials for, obtain approvals for and commercialize any of its product candidates for muscular dystrophy patients or other patient populations; the timing, progress and results of clinical trials for EDG-5506 ; Edgewise’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; negative impacts of the COVID-19 pandemic on Edgewise’s operations, including preclinical and clinical trials; the timing, scope and likelihood of regulatory filings and approvals; the potential for any clinical trial results to differ from preclinical, interim, preliminary, topline or expected results; Edgewise’s ability to develop a proprietary drug discovery platform to build a pipeline of product candidates; Edgewise’s ability to enroll and maintain patients in its ongoing and future clinical trials; Edgewise’s manufacturing, commercialization and marketing capabilities and strategy; the size of the market opportunity for Edgewise’s product candidates; the loss of key scientific or management personnel; competition in the industry in which Edgewise operates; Edgewise’s reliance on third parties; Edgewise’s ability to obtain and maintain intellectual property protection for its product candidates; general economic and market conditions; and other risks. Information regarding the foregoing and additional risks may be found in the section entitled “Risk Factors” in documents that Edgewise files from time to time with the
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