Edgewise’s Sevasemten Shows Positive Two-Year Results in Becker Muscular Dystrophy Trial

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a prominent biopharmaceutical company specializing in muscle diseases, has unveiled encouraging two-year topline results from the ARCH trial. The ARCH study, conducted at a single center, is an open-label investigation focusing on assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK), and functional measures using sevasemten (EDG-5506) among adults diagnosed with Becker muscular dystrophy.

Sevasemten is an orally administered small molecule meticulously crafted to mitigate contraction-induced muscle damage in various dystrophinopathies, encompassing Becker and Duchenne muscular dystrophy (Duchenne).

The ARCH trial, spanning two years, assessed the daily administration of sevasemten in adults diagnosed with Becker muscular dystrophy. Among the 12 participants, sevasemten demonstrated excellent tolerability, with no discontinuations or dose reductions due to adverse events.

Maintaining NSAA (North Star Ambulatory Assessment) functions, crucial for daily living activities in Becker patients, is paramount. Over the two-year treatment period, participants’ NSAA scores remained stable, contrasting with the functional declines typically seen in Becker’s natural history studies.

Furthermore, significant reductions in key biomarkers of muscle damage, including CK and TNNI2, were observed in sevasemten-treated participants, consistent with previous findings.

Dr. Joanne Donovan, Chief Medical Officer of Edgewise, expressed satisfaction with the consistent functional outcomes and favorable safety profile observed during the trial, thanking the Becker community for their engagement.

Dr. Barry J. Byrne, Chief Medical Adviser for MDA, highlighted the relentless disease progression in Becker and underscored the potential of sevasemten to address this unmet need based on the promising two-year results.

The positive outcomes from the ARCH trial further support the hypothesis that reducing contraction-induced muscle damage may preserve function and slow disease progression in Becker muscular dystrophy.

Several upcoming events will provide opportunities to delve deeper into the ARCH trial’s findings:

  1. Virtual Investor Event: Scheduled for Tuesday, April 16, members of Edgewise’s management team, along with Dr. Byrne, will discuss the two-year data in a live webcast. Registration for the webcast and replay is available on the Edgewise events page.
  2. American Academy of Neurology 2024 Annual Meeting Podium Presentation: Dr. Joanne Donovan will present the effects of EDG-5506 on function and muscle damage biomarkers in adults with Becker muscular dystrophy on April 16, 2024, during the session titled “Inherited Myopathies and Neuropathies: New Therapeutic Approaches and Observations.” The presentation will be accessible on the Edgewise website.
  3. Patient Community Webinar: Scheduled for Monday, May 13, at 1 p.m. ET, members of Edgewise’s management will host a webinar to discuss the data from the ARCH trial and the GRAND CANYON pivotal study.

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