Clinical-stage biopharmaceutical company Edgewise Therapeutics Inc. (EWTX:NASDAQ), which concentrates its effort on developing small molecule therapies for treating rare muscle disorders, today announced "positive topline results from the BMD, or Phase 1b, portion of a first-in-human Phase 1 clinical trial assessing the safety, tolerability, PK and pharmacodynamics (PD) of EDG-5506, an orally administered small molecule myosin inhibitor designed to protect injury-susceptible fast skeletal muscle fibers in dystrophinopathies such as Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD)."
Craig McDonald, M.D., Professor and Chair of the Department of Physical Medicine & Rehabilitation at UC Davis explained that "BMD is a rare, serious, progressive, permanently debilitating, and potentially fatal inherited neuromuscular disorder, which causes great hardship to individuals with BMD and their families and caregivers."
"I am encouraged by the EDG-5506 Phase 1b clinical data in individuals with BMD, for which there are no approved treatments. I look forward to working with fellow investigators and the BMD and DMD communities in the upcoming Phase 2 trials of EDG-5506," Dr. McDonald added.
Edgewise Therapeutics reported the that the Phase 1b clinical trial enrolled a total of seven adult participants who were diagnosed with BMD. Five patients in the study were administered 20 mg oral doses of EDG-5506 and two were given a placebo. The company advised that EDG-5506 was shown to be overall well-tolerated without any dose reductions or discontinuations required with only a few relatively mild and transient responses observed.
Another key objective of the Phase 1b BMD study was to investigate the relationship between dosage amount, muscle exposure, and biomarkers of muscle damage on a dystrophic background. The firm indicated that in the clinical trial, "treatment with EDG-5506 led to a significant and time-dependent decrease in key biomarkers of muscle damage when assessed by both standard laboratory assays and proteomic analysis using the SOMAscan 7,000 analyte set."
The company emphasized that it is import to note that "creatine kinase (CK) and fast skeletal muscle troponin (TNNI2) were reduced by 71% and 83%, respectively, to levels near those observed in healthy volunteers when evaluated by SOMAscan and that this reduction in muscle damage biomarkers supports the hypothesis that EDG-5506 meaningfully reduces damage from the excessive stress present in dystrophic muscle, thus potentially preserving muscle function and preventing disease progression in dystrophinopathies."
The company's Chief Medical Officer Joanne Donovan, M.D., Ph.D. stated, "The completion of this clinical trial in BMD adults is an important milestone for Edgewise. We're excited as we advance into Phase 2 clinical trials in BMD and DMD…We're also excited to announce that we are in the process of enrolling our follow-on open label study (ARCH), and we have been gratified by the excellent response from all participants from the Phase 1b as well as new participants."
Edgewise Therapeutics' President and CEO Kevin Koch, Ph.D. commented, "We believe the magnitude of reduction in multiple key biomarkers of muscle damage in adults with BMD after only two weeks of dosing is unprecedented…We expect EDG-5506 treatment to lead to reduced muscle damage, the root cause of dystrophinopathies and ultimately improve function in individuals with BMD and DMD."
The company stated that it is moving forward with its ARCH study evaluating EDG-5506 in adult males with BMD. The trial will include new enrollees along with those who already participated in the first-in-human Phase 1b study. The ARCH study is designed to assess safety, PK and changes in biomarkers of muscle damage based upon daily 10 mg doses of EDG-5506 administered over period of 3 months.
The firm explained that "BMD is a serious, progressively debilitating and potentially fatal inherited X-linked neuromuscular disorder." The condition mostly effects males and typically first presents with juvenile onset of muscle wasting and progressive proximal muscle weakness and other symptoms.
The company described EDG-5506 as "an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and BMD." The firm listed that EDG-5506 was granted Fast Track status by the U.S. Food and Drug Administration in August 2021 for the BMD treatment.
Edgewise Therapeutics is a clinical-stage biopharmaceutical company based in Boulder, Colo. that endeavors to discover, develop and commercialize new treatments for severe and rare neuromuscular and cardiac disorders. The firm's approach to drug discovery is structured to specifically target muscles as an organ. The firm is developing its lead pipeline candidate EDG-5506 to address the root cause of dystrophinopathies including BMD and DMD.
Edgewise Therapeutics Inc. started the day with a market cap of around $763.6 million with approximately 49.3 million shares outstanding and a short interest of about 5.4%. EWTX shares opened 16% higher today at $18.02 (+$2.53, +16.33%) over yesterday's $15.49 closing price. The stock has traded today between $16.21 and $20.71 per share and is currently trading at $19.25 (+$3.76 +24.27%).
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