BECKER MUSCULAR DYSTROPHY

Edgewise Therapeutics Announces Positive Topline Results From the EDG-5506 Phase 1b Clinical Trial in Adults With Becker Muscular Dystrophy (BMD)

Retrieved on: 
Wednesday, January 5, 2022
Biotechnology, Pharmaceutical, Health, Clinical Trials, the ARCH Open Label Study, Becker Muscular Dystrophy, EDG-5506 for DMD and BMD, Edgewise Therapeutics, THE ARCH OPEN LABEL STUDY, BECKER MUSCULAR DYSTROPHY, EDG-5506 FOR DMD AND BMD, EDGEWISE THERAPEUTICS

I am encouraged by the EDG-5506 Phase 1b clinical data in individuals with BMD, for which there are no approved treatments.

Key Points: 
  • I am encouraged by the EDG-5506 Phase 1b clinical data in individuals with BMD, for which there are no approved treatments.
  • The seven adults with BMD enrolled in the Phase 1b clinical trial were administered 20 mg oral doses of EDG-5506 (n=5) or placebo (n=2).
  • The most common adverse event, observed in all Phase 1b participants, was dizziness which was mild, transient and self-resolving.
  • The completion of this clinical trial in BMD adults is an important milestone for Edgewise.

Edgewise Therapeutics to Report Phase 1b Topline Results of EDG-5506 in Individuals with Becker Muscular Dystrophy (BMD) on January 5, 2022, and Initiates ARCH Follow-On Open Label BMD Study

Retrieved on: 
Wednesday, December 15, 2021
Health, Clinical Trials, General Health, Research, Pharmaceutical, Science, Therapy, U.S. Securities and Exchange Commission, LinkedIn, DMD, ARCH, Food, Protein, Company, Creatine kinase, Safety, Clinical trial, Muscular dystrophy, Securities Exchange Act of 1934, Drug Quality and Security Act, Myopathy, Adult, Conference, BMD, Duchenne muscular dystrophy, Industry, Biomarker, Incidence, PK, COVID-19, Degenerative disease, Securities Act of 1933, NASDAQ, Pharmacokinetics, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Marketing, Mortality, Disease, Patient, Security (finance), Neuromuscular Disorders, Mutation, Program, Disability, Fast Track, Twitter, PD, Duchenne, Muscle, Fibrosis, Webcast, Dystrophinopathy, Male, Pharmaceutical industry, Medical device, Dietary supplement, Facebook, Edgewise, the ARCH Open Label Study, Becker Muscular Dystrophy, EDG-5506 for DMD and BMD, Edgewise Therapeutics, THE ARCH OPEN LABEL STUDY, BECKER MUSCULAR DYSTROPHY, EDG-5506 FOR DMD AND BMD, EDGEWISE THERAPEUTICS

Additionally, the Company will provide details on the recently initiated follow-on open label study, EDG-5506-002 (ARCH).

Key Points: 
  • Additionally, the Company will provide details on the recently initiated follow-on open label study, EDG-5506-002 (ARCH).
  • We look forward to sharing the Phase 1b BMD topline results of EDG-5506 during our webcast, said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Edgewise.
  • We are also excited to initiate the ARCH study, which will enroll participants from our Phase 1b clinical trial as well as new participants with BMD.
  • Edgewises management will hold a conference call and webcast on January 5, 2022, at 9:00 am ET to discuss topline results from the Phase 1b clinical trial in individuals with BMD.

Edgewise Therapeutics Announces Positive Topline Results from the EDG-5506 Phase 1 Multiple Ascending Dose (MAD) Study in Healthy Volunteers (HVs) and Doses First Becker Muscular Dystrophy (BMD) Patients

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Thursday, October 28, 2021
Health, Other Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, Safety, Fibrosis, MAD, Muscular dystrophy, Mutation, Securities Act of 1933, LinkedIn, PD, Disease, Industry, Dizziness, Twitter, Muscle, Fast Track, Security (finance), U.S. Securities and Exchange Commission, Patient, NASDAQ, FDA, DMD, Degenerative disease, Creatine kinase, Food, Vital signs, Disability, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, PK, Adult, Protein, Marketing, Company, Dystrophinopathy, Myopathy, Clinical trial, Webcast, Biomarker, COVID-19, Leg, Male, Program, Pharmacokinetics, BMD, Somnolence, Drug Quality and Security Act, Therapy, Shoulder, Mortality, Neuromuscular Disorders, Securities Exchange Act of 1934, Incidence, Pharmaceutical industry, Dietary supplement, Vaccine, Becker Muscular Dystrophy, EDG-5506 for DMD and BMD, Edgewise Therapeutics, BECKER MUSCULAR DYSTROPHY, EDG-5506 FOR DMD AND BMD, EDGEWISE THERAPEUTICS

We are extremely encouraged by these multiple ascending dose data, which clearly demonstrate high levels of target engagement with a well-tolerated dosing regimen.

Key Points: 
  • We are extremely encouraged by these multiple ascending dose data, which clearly demonstrate high levels of target engagement with a well-tolerated dosing regimen.
  • The 40 healthy volunteers enrolled in the MAD study were administered oral doses of EDG-5506 (5 mg to 40 mg) or placebo.
  • At doses above 10 mg, exposures were above pharmacologically active levels observed across multiple preclinical models of DMD.
  • These compelling observations provide preliminary evidence of a dose dependent, selective inhibition of fast skeletal muscle fibers following multiple doses of EDG-5506.

Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Individuals with Becker Muscular Dystrophy (BMD)

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Monday, August 16, 2021
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The FDAs decision to grant EDG-5506 Fast Track designation underscores the urgency to address a significant unmet medical need for individuals with Becker muscular dystrophy, said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.

Key Points: 
  • The FDAs decision to grant EDG-5506 Fast Track designation underscores the urgency to address a significant unmet medical need for individuals with Becker muscular dystrophy, said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
  • We look forward to working closely with the FDA towards establishing EDG-5506 as the potential first muscle-directed therapy for individuals with Becker.
  • The designation is granted to therapeutics that offer potential to meaningfully impact survival, day-to-day functioning, or if left untreated, progression of the condition.
  • The decision to give EDG-5506 Fast Track designation was supported by a robust preclinical data package and an ongoing Phase 1 clinical trial (NCT04585464).

Italfarmaco Announces Topline Data from Phase 2 Trial with Givinostat in Patients with Becker Muscular Dystrophy

Retrieved on: 
Saturday, June 26, 2021
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Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the companys proprietary histone deacetylase (HDAC) inhibitor, in 51 adult males with Becker Muscular Dystrophy (BMD).

Key Points: 
  • Italfarmaco Group announced today topline data from its proof-of-concept Phase 2 trial with Givinostat, the companys proprietary histone deacetylase (HDAC) inhibitor, in 51 adult males with Becker Muscular Dystrophy (BMD).
  • The topline data was presented by Paolo Bettica on June 26, 2021, at the virtual Parent Project Muscular Dystrophy (PPMD) Annual Conference .
  • The Phase 2 trial investigating Givinostat in BMD patients was a randomized, double-blind, placebo-controlled study (ClinicalTrials.gov: NCT03238235 ).
  • Italfarmaco is also investigating the effect of Givinostat treatment in patients with Duchenne Muscular Dystrophy (DMD) in a Phase 3 trial (ClinicalTrials.gov: NCT02851797 ).