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Muscular Dystrophy News

Positive Phase 1 Results for Duchenne MD Drug Edasalonexent Presented in Webinar

Catabasis Pharmaceuticals, in a joint initiative with Parent Project Muscular Dystrophy (PPMD), recently hosted the webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy.”

The clinical-stage biopharma company, together with PPMD, a nonprofit organization founded in 1994 by parents of children with Duchenne and Becker Muscular Dystrophy, presented the results from the company’s recent MoveDMD clinical trial.

Dr. Joanne Donovan, chief medical officer at Catabasis, and Pat Furlong, at Parent Project Muscular Dystrophy, were the webinar presentation hosts. Donovan discussed the positive results from Part A (Phase 1) of the MoveDMD clinical trial and presented the trial’s design and inclusion criteria for Part B (Phase 2), which is currently ongoing.

The MoveDMD is a Phase 1/2 clinical trial testing the safety and efficacy of edasalonexent (CAT-1004) in boys ages 4 to 7 affected by DMD (carrying any confirmed mutation).

In Phase 1, researchers tested edasalonexent’s safety profile, tolerability, and pharmacokinetics, and its effects on its target protein NF-kB. The results of this phase were positive, with the drug being well tolerated with no safety signals triggered and successful NF-kB target engagement.Now, scientists are moving to Phase 2, and boys included in Phase 1 are asked to join this phase.Phase 2 will enroll 30 boys to evaluate both the safety and effectiveness of edasalonexent in Duchenne MD over a 12-week treatment period. This phase’s primary endpoint includes alterations to magnetic resonance scan (MRI) of boys’ leg muscles. Secondary endpoints include age-appropriate timed function tests such as the 10-meter walk/run, 4-stair climb, and time to stand. Additionally, researchers will determine muscle strength, the North Star Ambulatory Assessment, and the pediatric outcomes data collection tool (PODCI).Edasalonexent is an oral drug that researchers hope will revolutionize Duchenne MD treatment. Its effectiveness is independent of the type of mutation carried by patients. The target for edasalonexent is NF-kB, the protein that is activated in Duchenne MD, and which leads to inflammation and fibrosis and muscle degeneration, and suppresses muscle regeneration.In previous studies with animal models, edasalonexent successfully inhibited NF-kB, and the treatment led to a reduction in muscle degeneration while improving muscle regeneration and function. Its positive effects were also observed in skeletal, diaphragm, and cardiac muscle.In previous Phase 1 trials in adults, edasalonexent was also reported to be safe, well tolerated, and effective in reducing NF-kB activity.

First Duchenne Muscular Dystrophy Patient Treated in Follistatin Gene Therapy Trial

COLUMBUS, Ohio, April 13, 2015
Milo Biotechnology announced the first Duchenne muscular dystrophy (DMD) patient was treated with follistatin gene therapy at Nationwide Children’s Hospital. The therapy, delivered by intramuscular injection, is designed to maintain or restore muscle function in boys affected with DMD. This six patient clinical trial follows from a trial of the therapy in Becker muscular dystrophy patients that demonstrated initial safety and efficacy. Results of that study were published in the January 2015 issue of the Journal Molecular Therapy.

The trial is being led by Dr. Jerry Mendell, at Nationwide Children’s Hospital. According to Mendell, “The study in Becker was the first gene therapy clinical trial to demonstrate functional improvement in any form of muscular dystrophy, and a major advance for those suffering with muscle disease.” The DMD trial is being funded by the Duchenne Alliance, which coordinated funding from 15 disease foundations from around the world.

The therapy, developed at Nationwide Children’s Hospital by Dr. Mendell and Dr. Brian Kaspar, is based on adeno-associated virus delivery of follistatin 344 to increase muscle strength and prevent muscle wasting and fibrosis. Because follistatin’s mechanism of action is not mutation specific, it could potentially help other forms of muscular dystrophy. Another clinical trial of the follistatin gene therapy in patients with inclusion body myositis is also being conducted at Nationwide Children’s Hospital. “We are excited about the broad potential for this therapy and are working with foundations and patient advocates to launch additional studies,” said Milo CEO Al Hawkins.

Santhera Announces Successful Outcome of Phase III Study with Catena®/Raxone® in Duchenne Muscular Dystrophy
Liestal, Switzerland, May 13, 2014

The Phase III, double-blind, placebo-controlled DELOS study randomized 65 DMD patients who were 10-18 years of age and who were not using concomitant corticosteroids. The study met the primary endpoint, the difference between Catena®/Raxone® and placebo in the change from baseline to week 52 in Peak Expiratory Flow (p=0.04). Peak Expiratory Flow is a measure of respiratory muscle strength, the decline of which is a major contributing factor to morbidity and mortality in DMD. Catena®/Raxone® (900 mg/day) was safe and well tolerated withadverse event rates comparable to placebo. Other endpoint analyses are ongoing and results of these will be disclosed shortly.

DART Therapeutics Completes Study of New Biomarker for Duchenne Muscular Dystrophy Treatment
CAMBRIDGE, Mass., Oct. 10, 2012 /PRNewswire/

DART Therapeutics, LLC, an innovative, new-model biotechnology firm focused on developing therapies for Duchenne Muscular Dystrophy (DMD), announced today the successful completion of a study to evaluate a promising new biomarker for DMD. The biomarker, electrical impedance myography (EIM), is a non-invasive and simple-to-use technique that allows the operator to measure the health of a muscle and track its changes over time. The information can help researchers evaluate how well a treatment is working to halt disease progression, which could support more precise drug therapy for DMD boys and faster, less expensive drug development for the disease overall.DMD is a progressive and fatal neuromuscular disease for which there is no effective treatment. Current drug development efforts rely on patient outcomes measurements that are either invasive and poorly tolerated or highly subjective and prone to variability. This can have a negative impact on the value of the studies, which must generate results that can be measured and reproduced over time. EIM is based on the observation that as a muscle becomes progressively diseased, an applied weak electrical current moves through it differently. The technique could provide drug developers with a highly precise way to measure the effectiveness of experimental therapies earlier, potentially reducing the time and cost of clinical trials and propelling the availability of treatments for DMD.