Rare Disease Report

Based on Latest Data at World Muscle Society Conference, Catabasis to Begin Phase 3 DMD Study

OCTOBER 04, 2017
James Radke
The 22nd World Muscle Society Conference in St. Malo, France is the site of plenty of promising information this week, including encouraging data for the Duchenne muscular dystrophy (DMD) community.

Earlier today, Catabasis Pharmaceuticals announced the latest from the company’s ongoing Phase 1/2 MoveDMD study. Results show that its drug edasalonexent delays disease progression for up to 36 weeks of treatment.

DMD is a progressive degenerative muscle disease caused by low levels of dystrophin protein. Dystrophin acts as a shock-absorber for muscles and without it, muscles progressively die. Symptoms typically begin in patients around 4-to-5 years old, and those with the condition are commonly non-ambulatory by their teenage years.
Edasalonexent inhibits NF-kB, a protein that is active in DMD patients and appears to drive degeneration and suppress regeneration in the muscles.

In January of this year, things were looking pretty grim for the Catabasis' DMD program when  the MoveDMD study failed to reach its primary endpoint after 12 weeks of treatment. At that time, the company stated: “We observed potential treatment-associated effects at 12 weeks in the 100 mg/kg/day treatment group, which we believe warrant further evaluation to see if the signals strengthen in the longer-term data from the ongoing open-label extension. Following additional data analysis from the open-label extension, we will determine the next steps for edasalonexent in DMD.”

The new open-label 24- and 36-week data seems to have reignited hope that this drug will eventually gain approval and a Phase 3 clinical trial is being planned.

According to the company, improvements were observed in the rate of decline, using the North Star Ambulatory Assessment (NSAA) scale after 24 and 36 weeks of oral 100 mg/kg/day edasalonexent treatment compared to the rate of change in the control period for patients prior to receiving edasalonexent treatment.

Based on the results, Catabasis plans to begin a Phase 3 trial testing edasalonexent in patients with DMD regardless of mutation type in the first half of 2018 with results expected in 2020.

The Phase 3 trial will enroll approximately 125 patients ages 4 to 7 who have not been on steroids for at least 6 months. The primary endpoint will be change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo.

Per Catabasis: “We are extremely excited to see edasalonexent change the trajectory of disease in the MoveDMD trial with substantially slowed disease progression,” said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis. “Boys treated with edasalonexent stabilized; they experienced meaningful improvements in muscle function compared to the rates of change observed during the control period. Importantly, other supportive positive measures of muscle health were observed. We look forward to advancing edasalonexent as a disease-modifying therapy in a single Phase 3 pivotal trial as soon as possible with the goal of providing a meaningful impact on disease progression for all boys affected by Duchenne.”

“Our goal in treating boys with Duchenne is to slow the progression of the disease. It is tremendously encouraging to see boys taking edasalonexent stabilize in their functional abilities and MRI T2 measures along with its continued safety profile,” said Richard Finkel, M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours Children’s Health System and a Principal Investigator for the study. “I look forward to continuing to investigate edasalonexent as a potential therapy for the many boys affected by this devastating disease.”

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