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Edasalonexent: Positive results and no side effects

One of the objectives of our team is to inform you about new treatments. Here’s the latest news from a recent Catabasis press release about edasalonexent. Enjoy reading and thank you for sharing these articles within the DMD community.

The MoveDMD trial through 48 weeks of edasalonexent treatment

 

“ We believe that these effects will ultimately translate to boys with Duchenne maintaining functional abilities longer.”

Said Jill C. Milne, Ph.D., Chief Executive Officer of Catabasis, in a press release this week.

The fact that no evidence of side effects or safety has been observed after more than 37 patient-years of exposure to treatment is also encouraging.

What is Edasalonexent?

Edasalonexent (CAT-1004)  is being developed as a potential foundational disease-modifying therapy for all patients affected by DMD, regardless of their underlying mutation. It is an investigational oral small molecule. Edasalonexent inhibits NF-kB, a protein that is activated by DMD and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration.

You can watch our video here to have a more in-depth explanation about the basis of this treatment.

 

Positive results:

Statistically significant improvement was observed compared to the off-treatment control period. These improvements show a slowing of disease progression and are in addition to the improvements found in all assessments of muscle function through more than a year of edasalonexent treatment.

What’s next?

Catabasis is preparing for a Phase 3 trial that will enroll approximately 125 boys with DMD between the ages of 4-7 years old regardless of mutation type and who have not been on steroids for at least six months. It is planned to be a single, global, placebo-controlled Phase 3 trial with two boys receiving edasalonexent for every one boy receiving placebo. After 12 months in the trial, all boys are expected to receive edasalonexent in an open-label extension.

Approbation in the US: FDA Status

The FDA has granted orphan drug, fast track and rare pediatric disease designations and the European Commission has granted orphan medicinal product designation to edasalonexent for the treatment of DMD. For a summary of clinical results reported to-date, please visit www.catabasis.com.

About DMD

Duchenne muscular dystrophy (DMD) is a disease that almost exclusively affects boys and whose incidence is 1 in 3,500. It is extremely rare that Duchenne muscular dystrophy (DMD) will affect girls. Those affected are usually diagnosed around the age of five, but symptoms may be visible from early childhood. It is a degenerative disease of the muscles caused by a genetic mutation. The Duchenne muscular dystrophy (DMD) – for which no treatment is currently available – directly affects skeletal muscles. Without treatment, the consequences of the disease are dire for those afflicted and their families.

For more information:

More information about the trial:  DMDtrials@catabasis.com and Catabasis -clinical trials

More: Portrait of Duchenne – edasalonexent cat-1004 – La Force DMD

Press release: www.catabasis.com

Business Wire: www.businesswire.com

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