In Research

One of the objectives of our team is to inform you about new treatments. Here’s the latest news about Polaris DMD, the clinical trial of edasalonexent, developed by Catabasis. Thank you for sharing these articles in the DMD community.

 

Maria Mancini, vice president of clinical operations at Catabasis: We named the trial ‘Polaris’ because it is the brightest star in its constellation and it’s also known as the North Star.

 

What is Polaris DMD?

Polaris DMD is a phase 3 trial of edasalonexent, a potential treatment for Duchenne muscular dystrophy (DMD), regardless of mutation type, is enrolling boys ages 4 to 7 in the U.S. Registration information is available here > Clinicaltrials.gov.

The Polaris DMD Trial is a one-year, randomized, placebo-controlled trial. Catabasis began evaluating patients on October 2 at several sites in the United States. Catabasis plans to conduct the trial at nearly 40 sites around the world. The results are expected in June 2020.

 

Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis: We are very excited to advance edasalonexent through this potentially last phase of clinical development with the hope of providing a new treatment option to all boys affected by this disease. We believe that edasalonexent has great potential as a therapy to be taken on its own as well as in combination with other treatments.

 

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.

 

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.

 

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 Catabasis: www.catabasis.com

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

Muscular Dystrophy News: www.musculardystrophynews.com

More information about the Polaris DMD trial: Catabasis – Polaris DMD

Business Wire: www.businesswire.com

 

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