What’s next for edasalonexent in 2020?

La Force is happy to share the latest edition of the Catabasis Connection newsletter about the Phase 3 PolarisDMD trial enrolled in the expected patient population and their partnership with Duchenne UK to evaluate edasalonexent in non-ambulatory boys and men affected by Duchenne.

A new Phase 2 non-ambulatory clinical trial in partnership with Duchenne UK!

Catabasis and Duchenne UK are partnering to study edasalonexent in a Phase 2 non-ambulatory Duchenne muscular dystrophy trial. Duchenne UK has granted over $600,000 in funding to support patient and clinical trial site costs. The exploratory Phase 2 study is designed to be a 1-year, randomized, double-blind, placebo-controlled trial to assess safety, pharmacokinetics, and exploratory measures of function including cardiac, skeletal muscle, and pulmonary function in non-ambulatory DMD patients. > Read more here.

To learn more about Duchenne UK, please visit their website: https://www.duchenneuk.org/

Phase 3 PolarisDMD trial enrolled expected patient population

With the Phase 3 PolarisDMD trial fully enrolled, Catabasis is sharing measurements and information about the boys enrolled from the beginning of the trial. The analysis of baseline characteristics shows that patients enrolled in the Phase 3 trial have similar characteristics to the patients that enrolled in the previous Phase 2 MoveDMD trial. Both trials enrolled boys affected by Duchenne ages 4-7 (up to 8th birthday) with any mutation type who had not been on steroids for the past 6 months. Baseline age, North Star Ambulatory Assessment score, and timed function test values (time to stand, 4-stair climb, and 10-meter walk/run) were similar in both trials. There were no significant differences in these baseline characteristics between the two trials.

Top-line results from the Phase 3 PolarisDMD trial are expected in Q4 2020 and are anticipated to support an NDA filing in 2021.

About Catabasis

The mission of Catabasis Pharmaceuticals is to bring hope and life-changing therapies to patients and their families. There lead program is edasalonexent, an NF-kB inhibitor in Phase 3 development for the treatment of Duchenne muscular dystrophy. For more information on edasalonexent and the Phase 3 trial, please visit www.catabasis.com.

About La Force DMD

The Force’s mission is to unite the DMD community to raise awareness around a common objective: that of providing access to new treatments as fast as possible and to participate in the funding of promising research projects. Where access to treatments for rare diseases is concerned, it is essential that our community be strong: each member must be an active spokesperson who helps raise awareness for DMD among the general public, as well as for the challenges associated with access to treatment.

 

Edasalonexent is an investigational drug that is not yet approved in any territory.

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