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Positive Preclinical Research on the Edasalonexent (CAT-1004) Program

Posted on: January 6th, 2017 | 0 comments


Catabasis announced the publication of preclinical data on the edasalonexent program, a potential disease-modifying therapy for Duchenne muscular dystrophy. Edasalonexent (CAT-1004) is an oral small molecule that has the potential to be a disease-modifying therapy for all patients affected by Duchenne muscular dystrophy (DMD or Duchenne), regardless of their underlying mutation.

The preclinical data demonstrates that edasalonexent is effective in ameliorating the dystrophic process in two animal models (dog and mouse) with Duchenne. This research was led by H. Lee Sweeney, Ph.D., then at the University of Pennsylvania. He said the following about the drug:

“There remains a large unmet need in Duchenne for therapies that can treat all affected boys and slow disease progression. The orally bioavailable NF-kB inhibitors, edasalonexent and CAT-1041, improve the severe dystrophic phenotype found in both mechanically-damaged mdx mice and a GRMD dog and create an environment that can support more successful muscle regeneration. We believe that these in vivo preclinical results support edasalonexent as a candidate for the treatment of DMD.”

The full story can be found here. Action Duchenne will be following this closely and will keep the community updated on developments.

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