Searching for a cure, Improving lives

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.

Leave a Reply

Your email address will not be published. Required fields are marked *

Donate

Support Action Duchenne with a monthly or one off donation

Donate

Events

View our events

View All Events

Registry

Join the DMD registry for access to clinical trials

Register

Action Duchenne launches new Research Strategy for Duchenne muscular dystrophy

November 17th, 2017

  Action Duchenne, a leading UK-wide patient and parent-led organisation for Duchenne Muscular Dystrophy, is

Tell me more

Long-term microdystrophin pre-clinical work, moving towards a potential therapy restores muscle function in canines

July 26th, 2017

Promising microdystrophin pre-clinical work, moving towards a potential therapy has shown to restore muscle function

Tell me more

FDA new approach would reduce number of patients treated with placebo

December 11th, 2017

The U.S. Food and Drug Administration (FDA) has published its recommendation for a new approach

Tell me more

Results announced from survey ‘Juggling care and daily life’

December 11th, 2017

The 17th October 2017 was an important date for the rare disease community.  The highlights of the

Tell me more

Duchenne – How do you measure a life?

Living with Duchenne – an animation

Taking charge of Duchenne

wordpress counter

Stay informed with regular updates from Action Duchenne