Catabasis Pharmaceuticals Presents Information on Edasalonexent, a Potential Foundational Therapy for Duchenne Muscular Dystrophy, at the Virtual 25th International Congress of the World Muscle Society
-- New Preclinical Research Indicates that Edasalonexent Could Have Positive Effects on
“As we approach top-line Phase 3 PolarisDMD results, which we expect to report in the fourth quarter of this year, we are pleased to share new findings from preclinical studies with edasalonexent that could support its potential as a foundational therapy for those affected by Duchenne,” said
Persistent activation of NF-kB in DMD can drive cardiac dysfunction, which is the leading cause of mortality in DMD. In a preclinical study performed in the laboratory of
In DMD, NF-kB activation also drives inflammation and fibrosis, leading to loss of skeletal muscle function and disease progression. Reduced skeletal muscle function results in reduced bone strength. Steroids, which activate the glucocorticoid receptor (GR), can further negatively impact bone health. In a preclinical study led by
Information from the edasalonexent clinical program was also presented at the
An additional evaluation of data from patients at baseline (prior to any treatment) in the Phase 3 PolarisDMD trial was shared by
As Catabasis has previously presented, the Phase 2 MoveDMD trial and open-label extension was also shared showing that edasalonexent was well-tolerated for up to 150 weeks and associated with favorable growth patterns. The most common related adverse effect was diarrhea, generally mild and transient.
The posters are available to registered attendees during the conference and for three months afterwards. Select posters are available in the “Our Science” section of www.catabasis.com.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule designed to inhibit NF-kB that is being developed as a potential foundational therapy for all patients affected by DMD, regardless of their underlying mutation. In DMD the loss of dystrophin leads to chronic activation of NF-kB, which is a key driver of skeletal and cardiac muscle disease progression. The ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy and safety of edasalonexent for registration purposes. Edasalonexent is also being evaluated in the GalaxyDMD open-label extension trial. In the MoveDMD Phase 2 trial and open-label extension, the Company observed that edasalonexent preserved muscle function and substantially slowed disease progression compared to rates of change in a control period, and significantly improved biomarkers of muscle health and inflammation. The FDA has granted orphan drug, fast track, and rare pediatric disease designations and the
Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about the clinical program of edasalonexent, including the expected timing of release of Phase 3 data, edasalonexent potentially being a foundational therapy for DMD patients, and edasalonexent potentially having positive effects on cardiac function and preservation of bone health, along with other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements within the meaning of applicable securities regulations and laws. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties: inherent in the initiation and completion of clinical trials and clinical development; related to whether the results of pre-clinical studies will be predictive of results of clinical trials; related to whether the results of earlier stage clinical trials will be predictive of the results of later stage trials; related to the regulatory review and approval process; inherent in the commercialization of marketed products; related to competitive products, including those already approved and those in development; related to other matters that could affect the clinical development, regulatory status, availability or commercial potential of edasalonexent, as well as the risks and uncertainties discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended