Catabasis Pharmaceuticals Presents Data Supporting Edasalonexent as a Potential Foundational Treatment for Duchenne Muscular Dystrophy
-- Boys on Edasalonexent Grew More than Two Inches Taller per Year on Average --
“While the primary goal in treating boys with Duchenne is to slow the
progression of the disease, we recognize the significant negative impact
on boys’ quality of life when they do not grow and develop like their
friends. We are pleased to see boys on edasalonexent growing like their
unaffected peers while also demonstrating substantially slowed disease
In the MoveDMD trial, boys treated with edasalonexent grew an average of 2.1 inches taller per year and gained 2.9 pounds per year, and their overall body mass index decreased from 70th percentile of unaffected boys to the 55th percentile over 72 weeks of treatment, approaching the average body mass index for unaffected boys. Boys treated with corticosteroids, the standard of care in DMD, typically experience excess weight gain and curtailed growth.
“Using corticosteroids can be very beneficial to many patients with DMD.
However, alternatives are desperately needed as steroids come with a
long list of very unpleasant side effects, which includes stunted
growth. This can affect self-esteem and confidence. We are very
encouraged by this early data suggesting that boys on edasalonexent did
not have their growth affected, and we look forward to seeing more
Edasalonexent is being studied in the Phase 3 PolarisDMD trial, which is
actively enrolling patients. The PolarisDMD trial is evaluating the
efficacy and safety of edasalonexent in patients with DMD and is
intended to support an application for commercial registration of
edasalonexent. A total of 17 PolarisDMD clinical trial sites are now
open for enrollment across
The global Phase 3 PolarisDMD trial is a one-year, randomized, double-blind, placebo-controlled trial. Catabasis plans to enroll approximately 125 patients ages 4 to 7 (up to 8th birthday) regardless of mutation type who have not been on steroids for at least 6 months. Boys on a stable dose of eteplirsen may be eligible to enroll. The primary efficacy endpoint is change in the North Star Ambulatory Assessment score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints include the age-appropriate timed function tests: time to stand, 4-stair climb and 10-meter walk/run. Assessments of growth, cardiac and bone health are also included as important potential areas of differentiation. Two boys will receive 100 mg/kg/day of edasalonexent for each boy that receives placebo, and, after 12 months, all boys are expected to receive edasalonexent in an open-label extension study. The PolarisDMD trial design was informed by discussions with regulators as well as input from treating physicians, patient organizations and families of boys affected by Duchenne.
The Phase 3 PolarisDMD trial is designed to further validate the positive efficacy seen in the MoveDMD Phase 2 trial and open-label extension evaluating edasalonexent. In the MoveDMD trial, we observed that edasalonexent preserved muscle function and substantially slowed DMD disease progression across all four assessments of muscle function (the North Star Ambulatory Assessment, time to stand, 4-stair climb and 10-meter walk/run) through 72 weeks of treatment compared to an off-treatment control period. Preclinical data and clinical biomarker data from the MoveDMD Phase 2 trial suggest that edasalonexent could have potential benefits in skeletal muscle, diaphragm and heart. Edasalonexent has been well tolerated through more than 55 patient-years of treatment with no safety signals observed.
More information about the Phase 3 PolarisDMD clinical trial is available on clinicaltrials.gov and in a recently recorded webinar with PPMD. Contact the Catabasis clinical team with any questions at DMDtrials@catabasis.com.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential new standard of care for all patients affected by DMD, regardless of their underlying mutation. Edasalonexent inhibits NF-kB, which is a key link between loss of dystrophin and disease progression in DMD. NF-kB has a fundamental role in skeletal and cardiac muscle disease in DMD. We are currently enrolling our global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. In our MoveDMD Phase 2 trial and open-label extension, we 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. Edasalonexent continues to be dosed in the open-label extension of the MoveDMD trial. The
Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future clinical trial plans including, among other things, statements about the Company’s global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and safety of edasalonexent for registration purposes, and other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company’s product candidates; and general economic and market conditions and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the quarter ended
Catabasis Pharmaceuticals, Inc.
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