Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial for Edasalonexent in Duchenne Muscular Dystrophy
-- Pivotal Study of Novel Inhibitor of NF-kB, a Key Driver of Skeletal Muscle Disease and Cardiomyopathy in Duchenne --
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PolarisDMD clinical trial sites across
“We are very excited to initiate our Phase 3 PolarisDMD trial as we
believe edasalonexent has tremendous potential to become the new
standard of care for all affected by Duchenne, regardless of mutation
type and from the time of diagnosis throughout their lifespan,” said
The 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 will be 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. 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.
“We are so glad to bring edasalonexent into Phase 3 for boys affected by
Duchenne that could benefit from this potential therapy,” said
“PPMD has conducted preference studies of those impacted by Duchenne to
understand what is most important for novel therapies. Families
responded that their highest priority is to stop or even slow disease
progression and maintain muscle function,”
Edasalonexent has been shown to preserve muscle function and substantially slow 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) compared to control in the MoveDMD® Phase 2 trial and open-label extension. 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 45 patient-years of treatment with no safety signals observed.
More information about the Phase 3 PolarisDMD clinical trial will be available on www.clinicaltrials.gov in the coming days or contact the Catabasis clinical team via email 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 the key link between loss of dystrophin and disease progression. NF-kB has a fundamental role in skeletal and cardiac muscle disease in DMD. Catabasis is currently enrolling the single global Phase 3 PolarisDMD trial to evaluate the efficacy and safety of edasalonexent for registration purposes. Edasalonexent continues to be dosed in an open-label extension of the MoveDMD Phase 2 clinical 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
Investor and Media Contact
Andrea Matthews, 617-349-1971
Catabasis Pharmaceuticals, Inc.