Catabasis Pharmaceuticals Announces Top-Line Results for the Phase 3 PolarisDMD Trial of Edasalonexent in Duchenne Muscular Dystrophy
-- PolarisDMD Trial Did Not Achieve Primary or Secondary Endpoints --
“We are deeply saddened and disappointed by the results of our Phase 3 PolarisDMD trial,” said
The Phase 3 trial was a one-year placebo-controlled trial designed to evaluate the safety and efficacy of edasalonexent in boys ages 4-7 (up to 8th birthday) with DMD. The global trial enrolled 131 boys across eight countries, with any mutation type, who were not on steroids. Edasalonexent was well-tolerated, consistent with the safety profile seen to date. The majority of adverse events were mild in nature and the most common treatment-related adverse events were diarrhea, vomiting, abdominal pain and rash. There were no treatment-related serious adverse events and no dose reductions. The global COVID-19 pandemic had no meaningful impact on the trial or its results. Data from the PolarisDMD trial will be further analyzed and are expected to be presented at an upcoming scientific conference and published.
“These results are disheartening for the Duchenne community, and specifically for the boys who participated in this trial and their families. However, the results contribute to the natural history data of Duchenne and add to the knowledge base that will one day produce a foundational, long-term therapy for this disease,” said
The Company expects to report Q3 2020 financials in November of 2020. As of
Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about its exploration and evaluation of strategic options, the termination of activities related to the edasalonexent program, including the ongoing GalaxyDMD open-label extension trial, and plans to publish data from the Phase 3 PolarisDMD trial, and other statements containing the words “believes,” “anticipates,” “plans,” “hopes,” “expects,” and similar expressions, constitute forward-looking statements within the meaning of applicable securities laws and regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties related to: the impact of the COVID-19 pandemic and the effectiveness of the steps we have implemented to address the pandemic; the availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; unexpected costs or expenses that arise during the termination of activities related to the edasalonexent program; risks inherent in the Company’s exploration, evaluation and implementation of its review of strategic options; and general market and economic conditions; and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended