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Catabasis begins recruiting for Phase 3 PolarisDMD trial -125 participants which includes patients currently taking Exondys51Catabasis currently proposing reverse split see recent SEC documents anywhere from 1 to 5 to 1 to15. https://www.sec.gov/Archives/edgar/data/1454789/000104746918006698/a2236896zpre14a.htm Clinical trial listed October 12, 2018 From Catabsis second quarterly report with the SEC: "The POLARIS DMD trial will be a randomized, double-blind, placebo-controlled trial, and we anticipate enrolling approximately 125 patients between the ages of four and seven (up to eighth birthday), regardless of mutation type, who have not been on steroids for at least six months. Boys may be eligible to enroll in the trial if they are on a stable dose of EXONDYS 51®, also known as eteplirsen, Sarepta Therapeutics, Inc.’s exon skipping therapy and one of two therapies approved for the treatment of DMD in the United States. The primary efficacy endpoint will be change in North Star Ambulatory Assessment, or NSAA, score after 12 months of treatment with edasalonexent compared to placebo. Key secondary endpoints are planned to 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 planned. Enrolled boys will be randomized in a 2:1 ratio with two boys receiving edasalonexent for every boy that receives placebo, and we expect that after the initial 12-month treatment period all boys will be offered the opportunity to receive edasalonexent in an open-label extension. From Sarepta's docs: Catabasis 10-K - "In September 2016, we entered into a joint research collaboration with Catabasis Pharmaceuticals, Inc. (“Catabasis”) to explore a combination drug treatment approach for DMD. We will contribute our expertise to study an exon skipping treatment while Catabasis will contribute its oral NF-kB inhibition treatment for this combination study for DMD. The objective of the joint research is to study the safety and efficacy of combining these two treatment strategies using a mouse model of DMD, including evaluating the potential for additional or synergistic benefits." September 29, 2016 Press Release: "We are excited to work with Sarepta on this joint research collaboration, which to our knowledge is the first time two companies are testing a combination of investigational therapies to treat Duchenne. Although we believe edasalonexent (CAT-1004) has the potential to be a disease-modifying monotherapy, we think there is benefit to exploring innovative ways to make the most meaningful difference in this devastating disease”, said Jill C. Milne, Ph.D., chief executive officer of Catabasis. “In addition to our continued development of edasalonexent, we are pleased to take the first step via this collaboration to determine if edasalonexent may be complementary to an exon-skipping treatment strategy in the treatment of DMD using a preclinical model.” |
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