CytRx Corporation (OTCQB:CYTR) stock sheeded 33% after noting that Orphazyme A/S (NASDAQ:ORPH) (“Orphazyme”) announced that the ORARIALS-01 pivotal trial for arimoclomol in Amyotrophic Lateral Sclerosis (“ALS”) did not meet primary and secondary endpoints to show benefit in people living with the disease. According to Orphazyme, no important safety signals were reported in the trial. Topline data will be presented at the upcoming virtual European Network to Cure ALS meeting and complete data from the study will be published later this year. The stock closed at $5.75, down $2.81.
As part of the announcment, Orphazyme’s disclosed that the randomized, placebo-controlled Phase 3 trial was conducted among 245 patients at 29 sites in 12 countries in North America and Europe. Participants were randomized (2:1 ratio) to receive either arimoclomol (248 mg three times daily) or placebo for up to 76 weeks. The primary endpoint was to determine the efficacy of chronic treatment with arimoclomol compared to placebo in participants with ALS as assessed by the combined assessment of function and survival (CAFS). This endpoint was selected to illustrate the overall treatment effect based on survival and the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. Secondary endpoints included survival, change in ALSFRS-R, and slow vital capacity (SVC).
Notably, Orphazyme’s applications for arimoclomol (to be branded MIPLYFFA™) for Niemann-Pick disease type C are under priority review with the U.S. Food and Drug Administration, with an expected PDUFA action date of June 17, 2021, and with the European Medicines Agency, an opinion from their Committee for Medicinal Products for Human Use is expected later this year.
CytRx will continue to provide updates that are relevant to our agreement with Orphazyme.
