Orphazyme announces topline data from pivotal trial of arimoclomol in Amyotrophic Lateral Sclerosis (ALS)
Orphazyme A/S - American Depositary Shares (ORPH)
Company Research
Source: GlobeNewswire
Orphazyme A/SCompany announcementNo. 14/2021Inside informationCompany Registration No. 32266355 Pivotal trial did not meet primary and secondary endpoints evaluating impact on function and survival Orphazyme remains focused on commercial readiness and potential U.S. approval of arimoclomol for Niemann-Pick disease type C (NPC) in June Copenhagen – May 7, 2021 – Orphazyme A/S (ORPHA.CO; ORPH), a late-stage biopharmaceutical company pioneering the heat shock protein response for the treatment of rare diseases, today announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. No important safety signals were reported in the trial. Topline data will be presented at the upcoming virtual European Network to Cure ALS (ENCALS) meeting, May 12-14, and complete data from the study will be published later this year. “We are disheartened by these resu
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