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Orphazyme Shares Plummet As Arimoclomol Flunks In Amyotrophic Lateral Sclerosis Study

Orphazyme Shares Plummet As Arimoclomol Flunks In Amyotrophic Lateral Sclerosis Study
  • Orphazyme A/S (NASDAQ: ORPH) has announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints.
  • No important safety signals were reported in the trial.
  • Topline data will be presented at the upcoming virtual European Network to Cure ALS meeting next week.
  • Complete data from the study will be published later this year.
  • 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).
  • Orphazyme’s applications for arimoclomol for Niemann-Pick disease type C (NPC) are under priority review with the FDA, with an expected PDUFA action date of June 17, as well as with the European Medicines Agency, with an opinion from the Committee for Medicinal Products for Human Use (CHMP) expected later this year.
  • In March, Arimoclomol failed to meet the primary and secondary endpoints in Phase 2/3 trial in inclusion body myositis patients.
  • Price Action: ORPH shares are down 28.7% at $6.10 on the last check Friday.

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