Full dataset for Phase II/III trial of arimoclomol in Niemann- Pick disease.- Orphazyme A/S

Orphazyme A/S, announced the full data set for its Phase II/III clinical trial of arimoclomol in NPC (Niemann- Pick disease), a devastating rare genetic disorder. Treatment with arimoclomol adjunct to routine clinical care resulted in a 74% reduction in disease progression (p-value =0.0506) as measured by the primary endpoint, 5-domain NPC Clinical Severity Scale (NPC-CSS).

In the predefined subgroup of patients of 4 years and older (44 out of 50 randomized patients in the trial), the treatment difference was statistically significant with a minimal disease progression at month 12 in the arimoclomol-treated group (p-value =0.0219). A highly statistically significant treatment difference was observed in another predefined subgroup analysis, requested by the European Medicines Agency (EMA), that compared arimoclomol to placebo control in patients receiving miglustat as a part of routine clinical care (p-value =0.0071).

Comment: 24-month data is expected to become available from the on-going open-label extension of the trial in Q3 2019. Orphazyme plans for submission of filing in the US and EU in H1 2020, with potential approval in H2 2020.