Orphazyme: Follow-up Note to Failure in ALS

Redeye provides a follow-up note after listening to the presentation of the top-line results with arimoclomol in ALS. The results were presented at the ENCALS virtual meeting. While we are pending data on biomarkers and SOD1 subgroup analysis, it is unlikely to change our view of the neuromuscular projects. We continue to exclude arimoclomol-sIBM and arimoclomol-ALS and reiterate our Base Case.

All eyes are now on the FDA's decision to potentially approve Miplyffa (arimoclomol) in the ultra-rare disorder, Niemann-Pick Disease type C (NPC). This is due in just a few weeks.