Published Arimoclomol SOD1 Phase II trial results.

This trial employed a novel enrichment design to enroll an etiologically, biologically, and phenotypically homogeneous population.

The trial demonstrated that, using an appropriate enrichment design, it is possible to observe a therapeutic signal even with a small sample size.

The promising finding of this phase II trial has led to the launch of Orphazyme’s Arimoclomol Phase III trial in the general ALS population, with Benatar as the International Coordinating Investigator.