Enrollment is complete for a phase 3 clinical trial of arimoclomol (Orphazyme, Copenhagen, Denmark), an investigational therapy for treating amyotrophic lateral sclerosis (ALS). The ORARIALS-01 trial (NCT03491462) is a randomized, placebo-controlled study to evaluate the efficacy and safety of arimoclomol over a 76-week treatment period. Participants with ALS who had first appearance of symptoms (weakness) within the previous 18 months were eligible for screening and have been randomly assigned in a 2:1 ratio to receive either arimoclomol or placebo orally. After the first patient was enrolled in the trial in August 2018, recruitment was completed in 11 months from 30 centers in North America and Europe.
Arimoclomol is amplifies production of heat-shock proteins (HSPs) involved in rescuing defective misfolded proteins, clearing protein aggregates, and improving lysosomal function. Arimoclomol crosses the blood-brain barrier and has been studied in 7 Phase 1 trials and 3 Phase 2 trials.
Arimoclomol, which is in clinical development for Niemann-Pick Type C (NPC) disease, Gaucher disease, sporadic inclusion body myositis (sIBM), and ALS, has been granted Orphan Drug status in Europe and the US for the treatment of ALS.
Margaret Yu, MD; Christian Sikorski, MD; Craig M. Horbinski, MD; and Rimas V. Lukas, MD
Reginald Lafleur, MD; Melissa Lafleur, MD; Steven Mandel, MD; and Jason A. Ellis, MD
Catherine L. Gallagher, MD