A phase 3 trial (NCT03491462) of arimoclomol (CytRx, Los Angeles, CA), an investigational treatment for amyotrophic lateral sclerosis (ALS) has completed enrollment. The trial is a 76-week, placebo-controlled study being conducted at 30 centers of excellence in North America and Europe. A total of 213 patients have been randomized 2:1 to receive arimoclomol or placebo.
Arimoclomol increases production of heat shock proteins (HSPs) known to reccue defective misfolded proteins, clear protein aggregates, and improve lysosomal function. The drug is administered orally, crosses the blood-brain barrier, and has been studied in 7 phase 1 and 3 phase 2 clinical trials. Top-line results from this first phase 3 study are expected in the first half of 2021.
Arimoclomal is also being tested for other conditions, including Niemann-Pick disease type C (NPC), Gaucher’s disease, and sporadic inclusion body myositis (sIBM).
"We continue to be impressed with the progress our partner Orphazyme is making with the clinical development of arimoclomol," said Eric Curtis, president and CEO, CytRx. "ALS is a devastating disease with few treatment options currently available, and we remain hopeful that arimoclomol could become a life-changing drug for these patients.”