Orphazyme announced the phase 2/3 trial evaluating arimoclomol for the treatment of inclusion body myositis (IBM), a progressively debilitating muscle-wasting disease, did not meet its primary and secondary endpoints. The primary goal was to evaluate the treatment effect on disease progression as measured by the inclusion body myositis functional rating scale (IBMFRS).
The randomized, placebo-controlled trial enrolled 150 patients with IBM across 12 sites in North America and Europe. The trial was conducted in partnership with University College of London and the University of Kansas. Patients were randomized 1:1 to receive either arimoclomol (400 mg three times daily) or placebo for up to 20 months. No major safety concerns were detected in the trial.
Orphazyme is analyzing the data and will share the findings in a future scientific forum.
Arimoclomol amplifies the production of Heat Shock Proteins (HSPs). HSPs can rescue defective misfolded proteins, clear protein aggregates, and improve the function of lysosomes.
Arimoclomol has received orphan drug designation and fast-track designation from the U.S. Food and Drug Administration for IBM.
Inclusion Body Myositis (IBM) is a progressively debilitating muscle-wasting disease. IBM is characterized by a build-up of protein aggregates and atrophy of muscle cells, which leads to weakness and over time severe disability.