Arimoclomol is a treatment that is in development for ALS, Niemann-Pick disease type C (NPC), and inclusion body myositis (IBM). A recent Phase 2/3 trial has ended in failure, unable to meet primary or secondary endpoints within IBM patients. While this news is disappointing, Orphazyme will continue to investigate and develop arimoclomol for ALS and NPC.
About Inclusion Body Myositis (IBM)
Inclusion body myositis (IBM) is a progressive disorder that impacts the muscles, characterized by inflammation, atrophy, and feebleness. It typically impacts adults when they reach 50. Affected individuals will experience myalgia, rimmed vacuoles, tripping and falling, muscle weakness and atrophy, ragged-red muscle fibers, elevated serum creatine phosphokinase, and feeding difficulties in infants. The thighs, fingers, and wrists are the most heavily affected. Medical professionals are unsure as to what causes these symptoms, although they suspect that there are environmental, genetic, and immune-related factors. They do not think that it is hereditary, although one may be genetically predisposed. In terms of treatment, there is no cure. It is symptomatic. Affected individuals typically require occupational or physical therapy, walking aids, or other devices to aid with movement.
About the Study
Researchers were focusing on the primary endpoint of the Phase 2/3 study: arimoclomol’s impact on disease progression evaluated by the inclusion body myositis functional rating scale (IBMFRS). Not only was this goal unmet, but so were all secondary endpoints.
15o patients from 12 sites across the United States and Europe enrolled in the trial, which was run by Orphazyme in collaboration with the University of Kansas and University College of London. These patients were separated randomly into two groups; one half was given 400 mg of arimoclomol three times a day while the other was given a placebo. Both groups were followed for twenty months.
While the results of this trial are very disappointing for researchers and patients alike, the data collected is still helpful and can be applied to future research. Additionally, arimoclomol is still being investigated for ALS and NPC. Data from a Phase 3 trial with ALS patients is expected soon, and an application is under priority review with the FDA for the treatment of NPC. Looking to Europe, the Committee for Medicinal Products for Human Use (CHMP) is expected to give its opinion as well.
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