Orphazyme’s latest trial has ended in failure after arimoclomol, a possible treatment of inclusion body myositis (IBM), failed to meet any of its endpoints. As the drug has shown massive success in the past, especially when indicated for amyotrophic lateral sclerosis (ALS), this failure comes as a surprise to researchers. They do, however, believe that the data gathered during the trial will be helpful for further research and treatments of IBM.
About 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 Arimoclomol
This treatment is intended to strengthen the production of Heat Shock Proteins (HSPs), as their function is to get rid of protein aggregates, repair misfolded proteins, and improve the functioning of lysosomes. It has been indicated as a treatment for numerous neurodegenerative conditions besides IBM, such as amyotrophic lateral sclerosis, Niemann-Pick disease type C (NPC), and Gaucher disease.
Already, the FDA and European Commission have granted it the orphan drug designation as a medication for ALS, NPC, and IBM. Solely in America, it has received the fast track designation as well. Its last designations are the rare pediatric disease and breakthrough therapy, which were granted by the FDA for the treatment of NPC.
Failed Trial
150 participants across the United States and Europe participated in the trial, all of whom were randomized in a 1:1 ratio to receive either 400 mg of arimoclomol three times a day or a placebo. For up to 20 months, they were observed in relation to the primary endpoint, which was the drug’s effect on disease progression as measured by the inclusion body myositis functional rating scale (IBMFRS).
Unfortunately, arimoclomol did not meet this endpoint or any secondary endpoints. Researchers are disappointed by this outcome and recognize how it affects patients and their families as well. They know that there is an unmet medical need for these people, and they hoped to finally fill this gap with arimoclomol. While this did not happen, they believe that the data gathered will be useful for further research.
Looking Forward
While this trial failed, Orphazyme is not abandoning arimoclomol. Later this spring, they expect data from a Phase III trial of ALS patients. The FDA is currently reviewing it as a treatment for NPC as well, with their decision expected in June.
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