Arimoclomol is an ‘investigative medicinal product’, meaning it is not an approved medicine. We are conducting clinical studies to investigate how effective arimoclomol is as a treatment for a number of diseases. To be approved, all new therapies must be effective and must not cause undue harm. We have studied arimoclomol’s effects thoroughly in the laboratory and are now investigating its effects and safety in patients.
Arimoclomol is a small molecule, which readily distributes around the body, including crossing the blood-brain barrier. It is soluble, and has almost no taste or smell, which means it can easily be dissolved in a drink or suitable food.
Many lysosomal storage diseases are caused by mutations in a protein involved in lipid digestion and transportation. This causes an accumulation of lipid in compartments of the cell called lysosomes, which in turn leads to cell stress and toxicity. When stressed cells are treated with arimoclomol, they are stimulated to produce a natural chaperone called Heat Shock Protein 70 (HSP70), which helps to fold the digestive enzyme into a functional conformation, allowing the cell to process the accumulated lipid.
In rare circumstances, some proteins are vulnerable to aggregation, meaning they clump together and don’t get recycled properly. Aggregations of misfolded proteins cause cell stress, and eventually cell death. When stressed cells are treated with arimoclomol, they are stimulated to produce a natural chaperone called Heat Shock Protein 70 (HSP70), which helps to guide aggregated proteins into the recycling pathway and prevents newly-built proteins from aggregating.
The safety profile of arimoclomol has been extensively explored and confirmed in multiple pre-clinical and phase I clinical studies. The compound has been investigated in pre-clinical toxicity studies, in extensive Phase I studies in healthy adults as well as in Phase II studies in ALS. A total of more than 200 patients have been exposed to arimoclomol for up to a year. No major safety concerns were observed in any of the patients as a result of arimoclomol.