What does the drug do?
Arimoclomol works by protecting cells from accumulation of misfolded proteins by using the cell's natural defence mechanism, the heath shock response, resulting in production of protective heat-shock protein (HSPs) in stressed cells. HSPs can also direct the removal of the abnormal proteins when folding is not feasible. It is also able to cross the blood-brain barrier, which is often a roadblock for therapies that need to reach nerve cells in the brain.
The Phase 2 trial, which tested arimoclomol in 36 people with SOD1 MND, found that the treatment was safe and well tolerated by the participants. Indication of improvement in survival and ALSFRS-R was also observed. You can read more about the trial here.
The Phase 3 quadruple-blinded, placebo-controlled trial (ORARIALS-01) which initiated in August 2018, has fully enrolled 231 participants to test effectiveness of arimoclomol when given in form of oral capsules across 18 months. The primary outcomes will measure function and survival (using the Combined Assessment of Function and Survival (CAFS) measure), with additional outcomes of interest focusing on ALSFRS-R and SVC progression. Unfortunately, the trial did not meet its primary or secondary endpoints to show benefit in people living with MND. You can read more about the trial here.