Monepantel is a well-known veterinary drug. It has been shown to have an effect on the abnormal accumulation of protein in cells. This accumulation is associated with motor neurone disease. Monepantel works by increasing the recycling and removal of excessive or abnormal proteins.
Results from phase 1 of this trial suggest that monepantel was well tolerated. In February of 2024, the pharmaceutical running the trial, PharmAust Ltd, announced that the phase 1 trial had moved to an Open-Label Extension program. This means that the 12 patients who took part in the phase 1 trial will continue on the medication and will continue to be monitored.
A Phase 2/3 of this trial is commencing in 2024. Unfortunately, there are no Australia sites for this phase of the monepantel trial. The therapy is being included in the US-based Healey ALS Platform trial.
For additional information on how this therapy works you can read a summary from the ALS Therapy Development Institute (ALS TDI).
