What does the drug do?

Masitinib inactivates a cellular enzyme called tyrosine kinase, which is believed to be involved in tissue inflammation. While inflammation of the central nervous system may not be a direct cause of MND, it is likely to play a role in the speed of disease progression. Reducing the activity of inflammatory cells in the brain may reduce the damage to motor neurones.

Phase 3

The Phase 3 trial is a randomised, double blind, placebo controlled trial. It is looking to investigate the efficacy, measured by ALSFRS-R, of masitinib in combination of riluzole.  Participants will be given two doses (4.5 and 6mg/kg/day) in combination with riluzole. You can read more here.

This trial is recruiting in the US and Europe only.

Previous Clinical Trials

Phase 2/3

The Phase 2/3 trial examined the safety and effectiveness of masitinib in combination with riluzole in 394 people with MND, when taken for nearly a year. AB Science, the company behind masitinib, has published potentially promising results, showing improved ALSFRS-R score when given 4.5mg/kg of masitinib per day for people with MND with "normal" progression. You can read more about the trial here.

Additional analysis was conducted, including long-term survival. The long-term survival data was collected from participants who received either the drug or placebo, alongside riluzole, over an average of 34.1 months. Researchers placed participants from the trial into groups based on their rate of disease progression and disease severity. They defined normal progression as people living with ALS who had a change of less than 1.1 points per month on the ALSFRS-R scale. Moderate ALS was defined as people living with ALS with a starting ALSFRS-R score of greater than or equal to 2 in all categories. Participants who had both normal progression and moderate ALS were found to have a potential increase in survival of approximately 25 months when taking 4.5mg/kg of masitinib alongside riluzole, when compared to those who took placebo and riluzole. Results also suggested a potential slower decline in ALSFRS-R score per month. You can read more about the trial here.

Latest News


January 2024 - The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has requested AB Science to submit a written response to the List of Outstanding Issues. This has pushed back the timeline to a decision on whether to approve masitinib for the treament of MND in Europe. A decision is now expected before mid-2024.


Dec 2022 - Health Canada has asked for more information about Masitinib before continuing to review the request for its approval. Find out more here


May 2022 - AB Science confirmed that Health Canada is now reviewing the new drug submission application for Masitinib. You can read more here.


Feb 2022 - AB Science confirmed that Health Canada granted authorisation to file a new drug submission for Masitinib. You can read more here.


Sept 2021 - AB Science confirmed that the study was allowed to resume, with some enhanced measures in place to ensure participant safety. You can read more here.


June 2021 - AB Science filed for a temporary hold on all ongoing studies until concluding an investigation of a potential risk of ischemic heart disease associated with masitinib use. You can read more here.

Last updated: 29/01/2024