The protein kinase inhibitor masitinib is showing promise as an ALS therapy, according to a press release by the pharmaceutical company AB Science. However, the interim results were have not yet been disclosed, and will likely only be reported after the completion of the trial.
The company conducted interim analysis of an ongoing double-blind, placebo-controlled Phase II/III clinical trial in ALS after nearly 200 subjects (approximately 50% of the target enrollment) had reached the designated 48-week time point since treatment initiation. The company reports that ALS patients treated with masitinib and riluzole exhibited statistically significant improvements over placebo and riluzole-treated patients according to several endpoints, including the primary endpoint of change from baseline in Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) and the secondary endpoint of change from baseline in forced vital capacity (FVC).
Based on pre-clinical studies, the drug acts by blocking pathological mast cell and microglial activation, thereby exerting anti-inflammatory effects that improve motor neuron survival.