Drug intelligence / Profile preview

masitinib + riluzole

Development stage
Unknown
Lead developer
AB Science
Modality
Small Molecules
Administration
Oral
01

Overview

Masitinib + riluzole is a combination of two oral small molecule drugs used in the investigational treatment of amyotrophic lateral sclerosis (ALS)[1][2][3]. - Masitinib is a selective tyrosine kinase inhibitor that targets mast cells and macrophages, modulating neuroinflammatory responses and exerting neuroprotection in central and peripheral nervous systems by inhibiting various kinases implicated in disease pathogenesis[2][3]. - Riluzole is a benzothiazole derivative that acts mainly by inhibiting glutamatergic neurotransmission through antagonism of voltage-gated sodium channels and reducing presynaptic glutamate release, providing modest survival benefit in ALS[2][4]. This combination has shown a statistically significant delay in disease progression and improved survival in ALS patients compared to riluzole monotherapy in phase 2/3 trials, most notably for patients with mild-to-moderate disease[2][3]. The combination is under late-stage clinical development, with positive efficacy and safety signals but notable risk of severe adverse events associated with masitinib (such as hepatotoxicity and ischemic heart disease), requiring close monitoring[2][4].

Brand names
Alsitek (masitinib)Rilutek (riluzole)
Other names
masitinib + riluzole
02

Targets

PDGFRA (Platelet-derived growth factor receptor alpha)LYN (LYN proto-oncogene, Src family tyrosine kinase)FYN (Proto-oncogene tyrosine-protein kinase Fyn)CSF1R (Macrophage colony-stimulating factor receptor)PDGFRB (Platelet-derived growth factor receptor beta)KIT (c-KIT proto-oncogene receptor tyrosine kinase)

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