Masitinib in Phase III Trial for ALS Treatment

AB Science just announced that their Phase II clinical trial of masitinib for the treatment of ALS has been advanced into a Phase III trial. Masitinib (marketed as Kinavet in the US) is an inhibitor of the c-kit tyrosine kinase and was originally developed for the treatment of tumors in dogs. Now, masitinib is being tested in humans for the treatment of multiple indications, including cancer, inflammatory diseases, and central nervous system disorders, such as multiple sclerosis and ALS. Masitinib works by targeting and inhibiting “the survival, migration and activity of mast cells,” which are involved in the inflammatory response and regulate the permeability of the blood-brain barrier. Neuroinflammation is a hallmark of ALS, and potentially reducing neuroinflammation via masitinib might provide some therapeutic benefit in ALS. The original Phase II study in ALS involved the recruitment of 45 patients, and the Phase III will expand the recruitment to 210 patients and will evaluate the safety and efficacy of masitinib for the treatment of ALS. The results of the Phase III study are expected in 2015.

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