Kinase Inhibitor, Masitinib, Spotted for Potential to Treat Neurological Disorders Like PPMS

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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AB Science recently reported the publication of four peer-reviewed and independent research papers that add to the growing recognition of masitinib, the company’s lead compound, as a promising treatment for neurological and neurodegenerative diseases, including progressive multiple sclerosis (MS), Alzheimer’s disease (AD), and amyotrophic lateral sclerosis (ALS).

Tyrosine kinase inhibitors (TKIs) like masitinib are compounds that target a class of enzymes, tyrosine kinases, which are responsible for the activation of other proteins in a number of cell signaling pathways. Blocking the selected activation of such proteins has been shown to have therapeutic benefits in numerous diseases, such as cancer, inflammatory diseases, and central nervous system (CNS) disorders.

AB Science has been developing compound candidates that both inhibit targeted tyrosine kinases known to be involved in specific diseases and limit toxicity by avoiding the deactivation of kinases necessary for normal cell function.

According to a company press release, masitinib, an orally administered TKI, meets those two goals. It targets mast cells and macrophages, immune cells involved in numerous important, and sometimes harmful, immune responses. Moreover, mast cells and microglia are the main cells of the CNS immune system, so the drug has the potential to treat symptoms associated with diseases like primary progressive MS, which, unlike relapsing-remitting MS, currently has no approved disease-modifying therapies.

The reported publications noted masitinib as a promising compound in neurology based on mechanism of action data from animal models, published Phase 2 proof-of-concept studies, and analyses in ongoing Phase 3 studies, a key milestone for diseases such as progressive MS, ALS, and AD, which have seen little to no success in clinical studies in the last 10 years.

The publications include a recent review, “Therapeutic Advances and Future Prospects in Progressive Forms of Multiple Sclerosis,” which discussed therapeutics under Phase 2 or Phase 3 investigation for progressive forms of MS, and highlighted masitinib as the lead candidate in the class of TKIs.

Other publications include “The latest innovations in the drug pipeline for multiple sclerosis,” a review of investigational drugs in ongoing Phase 3 studies that may be significant additions to therapy with armamentarium. Masitinib is discussed due to its oral administration route and Phase 2 published data on the difficult-to-treat primary progressive multiple sclerosis (PPMS). The use of masitinib for multiple sclerosis is also discussed in the review, “Tyrosine Kinase inhibitor as a new therapy for ischemic stroke and other neurologic diseases: is there any hope for a better outcome?

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