The French Biotech AB Science has announced positive results of a final Phase III trial analyzing its lead asset masitinib in patients with amyotrophic lateral sclerosis (ALS).

AB Science is a pharmaceutical company specializing in the clinical development of protein kinase inhibitors. Its lead asset, masitinib, is in clinical development for several indications and has now passed a final Phase III study revealing the efficacy and safety of masitinib in patients with ALS.

ALS is a degenerative disorder, which affects about 50,000 people in Europe. Almost 80% of ALS patients die within 5 years, revealing the need for new therapeutic options. AB Science was already granted Orphan Drug Designation for masitinib by both FDA and EMA and filed for marketing authorization at the EMA in September 2016.

This is a very good news for the patients. These final data confirm findings from the study’s interim analysis and proves that masitinib is capable of slowing down motoneuron degenerative disease such as ALS, which is a devastating condition with an urgent unmet medical need.” commented Alain Moussy, CEO of AB Science

The compound is an orally administered protein kinase inhibitor, which targets mast cells and macrophages by inhibiting a number of protein kinases within these cells. In the brain, the inhibitor also targets microglia, the resident macrophages of the brain, and can thereby inhibit inflammatory processes within the central nervous system.

Stephen Hawking

The famous scientist Stephen Hawking is living with ALS.

Based on the high medical need, several competitors are also working on new treatments for ALS. The FDA recently accepted an NDA filed by Mitsubishi Tanabe Pharma for Edaravone. This drug is a free radical scavenger, which has passed phase III trials for ALS last year. The FDA decision on the new compound is expected this summer.

In addition, US-based Cytokinetics recently completed the enrollment of a Phase III trial for its orphan drug Tirasemtiv. This compound is a skeletal muscle activator aimed to increase muscle strength in patients with ALS.

There is definitely a lot happening in the ALS field, but AB Science seems to have a lot of options with masitinib. The inhibitor has already completed Phase III studies in patients with pancreatic cancer and is currently in Phase II/III clinical development for cancer, inflammatory diseases as well as other neurodegenerative diseases such as Alzheimer’s.

Images via shutterstock.com / Minerva Studio; Flickr.com / Lwp Kommunikáció

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