Ab Science in phase 3 of amytrophic lateral sclerosis treatment

AB Science pharmaceutical company, specialist in the protein kinase inhibitors (PKIs), said on Monday that it had the first successful phase 3 trial of a tyrosine kinase inhibitor in the treatment of amyotrophic lateral sclerosis (ALS). The company said the phase 2/3 study AB10015 of masitinib in ALS has met its pre-specified primary endpoint.

This finding signifies masitinib as first-in-class for ALS, with a unique mechanism of action against microglia cells, said AB Science.

Amyotrophic lateral sclerosis is a rare degenerative disorder that results in progressive wasting and paralysis of voluntary muscles. There are approximately 50,000 people with ALS in the European Union and in the US, with more than 16,000 new cases diagnosed each year in Europe and in the US. Almost 80% of ALS patients die within 5 years and 90% die within 10 years.

Study AB10015 was a double-blind, placebo-controlled phase 2/3 study to compare the efficacy and safety of masitinib in combination with riluzole, versus placebo in combination with riluzole in the treatment of patients suffering from ALS.

AB Science filed an application for marketing authorization of masitinib in ALS at EMA in September 2016.

Alain Moussy, CEO of AB Science said: “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.”

Professor Olivier Hermine, President of AB Science scientific committee said: “Perhaps the most impressive finding from this study is that masitinib has generated a significant difference in progression free survival with respect to the placebo treatment-arm. Similar to cancer studies when PFS is significantly improved, this indicates a clear clinical benefit in favor of masitinib.”

Dr. Jesús Mora Pardina, international coordinator of study AB10015 said: “Masitinib is one of the rare drugs developed for the treatment of ALS that has proved its efficacy through validated endpoints. These results are truly encouraging and can be considered as a major breakthrough for ALS treatment, a condition that has proved extremely challenging in the development of new effective medication. Masitinib may be the long-awaited addition to the therapeutic armamentarium of ALS.”

Masitinib received orphan drug designation for ALS from both EMA and FDA.