FDA clears masitinib IND in Amyotrophic Lateral Sclerosis (ALS), allowing U.S. patient enrollment to commence in Phase 3 study
AB Science SA (NYSE Euronext - FR0010557264 - AB) today announces that the U.S. Food and Drug Administration (FDA) has cleared the company's Investigational New Drug (IND) application, allowing the Company to initiate its masitinib Phase 3 study (AB19001) in amyotrophic lateral sclerosis (ALS).
“That is really a good news for all of us and in particular for the patients and we aim to initiate the Phase 3 confirmatory trial in ALS as soon as conditions at U.S. clinical sites stabilize post-the coronavirus pandemic,” said Alain Moussy, co-founder and CEO of AB Science.
Study AB19001 is an international, multicenter, randomized, double-blind, placebo-controlled, 3-parallel group, Phase 3 study to compare the efficacy and safety of masitinib in combination with riluzole versus placebo in combination with riluzole for the treatment of patients suffering from ALS.
The study’s primary endpoint is the absolute change from baseline in functional score as assessed using the Amyotrophic Lateral Sclerosis Functional Rating Scale-revised (ALSFRS-R) after 48 weeks of treatment. The main secondary endpoint is the Combined Assessment of Function and Survival (CAFS).
The study will enroll 495 patients randomized to one of three treatment groups in a 1:1:1 ratio.
Group 1: Masitinib titration starting at 3.0 mg/kg/day and escalating to 4.5 mg/kg/day, plus riluzole
Group 2: Masitinib titration starting at 3.0 mg/kg/day and escalating to 4.5 mg/kg/day and then to 6.0 mg/kg/day, plus riluzole
Group 3: Matching placebo, plus riluzole
Study AB19001 is intended to confirm the previously published results [1] from the first Phase 2/3 study (AB10015) which demonstrated that masitinib at 4.5 mg/kg/day in combination with riluzole significantly slowed ALSFRS-R decline by 27% compared to riluzole alone at week 48 (p-value < 0.05).
Evidence of a dose-response was observed in the previous AB10015 study with maintenance doses of 3.0 and 4.5 mg/kg/day, with an acceptable safety profile. Therefore, this confirmatory trial will evaluate an even higher 6.0 mg/kg/day dose in one of the two active arms.
The design of AB19001 Phase 3 trial has benefited from protocol assistance, the special form of scientific advice available for developers of designated orphan medicines, obtained from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency's (EMA).
The rationale to use masitinib in ALS patients is supported by a strong body of evidence demonstrating that the pharmacological action of masitinib in microglia and mast cells can slow microglial-mediated disease progression, reduce neuro-inflammation, and modulate the degenerative neuronal microenvironment in both central (CNS) and peripheral nervous systems (PNS) [2–5].
Prof Albert Ludolph, Professor of Neurology and Chairman of the Department of Neurology at the University Hospital and Medical Faculty of Ulm in Germany, and international coordinator of study AB19001 said “I am extremely pleased with this FDA clearance, which represents a significant milestone for the development of masitinib in ALS. Masitinib is the first drug that targets both microglia and mast cells to be evaluated in ALS. I am convinced that this approach represents a promising potential addition to our currently limited therapeutic options for patients with ALS.”
Goed nieuws. Masitinib loopt ook al in MS en Alzheimer, en in een paar oncologische en ontstekingsindicatoren. Een blockbuster in de maak, maar dan moet alles wel meezitten natuurlijk.
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