AB Science Publishes Promising Study on Masitinib in ALS

Significant Findings from Phase 2b/3 Study

According to AB Science, the post-hoc analysis of the phase 2b/3 study AB10015, published on MedRxiv, shows that masitinib is beneficial for patients with ALS prior to complete functional loss. The results indicate a significant improvement in functional decline measured by the ALSFRS-R score, with a difference of 4.04 points compared to placebo (p=0.0065). Additionally, a relative benefit of 20.2% on the CAFS and an extension of the median progression-free survival (PFS) by 9 months were observed. The overall median survival (OS) also increased by 12 months (p=0.0192), with an improved tolerance profile in the studied subgroup.

Influence on the Design of Confirmatory Study AB23005

AB Science states that these results influence the design of the confirmatory study AB23005, a multicenter, randomized phase 3 trial. This study aims to confirm the efficacy and tolerance of masitinib administered at 4.5 mg/kg/day in combination with riluzole, compared to riluzole with placebo, over a period of 48 weeks in 408 ALS patients. Participants must show a disease progression of less than 1.1 points per month on the functional score and no total functional decline. The inclusion of American patients treated with Edaravone is planned, and the protocol has received approval from European health authorities and the FDA.

Study Based on Masitinib’s Mechanism of Action

According to the press release, the AB23005 study is based on the mechanism of action of masitinib, which targets neuroinflammation. This project is part of AB Science's overall strategy to develop innovative treatments in response to unmet medical needs. The company asserts that the patient enrichment strategy, focused on early treatment before complete functional loss, aligns with the goal of neuromuscular preservation of masitinib.