ALS News TodayMasitinib Works Best in Moderate ALS, New Trial Analysis Shows

Researchers presented findings from the AB10015 Phase 2/3 clinical trial, demonstrating that masitinib, an oral add-on therapy to Rilutek, offers significant benefits for patients with mild to moderate amyotrophic lateral sclerosis (ALS). Although the entire study population did not experience improved survival with masitinib, patients with moderate ALS who received masitinib in addition to Rilutek had significantly longer survival compared to those who received a placebo. The combined treatment also slowed disease progression, improved lung function, and enhanced quality of life in these patients. Masitinib works by blocking specific tyrosine kinase enzymes that regulate immune cell activity and inflammation, aiming to slow disease progression and alleviate symptoms.

The analysis focused on normally progressing participants with moderate ALS at the study’s start, and in this subgroup, masitinib combined with Rilutek significantly slowed disease progression by 42% compared to Rilutek alone. Additionally, patients with moderate ALS experienced a median survival of 69 months with masitinib, representing a 44% reduction in mortality risk compared to placebo-treated patients. The combination therapy also showed positive effects on quality of life and lung function in patients progressing normally. These findings suggest that initiating masitinib at an earlier stage of the disease could lead to substantial reductions in disease progression for both normal and fast-progressing patients. AB Science is conducting the AB19001 Phase 3 clinical trial to further confirm these results and expects to announce top-line results this year. Masitinib is currently under review for conditional approval in Europe and Canada.