AB Science updates on conditional marketing authorisation application for masitinib in ALS

Paris, 28 June, 2024 – AB Science has announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a negative opinion on the conditional marketing authorization of masitinib for treating amyotrophic lateral sclerosis (ALS). Despite this setback, AB Science plans to request a re-examination of the decision.

The company's request for re-examination will be grounded on two primary points: firstly, the urgent necessity for patients to access promising treatments earlier, and secondly, the potential benefits of having the application reviewed by new rapporteurs and a Scientific Advisory Board.

AB Science acknowledges the difficulty of obtaining conditional marketing authorization for ALS treatments and does not guarantee a positive outcome after the re-examination. However, it intends to highlight several key arguments to support its case:

1. Acceptable Safety Profile: The CHMP has acknowledged that the safety of masitinib is acceptable, an essential factor for conditional marketing authorization which requires confirmatory evidence of efficacy.

2. Good Clinical Practice Compliance: According to EMA guidance, analyses of protocol deviations that couldn't be corrected were performed, showing no impact on the study's validity. This addresses the CHMP’s concerns about compliance with Good Clinical Practice standards.

3. Exclusion of Fast Progressors: The transition from phase 2 to phase 3 included an amendment to exclude fast progressors from the primary analysis population. This move aimed to create a more homogeneous group with a higher likelihood of reaching the 48-week time point, thus minimizing missing data. The amendment was implemented early and while the study was still blinded, ensuring no methodological biases.

4. Treatment of Missing Data: Multiple sensitivity analyses of the primary data, using methods other than LOCF (Last Observation Carried Forward) for imputing missing data, showed positive and consistent results. These analyses, including two previously recommended by the CHMP, demonstrate the robustness of the primary analysis and address the objection concerning missing data treatment.

5. Subgroup Data Analysis: There was an imbalance in a subset of patients experiencing complete loss of function, with 20% in the masitinib arm versus 8% in the placebo arm. The ALSFRS-R score was minimized but not stratified by severity category. However, excluding this biased subset, which constituted 86% of the population, revealed compelling results, including a significant 12-month survival benefit. This subgroup analysis strictly adheres to EMA guidelines applicable to post hoc analysis and single pivotal study registration, thus addressing the objection regarding subgroup data.

AB Science, founded in 2001, specializes in the development of protein kinase inhibitors (PKIs), targeting diseases with high unmet medical needs. The company's portfolio includes molecules developed for oncology, neurological, inflammatory, and viral diseases. Masitinib, its lead compound, is already registered for veterinary use and is under development for human medicine.

AB Science is headquartered in Paris, France, and is listed on Euronext Paris (ticker: AB). The company remains committed to advancing its pipeline and addressing critical medical needs through innovative therapies.

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