OREANDA-NEWS. AB Science SA, a pharmaceutical company specialized in the research, development and marketing of protein kinase inhibitors (PKIs), announced today that Rapporteurs appointed by the European Medicines Agency (EMA) have recommended the filing of masitinib in combination with riluzole in the treatment of adult patients with amyotrophic lateral sclerosis (ALS) for conditional marketing authorization.

This request for filing was based on clinical data from the phase 2/3 study AB10015, which was successful on its pre-specified primary endpoint at the interim analysis, as well as preclinical data on masitinib mechanism of action in ALS, and manufacturing process.

Three pre-submission meetings were held in May 2016, one with the Rapporteur and his team, one with the co-Rapporteur and his team, and one with the EMA coordinator, to assess the request for filing of masitinib in combination with riluzole in the treatment of adult patients with ALS.

The following points were reviewed during these pre-submission meetings:

  • Compliance of the study design with the EMA guidance on clinical trials in ALS
  • Clinical relevance of the treatment effect based on the study's primary endpoint data, in particular the change in slope of decline in ALSFRS between masitinib and placebo
  • Significance of the study results :
    • the p-value of the primary analysis
    • the consistency of sensitivity analyses on the primary analysis
    • the convergence of treatment effects on secondary analyses, including quality-of-life which is an important endpoint for EMA
  • The absence of bias due to center effect or baseline characteristics
  • The safety profile based on frequency of serious adverse events, and advent events leading to treatment discontinuation
  • Plausibility of the mechanism of action of masitinib in ALS
  • Timing of availability of final data from study AB10015
  • The resolution of objections concerning the manufacturing raised by EMA during the previous evaluation in GIST

Based on the review of these points, the Rapporteurs have recommended AB Science to submit its application for conditional marketing authorization for masitinib in combination with riluzole in the treatment of adult patients with amyotrophic lateral sclerosis.

AB Science expects to file this application for conditional marketing authorization by the end of Q3 2016.