(Boursier.com) — AB Science SA provides a summary of the virtual conference held on May 30, 2024, providing an update on the conditional marketing authorization application for masitinib in the treatment of amyotrophic lateral sclerosis (ALS).
The presentation focused on three topics:
Request for conditional authorization of masitinib in ALS by the European Medicines Agency (EMA).
Request for conditional authorization of masitinib in ALS by Health Canada.
Current preclinical and clinical data in the context of a full authorization application.
Regarding the request for conditional authorization of masitinib in ALS by the EMA, AB Science presented the voting trend of the CHMP, concluding that the tolerability of masitinib is considered acceptable but that outstanding questions remain, preventing a favorable evaluation of the profit.
These questions and the main justifications provided by AB Science are detailed in the presentation. The EMA’s decision will be made public at the next CHMP meeting to be held from June 24 to 27, 2024.
AB Science indicated that it had the possibility of requesting a review and that it would contact the EMA in order to define the procedure towards registration. In the event of a re-examination, a new Rapporteur and a new co-Rapporteur are appointed to evaluate the file, and a Scientific Advisory Group may be appointed to provide recommendations on key points.
These key points could include: Application of the EMA guidelines on Good Clinical Practice (GCP), application of the EMA guidelines on subgroups, application of the two recommendations on the management of missing data, and the question of determine whether it is justified to exclude Fast progressors patients from the primary analysis.
Regarding Health Canada’s request for conditional authorization of masitinib in ALS, AB Science presented Health Canada’s main clinical objections, which are slightly different from those of the EMA.
Finally, AB Science clarified that a distinction should be made between conditional marketing authorization, which requires very convincing data from a single study, and the current preclinical and clinical data from the masitinib program, which are robust. and which will support full authorization, provided that the confirmatory study is positive.
Masitinib has a validated mechanism of action, targeting the innate immune system, via modulation of mast cells and microglia. Masitinib exerts a protective effect on the central nervous system and the peripheral nervous system. Additionally, masitinib demonstrated the ability to reduce blood levels of neurofilament light chain (NfL) in a neurodegenerative disease model (EAE model).
Study AB10015 is a 48-week study, which is stronger evidence than a 24-week study, and this is the same duration as the confirmatory study. Additionally, study AB10015 demonstrated a significant treatment effect in the primary analysis population.
The study data are very solid in the patient population with the inclusion criteria close to the confirmatory study and this could be the new indication to be proposed, with a significant benefit on the functional score, a significant benefit on the quality of life, and a significant benefit of more than 12 months on long-term overall survival.
AB Science has asked Euronext Paris to resume trading of its shares from the opening of the markets on Monday June 3, 2024.