AB Science Summarizes May 30 Webcast on Masitinib ALS Marketing Authorization

AB Science SA provided a detailed summary of their recent webcast held on May 30, 2024, which focused on the conditional marketing authorization application for masitinib in treating amyotrophic lateral sclerosis (ALS). The presentation covered the conditional approval process with both the European Medicines Agency (EMA) and Health Canada, alongside available preclinical and clinical data supporting full approval.

Key Presentation Highlights

EMA Conditional Approval:

AB Science discussed the Committee for Medicinal Products for Human Use (CHMP) trend vote, noting that while the safety of masitinib was considered acceptable, certain issues need to be resolved before a favorable benefit assessment can be achieved. These unresolved issues are elaborated upon in the presentation. The EMA’s final decision is expected during the CHMP meeting scheduled for June 24-27, 2024.

AB Science plans to potentially request a re-examination, which involves appointing a new Rapporteur and Co-Rapporteur to reassess the dossier. A Scientific Advisory Group might also be involved to provide recommendations on several key points, such as the application of Good Clinical Practices (GCP) guidelines, EMA guidelines on subgroup analyses, handling of missing data, and the justification for excluding fast progressors from the primary analysis.

Health Canada’s Conditional Approval:

The webcast also highlighted Health Canada’s main clinical objections, which differ slightly from those raised by the EMA. Specific details on these objections were presented.

Distinction Between Conditional and Full Approval:

AB Science emphasized the difference between conditional marketing authorization, which requires compelling evidence from a single study, and the full approval that robust preclinical and clinical data can support, provided a confirmatory study yields positive results.

Mechanism and Efficacy of Masitinib:

Masitinib, targeting the innate immune system through modulation of mast cells and microglia, has shown a protective effect on both the central and peripheral nervous systems. It also demonstrated the ability to lower blood levels of neurofilament light (NfL) in a neurodegenerative disease model (EAE model).

Study AB10015, a 48-week study, provided stronger evidence compared to a 24-week study, aligning with the timeline of a confirmatory study. This study showed significant treatment effects in the primary analysis population, with notable benefits in functional score, quality of life, and an increase of over 12 months in long-term overall survival.

Next Steps:

AB Science has requested the resumption of trading of its shares on Euronext Paris from June 03, 2024.

About AB Science
Founded in 2001, AB Science is a pharmaceutical company that focuses on the research, development, and commercialization of protein kinase inhibitors (PKIs). These targeted proteins play a crucial role in cellular signaling pathways. The company’s programs target severe diseases with high unmet medical needs, often lethal or rare and refractory to previous treatments. AB Science has a proprietary portfolio of molecules, with its lead compound, masitinib, already registered for veterinary use and under development for human applications in oncology, neurological, inflammatory, and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris (ticker: AB).

How to obtain the latest research advancements in the field of biopharmaceuticals?

In the Synapse database, you can keep abreast of the latest research and development advances in drugs, targets, indications, organizations, etc., anywhere and anytime, on a daily or weekly basis. Click on the image below to embark on a brand new journey of drug discovery!