AB Science: Muscle & Nerve Publication Validates ALSFR-S Decline Rate for ALS Clinical Studies and Treatment

AB Science SA has announced a significant publication in the medical journal Muscle & Nerve, which emphasizes the importance of the rate of decline of the ALSFRS-R score (ΔFS) in designing clinical studies and choosing treatments for Amyotrophic Lateral Sclerosis (ALS) patients. This validation is particularly relevant to the design of AB Science's masitinib Phase 2/3 study AB10015 and its confirmatory study in ALS.

The article, featured in Muscle & Nerve's Issues & Opinions section, highlights the ΔFS parameter, which measures the decline rate of the ALSFRS-R score over time starting from the onset of ALS symptoms. This measure is considered a valuable tool for both research design and clinical practice. According to the publication, post-onset ΔFS serves as a critical factor for patient stratification and enrichment. It allows researchers to identify subgroups of patients who respond differently to treatments, facilitating the optimization of therapeutic effectiveness and patient management.

Professor Albert Ludolph, MD, PhD, a senior author of the article and Chairman of the Department of Neurology at the University Hospital and Medical Faculty of Ulm, emphasized the relevance of post-onset ΔFS in ALS study design. He stated that ΔFS is an independent predictor of survival and can distinguish between patient subgroups with varying disease progression. This categorization helps in optimizing drug effectiveness and patient management, making it an important consideration for clinical trial designs.

Professor Olivier Hermine, MD, PhD, President of the Scientific Committee of AB Science and member of the Académie des Sciences in France, echoed these sentiments. He noted that while the article is not specifically focused on the masitinib study AB10015, it validates the study's design philosophy by highlighting the consensus among key opinion leaders on the utility of post-onset ΔFS. The measure is simple to use and integrates seamlessly into routine clinical practice, making it a practical tool for patient selection.

Key points from the article include the clinical relevance of ΔFS as an independent predictor of survival, irrespective of disease progression stages. The use of post-onset ΔFS allows for innovative ALS trial designs that can optimize drug effectiveness and patient management. Moreover, the article suggests there are no significant barriers to applying post-onset ΔFS in clinical practice, making it an attractive tool for enriching ALS clinical trials.

The publication underscores the importance of ΔFS in both clinical settings and research, offering a robust framework for future ALS studies. The findings support the ongoing efforts to improve the design and efficacy of clinical trials for ALS, particularly those involving the drug masitinib.

Masitinib, an orally administered tyrosine kinase inhibitor developed by AB Science, targets mast cells and macrophages, which play crucial roles in immunity. Its unique mechanism of action allows it to be used in various conditions, including oncology, inflammatory diseases, and certain central nervous system diseases. In oncology, masitinib can enhance survival rates, either alone or in combination with chemotherapy. In inflammatory and central nervous system diseases, it can alleviate symptoms and slow disease progression.

Founded in 2001, AB Science is a pharmaceutical company specializing in the development of protein kinase inhibitors (PKIs). These inhibitors are essential in cellular signaling pathways and target diseases with high unmet medical needs. The company's lead compound, masitinib, is being developed for human medicine in oncology, neurological diseases, inflammatory diseases, and viral infections. AB Science is headquartered in Paris, France, and is listed on Euronext Paris.