Paris, France – AB Science SA (Euronext – FR0010557264 – AB) announced the identification of a potential biomarker for assessing the activity of masitinib in pathological microglial involvement in Amyotrophic Lateral Sclerosis (ALS).
The key characteristics of this newly identified biomarker are as follows:
- It is a blood-based (plasmatic) biomarker, which has the advantages of being easy to collect and accurately evaluated using ELISA (enzyme-linked immunoassay).
- It is produced by proinflammatory microglia.
- It activates microglia and astrocytes and is therefore an activator contributing to a vicious neuroinflammation feedback loop.
- It is also released by mast cells, establishing a link between mast cells and microglia, which are two major cellular targets of masitinib.
- It is predictive of survival in ALS, potentially explaining why masitinib could extend survival in some specific patients.
- In-house experiments showed that this biomarker was reduced by masitinib when mast cells and microglia were activated in vitro, underscoring the specific and potent activity of masitinib on mast cells and microglia.
Professor Olivier Hermine, President of AB Science’s Scientific Committee, member of the French Academy of Sciences and Head of the Hematology Department at Necker Hospital, commented: “Interestingly, this biomarker could be used in ALS but also in other neurodegenerative diseases of interest, namely progressive forms of multiple sclerosis (MS) and Alzheimer’s disease. In multiple sclerosis, for instance, this biomarker has normal plasmatic levels in clinically isolated syndrome (CIS), is elevated in RRMS during relapse, and is high in progressive forms, consistent with what we know about the involvement of microglia in MS”.
This biomarker will be introduced in the phase 3 program of masitinib in ALS, as well as in progressive MS and Alzheimer’s disease, to validate the mechanism of action of masitinib in humans and its clinical relevance. Once validated, it could facilitate registration by determining which patients respond best to treatment and serve as a surrogate endpoint of efficacy if necessary. Indeed, FDA Guidance on ALS states that “FDA encourages sponsors to incorporate exploratory biomarkers in all phases of development of ALS drugs. In the future, greater scientific understanding of ALS may provide opportunities for discussion of surrogate endpoints that are reasonably likely to predict clinical benefit and that might serve as a basis for accelerated approval.”
This biomarker remains undisclosed for patent protection reasons.
About AB Science
Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development, and commercialization of protein kinase inhibitors (PKIs), a class of targeted proteins whose action is key in signaling pathways within cells. Our programs target only diseases with high unmet medical needs, which are often lethal with short-term survival or rare or refractory to previous lines of treatment.
AB Science has developed a proprietary portfolio of molecules, and the Company’s lead compound, masitinib, has already been registered for veterinary medicine and is being developed for human medicine in oncology, neurological diseases, inflammatory diseases, and viral diseases. The company is headquartered in Paris, France and is listed on Euronext Paris (ticker: AB).
Further information is available on AB Science’s website: www.ab-science.com.
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