AB Science Shares Progress on Masitinib for MS Treatment
AB Science Provides Insight into Masitinib for Multiple Sclerosis
AB Science SA (Euronext - FR0010557264 - AB) has recently shared valuable updates regarding the development of its investigational treatment, masitinib, for progressive forms of multiple sclerosis (MS). This announcement follows substantial discussions and findings presented at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) 2024 conference.
Understanding Masitinib and Its Development Journey
Masitinib is currently being assessed through the MAXIMS study (AB20009), a randomized, double-blind, phase 3 clinical trial that focuses on its efficacy in treating patients with primary progressive multiple sclerosis (PPMS) and non-active secondary progressive multiple sclerosis (nSPMS). Participants in this study exhibit a range of Expanded Disability Status Scale (EDSS) scores between 3.0 and 6.0, have experienced disease progression over the last two years, and do not exhibit T1 Gadolinium-enhancing brain lesions.
Key Findings from the Study
The MAXIMS study primarily aims to determine how effectively masitinib can slow the progression of disability in MS patients. Recent findings reported at ECTRIMS 2024 have reinforced the scientific premise that treating microglia in nSPMS represents a promising therapeutic pathway. Such research notably included the drug tolebrutinib, which operates through Bruton's Tyrosine Kinase (BTK) targeting.
Masitinib offers a different approach by targeting microglia through the M-CSFR1 pathway (Macrophage Colony Stimulating Factor Receptor-1). This mechanism of action yielded encouraging results from earlier phase 2B trials (AB07002), aligning with the outcomes observed in studies involving tolebrutinib.
Comparative Efficacy of Masitinib and Tolebrutinib
Further exploration of the comparative effectiveness reveals significant data:
- The reduction in EDSS progression confirmed at the three-month mark was 37% with masitinib versus a 23% reduction with tolebrutinib, though the trial conditions differed in terms of participant numbers.
- At six months, masitinib led to a 32% decrease in EDSS progression, closely followed by tolebrutinib's 31% reduction.
Masitinib’s Unique Advantages
Masitinib's benefits do not stop with disease progression. The study demonstrated significant enhancements in manual dexterity, as measured by the 9-hole Peg test, with masitinib outpacing expectations. Additionally, it has shown potential for reducing serum neurofilament light chain (NfL) concentrations in MS animal models, which may indicate a decrease in neuronal damage.
Beyond microglia, masitinib also interacts with mast cells, which are crucial in the pathology of progressive MS. This multi-target approach is supported by a vast array of research illustrating the importance of mast cells in neuroinflammation and other related CNS disorders.
A Robust Safety Profile
Masitinib benefits from a strong safety database, accumulated from more than 2,200 patients who have received at least one dose across various conditions. Notably, over 1,300 patients have been treated for periods exceeding six months, and nearly 1,000 individuals have continued treatment for over a year, allowing for a comprehensive assessment of its safety profile. In contrast, BTK inhibitors have demonstrated potential safety concerns, such as increased risks of liver injuries and infections, suggesting that masitinib could be a safer therapeutic alternative.
Looking Ahead: Masitinib’s Potential
In summary, masitinib holds promise as a viable option compared to existing BTK inhibitors in addressing unmet medical needs in both primary and non-active secondary progressive forms of MS. Comments from Professor Patrick Vermersch, MD, the principal investigator of the MAXIMS study, highlight the importance of these findings. He noted that masitinib, along with BTK inhibitors, represents significant potential in addressing these challenging forms of MS.
Frequently Asked Questions
What is masitinib?
Masitinib is a protein kinase inhibitor being investigated for the treatment of various diseases, including progressive multiple sclerosis.
How does masitinib work in treating multiple sclerosis?
Masitinib targets microglia through the M-CSFR1 pathway, potentially reducing disease progression and improving neurological function.
What were the key findings of the MAXIMS study?
The MAXIMS study indicated that masitinib significantly reduced EDSS progression compared to placebo, demonstrating its effectiveness in treating progressive MS.
What is the safety profile of masitinib?
Masitinib has a robust safety profile, with extensive long-term exposure demonstrating no elevated risks typically associated with BTK inhibitors.
How does masitinib compare to other treatments for MS?
Masitinib offers a unique approach by targeting both microglia and mast cells, potentially making it a safer and effective alternative to existing treatments.
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