Jun 14 2012
AB Science SA (Paris:AB)(NYSE-Euronext-FR0010557264-AB), a
pharmaceutical company specializing in the research, development and
commercialization of protein kinase inhibitors, announces the
publication of results from the human phase 2 study of masitinib
carried-out in the treatment of progressive multiple sclerosis. The
article, 'Masitinib treatment in patients with progressive multiple
sclerosis: a randomized pilot study', is freely accessible online
from BioMed Central's peer-reviewed journal BMC Neurology (http://www.biomedcentral.com/1471-2377/12/36/abstract).
• Phase 2 study establishes proof-of-concept that oral
masitinib has potential therapeutic benefits in patients with
progressive forms of multiple sclerosis
• Overall, results add new scientific data to the important
question of the potential role of anti inflammatory agents in the
management of progressive multiple sclerosis
• A phase 3 study has been initiated based upon these
promising results
Reported are results from a phase 2 study of 30 patients, conducted by
Professor Patrick Vermersch (CHRU Lille - Hôpital Roger Salengro,
France) and colleagues from six study centers across France,
investigating the hypothesis that masitinib's targeted inhibitory action
on mast cells can delay the onset of symptoms associated with
progressive forms of multiple sclerosis. There is currently no
satisfactory treatment for primary progressive multiple sclerosis or
relapse-free secondary progressive multiple sclerosis, which represent
approximately 60% of patients diagnosed with multiple sclerosis.
Neuroinflammation is thought to be important in progressive multiple
sclerosis pathogenesis. Mast cells are a key component of the
inflammatory network and participate in the regulation of the
blood-brain barrier's permeability. Masitinib is an oral tyrosine kinase
inhibitor that effectively down-regulates mast cell functions and
therefore, represents a different approach to those therapeutic
strategies currently developed. The results showed that for the primary
endpoint of Multiple Sclerosis Functional Composite (MSFC) score (which
measure symptoms of patients on three aspects: movement of the lower
limbs, movement of the upper limbs, and cognitive tests) 32% of patients
treated with masitinib were responders against 0% under placebo.
Responses were seen in the third month and were more-or-less sustained
over the study's 18-month duration. These results suggest that daily
administration of masitinib is of therapeutic benefit to progressive
forms of multiple sclerosis and could therefore represent an innovative
avenue of treatment for this disease.
Professor Vermersch commented: "Masitinib is a selective inhibitor of
specific kinases that play a major role in the activation of mast cells,
which are involved in the immune response, in the recruitment of
lymphocytes to the brain, and also in inflammatory processes associated
with multiple sclerosis and many of its resulting symptoms. Masitinib
therefore represents an oral treatment different from those drugs
already on the market for this indication, with a unique mechanism of
action in blocking mast cells. Masitinib showed promising signs of
retarding the onset of symptoms associated with progressive multiple
sclerosis as compared to placebo, with an acceptable tolerance profile.
Although the number of patients in this study was too small to make any
definitive conclusions about treatment efficacy, the evidence is
sufficiently compelling to warrant further phase 3 investigation."
Professor Olivier Hermine, President of the scientific committee of AB
Science and co-corresponding author on this paper declared: "Masitinib
differs from those treatments currently available or under development
in multiple sclerosis. It has a weak immunosuppressive activity,
although by inducing a reduction in the number of lymphocytes
infiltrating the brain it helps prevent injuries. Masitinib's
characteristic selectivity against mast cells also means that it is not
associated to date with major toxicities; for example, cardiac toxicity
as seen with mitoxantrone, a drug sometimes used in severe progressive
multiple sclerosis, or opportunistic infections as seen with Tysabri or
Gilenya, which are associated with an increased risk of infection."