PRESS RELEASE
Study AB20006 is titled ‘A 24-week, multicenter, randomized, double blind, placebo-controlled, dose-range finding phase 2 study to compare efficacy and safety of oral masitinib to placebo in treatment of patients with severe mast cell activation syndrome (MCAS) or severe smoldering or indolent systemic mastocytosis (SSM/ISM) with handicap unresponsive to optimal symptomatic treatment’. The study will enroll 60 patients from numerous study centers. The treatment objective in severe MCAS is to reduce symptoms (pruritus, flush, depression) and improve impaired quality-of-life.
Study AB20006 has also been approved by the
Professor
MCAS is a disease caused by inappropriate activation of mast cells, which can lead to mast cell mediator release symptoms with a severity ranging from mild to life-threatening. In this aspect, MCAS is similar to indolent and smoldering systemic mastocytosis (ISM/SSM), however, important differences exist that make MCAS a distinct entity from systemic mastocytosis. In mastocytosis, well-defined mutations result in an aberrant population of mast cells with a marked increased proliferation in tissues, whereas MCAS is driven by greater (ill-defined) mutational heterogeneity that is associated with aberrant mast cell activation but only modest increases in mast cell numbers due to reduced apoptosis [3]. Another striking difference between systemic mastocytosis and MCAS is the prevalence of these diseases. Systemic mastocytosis is considered to be a rare, orphan disease affecting about 1/100,000 people, whereas MCAS has an estimated prevalence of 1–17% of the population (i.e., at least a 1000-fold difference) [4,5].
Because masitinib has been designed to be a potent inhibitor of mast cell activation (through its action against wild-type c-Kit, Lyn and Fyn tyrosine kinases), it is uniquely well-suited for the treatment of severe MCAS, unlike other c-Kit tyrosine kinase inhibitors that typically target specific c-Kit mutations that are associated with systemic mastocytosis. There are currently no approved therapies for severe MCAS or drugs in clinical development for this indication.
Reference
[1] Lortholary O, Chandesris MO, Bulai Livideanu C, et al. Masitinib for treatment of severely symptomatic indolent systemic mastocytosis: a randomised, placebo-controlled, phase 3 study.
[2] Paul C, Sans B, Suarez F, et al. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010;85:921–25.
[3] Afrin LB, Ackerley MB, Bluestein LS, et al. Diagnosis of mast cell activation syndrome: a global "consensus-2". Diagnosis (Berl). 2020;8(2):137-152. Published 2020 Apr 22.
[4] Molderings GJ, Haenisch B, Bogdanow M, Fimmers R, Nöthen MM. Familial Occurrence of Systemic Mast Cell Activation Disease. PLoS One. 2013;8:e76241.
[5] Haenisch B, Nöthen MM, Molderings GJ. Systemic mast cell activation disease: the role of molecular genetic alterations in pathogenesis, heritability and diagnostics. Immunol. 2012; 137:197–205.
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