Mastocytosis is a rare disease characterized by proliferation and accumulation of mast cells in various tissues, causing a wide variety of clinical symptoms. Indolent systemic mastocytosis, including the subvariant of smouldering systemic mastocytosis, is a lifelong condition associated with reduced quality-of-life.
Masitinib is being developed in severely symptomatic indolent systemic mastocytosis, which accounts for about 35%of disease’s total population. AB Science has completed a phase 3 study in this patient population with promising results. These findings have been published in The Lancet medical journal. A confirmatory phase 3 study will be launched in 2019. Masitinib is currently the only drug in advanced clinical development for treatment of indolent systemic mastocytosis with severe symptoms.
In recognition of the critical need for new treatments, masitinib received orphan drug designation for mastocytosis from both the European Medicine Agency (EMA) and the U.S. Food and Drug Administration (FDA).
Scientific rationale for masitinib development in mastocytosis
Summary of mastocytosis clinical program with masitinib
|Phase||Design||Population||Primary Target||Patient Target||IDMC recommandation||Study Status||Related publications|
|Patients with mastocytosis with handicap and bearing activating point mutations in the phosphotransferase domain of c-Kit (D816V)||Masitinib efficacy on 2 out of these 4 variables : Pruritius, Flush, Hamilton score and Fatigue Impact Scale||21||NA||
2-parallel group study
|Patients with systemic indolent mastocytosis with handicap and not bearing activating point mutations in the phosphotransferase domain of c-Kit (D816V)||
Masitinib efficacy on Pruritius, Flush, Pollakyuria, Number of stools, QLQ-C30 score and Hamilton Score
2-parallel group study
|Patients with documented smouldering or indolent systemic mastocytosis with severe handicap||Cumulative response by handicap (Pruritius, Flush, Hamilton score and Fatigue Impact Scale). Response on a handicap is defined as an improvement ≥ 75%||130||NA||Study completed||
Phase 2a proves of concept
Phase 3 study
Lortholary O, Chandesris MO, Bulai Livideanu C, Paul C, Guillet G, Jassem E, Niedoszytko M, Barete S, Verstovsek S, Grattan C, Damaj G, Canioni D, Fraitag S, Lhermitte L, Georgin Lavialle S, Frenzel L, Afrin LB, Hanssens K, Agopian J, Gaillard R, Kinet JP, Auclair C, Mansfield C, Moussy A, Dubreuil P, Hermine O.
Lancet. 2017 Feb 11;389(10069):612-620. doi: 10.1016/S0140-6736(16)31403-9. Epub 2017 Jan 7.
Depression in patients with mastocytosis: prevalence, features and effects of masitinib therapy.
Moura DS, Sultan S, Georgin-Lavialle S, Pillet N, Montestruc F, Gineste P, Barete S, Damaj G, Moussy A, Lortholary O, Hermine O.
PLoS One. 2011;6(10):e26375. doi: 10.1371/journal.pone.0026375. Epub 2011 Oct 21.
Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study.
Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmérini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O.
Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.
Mast cell leukemia: identification of a new c-Kit mutation, dup(501-502), and response to masitinib, a c-Kit tyrosine kinase inhibitor.
Georgin-Lavialle S, Lhermitte L, Suarez F, Yang Y, Letard S, Hanssens K, Feger F, Renand A, Brouze C,
Canioni D, Asnafi V, Chandesris MO, Aouba A, Gineste P, Macintyre E, Mansfield CD, Moussy A, Lepelletier Y, Dubreuil P, Hermine O.
Eur J Haematol. 2012 Jul;89(1):47-52. doi: 10.1111/j.1600-0609.2012.01761.x. Epub 2012 Apr 28.
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