OREANDA-NEWS. AB Science SA, a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), today announced that results from its phase 3 trial in severe systemic mastocytosis will be presented as an oral presentation at the 21st Congress of the European Hematology Association (June 9 - 12, 2016, Copenhagen, Denmark).

Professor Olivier Hermine, President of the Scientific Committee of AB Science and coordinator of the Reference Center for Mastocytosis (CeReMast, Paris, France), will deliver this oral presentation at the EHA 2016 Congress on Sunday, 12th June. Abstracts were selected by the EHA Scientific Program Committee based on scientific quality, with just 8% of the total submitted being selected for oral presentation.

Professor Olivier Hermine said: "We will present results from the masitinib international AB06006 study, which was the first ever phase 3 prospective, randomized, placebo-controlled study of a treatment for severe indolent systemic mastocytosis, including the subvariant of smouldering systemic mastocytosis. There is currently no registered or established standard treatment for this rare condition with high unmet medical need. Study AB06006 successfully achieved all of its primary and secondary objectives. The study also generated positive results on the pre-specified objective markers of mast cell activation. Masitinib may therefore be an important new treatment option for these patients; moreover, both efficacy and safety data indicate a possibility for effective long-term management of this difficult-to-treat condition."

  • Abstract and schedule

Masitinib for the treatment of severely symptomatic indolent and smouldering systemic mastocytosis: a randomized, placebo-controlled, international, phase 3 study
Abstract: #S828
Session Title: Non-malignant hematopoietic disorders
Date, Location: Sunday, June 12 (8am), Room H5

  • About the phase 3 study in severe systemic mastocytosis

The phase 3 study was designed to evaluate masitinib efficacy and safety in severe systemic mastocytosis patients, with or without D816V mutation of c-Kit. The primary objective of the phase 3 study was to detect a statistically significant difference between masitinib (plus optimal concomitant symptomatic treatments) and placebo (plus optimal concomitant symptomatic treatments) in cumulative response on four severe symptoms, referred to also as handicaps (pruritus, flush, depression and asthenia).

  • Targeted population with masitinib in in severe systemic mastocytosis

Mastocytosis is an orphan disease characterized by an abnormal proliferation or activation of mast cells either in the skin or in bone marrow or other organs. Mastocytosis comes in two main forms: indolent and aggressive. Indolent forms of mastocytosis can be either cutaneous or systemic. The prevalence of indolent systemic mastocytosis, including smoldering systemic mastocytosis, is estimated to be 1/26,000 in Europe1. The symptoms and handicaps are severe in about one third of the patients; hence, an estimated target population for masitinib of approximately 1/78,000 of the general population.

Since the prevalence of indolent forms of systemic mastocytosis is reputed to be comparable across countries, the target population for masitinib could reach 10,000 adult patients in the USA and in Europe.