AB Science receives FDA authorization to start clinical development
program of masitinib in mast cell activation syndrome (MCAS)
PRESS RELEASE
AB SCIENCE
RECEIVES U.S. FOOD AND DRUG ADMINISTRATION
(FDA) AUTHORIZATION TO START
CLINICAL DEVELOPMENT PROGRAM
OF MASITINIB IN MAST CELL
ACTIVATION SYNDROME (MCAS)
MCAS IS A
NEWLY RECOGNIZED
DISORDER, DISTINCT FROM
BUT CLOSELY RELATED TO SYSTEMIC MASTOCYTOSIS AND
FOR WHICH THERE IS
A FAR GREATER PREVELANCE
IN THE GENERAL POPULATION
Paris, October 04, 2021, 6pm CET
AB Science SA (Euronext -
FR0010557264 - AB) today announced that its clinical development
program of masitinib in adult patients with mast cell activation
syndrome (MCAS) has been approved by the U.S. Food and Drug
Administration (FDA). The Investigational New Drug (IND) approval
letter received from the FDA provides authority to proceed with a
Phase II study (AB20006) in patients with severe mast cell
activation syndrome.
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.
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 [1]. 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) [2,3].
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.
Professor Mariana Castells (Professor of
Medicine at Harvard Medical School and the Director of the Brigham
and Women’s Hospital Mastocytosis Center, Boston, USA) said, “We
are very excited about the news of FDA approval to begin masitinib
clinical trials in mast cell activation syndromes. Masitinib
represents a path forward for severe MCAS patients, for whom there
is a significant unmet medical need and no clinical trials”.
Dr Lawrence Afrin (AIM Center for Personalized
Medicine, USA) a leading expert in MCAS said, “MCAS is frequently
unrecognized and misdiagnosed because symptoms associated with it
are often present in other medical conditions and are highly
variable among patients. Nevertheless, it is now apparent that
mastocytosis, a malignancy of the mast cell, comprises only a very
small proportion of the overall burden of mast cell activation
disease, with a far larger bulk of this population consisting of
MCAS patients. Given the comparatively large number of MCAS
patients suffering severe symptoms, the development of a targeted
drug such as masitinib in this indication is of enormous
importance.”
Professor Olivier Hermine, President of the
Scientific Committee of AB Science and member of the Académie des
Sciences in France said, “Based on our extensive knowledge of
masitinib’s mechanism of action in mast cell disease and clinical
experience of treating indolent systemic mastocytosis, we believe
that masitinib is particularly well-suited for the treatment of
severe MCAS, for which there are currently no registered
therapeutic drugs. Indeed, masitinib has already shown potential
efficacy in a population that closely matches the targeted
population of severe MCAS having substantially reduced severe mast
cell mediator release symptoms in mastocytosis, regardless of the
patient's c-Kit mutational status [4,5].”
Reference[1] 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.
[2] Molderings GJ, Haenisch B, Bogdanow M,
Fimmers R, Nöthen MM. Familial Occurrence of Systemic Mast Cell
Activation Disease. PLoS One. 2013;8:e76241.
[3] Haenisch B, Nöthen MM, Molderings GJ.
Systemic mast cell activation disease: the role ofmolecular genetic
alterations in pathogenesis, heritability and diagnostics. Immunol.
2012;137:197–205.
[4] 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. Lancet. 2017;389(10069):612-620.
[5] 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.
About masitinibMasitinib is an
orally administered tyrosine kinase inhibitor that targets mast
cells and macrophages, important cells for immunity, through
inhibiting a limited number of kinases. Based on its unique
mechanism of action, masitinib can be developed in a large number
of conditions in oncology, in inflammatory diseases, and in certain
diseases of the central nervous system. In oncology due to its
immunotherapy effect, masitinib can have an effect on survival,
alone or in combination with chemotherapy. Through its activity on
mast cells and microglia and consequently the inhibition of the
activation of the inflammatory process, masitinib can have an
effect on the symptoms associated with some inflammatory and
central nervous system diseases and the degeneration of these
diseases.
About AB ScienceFounded in
2001, AB Science is a pharmaceutical company specializing in the
research, development and commercialization of protein kinase
inhibitors (PKIs), a class of targeted proteins whose action are
key in signaling pathways within cells. Our programs target only
diseases with high unmet medical needs, often lethal with short
term survival or rare or refractory to previous line of treatment.
AB Science has developed a proprietary portfolio of molecules and
the Company’s lead compound, masitinib, has already been registered
for veterinary medicine and is developed in human medicine in
oncology, neurological diseases, inflammatory diseases and viral
diseases. The company is headquartered in Paris, France, and listed
on Euronext Paris (ticker: AB).
Further information is available on AB Science’s website:
www.ab-science.com.
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