PRESS RELEASE
AB SCIENCE RECEIVES NOTICE OF ALLOWANCE
FOR EUROPEAN PATENT COVERING MASITINIB UNTIL 2040 IN THE TREATMENT
OF SICKLE CELL DISEASE
THIS DECISION FURTHER STRENGTHENS
MASITINIB’S INTELLECTUAL PROPERTY ADDING ONE MORE INDICATION WITH
LONG TERM PROTECTION
MASITINIB IS IN PHASE 2 IN SICKLE CELL
DISEASE
Paris, October 28, 2024, 5.45pm CET
AB Science SA (Euronext -
FR0010557264 - AB) today announced that the European Patent Office
has issued a Notice of Allowance for a patent relating to methods
of treating sickle cell disease (i.e. a medical use patent) with
its lead compound masitinib, based on preclinical data. This new
European patent provides intellectual property protection for
masitinib in this indication until November 2040.
Professor Olivier Hermine, President of the AB
Science Scientific Committee and member of the French Academy of
Sciences and Head of Hematology Department at Necker Hospital
commented: “Masitinib represents a promising novel strategy for
treating sickle cell disease and its serious complication of acute
chest syndrome, which can lead to the development of chronic lung
disease and is a common cause of hospitalization or even death.
There is an increasing need in sickle cell disease with several new
drugs being retrieved from market for lack of efficacy or toxicity;
however, unlike masitinib, none of these drugs target mast
cells”.
Sickle cell disease (SCD) is a group of
inherited red blood cell disorders, with masitinib being developed
to treat the severest forms of the disease, accounting for about
65% of SCD. Severe SCD represents a major public health challenge
and leads to early death. Treatment for SCD can be curative based
on gene therapy (targets the HbS mutation), however, such an option
remains extremely limited due to scarcity of donors, unresolved
safety challenges, and very high costs. Standard treatment for SCD
includes red blood cell transfusions and treatment with
hydroxyurea to manage complications, but significant unmet need
remains.
Mast cells, a major target for masitinib, appear
to play a critical role for the severe forms of SCD and its
complications, such as vaso-occlusive crises (VOC), acute chest
syndrome (ACS), and pain [1-2]. Masitinib has demonstrated survival
benefit in an SCD mouse model: all control SCD mice experienced VOC
and 83% died in the first 3 hours, whereas SCD mice pretreated with
masitinib for 4 days, experienced no VOCs and no death.
Furthermore, lung histology and immune immunohistochemistry showed
that masitinib protects from acute lung injuries and mast cell
infiltration in an SCD mouse model.
Masitinib clinical development in SCD is being
conducted as part of the SICKMAST collaborative program, funded
with 9.2 million euros [3], which aims to demonstrate in a phase 2
clinical trial the efficacy of masitinib in the treatment of acute
and chronic complications of SCD in patients identified based on
biomarkers. The Assistance Publique-Hôpitaux de Paris (AP-HP) will
be the promoter of these phase 2 studies. AB Science will mainly be
involved in supplying masitinib and monitoring masitinib
pharmacovigilance data. AB Science remains free to carry out, as it
sees fit, any potential phase 3 development following the success
of phase 2.
Sickle cell disease
Sickle cell disease (SCD) is an autosomal
recessive disorder affecting millions of people worldwide. Although
life expectancy has increased over the last 20 years, acute and
chronic complications still result in comorbidities, high social
burden and premature death at around 40 years. Approximately 1.1%
of couples worldwide are at risk of having a child with a
hemoglobin disorder (sickle cell disease or thalassemia), and 2.3
conceptions per 1,000 are affected by sickle cell disease.
Estimates suggest that each year, around 300,000 children are born
with sickle cell disease, and this number could reach 400,000 by
2050 [4]. Sickle cell disease affects over 100,000 children and
adults in the United States. In France, approximately 26,000
patients are affected (50% children, 50% adults).
Rationale for the use of masitinib in this
indication
Inflammation mediated by innate immune cells and
promoting vaso-occlusion has recently been shown to play a major
role in sickle cell disease. In particular, our clinical
observations and experimental work in mice, have revealed the
involvement of mast cells and basophils in complications associated
with sickle cell disease:
-
The degree of mast cell activation in patients with sickle cell
disease may contribute to the heterogeneity of inflammation and
chronic and acute complications.
-
The potential role of basophils in sickle cell disease has not been
studied, however, given their role in various diseases and their
ability to release substance P and histamine, they could also play
important roles in the pathophysiology of sickle cell disease.
Masitinib is an inhibitor of KIT, LYN, and FYN,
three major kinases involved in the activation of mast cells and
basophils.
Medical need
The classic view of sickle cell disease
pathophysiology involves polymerization of mutated hemoglobin (HbS)
leading to red blood cell (RBC) sickling with subsequent hemolytic
anemia, painful vaso-occlusive crisis (VOC) and acute chest
syndrome (ACS).
Current treatment options such as
hydroxycarbamide, chronic transfusion or anti-P-selectin
antibodies, do not fully prevent life-threatening acute and chronic
complications of sickle cell disease. Allogeneic stem cell
transplantation and gene therapy are available only for a minority
of patients, are associated with toxicity and are very expensive,
which limits their use.
There is a significant medical need to prevent
the acute and chronic complications of sickle cell disease.
References
[1] Allali S, Lionnet F, Mattioni S, et al. Br J
Haematol. 2019;186(1):125-129.
[2] Allali S, Maciel TT, Hermine O, de
Montalembert M. Haematologica. 2020;105(2):273-283.
[3] AB Science Press Release, 27th November
2023.
https://www.ab-science.com/the-clinical-development-of-masitinib-in-sickle-cell-disease-is-among-the-19-winning-projects-under-the-sixth-call-for-hospital-inuversity-research-in-health-rhu/
[4] Piel FB, Steinberg MH, Rees DC. Sickle Cell
Disease. N Engl J Med. 2017;376(16):1561-1573.
About AB Science
Founded 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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While AB Science believes these forward-looking statements are
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and which may imply that results and actual events significantly
differ from those expressed, induced or anticipated in the
forward-looking information and statements. These risks and
uncertainties include the uncertainties related to product
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identified in the public documents published by AB Science. AB
Science disclaims any obligation or undertaking to update the
forward-looking information and statements, subject to the
applicable regulations, in particular articles 223-1 et seq. of the
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Relations
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- Sickle Cell Patent EPO vENG VF
Ab Science (EU:AB)
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부터 10월(10) 2024 으로 11월(11) 2024
Ab Science (EU:AB)
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부터 11월(11) 2023 으로 11월(11) 2024
