AB Science: 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)”

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press release

The clinical development of masitinib in sickle cell disease, a widespread genetic condition, is among the 19 winning projects under the sixth call for “Hospital-University Research in Health (RHU)”.

This program will fund a phase 2 clinical trial of masitinib

Father Science WEBCAST will conduct this research program on Thursday, November 30, 2023 from 3:00 AM to 4:00 PM CET.

Paris, November 27, 2023, 8 am CET

AB Science S.A (Euronext – FR0010557264 – AB) announced today that it is one of the 19 winning projects funded by the “Recherche Hospitalo-Universitaire en santé” (Recherche Hospitalo-Universitaire en santé) under the sixth call for a new clinical development program for masitinib in sickle cell disease. ) projects that are part of the Future Investment Program.

“Hospital-University Research in Health” (RHU) managed by the National Research Agency calls for future investment program projects and aims to support innovation and large-scale research projects in the field of health. Focused on translational research, that is, turning the results of basic research into outcomes that directly benefit people, RHU projects involve academic, hospital and commercial stakeholders.

For this sixth wave, international judges evaluated 62 applications based on scientific quality, innovation and medical and socio-economic impact. He proposed to select and fund 19 projects covering various medical areas and medical needs. These 19 projects will receive a special grant of 160 million euros from the Future Investment Program.

As part of this call for projects, the SICKMAST project, funded with 9.2 million euros, aims to:

  • First, confirm biomarkers from a database of 1500 patients (including 700 already known) highlighting the role of mast cells and basophils in the combination of acute and chronic complications of sickle cell disease.
  • Second, demonstrate the efficacy of masitinib in the treatment of acute and chronic complications of sickle cell disease in patients stratified based on biomarkers in a phase 2 clinical trial.

Aid Publique-Hôpitaux de Paris (AP-HP) will be the promoter of these phase 2 studies. AB Science is primarily involved in monitoring masitinib availability and masitinib pharmacology data. AB Science remains free to conduct Phase 3 development as it sees fit following the success of Phase 2.

A new patent has been filed that, if granted, would extend the worldwide protection of masitinib in sickle cell disease until 2040. As part of the joint patent agreement, AB Science will pay APHP royalties upon commercialization. Masitinib in sickle cell disease.

SICKMAST Program Coordinator Professor Olivier Hermine Necker-Infants Malads AP-HP “We are delighted to be among the awardees of this program, which has been recognized by a selection of innovative and medically impactful projects. Sickle cell disease represents a major public health challenge due to the number of individuals affected and the resulting premature death. Biomarkers allow us to demonstrate the importance of identifying patients who are likely to respond.

Alain Moses, President and Co-Founder of AB Science; “We are very pleased with the collaboration with APHP, one of the largest promoters of clinical studies in France, whose research excellence is internationally recognized. This is a very promising new development path for masitinib and the collaboration we are exploring to enable the registration and commercialization of masitinib in this indication.” .

A webcast with sickle cell disease experts will be held on November 30, 2023 to provide a more detailed description of this project. Login details for this live stream will be provided later.

It has recently been shown to play an important role in sickle cell disease, which promotes innate immune cell-mediated inflammation and vaso-occlusion. In particular, our clinical observations and experimental work in mice have demonstrated the involvement of mast cells and basophils in the complications associated with sickle cell disease.

  • In patients with sickle cell disease, the degree of mast cell activation may contribute to a variety of inflammatory and chronic and acute complications.
  • The potential role of basophils in sickle cell disease has not been studied, but given their role in various diseases and their ability to release substance P and histamine, they may play an important role in the pathophysiology of sickle cell disease.

Masitinib inhibits KIT, LYN, and FYN, three major kinases involved in the activation of mast cells and basophils.

Sickle cell disease (CD) is an autosomal recessive disorder that affects millions of people worldwide. Although life expectancy has increased over the past 20 years, acute and chronic complications still cause comorbidities, high social burdens, and premature death around 40 years of age. Approximately 1.1% of couples are at risk of having a child with a hemoglobin disorder (sickle cell disease or thalassemia), and 2.3 conceptions in 1,000 are affected by sickle cell disease. Estimates indicate that approximately 300,000 children are born with sickle cell disease each year and this number may reach 400,000 by 2050. [1]. Sickle cell disease affects more than 100,000 children and adults in the United States. In France, about 26,000 patients (50% children, 50% adults) are affected.

The classic view of the pathophysiology of sickle cell disease involves polymerization of hemoglobin (HbS) to red blood cells (RBC) with subsequent hemolytic anemia, painful vaso-occlusive crisis (VOC), and acute chest syndrome (ACS).

Current treatment options, such as hydroxycarbamide, chronic blood transfusions, or anti-P-selectin antibodies, do not completely prevent life-threatening complications of acute and chronic sickle cell disease. Allogeneic stem cell transplants and gene therapy are only available to a minority of patients, are associated with toxicity and are expensive, limiting their use.

There is a great need for treatment to prevent the acute and chronic complications of sickle cell disease.

Ref

[1] Piel FB, Steinberg MH, Rees DC Sickle cell disease. N Engl J Med. 2017;376(16):1561-1573.

About AB scienceIn the year Founded in 2001, AB Science is a pharmaceutical company specializing in the research, development and commercialization of protein kinase inhibitors (PKIs), a class of target proteins that are key to signaling pathways in cells. Our programs target only diseases with high unmet medical needs, often fatal with short survival, or rare or refractory diseases. AB Science has developed a proprietary portfolio of molecules and the company’s lead compound, masitinib, is already registered for animal therapy and has been developed for human treatment in oncology, neurological diseases, infectious diseases and viral diseases. The company is headquartered in Paris, France, and is listed on Euronext Paris. AB).

More information is available on the AB Science website. www.ab-science.com.

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