AB Science granted European patent for masitinib in sickle cell disease treatment until 2040

AB Science SA has recently announced that the European Patent Office has issued a Notice of Allowance for a patent covering methods of using masitinib to treat sickle cell disease. This patent provides protection for masitinib in this indication until November 2040. Masitinib, AB Science’s leading compound, is currently in Phase 2 of clinical trials for treating sickle cell disease.

Professor Olivier Hermine, the President of the AB Science Scientific Committee, emphasized that masitinib represents a new approach to treating sickle cell disease and one of its severe complications, acute chest syndrome. This condition can lead to chronic lung disease, hospitalization, or even death. The need for effective treatments in sickle cell disease is growing, particularly as some recent drugs have been withdrawn from the market due to inefficacy or toxicity. Unlike these drugs, masitinib targets mast cells.

Sickle cell disease (SCD) is a genetic disorder affecting red blood cells. Masitinib is being developed to address the most severe forms of SCD, which account for about 65% of all cases. Severe SCD poses significant public health challenges and leads to early mortality. While gene therapy offers a potential cure by targeting the HbS mutation, it is limited by donor scarcity, safety issues, and high costs. Current standard treatments, such as red blood cell transfusions and hydroxyurea, help manage complications but do not fully address the unmet medical needs.

Mast cells are crucial in the severe forms of SCD and its complications, including vaso-occlusive crises (VOC) and acute chest syndrome (ACS). In a mouse model of SCD, masitinib demonstrated a significant survival benefit. All control mice experienced VOC and 83% died within the first three hours, while none of the mice treated with masitinib experienced VOC or death. Further studies showed that masitinib protected against acute lung injuries and mast cell infiltration in SCD mice.

The clinical development of masitinib in SCD is part of the SICKMAST collaborative program, funded with 9.2 million euros. This program aims to demonstrate the efficacy of masitinib in a phase 2 clinical trial, focusing on acute and chronic complications of SCD. The Assistance Publique-Hôpitaux de Paris (AP-HP) will lead these phase 2 studies, while AB Science will supply masitinib and monitor its pharmacovigilance data. AB Science retains the right to pursue phase 3 development if phase 2 is successful.

Sickle cell disease affects millions globally, with a significant percentage of at-risk couples potentially having a child with a hemoglobin disorder. Each year, approximately 300,000 children are born with SCD, a number expected to rise to 400,000 by 2050. In the United States, over 100,000 children and adults live with SCD, and in France, around 26,000 patients are affected.

The role of inflammation mediated by innate immune cells, particularly mast cells and basophils, is increasingly recognized in the pathophysiology of SCD. Mast cell activation contributes to the variability of inflammation and complications in SCD. Although the role of basophils has not been extensively studied, their involvement in other diseases suggests they may also play a role in SCD. Masitinib targets kinases involved in the activation of these cells, making it a promising therapeutic candidate.

Current treatments for SCD, including hydroxycarbamide, chronic transfusions, and anti-P-selectin antibodies, do not fully prevent the disease's life-threatening complications. Allogeneic stem cell transplantation and gene therapy are available for only a few patients due to toxicity and high costs. Therefore, there is a significant need for new treatments to prevent both acute and chronic complications of SCD.

AB Science, founded in 2001, specializes in developing protein kinase inhibitors (PKIs) for diseases with high unmet medical needs. Their leading compound, masitinib, has shown promise in various medical fields, including oncology, neurological, inflammatory, and viral diseases. The company is headquartered in Paris, France, and listed on Euronext Paris.