AB Science Granted US Patent for Masitinib in Sickle Cell Disease
AB Science announces that the United States Patent Office has issued a notice of allowance for a patent on the use of masitinib in the treatment of sickle cell disease. This intellectual property protection extends until November 2040.
Patent Approval for Medical Use
The French pharmaceutical group has received a notice of allowance from the US Patent Office for a medical use patent (US12,472,164) covering the treatment of sickle cell disease with masitinib, according to the press release. This decision, based on preclinical results, protects the intellectual property of the molecule for this indication until November 2040. Masitinib is being developed to treat the most severe forms of the disease, which represent about 65% of sickle cell cases, according to the company. These severe forms pose a major public health challenge and can lead to premature death of patients.
Demonstrated Survival Benefit in Preclinical Model
According to the press release, masitinib demonstrated a survival benefit in a murine model of sickle cell disease. All control mice affected by the disease exhibited vaso-occlusive crises, and 83% of them died within the first three hours, while mice pre-treated with masitinib for four days showed neither vaso-occlusive crises nor deaths, the company specifies. Histological and immunohistochemical analyses showed that the molecule protects against acute lung injuries and mast cell infiltration. The group explains that mast cells, a major target of masitinib, appear to play a crucial role in severe forms of sickle cell disease and its complications, such as vaso-occlusive crises, acute chest syndrome, and pain.
Clinical Development under SICKMAST Collaborative Program
The clinical development of masitinib in this indication is part of the SICKMAST collaborative program, funded with 9.2 million euros, AB Science reports. The Public Assistance-Paris Hospitals is the sponsor of this phase 2 study, which aims to demonstrate the efficacy of masitinib in treating the acute and chronic complications of sickle cell disease in patients identified based on biomarkers. The trial is designed in two stages: the identification and validation of biomarkers highlighting the role of mast cells and basophiles, followed by the demonstration of treatment efficacy. According to the press release, sickle cell disease affects more than 100,000 people in the United States and about 26,000 patients in France. Current therapeutic options do not fully prevent the complications of the disease, the company notes.
