AB Science has announced that the United States Patent Office has formally granted a patent for the use of its lead compound, masitinib, in the treatment of sickle cell disease (SCD), providing intellectual property protection until November 2040. 

“This decision strengthens masitinib’s intellectual property portfolio with long-term protection for an additional indication with a high unmet medical need,” the company said. 

Masitinib targets mast cells, which play a critical role in severe forms of SCD and its complications, including vaso-occlusive crises, acute chest syndrome, and pain. Preclinical studies have shown striking results: “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.” Lung tissue analyses further revealed that masitinib protects against acute lung injury and mast cell infiltration. 

SCD affects millions worldwide, with severe cases accounting for roughly 65% of patients. Current treatments, including red blood cell transfusions and hydroxyurea, fail to fully address the disease’s complications, while gene therapy remains limited by donor scarcity, high cost, and safety challenges. 

AB Science confirmed that masitinib’s Phase 2 clinical development is fully financed through the SICKMAST collaborative program, funded with €9.2 million. The study, sponsored by Assistance Publique-Hôpitaux de Paris (AP-HP), will proceed in two stages: 

Stage 1 involves identification and validation of biomarkers highlighting mast cells’ and basophils’ role in SCD complications. Stage 2 tests masitinib’s efficacy in treating acute and chronic SCD complications in patients selected based on biomarkers. 

AB Science emphasized that it “remains free to carry out, as it sees fit, any potential phase 3 development following the success of phase 2.” 

With this U.S. patent in hand, masitinib now has long-term intellectual property coverage for an indication with significant unmet medical needs, positioning AB Science at the forefront of sickle cell disease research.