AB Science Secures US Patent for Masitinib in Sickle Cell Disease Treatment Through 2040
Key Insights
AB Science (search) received formal US patent approval (US12,472,164) for masitinib in treating sickle cell disease (search), providing intellectual property protection until November 2040.
Preclinical studies demonstrated masitinib's survival benefit in sickle cell disease (search) mouse models, with 100% survival in treated mice compared to 83% mortality in controls within three hours.
The company is developing masitinib for severe forms of sickle cell disease (search) representing approximately 65% of cases, targeting mast cell-mediated complications through a fully funded €9.2 million Phase 2 trial.
AB Science (search) announced that the United States Patent Office has formally granted a patent for methods of treating sickle cell disease (search) using its lead compound masitinib, based on preclinical data. The new US patent (US12,472,164) ensures intellectual property protection for masitinib until November 2040, strengthening the company's portfolio for this high unmet medical need indication.
Targeting Severe Forms of Sickle Cell Disease
Masitinib is being developed to treat the most severe forms of sickle cell disease (search), which account for approximately 65% of cases. According to AB Science (search), severe sickle cell disease poses a major public health challenge and often leads to early death. The company explains that mast cells (search), a major target of masitinib, appear to play a crucial role in severe forms of sickle cell disease and its complications, including vaso-occlusive crises (search), acute chest syndrome (search), and pain.
Current treatment options such as hydroxycarbamide and chronic transfusion do not fully prevent life-threatening acute and chronic complications of sickle cell disease (search). While gene therapy targeting the HbS mutation can be curative, this option remains extremely limited due to donor scarcity, unresolved safety challenges, and high costs.
Promising Preclinical Results
Masitinib demonstrated significant survival benefits in a murine model of sickle cell disease (search). According to the company's data, all control mice affected by the disease exhibited vaso-occlusive crises (search), and 83% of them died within the first three hours. In contrast, mice pre-treated with masitinib for four days showed neither vaso-occlusive crises nor deaths.
Histological and immunohistochemical analyses revealed that masitinib protects against acute lung injuries and mast cell infiltration in the sickle cell disease (search) mouse model. The drug functions as an inhibitor of KIT (search), LYN (search), and FYN (search), three major kinases involved in the activation of mast cells (search) and basophils.
Fully Funded Clinical Development Program
Masitinib's clinical development in sickle cell disease (search) is being conducted as part of the SICKMAST collaborative program, funded with €9.2 million. The program aims to demonstrate in a Phase 2 clinical trial the efficacy of masitinib in treating acute and chronic complications of sickle cell disease in patients identified based on biomarkers.
The Assistance Publique-Hôpitaux de Paris (AP-HP) serves as the sponsor of this Phase 2 study, which is designed in two parts. Part 1 focuses on identification and validation of biomarkers highlighting the role of mast cells (search) and basophils in orchestrating acute and chronic complications of sickle cell disease (search). Part 2 will demonstrate masitinib's efficacy in treating these complications in biomarker-identified patients.
Addressing Global Disease Burden
Sickle cell disease (search) affects millions of people worldwide, with estimates suggesting that around 300,000 children are born with the condition each year, potentially reaching 400,000 by 2050. The disease affects over 100,000 children and adults in the United States, while approximately 26,000 patients are affected in France.
The disease extends beyond episodic pain crises, with chronic hemolysis, vaso-occlusion, and endothelial dysfunction leading to progressive organ damage affecting the brain, kidneys, lungs, and spleen. This cumulative damage reduces life expectancy by 20-30 years even in high-income settings, and patients often develop complications that current therapies cannot reverse.
Recent treatment failures have highlighted the continued unmet need in this space. Anti-P-selectin antibodies, once considered promising treatment options, have failed to confirm their efficacy. Voxelotor, a hemoglobin modifier that was conditionally approved, was withdrawn from the market due to increased occurrence of fatal stroke in post-marketing trials.