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)” - Seite 2

A new patent has been filed, which, if granted, will extend the international protection of masitinib in sickle cell disease until 2040. As part of the consortium agreement established for its patents, AB Science will pay royalties to APHP in the event of commercialization of masitinib in sickle cell disease.

Prof. Olivier Hermine, Necker-Enfants malades AP-HP, coordinator of the SICKMAST program, said, "We are delighted to be among the laureates of this program, recognized for its extremely rigorous selection of innovative and medically impactful projects. Sickle cell disease represents a major public health challenge due to the number of affected individuals and the early mortality it causes. This program should allow us to demonstrate the relevance of selectively targeting two key immune cells in the treatment of this disease and identifying patients most likely to respond through new biomarkers."

Alain Moussy, President and co-founder of AB Science, said, "We are very pleased with this collaboration with APHP, one of the largest promoters of clinical studies in France, whose research excellence is globally recognized. This project demonstrates the full potential of masitinib in targeting certain key immune cells involved in numerous pathologies. It opens an extremely promising new development avenue for masitinib and offers partnership prospects that we will explore to enable the registration and commercialization of masitinib in this indication under the best conditions."

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

• Scientific rationale
  
  

Inflammation mediated by innate immune cells and promoting vaso-occlusion has recently been shown to play a major role in sickle cell disease. In particular, our clinical observations and experimental work in mice, have revealed the involvement of mast cells and basophils in complications associated with sickle cell disease:

• The degree of mast cell activation in patients with sickle cell disease may contribute to the heterogeneity of inflammation and chronic and acute complications.
• The potential role of basophils in sickle cell disease has not been studied, however, given their role in various diseases and their ability to release substance P and histamine, they could also play important roles in the pathophysiology of sickle cell disease.

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