AB Science gets European patent covering masitinib for SCD
Notice of allowance follows data showing treatment improved survival in mice
AB Science said it received a notice of allowance from the European Patent Office for a patent covering the use of the experimental oral therapy masitinib to treat sickle cell disease (SCD).
The decision was based on preclinical data demonstrating that masitinib was able to prevent vaso-occlusive crises (VOCs), a common complication of the disease, and increase survival in a mouse model of SCD. The therapy also protected mice from acute lung damage and inflammation.
The patent covers the use of masitinib for SCD until November 2040, said AB Science. The company holds patents covering the treatment’s use for other indications.
“Masitinib represents a promising novel strategy for treating sickle cell disease and its serious complication of acute chest syndrome, which can lead to the development of chronic lung disease and is a common cause of hospitalization or even death,” Olivier Hermine, MD, PhD, president of the AB Science scientific committee and head of Necker Hospital’s hematology department, said in a company press release.
In SCD, red blood cells stiffen and acquire a sickle-like shape that causes them to break down easily. Sickled red blood cells are also more prone to stick together and to other cells, so they get trapped inside blood vessels, blocking blood flow and compromising oxygen delivery to tissues and organs.
Blocking enzymes
Anemia, or a shortage of red blood cells, is one of the hallmark symptoms of SCD. Patients may also experience VOCs, episodes of acute pain and organ damage that occur when tissues become deprived of oxygen as a result of blood vessel blockage. Another potentially severe and fatal complication of the disease is acute chest pain syndrome, which occurs when blood vessels in the lungs become obstructed.
Masitinib is an orally available small molecule that blocks the activity of tyrosine kinases, enzymes that activate certain proteins involved in cellular processes including cell maturation, growth, and death.
It blocks the activity of tyrosine kinases that are essential for the activity of mast cells — a type of immune cells involved in inflammatory responses — that are thought to play a role in severe forms of SCD and its complications.
The treatment’s clinical development is being conducted as part of the SICKMAST collaborative program, which aims to demonstrate the efficacy of masitinib in treating acute and chronic SCD complications in patients identified based on biomarkers in a Phase 2 clinical trial.
AB Sciences is also testing masitinib for several neurodegenerative diseases, including amyotrophic lateral sclerosis, multiple sclerosis, and Alzheimer’s disease, as well as some types of cancer and inflammatory diseases.
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