AB Science to win patent covering medical use of masitinib for SCD
Patent valid until 2040, giving developer exclusive rights to advance therapy
The U.S. Patent Office has given AB Science notice that it will allow a patent to be issued covering the use of the experimental therapy masitinib for sickle cell disease (SCD).
This medical use patent will be valid until November 2040, giving AB Science exclusive rights to develop masitinib for SCD until that time.
A similar patent application was approved in Europe late last year, also covering the treatment candidate through 2040. Both patent decisions were backed by preclinical data showing that masitinib, which targets a type of immune cells called mast cells that are believed to play a key role in driving SCD complications, had numerous benefits in a mouse model of SCD.
“Masitinib is quite unique in the therapeutic landscape of sickle cell disease because no other development drug targets mast cells,” Olivier Hermine, MD, PhD, president of AB Science’s scientific committee and head of hematology at Necker Hospital in Paris, said in a company press release. “Masitinib therefore represents a promising novel strategy for treating sickle cell disease.”
Masitinib targets immune cells thought to drive SCD complications
In SCD, typically round and flexible red blood cells become rigid and sickle-shaped, making them prone to clumping up in blood vessels and obstructing blood flow. As such, compromised oxygen delivery to the body’s tissues can lead to pain episodes known as vaso-occlusive crises (VOCs), a hallmark SCD symptom. Sickle-shaped cells also break apart and die off more easily than healthy red blood cells.
People with severe forms of SCD can also experience a range of other complications, such as acute chest syndrome, or ACS, a medical emergency in which sickled red blood cells block blood vessels in the lungs.
Many currently available treatment options for sickle cell disease don’t adequately address the serious complications that can arise with SCD, according to AB Science. Other treatments, like gene therapy or stem cell transplants, can be expensive and are not accessible for all patients.
Mast cells, a class of immune cells involved in inflammatory responses, are believed to play a key role in driving the complications of severe SCD, including ACS and VOCs.
Masitinib is an oral therapy designed to block enzymes called tyrosine kinases that are important for immune cell activation — including mast cells. By reducing the activation of these cells, the company believes masitinib will be able to prevent SCD-related complications.
According to the company, in an SCD mouse model, all untreated mice experienced VOCs, and 83% died. But four days of treatment with masitinib completely prevented VOCs and death in another group of mice.
Moreover, the treatment protected against acute lung injury and mast cell infiltration into the lungs in a mouse model.
The therapy’s clinical development in SCD is being conducted via the SICKMAST collaborative program, which is backed by €9.2 million (nearly $10.5 million) in French public funding.
SICKMAST’s goal is to support a Phase 2 trial that will demonstrate masitinib’s ability to ease acute and chronic complications among SCD patients identified based on certain biomarkers. It would be sponsored by the Assistance Publique-Hôpitaux de Paris.
AB Science’s role is to provide masitinib and monitor safety data. The company would be free to later carry out any Phase 3 studies should the Phase 2 studies be successful.
The company is also developing masitinib for a range of other conditions, such as amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer’s disease, certain cancers, and other inflammatory diseases. It holds patents protecting masitinib’s use for some of these indications.
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