PARIS—Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, is a rare degenerative disorder that results in progressive wasting and paralysis of voluntary muscles. There are approximately 50,000 people with ALS in the European Union and in the United States, with more than 16,000 new cases diagnosed each year. Almost 80 percent of ALS patients die within five years, and 90 percent die within 10 years.
AB Science SA, a pharmaceutical company specializing in the research, development and marketing of protein kinase inhibitors (PKIs), thinks it has an answer. The company announced that new mechanistic data demonstrate the unexpected protective effect of its compound, masitinib, on muscles and nerves in ALS.
Masitinib is a new orally administered tyrosine kinase inhibitor that targets mast cells and macrophages, important cells for immunity, through inhibiting a limited number of kinases. Based on its unique mechanism of action, masitinib can be developed in a large number of conditions in oncology, in inflammatory diseases and in certain diseases of the central nervous system.
The findings were presented at the 27th International Symposium on ALS/MND, the largest medical and scientific conference specific to ALS, in December 2016 in Dublin, Ireland. They confirmed previous findings demonstrating the inhibiting effect of masitinib on microglial cells and survival prolongation in post-paralytic SOD1G93A rats, as well as clinical data from the phase 2/3 interim analysis showing the positive effect of masitnib on the progression of disability in patients with ALS, improving quality of life and survival.
Prof. Luis Barbeito, head of the Montevideo, Uruguay-based Neurodegeneration Laboratory of the Institut Pasteur, delivered the presentation. He explained, “Beyond our recently published findings, we have acquired additional preclinical data showing neuroprotective effects of masitinib in ALS. We have now shown that masitinib generates its observed neuroprotective effect in ALS by regulating neuroinflammation in the peripheral nervous system as well as the central nervous systems, and that it also penetrates the blood-brain barrier to a greater extent than previously thought. Overall, these data provide further compelling pharmacological rationale for the recently reported positive Phase 3 interim analysis.”
“AB Science was founded in 2001 by a team of researchers, clinicians and entrepreneurs to discover key mechanisms in life science and develop new drugs targeting these mechanisms to dramatically change the life of patients in the most unmet medical needs,” according to Alain Moussy, co-founder and CEO. “Our group is specialized in targeted therapies through tyrosine kinase inhibitors for treating diseases with high medical needs in the field of cancers, chronic inflammatory diseases and neurological degenerative disorders. Our pipeline is both in human and veterinary medicine. Our drugs are aimed to be used by hospitals and specialist physicians.”
He added, “Our strategy is aimed at becoming an integrated pharmaceutical company with strong drug discovery, development and marketing capabilities in the specialty pharmaceutical sector present in the USA and Europe.”
AB Science is headquartered in Paris and has a subsidiary in New Jersey. It is the only small independent pharmaceutical company to have registered a tyrosine kinase inhibitor. AB Science has developed a lead compound, masitinib, registered in veterinary medicine and developed in several indications in human medicine. Tyrosine kinase inhibitors are the second generation of targeted therapies and represent a new and potent therapeutic option for numerous diseases.
AB Science is currently pursuing 13 Phase 3 studies in human medicine in metastatic prostate cancer, metastatic pancreatic cancer, relapsing metastatic colorectal cancer, relapsing metastatic ovarian cancer, first- and second-line gastrointestinal stromal tumors, metastatic melanoma expressing JM mutation of c-Kit, relapsing multiple myeloma, relapsing T cell lymphoma, severe asthma, amyotrophic lateral sclerosis, Alzheimer’s disease and progressive forms of multiple sclerosis.
In other recent AB Science news, the company announced several days after the ALS/masitinib news that results from the pivotal Phase 3 study of masitinib in severe indolent systemic mastocytosis were published in The Lancet, with the company noting that masitinib is the first drug to demonstrate efficacy in a Phase 3 study of adult patients with severe indolent systemic mastocytosis, including the subvariant of smouldering systemic mastocytosis—patients who are unresponsive to existing, optimal symptomatic treatments.