AB Sciences has more reasons to believe that its protein kinase inhibitor (PKI) masitinib could have further clearances, as the results from the pivotal phase 3 study have been published in highly regarded independent general medical journal, The Lancet.
Based on the phase 3 study results, AB Science filed for registration to the European Medicines Agency (EMA) in April 2016 for masitinib in severe indolent systemic mastocytosis. In July, the FDA granted a Single Patient IND for Compassionate Use of masitinib for patient with amyotrophic lateral sclerosis (ALS).
AB presented in December in Dublin recent data about masitinib at the International Symposium on ALS/MND. The mechanistic data showed some unexpected protective effect of masitinib on muscles and nerves in amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
The European Medicines Agency (EMA) has also recently accepted the filing of masitinib for Lou Gehrig’s disease. In August, EMA Committee for Orphan Medicinal Products (COMP) granted orphan
drug status to Masitinib.
Dr Michel Arock from the Ecole Normale Supérieure de Cachan (Paris, France), an expert in mastocytosis commissioned by The Lancet to comment on the published article concluded “These data offer high hopes for masitinib, which could become an attractive alternative to the therapeutic arsenal available for patients with severely symptomatic indolent or smouldering systemic mastocytosis.”
Alain Moussy, Chief Executive Officer of AB Science said “Publication of these pivotal phase 3 data in a prestigious medical journal such as The Lancet marks another important milestone in the path to bring masitinib to patients with severely symptomatic indolent systemic mastocytosis, an indication for which there is currently no registered or established standard treatment and a high unmet medical need. Considering that The Lancet sets extremely high standards for peer review and acceptance, its publication of these data is an indication that Key Opinion Leaders consider masitinib as a potential new treatment option for adult patients with severely symptomatic indolent systemic or smouldering systemic mastocytosis, who are unresponsive to existing symptomatic treatments.”
Professor Olivier Hermine M.D., senior author of the paper, said: “The AB06006 study data now published in The Lancet show, for the first time, a new therapy that can substantially reduce severe symptoms associated with indolent systemic mastocytosis, regardless of a patient`s c-Kit mutational status. Study AB06006 successfully achieved its primary and secondary objectives; moreover, both efficacy and safety data indicate a possibility for effective long-term management of this difficult-to-treat condition. This observation is important, given that indolent systemic mastocytosis is a chronic condition that requires lifelong management. Masitinib may therefore be an important new treatment option for these patients.”