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The study tested the effectiveness of orally administered masitinib in slowing the progression of symptoms in patients with primary progressive MS (PPMS) and non-active secondary progressive MS (nSPMS). Generally, these are the most severe forms of the condition. The patient outcomes were measured using the Expanded Disability Status Scale (EDSS), a benchmark scale within the field for monitoring changes in level of disability over time.
The lead investigator for this study was Prof. Patrick Vermersch, of the Department of Neurology at the University of Lille, considered a key opinion leader on the disease and its treatment.
Professor Vermersch has authored numerous studies on drugs for diseases in the field of neurology and, in particular, multiple sclerosis, with notable involvement in the development of Roche’s Ocrevus (ocrelizumab) and Novartis’s Mayzent (siponimod). He also lead the pilot study for masitinib in the treatment of patients with progressive MS.
The new pivotal study results showed the success of masitinib in the treatment of progressive MS, Prof. Vermersch explained during the conference. In particular, he drew attention to the delay in the progression of disability, measured in average overall change to level of disability.
The probability of patients treated with masitinib having either more disease improvements or fewer disease progressions was increased by 39%, the professor highlighted.
Notably, masitinib compared favourably to the few other drugs currently available for progressive forms of MS, the professor added, with this point being echoed by other experts at the conference.
The lead investigator for this study was Prof. Patrick Vermersch, of the Department of Neurology at the University of Lille, considered a key opinion leader on the disease and its treatment.
Professor Vermersch has authored numerous studies on drugs for diseases in the field of neurology and, in particular, multiple sclerosis, with notable involvement in the development of Roche’s Ocrevus (ocrelizumab) and Novartis’s Mayzent (siponimod). He also lead the pilot study for masitinib in the treatment of patients with progressive MS.
The new pivotal study results showed the success of masitinib in the treatment of progressive MS, Prof. Vermersch explained during the conference. In particular, he drew attention to the delay in the progression of disability, measured in average overall change to level of disability.
The probability of patients treated with masitinib having either more disease improvements or fewer disease progressions was increased by 39%, the professor highlighted.
Notably, masitinib compared favourably to the few other drugs currently available for progressive forms of MS, the professor added, with this point being echoed by other experts at the conference.
Expert opinions
The results were positive, with the masitinib study showing clear efficacy, explained the researchers and the commenting medical experts of the panel. Additionally, the mechanism of action which allow masitinib to function are reportedly unique in the field of MS treatment.
Masitinib targets a different manifestation of MS, at the later stages when the typical inflammation caused by antibodies of the active immune system has subsided, explained Prof. Bob Fox, who is the staff neurologist at the Mellen Center for Multiple Sclerosis and Professor of Neurology at Cleveland Clinic Lerner College of Medicine.
“What it is replaced by is the innate immune system – the immune system that lives within the brain. What is clear is that [with masitinib] there is activity on the innate immune system, and the innate immune system is thought to be involved in progressive MS,” Prof. Fox said.
What is exciting for the MS community is that masitinib works differently to any progressive MS therapy currently available, Prof. Fox said. The compound acts on mast cells and microglia in the brain, a characteristic that has also been demonstrated through a number of separate studies with masitinib in the treatment of other neurological disorders.
Researchers saw “very compelling differences in the progression” of patients’ disability over the course of treatment with masitinib, particularly patients with more advanced symptoms, Prof. Fox concluded.
The strong safety profile of the drug, and the data were very convincing when compared to the other compounds for the treatment of progressive forms of MS, explained Prof. Friedmann Paul, Professor of Clinical Neuroimmunology and head of the neuroimmunology outpatient clinic at the Experimental and Clinical Research Centre in Berlin.
During the conference, he too expressed interest in the unique mode of action which, he said, is new to the field of MS therapy.
“What I find appealing about this study and the results...is that the efficacy is convincing...in a population with advanced disease [symptoms]. This is something that is unique to the study,” he said.
The experts were in agreement that this was because other drugs for progressive forms of MS target inflammation caused by the active immune system, which becomes less of a factor as the disease progresses to advanced stages.
What happens next?
Following results of the phase 3 trial, AB Science has filed a second patent for the novel therapeutic application of masitinib in the treatment of progressive forms of MS. If granted, this will provide market protection until 2040.
At present, the company has protection for the use of masitinib in the treatment of multiple sclerosis until 2031. Following the success of the phase 3 trial, AB Science will coordinate with regulators in the US and Europe, with the possibility of a confirmatory study to be conducted in the near future.
