Masitinib demonstrates efficacy, safety in Alzheimer’s disease
Click Here to Manage Email Alerts
Masitinib, an oral tyrosine kinase inhibitor, improved measures of cognition, memory and activities of daily living in a phase 2b/3 study of patients with mild and moderate Alzheimer’s disease, according to a press release.
Moreover, fewer patients who received masitnib progressed to a severe stage of dementia, according to the release. The drug demonstrated an acceptable safety profile, one that was consistent with the known tolerability of the agent.
“The preliminary results from this study support efficacy on important outcomes assessing both cognition and function. The observed patient tolerability is encouraging,” Jeffrey L. Cummings, MD, director of the Chamber-Grundy Center for Transformative Neuroscience at University of Nevada, Las Vegas, said in the release. “Masitinib's mechanism is novel in its targeting of the innate immune system via mast cells and microglia. A growing body of evidence suggests that microglia play a central role in [AD] and other neurodegenerative disorders."
The phase 2b/3 ABO9004 study, an international, randomized, placebo-controlled trial, examined different doses of masitinib (4.5 mg/kg/day and a titrated dose from 4.5 to 6 mg/kg/day) in patients with confirmed mild to moderate AD. The researchers compared the efficacy and safety of masinitib with placebo following 24 weeks of treatment. They administered masitinib as an add-on to a cholinesterase inhibitor (donepezil, rivastigmine or galantamine) and/or memantine.
Masitinib 4.5 mg/kg per day, which was given to 182 patients, provided a significant treatment effect compared with the control arm (n = 176) regarding the primary endpoint of change from baseline in the AD Assessment Scale-Cognitive Subscale (P = .0003). Masitinib 4.5 mg/kg per day also resulted in a significant change from baseline on the AD Cooperative Study Activities of Daily Living score (P = .0381).
Significantly fewer patients treated with masitinib 4.5 mg/kg/day progressed to a severe stage of dementia after 24 weeks, according to the press release (P = .0446).
“The fact that masitinib could significantly reduce the proportion of patients reaching the stage of severe dementia is particularly interesting because this stage of the disease represents a significant burden for the society,” Bruno Dubois, MD, PhD, professor of neurology at the Neurological Institute of Salpetriere University Hospital at Paris and coordinating investigator of the study, said in the release.
The study results demonstrated an acceptable safety profile for masitinib 4.5 mg/kg per day, one consistent with the known tolerability profile for this agent, according to the release. At least one adverse event occurred in 87% of patients in the masitinib arm compared with 77.5% of patients in the control arm. More patients in the masitinib arm (13%) experienced at least one serious, non-fatal adverse event compared with the control arm (5.4%). Researchers reported at least one severe adverse event in 26.5% of participants in the masitinib arm vs. 19.3% of participants in the control arm.
“There is a vacuum of treatment options for patients with [AD] and today very few attempts to address the population with confirmed mild or moderate dementia associated with [AD],” Dubois said. “These data are very encouraging and may provide new hope for patients with [AD].”
Reference:
- AB Science. AB Science announces that phase 2B/3 study evaluating masitinib in Alzheimer’s disease met its primary endpoint. Available at: https://www.ab-science.com/positive-confirmatory-phase-2-3-study-ab09004-with-masitinib-in-alzheimers-disease/. Accessed Dec. 16, 2020.
Editor’s note: This story was updated on Thursday, Dec. 17, 2020, to reflect revised safety data.