Ionis Pharmaceuticals Starts Phase 1/2 Clinical Trial of Alzheimer’s Therapy IONIS-MAPTRx

Ionis Pharmaceuticals Starts Phase 1/2 Clinical Trial of Alzheimer’s Therapy IONIS-MAPTRx
Ionis Pharmaceuticals has started a Phase 1/2 clinical trial to evaluate the Alzheimer’s therapy IONIS-MAPTRx. IONIS-MAPTRx is aimed at reducing the production of tau protein, whose accumulation in the brain is a hallmark of the disease. The trial will evaluate the therapy in patients with mild forms of Alzheimer's. Therapies targeting clusters of a toxic form of tau could prove a valuable way to treat Alzheimer's, according to a study published in February. The article, “Tau Reduction Prevents Neuronal Loss And Reverses Pathological Tau Deposition And Seeding In Mice With Tauopathy,” appeared in the journal Science Translational Medicine i Tau misfolding leads to an accumulation of toxic tangles inside brain cells. The tangles are a key feature of neurodegenerative diseases known as tauopathies. They include Alzheimer's and some forms of frontotemporal dementia, a rare early-onset disease that can result from neuronal damage to the brain's frontal and temporal lobes. "In contrast to amyloid plaques that may begin to deposit in the brain for up to 20 years before the onset of AD [Alzheimer's], tau deposits are spatially and temporally associated with the brain regions where atrophy occurs and neurocognitive deficits originate," C. Frank Bennett, Ionis' senior vice president of research, said in a press release. "IONIS-MAPTRx is designed to reduce the production of all of the many forms of tau in all regions of the brain." The trial (NCT03186989) will be a three-month randomized, placebo-controlled, dose-escalation study. It will cover the safety, characteristics and behavior of IONIS-MAPTRx, and patients' ability to tolerate it. About 44 patients are expected to take part in the trial. IONIS-MAPTRx will be injected once a month into par
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