#AAN2018 – Early Data Supports Clinical Trial Now Testing IONIS-MAPTRx in Alzheimer’s Patients

#AAN2018 – Early Data Supports Clinical Trial Now Testing IONIS-MAPTRx in Alzheimer’s Patients
An injection of IONIS-MAPTRx resulted in a 70 percent decrease in tau protein produced in the brains of non-human primates, researchers at  Ionis Pharmaceuticals, the potential treatment's maker, are reporting. These positive pre-clinical findings helped in the design of a first clinical study, now underway and enrolling, that will assess IONIS-MAPTRx in people with mild Alzheimer’s disease. The results are in the oral presentation “Design of the First-in-Human Study of IONIS-MAPTRx, a Tau-lowering Antisense Oligonucleotide, in Patients With Alzheimer Disease” set to be given April 22 at the 2018 American Academy of Neurology (AAN) Annual Meeting, which runs April 21—27 in Los Angeles. Ionis recently launched a Phase 1/2 clinical trial  (NCT03186989) to evaluate multiple doses of IONIS-MAPTRx as a potential therapy for Alzheimer’s disease. In preclinical studies, in which Ionis researchers tested IONIS-MAPTRx's safety, pharmacokinetic (the movement of the drug once inside the body) and pharmacodynamic (the therapy’s effects on the body) profiles in rodents and non-human primates. IONIS-MAPTRx is an antisense oligonucleotide (ASO) that targets a messenger RNA molecule — called MAPT mRNA — that carries information coded by our DNA to ultimately produce a functional tau protein. As a result, IONIS-MAPTRx is designed to reduce pro
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