Alzheimer’s: Ionis Initiates phase I/II trial for antisense drug

Ionis Pharmaceuticals has initiated its Phase I//IIa clinical trial designed to test IONIS-MAPTRx in patients with moderate Alzheimer’s disease. Having initiated this study, Ionis will receive a $10m milestone payment from Biogen. IONIS-MAPTRx is an antisense drug designed to selectively reduce the production of MAPT (microtubule-associated protein tau), or tau protein, in the brain. Alterations to this protein are believed to be a hallmark feature of some neurodegenerative diseases known as tauopathies, which include Alzheimer’s disease and some forms of frontotemporal dementia. The new trial, therefore, follows an alternative research pathway to the common one, targeting amyloid plaques. The study is a 3-month, randomized, placebo-controlled, dose escalation trial, designed to evaluate the safety and activity of the product in 44 patients. IONIS-MAPTRx will be administered as a once-monthly intrathecal injection directly into the cerebral spinal fluid. Biogen has an option to develop and commercialize the drug.
(Source: Ionis Pharmaceuticals press release)