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New data presented at AD/PD™2023 show IONIS-MAPT Rx (BIIB080) substantially reduced tau protein in patients with early-stage Alzheimer's disease
Phase 1b study showed dose-dependent and sustained reduction of tau protein in cerebrospinal fluid through open-label long-term extension (LTE)IONIS-MAPTRx
About this update from Ionis Pharmaceuticals, Inc.
[{"type":"text","content":"Phase 1b study showed dose-dependent and sustained reduction of tau protein in cerebrospinal fluid through open-label long-term extension (LTE)IONIS-MAPTRx reduced aggregated tau pathology across all brain composites assessed starting as early as week 25 through week 100CARLSBAD, Calif., March 29, 2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today reported that its partner Biogen presented new Phase 1b clinical data showing that IONIS-MAPTRx (BIIB080) reduced soluble tau protein in cerebrospinal fluid (CSF) in a dose-dependent and sustained manner in patients with early-stage Alzheimer's disease (AD). IONIS-MAPTRx also reduced aggregated tau pathology, as measured by positron emission tomography (PET) in all brain composites assessed. The primary endpoint of the Phase 1b study (25 weeks) and open-label long-term extension study (through week 100) was safety and tolerability, with biomarker data as an exploratory endpoint. The results were presented today at the International Conference on Alzheimer's and Parkinson's Diseases (AD/PD™ 2023). \n\n \n \n \n \n \n \n\n \nIn patients with AD, tau protein can form \"tangles\" that progressively accumulate in brain regions involved in cognition. The accumulation of pathological tau tangles has been shown to promote neuronal damage and death. IONIS-MAPTRx is an investigational antisense medicine discovered by Ionis that is being developed by Biogen. It is designed to target microtubule-associated protein tau (MAPT) mRNA and prevent production of tau protein.\n\"This Phase 1b clinical study of IONIS-MAPTRx is the first to demonstrate this magnitude of a reduction of tau pathology across important brain regions. These encouraging findings are the latest example of the potential of the Ionis RNA-targeting platform to selectively modulate proteins linked to CNS disease,\" said Eugene Schneider, M.D., executive vice president and chief clinical development officer at Ionis. \"Ionis is pioneering potential therapies for serious neurological diseases with high unmet need. In addition to Alzheimer's disease, our clinical stage neurology programs include ATTR polyneuropathy, ALS, Alexander disease, Parkinson's disease and Angelman syndrome.\"\nThe Phase 1b trial and its open-label long-term extension (LTE) were designed to evaluate the safety and tolerability of multiple dose l...