Ionis Pharmaceuticals’ IONIS-MAPTRx shows tau reduction efficacy in Alzheimer’s

Ionis Pharmaceuticals (IONS) reported that its partner Biogen (BIIB) presented new Phase 1b clinical data showing that IONIS-MAPTRx reduced soluble tau protein in cerebrospinal fluid in a dose-dependent and sustained manner in patients with early-stage Alzheimer’s disease. IONIS-MAPTRx also reduced aggregated tau pathology in all brain composites assessed. The primary endpoint of the Phase 1b study and long-term extension study was safety and tolerability, with biomarker data as an exploratory endpoint. 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 mRNA and prevent production of tau protein. In this study, the majority of adverse events were mild or moderate in severity, of which the most common were headache, back pain, and post-lumbar puncture syndrome. The results showed that IONIS-MAPTRx reduced biomarkers of soluble tau in CSF in a dose-dependent and sustained manner, with all dose groups showing approximately a 60% reduction from baseline CSF tau levels by the end of the LTE. The Phase 2 study of IONIS-MAPTRx is in progress and currently recruiting participants in the U.S.