Investigational Treatment for Alzheimer Disease Reports Positive Topline Clinical Data


The study demonstrated robust time and dose-dependent lowering of tau protein in cerebrospinal fluid (CSF) over the course of the 3-month treatment period.

The investigational therapy BIIB080/IONIS-MAPTRx (Biogen Inc and Ionis Pharmaceuticals) met its primary objective of safety and tolerability in patients with mild Alzheimer disease in a phase 1b placebo-controlled, multiple ascending dose clinical study, according to a press release from Biogen. The study further demonstrated robust time and dose-dependent lowering of tau protein in cerebrospinal fluid (CSF) over the course of the 3-month treatment period. These reductions were sustained over the 6-month post-treatment period.

Until recently, the treatment of Alzheimer disease was limited to symptom management. According to the investigators, there is growing evidence that suggests aggregated, hyperphosphorylated tau may be a key driver of neurodegeneration in Alzheimer disease, in addition to other tauopathies including progressive supranuclear palsy and frontotemporal degeneration. BIIB080 is an investigational antisense therapy designed to target microtubule-associated protein tau mRNA and prevent production of tau protein.

“There is clearly an urgent need to develop and deliver effective treatments for Alzheimer disease, a devastating disorder for which there currently are limited therapeutic options,” said C. Frank Bennett, PhD, chief scientific officer and franchise leader for neurological programs at Ionis, in a press release. “We are encouraged by the topline results from this study of BIIB080, which demonstrate the potential of Ionis’ antisense technology to successfully target what we believe is a root cause of Alzheimer disease. These study results support further investigation of BIIB080 for the treatment of Alzheimer disease and suggest that antisense-mediated suppression of tau protein may be a feasible therapeutic approach for other tauopathies.”

Dose-dependent decreases in the concentration of total tau in CSF were seen in patients receiving BIIB080 8 weeks after the final dose. Mean percentage reduction of 30%, 40%, and 49% were seen in the low, medium, and high dose groups treated every 4 weeks, respectively, and 42% in the group treated every 12 weeks. Total tau continued to decline 16 weeks after the final dose was administered in the high dose 4-week and 12-week dose groups, showing a 55% and 49% mean reduction from baseline, respectively.

“Biogen is encouraged by the results of this trial, and we look forward to our continued research in future clinical studies with this promising investigational asset,” said Alfred Sandrock, Jr., MD, PhD, head of Research and Development at Biogen, in the release.


Biogen and Ionis report positive topline clinical data on investigational Alzheimer’s disease treatment at AAIC [news release]. Biogen; July 26, 2021. Accessed July 29, 2021.

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