Biogen and Ionis have revealed topline data from an early-stage study evaluating its investigational Alzheimer’s disease drug candidate BIIB080 (IONIS-MAPT Rx).
The phase 1b study of BIIB080 met its primary objective, demonstrating an encouraging safety and tolerability profile in patients with mild Alzheimer’s disease.
In this study, investigators also noted that BIIB080 demonstrated both robust time- and dose-dependent lowering of tau protein in cerebrospinal fluid (CSF) over the three-month treatment period, with sustained reductions also observed in the six-month post-treatment period.
For BIIB080-treated patients, there were dose-dependent decreases in the concentration of total-tau in CSF eight weeks after their last dose. The mean percentage reduction was 30%, 40% and 49% in the low-, medium- and high-dose groups treated every four weeks respectively.
For patients in the group treated every 12 weeks, BIIB080 treatment led to a 42% reduction in the concentration of total-tau in CSF.
After 16 weeks post-dose, total tau in CSF also continued to decline in BIIB080-treated patients in both the high-dose four-week and 12-week groups, with a 55% and 49% reduction from baseline respectively.
“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’s disease,” said C Frank Bennett, chief scientific officer and franchise leader for neurological programmes at Ionis.
“These study results support further investigation of BIIB080 for the treatment of Alzheimer’s disease and suggest that antisense-mediated suppression of tau protein may be a feasible therapeutic approach for other tauopathies,” he added.
“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, head of research and development at Biogen.
In June, Biogen’s aducanumab was approved with the brand name Aduhelm for the treatment of Alzheimer’s disease, in a landmark moment for the therapy area.
The approval did not go without criticism, however, with The Institute for Clinical and Economic Review (ICER) – among others – maintaining that “the evidence on aducanumab is insufficient to be able to demonstrate that patients get benefits that would outweigh the risks and harms of this treatment”.