Briefing documents published by the US Food and Drug Administration (FDA) before an advisory committee meeting on Friday have initially backed Biogen’s potential Alzheimer’s treatment aducanumab.
The briefing documents will come before an advisory committee meeting which will help to determine if Biogen’s drug could become the first Alzheimer’s drug to be approved in 15 years.
The field of Alzheimer’s research has been littered with late-stage pipeline failures over the past decade, a fate that almost befell aducanumab last year.
Biogen initially scrapped the Eisai-partnered drug in phase 3, on advice from an independent data committee.
However, Biogen announced in a shock turnaround that results from a subset of patients in the phase 3 EMERGE study actually warranted taking the drug to regulators, bolstering hopes that it could be the first new treatment for the devastating neurodegenerative disease in over a decade.
Since then, Biogen has faced twists and turns on its way to achieving regulatory approval for the controversial drug, defending the efficacy of the treatment from critics as it submitted data to the FDA and the European Medicines Agency, respectively.
The FDA filing for aducanumab is supported by data from two phase 3 studies – EMERGE and ENGAGE – which evaluated the drug in patients with early-stage and mild Alzheimer’s disease, as well as a phase 1 study.
In the EMERGE study, patients who received the highest dose of the drug demonstrated a statistically significant improvement on a clinical dementia scale.
However, in the ENGAGE study, the drug didn’t show clinical efficacy, with patients who received the highest dose performing worse than individuals on the placebo arm.
Biogen has asserted that the effects of aducanumab were stronger as exposure to the drug accumulated, which if correct could mean that the divergence from placebo gets stronger with further follow-up.
After initially failing the futility analysis, Biogen collected additional data for aducanumab, which it says showed the drug reduced clinical decline, which convinced the company to submit the treatment for FDA review.
In the briefing documents, the FDA said that it ‘agrees that the results of Study 302 (EMERGE) are highly persuasive and the study is capable of providing the primary contribution to a demonstration of substantial evidence of effectiveness of aducanumab’.
The EMERGE trial ‘is a strongly positive study on multiple distinct and important clinical measures, robust to numerous sensitivity analyses, and supported by well-characterised biomarker data’, the agency added.
That’s positive news for Biogen and shares in the biotech climbed by 44% following the initial assessment.
Independent experts are set to review the drug in a Peripheral and Central Nervous System Drugs Advisory Committee meeting, scheduled for 6 November.