Biogen recently unveiled the new results of studies on aducanumab, an investigational drug for early-stage Alzheimer’s disease, and said it would seek the U.S. Food and Drug Administration’s approval.
If the company wins the FDA’s nod, aducanumab will be the first treatment to mitigate the cognitive decline in Alzheimer’s and prove that the elimination of amyloid-beta has clinical effects on Alzheimer's.
Biogen’s plan to seek regulatory approval was based on the results of phase-3 trials EMERGE and ENGAGE on aducanumab. The two were multi-center, randomized, double-blind, and placebo-controlled studies, conducted on 1,638 and 1,647 patients with Alzheimer's disease, respectively.
In March, the company scrapped the two studies because the futility analysis of aducanumab data showed that the trials had little chance of succeeding. However, it secured massive additional data on 2,066 out of 3,285 participants of the existing trials who continued the treatment for 18 months, and announced the experimental drug was effective to curb the cognitive decline of the patients.
Aducanumab was pharmacologically and clinically active, showing dose-dependent effects in lowering brain amyloid and clinical decline, according to Biogen.
Patients who received aducanumab experienced significant benefits in cognition and function, such as memory, language, and orientation, the company said. Patients also experienced benefits on activities of daily living such as managing personal finances, performing household chores (cleaning, shopping, and doing laundry), and independently traveling out of the home.
“This large dataset represents the first time a phase-3 study has demonstrated that clearance of amyloid-beta can reduce the clinical decline of Alzheimer’s disease, providing new hope for the medical community, the patients, and their families,” said Dr. Anton Porsteinsson, principal investigator and a professor of psychiatry, neurology and neuroscience at the University of Rochester. “Alzheimer’s disease community has been waiting for this moment. I commend Biogen, the FDA, the medical community, and the patients and their study partners for their persistence in working to make today’s announcement a reality.”
After consultations with the FDA, Biogen plans to submit a Biologics License Application (BLA) in early 2020 and continue dialogue with regulatory authorities in international markets, including Europe and Japan. The BLA submission will include data from the phase-1/1b studies as well as the complete set of data from the phase-3 trials.
Biogen said it would present further detail on the new analysis of the data from EMERGE and ENGAGE at the Clinical Trials on Alzheimer's Disease (CTAD) meeting next month.
Michel Vounatsos, CEO of Biogen, said the company’s announcement was “truly heartening” in the fight against Alzheimer’s, which affects tens of millions worldwide.
“We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer’s disease and the potential implication of these results for similar approaches targeting amyloid-beta,” he added.