Biogen, Eisai complete submission of controversial Alzheimer’s drug. The ball is in FDA’s court now.

The FDA has 60 days to decide whether or not to accept the application. An analyst wrote that the agency accepting it with a priority or standard review may signal whether the agency is seriously considering approval or has continued reservations about the amyloid beta-targeting drug’s clinical benefit.

Dementia or brain damage and injury as a mental health and neurology medical symbol with a thinking human organ made of crumpled paper torn in pieces as a creative concept for alzheimer disease.

Two companies that developed an Alzheimer’s disease drug that has attracted significant controversy have completed their submission of data to the Food and Drug Administration, which has 60 days to determine whether or not to accept it.

Cambridge, Massachusetts-based Biogen and Tokyo-based Eisai said Wednesday that they had finished their biologics license application, or BLA, for aducanumab, a drug designed to treat Alzheimer’s by attacking amyloid beta plaques in the brain. The companies said that if aducanumab is approved, it would be the first drug with potential meaningfully change the course of the disease. The BLA package includes data from the Phase III EMERGE and ENGAGE studies and the Phase Ib PRIME study.

Shares of Biogen were up about 5% in late-morning trading on the Nasdaq following the news. Shares of Eisai were up nearly 9% on the over-the-counter markets, where they trade in the U.S.

“Alzheimer’s disease remains one of the greatest public health challenges of our time,” Biogen CEO Michel Vounatsos said in a statement. “The aducanumab BLA is the first filing for FDA approval of a treatment that addresses the clinical decline associated with this devastating condition, as well as the pathology of the disease.”

The 1,638-patient EMERGE study met its primary endpoint, with patients receiving high-dose aducanumab showing a statistically significant reduction in clinical decline, as measured by the Clinical Dementia Rating-Sum of Boxes score, after 78 weeks, in comparison with those receiving placebo. Statistically significant reductions were also seen in the Mini-Mental State Examination and Alzheimer’s Disease Cooperative Study-Activities of Daily Living Inventory Mild Cognitive Impairment Version. Deposits of amyloid beta, or abeta, were shown through imaging to be statistically significantly lower in the aducanumab group than in the placebo group. The companies said that while the 1,647-patient ENGAGE study missed its primary endpoint, they are of the view that certain subset data support the outcome in EMERGE.

In a note to investors, Cowen analyst Phil Nadeau wrote that while the filing is in line with third-quarter guidance, investors may be “relieved” that there was no disruption from the companies’ recent pre-BLA meeting with the FDA. Citing an FDA regulatory consultant, Nadeau wrote that if the agency accepts the application and grants it priority review, it would be the first tangible sign that it is seriously considering approval. On the other hand, a standard review would be “disappointing” and signal that the agency has reservations about Biogen and Eisai’s claims regarding aducanumab’s clinical benefit.

Still, the drug has not been without controversy. Biogen and Eisai halted EMERGE and ENGAGE in March 2019 following a futility analysis by the independent data monitoring committee that showed the trials were likely to fail. Given aducanumab’s demonstrated potency against amyloid beta, the program’s ostensible failure reinforced growing doubts about the hypothesis that it would be possible to treat the disease by targeting plaques in the brain, especially after a string of failures of other abeta-targeting drugs. But the companies revived the drug soon thereafter, when a subsequent analysis indicated a benefit. Nevertheless, a presentation of data in December at the Clinical Trials on Alzheimer’s Disease conference mostly failed to sway analysts.

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