Biogen stock skidded Wednesday as the company scrambled to respond to a document release outlining division at the Food and Drug Administration over Biogen’s Alzheimer’s drug, Aduhelm.
On the stock market today, Biogen (BIIB) shares fell 0.7% to 371.90. That added to a nearly identical fall on Tuesday after the FDA released documents showing the internal strife the drug formerly known as aducanumab caused at the agency. FDA biostatisticians objected to the approval, saying the data didn’t support it.
Tristan Massie, a biostatistics reviewer at the FDA, “does not agree that the totality of the data provides sufficient evidence to support the (effectiveness) of aducanumab in Alzheimer’s disease and does not recommend approval,” according to one document.
In response, Biogen noted in a written statement it has nine years to complete a study confirming the benefit of the Alzheimer’s treatment.
Aduhelm gained accelerated approval on the basis that it removes beta amyloid, a plaque that builds up in the brains of Alzheimer’s patients. But the drug’s clinical benefit has yet to be established.
Biogen Stock Falls On Aduhelm Strife
Aduhelm is the first approved Alzheimer’s treatment that attempts to correct an underlying pathology of the disease. Biogen stock surged to a record high on the approval.
On that measure, Aduhelm works. But experts don’t agree that removing plaque slows the cognitive decline in Alzheimer’s patients. Internal documents suggest the data were inconclusive but strong enough to allow doctors and patients to make their own decision.
The FDA worked with Biogen for over two years to parse through the data. Aduhelm, formerly known as aducanumab, failed to pass muster in one of two final-phase studies. In the successful study, patients who received a high dose of Aduhelm declined slightly more slowly than those who received the placebo.
Making things more complicated, Biogen stopped both Phase 3 studies early — deciding they were unsuccessful — and that caused Biogen stock to collapse in 2019. That also led the FDA and Biogen to complete an ad hoc analysis to determine what they thought would have happened, if the studies had been completed.
Confirmatory Study To Begin
The FDA used the successful Phase 3 study and a smaller midstage test as its basis for accelerated approval. The move buoyed Biogen stock. Biogen now has nearly a decade to complete a confirmatory study.
Peter Stein, director of the FDA’s Office of New Drugs, acknowledged Biogen didn’t provide evidence for a standard approval.
“FDA, then, is faced with a situation where the available evidence on the clinical endpoints is short of what we would require for standard approval of aducanumab,” he said in a memo. “At the same time, patients with (Alzheimer’s) have a serious, progressive, ultimately fatal disease and are desperate for treatments that delay progression and prolong well-functioning survival.”
But the accelerated approval flew in the face of an advisory committee that voted overwhelmingly against recommending Aduhelm’s approval. As a result, several members of the panel resigned following the approval. Biogen stock tumbled on the advisory committee’s vote.
Potential Blockbuster Treatment
Aduhelm stands a chance of being a major blockbuster for Biogen. The drug costs $56,000 for a year of maintenance treatment. By 2025, Biogen stock analysts polled by FactSet expect Biogen to bring in $15.97 billion in total sales. That would grow almost 17% from 2020.
About 6 million Americans suffer from Alzheimer’s disease — a number expected to increase to 13 million by 2050. Biogen says Aduhelm could benefit 1 to 2 million of them.
Part of the controversy for Biogen stock stems from the additional cost of scans for brain swelling. That’s one side effect of Aduhelm. In addition, patients need to undergo semiregular cognitive testing. Biogen acknowledged the potential out-of-pocket costs for patients and families.
Biogen also noted the Black and Latinx communities are more likely to develop the disease. In its statement, the company said it would have signed agreements with CVS Health and the National Association of Free and Charitable Clinics to address the disparities in health care access.
Follow Allison Gatlin on Twitter at @IBD_AGatlin.
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