Top Biogen executives pushed back strongly against criticism of the company's new Alzheimer's medicine Aduhelm, decrying what they described as "extensive misinformation" around the drug and the process by which it was approved.
Speaking on a conference call Thursday, Biogen CEO Michel Vounatsos said the clinical trial results for Aduhelm justified the accelerated OK controversially granted by the Food and Drug Administration last month and denied that there were any irregularities in the regulator's review.
"Biogen stands behind our clinical data from eight studies with more than 3,000 patients that supported accelerated approval," Vounatsos said on the call, which was held to discuss the company's second quarter earnings results. "I want to be clear that Biogen stands behind the integrity of the review process."
The FDA approved Aduhelm despite contradictory clinical trial results that have left Alzheimer's doctors with significant uncertainty about whether the drug actually helps improve patients' lives. A group of advisers to the agency overwhelmingly rejected Biogen's case for the drug in a meeting late last year and documents released since showed FDA statisticians consistently opposed approval.
Recent reports from STAT and The New York Times, meanwhile, documented an unofficial meeting between a top FDA official and Biogen's head of research Al Sandrock — raising questions about the two organizations' subsequent close collaboration on Aduhelm — as well as an internal FDA council's conclusion that Biogen's evidence was insufficient.
Citing concerns about the FDA's review, acting commissioner Janet Woodcock this month requested an inspector general investigate the agency's decisionmaking process.
Speaking on the Thursday call, Sandrock said he welcomed the investigation. "A better understanding of the facts is good for everyone involved to assure confidence in both the therapy and the process by which it was approved," he said.
But Sandrock and Vounatsos also responded forcefully to the ongoing controversy around Aduhelm, blaming media coverage and what Sandrock described in an open letter as "a turn outside the boundaries of legitimate scientific deliberation."
"The background noise and the controversy are unfortunate and not helpful, mostly for the patients," said Vountasos. "Overall, [the launch] is a bit slower than what we assumed, but we are making tremendous progress with some positives and some headwinds."
In the few weeks between Aduhelm's June 7 approval and the close of the second quarter, Biogen recorded $1.6 million in sales for the drug. But some of that figure was the result of medical centers building inventory of Aduhelm, according to Vounatsos.
While several million Alzheimer's patients are estimated to fit the criteria for Aduhelm, Biogen expects sales will be gradual as insurance coverage is sorted out. As most people with the disease are older, coverage will mainly be given through Medicare, which this month began a nine-month process weighing whether to set a single, nationwide policy.
Biogen expects regional Medicare contractors to provide coverage while the national coverage determination process is underway, Vounatsos said, adding that the company has seen "first examples" of Medicare Advantage plans preauthorizing Aduhelm.
But some commercial insurers have said they won't cover the drug. UnitedHealth Group, the largest insurer in the country, is waiting on guidance from Medicare, while Anthem said this week it's continuing to evaluate Aduhelm.
A number of high-profile medical centers, including the Cleveland Clinic and Mount Sinai, have also indicated they won't administer the medicine, citing uncertainty over data and the investigation requested by the FDA.
Those decisions are "disappointing," said Biogen executive Alisha Alaimo, who noted physicians at those centers could still write prescriptions and that Biogen would help them find new sites that would allow administration.
Overall, Biogen said about a third of the 900 U.S. hospitals they are targeting have either cleared use of Aduhelm or indicated they would do so without an internal review by their pharmacy teams.
Investors are eager to see Aduhelm do well, as sales are declining for many of Biogen's other products. The company reported $2.8 billion in revenue between April and June, a 26% drop from the same period last year. U.S. sales of Tecfidera, a multiple sclerosis pill that lost patent protection in June 2020, plummeted by 80%.
Wall Street analysts were expecting even worse, however, and shares in the company were trading flat by mid-morning Thursday. Biogen raised its revenue forecast for the year to a range of $10.65 to $10.85 billion, reflecting estimated "modest Aduhelm revenue."