A crucial study evaluating Biogen's approved drug for Alzheimer's disease is set to begin in May, the company disclosed Thursday, and if enrollment goes as expected, initial results could arrive in four years.
The drug, known as Aduhelm, was cleared for the U.S. market in early June, making it the first new Alzheimer's treatment in nearly two decades. But in spite of that milestone, Aduhelm has been steeped in controversy due to its equivocal performance in clinical testing, its high price and the unusual way it was reviewed by the Food and Drug Administration.
In granting approval, the FDA stipulated that for Aduhelm to stay on the market, Biogen would need to run an additional clinical trial to affirm its drug works. The agency, though, drew further criticism by giving Biogen roughly nine years — or until 2030 — to submit final results from the trial.
Biogen now plans to have results in about half as long of a timeline. The company said Thursday that its study will be global, placebo-controlled and attempt to recruit more than 1,300 patients with early Alzheimer's disease. Each patient will be evaluated on the study's main measure 18 months after they've started treatment with Aduhelm. Biogen did not specify in its statement what that primary goal would be.
"We are delivering on our commitment to accelerate the timelines with the goal to complete the confirmatory study well ahead of schedule," said Priya Singhal, Biogen's interim head of research and development, in a Dec. 16 statement. Singhal became Biogen's head of R&D after Alfred Sandrock, the company's top scientist and chief defender of Aduhelm, unexpectedly retired last month.
A final study plan, or protocol, will be submitted to the FDA for review by March, Biogen said.
Alongside the confirmatory trial, Biogen is sponsoring other studies to assess Aduhelm when it's redosed or given in clinical practice. Together, such studies should generate data that "further inform patient and physician decisions about treatment."
Since launching Aduhelm, Biogen has had trouble convincing patients and their healthcare providers that the possible benefits of its drug outweigh the known safety concerns. To the consternation of doctors, Biogen has yet to publish in a peer-reviewed journal results from the two large clinical trials that led to Aduhelm's approval, one of which failed to show a benefit.
Insurers have also balked at Aduhelm's annual list price. At $56,000 on average, the drug could have an enormous impact on payer budgets if it's given to even a fraction of the estimated 6 million U.S. Alzheimer's patients. The Institute for Clinical and Economic Review, a watchdog group focused on drug costs, has said that, assuming the data supporting Aduhelm are true, the drug's price would still need to come down by around 90% to be considered cost-effective.
So far, the questions surrounding Aduhelm's effectiveness and value have hampered the drug commercially. In its first full quarter on the market, Aduhelm brought in just $300,0000 for Biogen, far below the $16 million Wall Street analysts had expected.
Use of the drug could grow if Medicare, which covers many of the people who would be eligible for treatment, decides to cover it. A draft decision is due in January and set to be finalized by April.
Biogen's new timeline means confirmation of Aduhelm's effectiveness is coming sooner than originally planned, but still years after Medicare and other insurers must make decisions on coverage. Next year will also bring results for trials of other experimental Alzheimer's drugs from Eli Lilly, Roche and Biogen's research partner Eisai, which could change how Aduhelm, and its supporting data, are perceived.