Biogen's Aduhelm can't catch a break even in Alzheimer's trials as neurologists worry about safety, efficacy

With federal payment for Biogen’s controversial Alzheimer’s disease drug, Aduhelm, restricted to clinical trials only, neurologists don’t even want to use the therapy in this limited setting.

In a survey of 81 U.S.-based neurologists, 52% said they were “not likely” to allow their patients to go into a trial to access Aduhelm via Medicare, while 46% said they were “somewhat likely,” according to a new report out by consultants at Spherix.

And only 12% of respondents have Alzheimer’s patients who have received at least one Aduhelm infusion. Concerns about safety (60%) and efficacy (57%) were the top reasons respondents didn’t want to use the drug.

This comes despite there being only a handful of old drugs on the market, all of which have limited efficacy. The disease affects nearly 7 million Americans.

The survey was undertaken last month, just after the Centers for Medicare & Medicaid Services (CMS) ruled that Medicare will only pay for the drug for patients enrolled in approved studies. This means it won’t be paid for in a normal commercial setting, severely cutting its already meager sales potential.

Aduhelm works by removing so-called amyloid plaques, misfolded proteins that are believed to be a central culprit in the memory-wasting disease. The problem is that clearing these plaques has not been shown to significantly improve disease progression in Alzheimer’s patients.

The drug was also approved on questionable efficacy data and was rejected by the FDA’s expert committee. But in a turnaround, the FDA went ahead and approved Aduhelm, but the med has struggled to make any sales headway. It brought in less than $10 million over the past two quarters. 

Besides the debatable efficacy, Aduhelm also comes with some potentially serious side effects including ARIA-E, a form of cerebral edema.

“None of my patients are willing to take the risk [with Aduhelm]: 40% chance of ARIA-E or ARIA-H,” said one neurologist, quoted in the report. “And when I tell people there was minimal, if any, benefit and it is only for early Alzheimer’s, they’re like ‘Yeah, no thanks, we’re not interested.’”

Biogen has now all but abandoned the Aduhelm ship, slashing its commercial team and exiting its CEO, who is expected to be replaced this year.

But the fallout from CMS' decision goes further than Aduhelm. There are some other major anti-amyloid therapies in late-stage trials that could be approved in the near-future. These include Eli Lilly’s donanemab, Roche’s gantenerumab and Biogen and Eisai’s Aduhelm backup lecanemab.

CMS' decision extends to all these drugs as well, though this could be reversed.

The future is not clear cut, and the Spherix report found neurologists had a mixed response when it came to making a final decision on whether to use them or not.

The report found that one-third of neurologists view the CMS’ final decision as having a “negative impact” on their willingness to prescribe any of the three emerging anti-amyloid meds should they be approved through the FDA’s accelerated pathway, which can green-light a drug based on biomarker evidence and ask questions on true clinical benefit later.  

But traditional approval “decreases this negative impact somewhat,” the report noted, indicating a slower but more rigid approach from the FDA and pharma may well sway neurologists back into the anti-amyloid fold.

For them, it’s about the data: Half the respondents believe that if all three emerging anti-amyloid drugs were to demonstrate a positive impact on cognitive and functional outcomes, the CMS would be likely to expand coverage.