Biogen is submitting a key Alzheimer's drug it once abandoned for approval 'as soon as possible.' Here's what we just learned and still need to know.
- Biogen is planning to file its leading experimental Alzheimer's drug for US approval "as soon as possible," the biotech's CEO said Thursday.
- Whether or not the Food and Drug Administration approves the drug, called aducanumab, it's set to be "the mother of binary approvals," one Wall Street analyst wrote in a research note. That is, the decision one way or the other will be a defining one for both Biogen and the agency.
- Biogen's drug, if approved, would be the first treatment to reduce the clinical decline of Alzheimer's disease.
- Executives for the $50 billion biotech answered - and dodged - Wall Street's questions on its fourth quarter earnings call about about the company's plans in Alzheimer's.
- The drug's controversial clinical results have split Wall Street. Some analysts anticipate approval, while others see virtually no chance the drug is cleared. It remains unknown how the FDA plans to treat the application, including if it will grant a speedy review or summon a panel of experts.
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Biogen executives were tight-lipped Thursday about specifics in 2020 for its leading Alzheimer's disease drug. But they did provide a few details about the company's next steps.
The Cambridge, Massachusetts-based biotech shocked the Alzheimer's research community and drug industry in the last months of 2019. After prematurely ending a late-stage clinical trial of aducanumab in March - acknowledging the study had failed - the firm's executives revived the prospects of the drug months later. They claimed an additional review of the data showed the drug worked.
Biogen's stock dove initially - erasing $18 billion in market value - after the discontinatuon in March, but then shot up by more than one-third in October.
Now, aducanumab is set to become one of the most important approval decisions facing the Food and Drug Administration in years. If approved, it would be the first disease-modifying therapy in Alzheimer's, the widespread neurodegenerative disease that drugmakers have spent billions on trying to treat.
"We are all curious about the timing of filing, which will set up the mother of binary approvals in the biggest therapeutic space in biotech in a long time," Cantor Fitzgerald analyst Alethia Young wrote in a Thursday note to investors.
Here's what we learned Thursday on Biogen's fourth quarter earnings call about the state of aducanumab's application, and the biotech's broader Alzheimer's ambitions.
What we learned
Aducanumab hasn't been submitted yet to the FDA, with Al Sandrock - Biogen's research head - calling it "a matter of putting together documentation."
CEO Michel Vounatsos said the company plans to file with US, European, and Japanese regulators, and is prioritizing the US application first.
"We look forward to completing a regulatory filing in the US as soon as possible," Vounatsos said Thursday.
Biogen did disclose an aducanumab filing, just not the one everyone had their eyes on. The company submitted plans to the FDA to run a new clinical study of aducanumab in patients with Alzheimer's.
Sandrock said Biogen is running the study mainly for "humanitarian reasons." It will enroll patients who were previously in an aducanumab trial and re-dose them with the therapy.
"We felt we owed something to them [the patients]," he said. "Many of them expressed a desire to the investigators that they wanted the drug."
For those looking for clarity on the drug's effectiveness, this study likely won't help. It's a single-arm trial, meaning there is no placebo group to compare results against. Sandrock said the study will mainly look at safety and tolerability.
There was also one hint at Biogen's coming efforts: the biotech expects to spend significantly more in 2020 on expenses related to selling its products and managing the company - more than what Wall Street analysts were expecting.
According to its forecast, Biogen could spend as much as $2.93 billion in this area, an increase of roughly 25% from last year. That boost likely comes from "laying the groundwork" to sell aducanumab, wrote RBC Capital Markets analyst Brian Abrahams.
What we still need to know
Specifics on the application and what the FDA would request for its review remain unclear.
Bernstein analyst Ronny Gal asked if the company expects to receive a normal or priority review. The latter shortens the decision timeline from 10 months to 6 months. Biogen executives didn't answer.
Evercore ISI analyst Umer Raffat inquired about what the clinical results look like if all the aducanumb studies are pooled together. Sandrock replied by saying the company hasn't disclosed what that data looks like.
Raffat also asked how many post-hoc analyses were done before Biogen settled on the one it presented in December to argue for the drug's benefit. Sandrock said he didn't remember.
Many of the analysts' questions were designed to get specifics on the core debate on aducanumab: Will a post-hoc analysis of a study that was stopped early be enough to gain FDA approval? Biogen provided few specifics Thursday to further inform opinions on that question.
Biogen's vision in Alzheimer's goes beyond aducanumab
In the eyes of Biogen executives, aducanumab is just the beginning of a multi-drug franchise in Alzheimer's.
"One of the reasons why we built the pipeline the way we did is we always envisioned combination therapies," Sandrock said.
He said combining aducanumab with another Alzheimer's drug that targets tau, a protein found in nerve cells, is an obvious next step. Biogen has two experimental drugs in early-stage testing that target tau.
The company also has another Alzheimer's drug targeting amyloid beta, the same protein aducanumab targets, in late-stage testing called BAN2401.
Biogen, along with its research partner Eisai, plan to additionally test that drug against placebo in the earliest stages of Alzheimer's disease, Sandrock said. That would include patients who have yet to show signs of cognitive decline.
That long-term R&D vision, however, hinges on aducanumab's approval in the near term. The next significant update from Biogen is expected to be officially submitting the application, which could provide the first timeline for an approval decision.
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