Eli Lilly (LLY) reported positive results for its Alzheimer's drug, donanemab, and plans to file for approval with the FDA by the end of the quarter.
The drug slowed clinical decline by 35% in the patient population and 40% less decline in the ability to perform daily activities, according to a statement from the company Wednesday.
Wall Street seemed to be pleased. Eli Lilly stock closed up 6.68% to $431.19 on Wednesday.
There is, however, some concern among experts. Two patients died from the drug, and a third died during the clinical trials, the company said. The reason behind those deaths is unclear, company executives said. There are also side effects like brain swelling, which was observed in about a third of the 1,700 patients in the trial.
But, overall, the reaction to the announcement has been positive, largely because the company has spent more than two decades on the drug's development, and other, older Alzheimer drug candidates have failed.
Wall Street analysts believe the drug will compete well with rival Biogen's (BIIB) Leqembi, which is awaiting full FDA approval, potentially by July.
Some differences between the rivals: Lilly's drug is given by infusion, once monthly. By comparison, Biogen's Leqembi slowed cognitive decline by 29% and is infused once every two weeks.
Donanemab was developed to target plaque buildup in the brain, a theory that Biogen, with partner Eisai (ESAIY), pursued in their trials of Leqembi. But the theory is controversial within the scientific and medical communities.
The reason: Leqembi targets what are called beta amyloid plaques, which build up in the brain and have been seen in Alzheimer's patients. But not all scientists agree that those plaques are the right target to help treat the disease.
Lilly's drug targets the same plaque, as does Leqembi and its predecessor Aduhelm — both Biogen drugs that have been mired in controversy over the theory that the levels of plaque buildup correlate to cognitive decline for Alzheimer's patients. Some experts don't think it's the right target and that side effects, like brain swelling, are too common.
In addition, the Biogen drugs are facing skepticism after the company was also accused of being too cozy with the FDA throughout the development of Aduhelm, which also came under fire for its high price of $56,000 per year. Even after dropping the price, the drug has struggled.
Though Biogen/Eisai's drug is on the market, it is not fully approved, which has hampered the duo's revenue potential from Medicare, the country's largest payer, which will not cover it until it gets full approval. That would stand true for Lilly's new drug as well.
It's why Eli Lilly provided an unusually large amount of detail of the trial results in its statement Wednesday, said Dan Skovronsky, Lilly's chief scientific officer. He spoke at STAT's Breakthrough Summit.
"There's just so much skepticism in Alzheimer's. Maybe because of the history of failure, maybe because of lecanemab (Leqembi). We didn't want to be in another situation like that," Skovronsky said.
He added the details were to prove that the results were "not noisy and make sure we don't give the skeptics any extra ammunition."
Eli Lilly's drug would be the third in the new class of drugs targeting the plaques, and Skovronsky hopes Medicare will change its stance on restricting coverage, which he says is unfair and costly to elderly Americans. The Centers for Medicare and Medicaid Services (CMS) is requiring patients to pay out of pocket — or forces them to participate in clinical trials.
"That doesn't feel fair. That's not how we should treat elderly Americans. That's not the reason why we have Medicare, it's not to coerce people to do research. I'm all for clinical research, I want more patients to participate in research, but it needs to be free choice, not holding [them] hostage by withholding government benefits," Skovronsky said
Skovronsky said the next step is to "get this submitted as quickly as possible" for regulatory approval.
When asked about the three deaths in the clinical trials, Skovronsky said he believes it could be related to an overlapping disease that some Alzheimer's patients have, but more research is required.
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