- Biogen and Eisai have just released full phase 3 data for a new drug for Alzheimer’s disease, lecanemab.
- The drug slowed the rate of cognitive decline by 27%, with few side effects.
- The study is based on a controversial hypothesis around the disease.
Alzheimer’s disease affects approximately 6.5 million Americans – and there is no cure.
It’s a complicated and debilitating disease and drug companies have struggled for years to find viable treatments.
A recent study of a new treatment for Alzheimer’s disease could help companies and doctors determine whether a key assumption underlying years of treatment is worth investing in. develop, called lecanemab. The study confirmed previous reports that the drug slowed the rate of cognitive decline in patients with Alzheimer’s disease by 27%. It was also accompanied by side effects, including infusion site reactions and brain swelling in about 13% of patients. Analysts say the positive result could make it a blockbuster drug for companies.
The long-awaited findings come from Biogen, a Cambridge-based biotech company focused on neurological diseases, and Eisai, a Japanese pharmaceutical company focused on developing drugs for cancer and brain diseases. Biogen previously developed an Alzheimer’s drug called Aduhelm, which was approved by regulators in 2021 but failed commercially after providers and insurers pushed back the prescription of the drug due to mixed clinical trial results. .
Now, lecanemab could pave the way for the validity of a hypothesis that has been a source of controversy in Alzheimer’s disease research for years.
Lecanemab’s slight success comes after series of Alzheimer’s disease treatment failures
At the center of the science behind the drug is the amyloid hypothesis: a decades-old theory that the buildup of beta-amyloid, or proteins also known as Aβ clusters, in the brain may be responsible for the disease of Alzheimers. These clumps build up in the brain and are thought to eventually kill brain cells, leading to disease progression.
A handful of biotech companies focused on treating Alzheimer’s disease have used this theory to develop drugs that block the effects of these proteins on the brain. But amid growing skepticism around the amyloid hypothesis, coupled with a string of failed drugs from companies like Eli Lilly and AstraZeneca, Pfizer and Roche, Biogen and Eisai’s Phase 3 study results have paved the way. in the future of the domain.
Lecanemab has been shown to successfully remove beta-amyloid from the brain, but Biogen’s study had to “either confirm the hypothesis…or make it even more questionable if the data is poor,” the Cowen analyst said. , Phil Nadeau, to Insider.
The late-stage study included 1,795 participants who received lecanemab or a placebo and were surveyed over an 18-month period to see if the drug could slow cognitive decline associated with Alzheimer’s disease. The drug is intended to treat people in the early stages of the disease.
Nadeau predicts a modest decline in Biogen’s revenue over the next few years due to competition in other pharmaceutical areas, so a resounding success for the company, such as the FDA approval of lecanemab, will be important. to drive revenue growth. Insider reached out to Biogen for comment, which referred lecanemab-related questions to Eisai.
But since the study showed promising results, lecanemab could take over about 11% of the Alzheimer’s disease market three years after its launch, Nadeau wrote in a recent analyst note, which would translate to more than $3 billion in annual revenue from the drug.
Previously, some thought the likelihood of a successful study was low. Doctors surveyed at the annual Cowen Health Conference in March estimated a 36% chance that lecanemab would be successful.
In a July note to investors, Needham analysts predicted that lecanemb had a 43% chance of being an effective treatment for Alzheimer’s disease.
If the test had been negative, it would not have been the end of the amyloid hypothesis
Negative results, which would have shown the drug failed to reduce the severity and progression of Alzheimer’s disease over an 18-month period, would not necessarily have meant the end of companies working on similar treatments. , said Nadeau.
Other big pharma companies such as Roche Holdings and Eli Lilly are pursuing similar treatments, with results expected later this year and early next year, respectively.
“I think people’s confidence in the amyloid hypothesis would drop even more, but ultimately people would wait to see what these other data events would show,” he said.
Negative results could have given a boost to companies exploring other ways to treat Alzheimer’s disease. As the amyloid hypothesis comes under increasing scrutiny, a handful of biotech companies have started looking to different strategies, like gene editing, to find better treatments for the disease. . And although Biogen’s drug is the most advanced in its development, it is far from the only treatment tested for Alzheimer’s disease. According to a recent report by Fierce Biotech, there are 143 disease-targeting drugs in 172 clinical trials.
The positive result can mean a successful financial success and a redemption arc for the hypothesis
The study had a positive result, which means it could be a resounding success for Biogen.
But a major hurdle lecanemab will face is the Centers for Medicare and Medicaid Services (CMS), which oversees more than 100 million people on Medicare and Medicaid. Biogen’s Aduhelm has come under intense scrutiny from CMS, which has the right to review drugs that have received expedited approval from regulators, a status that Aduhelm and lecanemb have received from the Food and Drug Administration. Today, CMS only covers Aduhelm for patients in clinical trials, meaning those who want the drug in real life will have to pay for it entirely out of pocket – to the tune of $28,200 a year.
So there’s still a chance the drug could be approved by the FDA but still not receive adequate reimbursement from Medicare, Nadeau said.
Eisai CEO Ivan Cheung told Insider that his trial with Biogen on lecanemab is designed to give patients and the wider medical community a “definitive” answer as to whether the drug really works.
“There shouldn’t be a lot of interpretations” about whether the drug should be approved for wide use to treat patients, he said.