Wall Street Journal
December 2, 2014
Biogen Notes Progress With Alzheimer’s Drug Shares Rise as Comments Renew Debate About Drugs That Target Plaque in Brains
Dr. Peter Davies, Director, Litwin-Zucker Research Center for the Study of Alzheimer’s Disease, Feinstein Institute for Medical Research
Biogen Idec Inc. reported positive interim results from an early-stage study of an Alzheimer’s disease drug, sending the company’s shares higher and renewing debate about drugs that attack amyloid plaques in the brain.
The study, which is still under way, is testing Biogen’s drug against a placebo in 200 patients with early forms of Alzheimer’s. Interim results showed the drug reduced beta amyloid levels in the brain, and had a “statistically significant effect on cognition” after 54 weeks of treatment, Biogen’s research and development chief, Doug Williams, told an investor conference in Boston, according to a company spokeswoman.
Dr. Williams’s discussion of the trial was brief, and Biogen doesn’t plan to present full results of the early-stage study until a medical conference next year. Still, Biogen’s stock rose 6.4% Tuesday on the Nasdaq exchange, adding about $4.7 billion to the company’s overall market value.
Beta amyloid protein forms sticky plaques in the brains of Alzheimer’s patients. Biogen said its drug, BIIB037, is designed to eliminate these plaques.
Many scientists believe amyloid plaques play a big role in causing Alzheimer’s—a theory known as the “amyloid hypothesis”—but a string of drugs designed to attack amyloid have failed to effectively treat the disease in clinical trials. Several high-profile failures in recent years have caused some researchers to question the value of pouring more money into amyloid-clearing therapies. Other researchers say the drugs must be tested in patients at an earlier stage of disease.
Many of the failed trials have tested amyloid-clearing drugs in patients with mild and moderate Alzheimer’s. Biogen’s study includes earlier-stage patients, with either mild or “prodromal” Alzheimer’s, which typically involves mild cognitive impairment.
Alzheimer’s researchers said they were intrigued by Biogen’s update but that they needed to see more data from the early-stage trial—and from a larger clinical trial—to judge the drug. Biogen said it is started planning for a larger late-stage clinical trial of BIIB037.
“I would have to see the ‘statistically significant’ data on cognition because I have heard this many times before in early development,” said Allen Roses, a neurology professor and Alzheimer’s expert at Duke University School of Medicine. The amyloid hypothesis “would be bolstered by clear data that needs to come from the properly conducted [late-stage] study using appropriate neuropsychologic tests,” he said.
Biogen’s Dr. Williams said the interim results from the early-stage study showed the drug reduced amyloid levels in the brain “in both a dose- and time-dependent fashion,” meaning that “the greater the dose, the more you remove” and “the longer you treat, the more you remove.” A dose-dependent result typically suggests that the drug, rather than some other factor, is responsible for the clinical outcome.
The study was primarily designed to assess BIIB037’s safety, which Dr. Williams called “acceptable.” Biogen said some patients in the study exhibited amyloid-related imaging abnormalities, or ARIA, which can suggest the presence of problems including vasogenic edema, a type of brain swelling. These abnormalities occurred mostly in people who carry a variant of the APOE gene that increases a person’s risk of developing Alzheimer’s. Biogen said the abnormalities were “largely mild to moderate and self-resolving.”
Peter Davies, director of the Litwin-Zucker Research Center for the Study of Alzheimer’s Disease at the Feinstein Institute for Medical Research in Manhasset, N.Y., said “it’s impossible without seeing the data” to draw larger conclusions about the drug. “To me, the most provocative thing [Biogen] said was about having a dose-dependent reduction in amyloid in the brain,” Dr. Davies said.