Eli Lilly and Co. on Wednesday disclosed the failure of an experimental drug aimed at slowing Alzheimer's, clouding the outlook for research programs but also thrusting Cambridge's Biogen Inc. to the front in the race to develop the first treatment for the devastating disease.
Lilly said a late-stage clinical trial of its drug, called solanezumab, didn't significantly slow cognitive decline in patients with mild dementia due to Alzheimer's. It was the latest in a long trail of crushing setbacks to finding a treatment for the neurodegenerative disorder, which gradually robs people of their memory and cognition and afflicts about 47 million people worldwide.
"We didn't get the results we wanted or expected," said Lilly distinguished medical fellow Eric Siemers, who leads the company's Alzheimer's drug program. He said the company was continuing to test the compound in patients with earlier stages of the disease.
"We're saddened and disappointed," said Maria Carrillo, chief science officer at the Alzheimer's Association in Chicago, which funds research programs and lobbies for more federal money to finance studies. "On behalf of over 5 million Americans with this disease and 15 million caregivers, we need an effective treatment."
The news pushed down the stocks of Lilly, Biogen, and other drug makers that are working on Alzheimer's therapies. Biogen shares dropped 3.8 percent as investors attempted to gauge how the Lilly setback might affect other Alzheimer's drug development programs. Lilly's shares tumbled 10.5 percent.
Like the Lilly compound, Biogen's experimental drug is based on the so-called amyloid hypothesis — researchers believe the buildup of amyloid plaque in the brain plays a pivotal role in the development of Alzheimer's symptoms. But Geoffrey Porges, biotech analyst at Boston health care investment bank Leerink Partners, stressed in a note to investors that Biogen's drug is "a materially different antibody" that works in a different way and is being tested on a different set of patients than Lilly's drug.
Biogen, the largest Massachusetts biotech company based on market value, is currently the world's leader in multiple sclerosis medicines. But it considers Alzheimer's therapies key to its growth. The company reported last year that, in an early-stage trial, its lead Alzheimer's drug, called aducanumab, slowed the mental decline of a small number of patients who had mild cases of the disease. Biogen is scheduled to report further data from that study next month. It's also evaluating the medicine in two later-stage studies.
"We're going to continue our studies as planned," said Samantha Budd Haeberlein, Biogen's vice president of clinical development. "Based on the data we've seen to date, we believe that aducanumab has potential for the treatment of Alzheimer's disease."
In all, Biogen is conducting clinical trials on three Alzheimer's compounds and is about to move two others into trials. In two of the drug programs, the company is collaborating with Eisai Inc., the pharmaceutical arm of Tokyo's Eisai Company Ltd.
Lilly's Siemers said his Indianapolis-based company has been researching Alzheimer's treatments for three decades and has no intention of giving up. It has a half-dozen experimental drugs in clinical trials — including a late-stage one in partnership with British drug maker AstraZeneca PLC. "We have a very robust pipeline of molecules that attack the disease in a number of different ways," he said.
Drug giants Merck & Co., Takeda Pharmaceutical Co., and the Genentech division of Roche AG also are developing Alzheimer's treatments. In all, there are 12 to 15 experimental therapies in late-stage trials, according to the Alzheimer's Association.
"This reinforces our need to redouble our efforts," the association's Carrillo said of the Lilly results. "We need to explore not only the amyloid hypothesis, but other therapeutic approaches and targets. The jury's out about amyloid, certainly."
The aging of baby boomers, many of whom are caring for parents suffering from Alzheimer's, has created a sense of urgency in the medical community.
"It is important that we continue to explore new ways to detect, diagnose, and intervene at all stages of this tragic disease — from before the onset of memory symptoms to patients in the throes of dementia," said Dr. Mark W. Albers, a neurologist at Massachusetts General Hospital in Boston.
Salim Syed, biotech analyst at Mizuho Securities USA in New York, said Lilly's Alzheimer's setback might have unfairly reflected on a Biogen drug that continues to show promise. In fact, he said it might be a good time to invest in the company, given the lower stock price. Biogen has "the only Alzheimer's drug that has prospectively hit" its goal in a clinical study, Syed wrote in a note to investors.
Doctors who work with Alzheimer's patients were troubled by the news of the unsuccessful Lily trial but continued to press for new treatments.