CHICAGO – For the first time, a large clinical trial of an Alzheimer's drug significantly slowed patients' cognitive decline and offered promising and unexpected hope from researchers and drug makers at a conference on Wednesday.
The drug from Cambridge, a Japanese company called Biogen and Eisai, showed a significantly better effect than a placebo in delaying the memory-destroying effects of Alzheimer's over a period of 1
The data presented at the International Conference of the Alzheimer's Association was a positive surprise for neurologists who have been observing for years how promising therapies fail in clinical trials.
The therapy slowed the mental decline by 30 percent in patients "We have received the highest dose in the study, and it has removed much of the sticky plaque that sticks to their brains," said the manufacturer of the drug on Wednesday.
If the results are confirmed, the drug may be the first to successfully attack both brain changes and the symptoms of Alzheimer's.
This study shows that you can both remove plaque and change perceptions, "said Dr. Reisa Sperling, director of the Center for Alzheimer's Disease and Treatment at Brigham and Women's Hospital in Boston Study. "I do not know that we have already made a homerun. It is important not to close the data too much. But as evidence of the concept, I feel very encouraging. "
But there are reservations: The Phase 2 study, which used multiple statistical measures, failed to meet its primary goal: four doses of the drug, called BAN2401, did not surpass a placebo, and the high dose was only on In addition, the metrics Biogen and Eisai are used to measure mental sharpness, a self-grown compound that has never been used to gain Food and Drug Administration approval.
"I will remain cautiously optimistic" said Dr. Ronald Petersen, director of Mayo Clinic Alzheimer's Disease Research Center. "I think the data is intriguing. The effect sizes sound reasonable, the drug seems safe, and on the biological side, the drug seems to work.
But, he added, "You would really want to see a Phase 3 to replicate these results."
Whether the drug will go into a Phase 3 trial remains an open question.
Despite ongoing Wall Street issues, Biogen and Eisai have not said whether they will take up a larger study to bring benefits to BAN2401 directly to the FDA with Phase 2 results. Lynn Kramer, chief medical officer of Eisai's Neurology Department, said that the company considers the Phase 2 trial as a "late stage" and is discussing the possibility of using it to get approval this year
It is unclear whether the FDA will lower the traditionally high limit for Alzheimer's therapies, for which two Phase 3 trials have been required in the past.
When BAN2401 expires, it could stop a generation of patients bitter disappointment in Alzheimer's who has not seen any new approved treatment for more than 15 years. Again and again, therapies have shown promise in early tests but are empty and frustrating patients, doctors and scientists. Meanwhile, the disease occurs more frequently, affects more than 5.7 million Americans and costs the country more than $ 250 billion a year, according to the Alzheimer's Association.
Biogens share price rose nearly 30 percent last month as analysts predicted modestly positive data from the Phase 2 study. Actual results surpassed Wall Street consensus and put Biogen on a path to further growth.
The Biogen share has moved after the publication of the study results in aftermarket trading. After several changes between profits and losses, he fell by 6.5 percent.
BAN2401 is part of a cadre of Alzheimer's treatments based on the idea that toxic plaques, called amyloids, are responsible for the corrosive effects of the disease on the brain. The so-called amyloid hypothesis is supported by numerous genetic data, but no drug targeting these plaques has ever made a major attempt to significantly affect the disease.
That surprised BAN2401. In the Phase 2 study, the highest dose of the drug had a pronounced effect on amyloid deposition in the brain, with 81 percent of patients switching from amyloid-positive to amyloid-negative. And in addition to its effect on Eisai's own level of cognition, the dose registered a 47 percent reduction in cognitive decline compared to placebo on a well-respected metric called ADAS-cog.
"That's the thing that really makes me feel like you're on something, that's a real effect we're seeing," Dr. Jim Hendrix, director of global scientific initiatives at the Alzheimer's Association.
The next step, he said, should be to enroll another, larger trial that isolates the best BAN2401 dose and tests whether it can replicate its promise.
Material used by the New York Times and the Associated Press was used in this report.