In Na shocking reversal, Biogen (BIIB) said on Tuesday it would resurrect an Alzheimer's drug that the company previously said had failed, and would ask the Food and Drug Administration to approve it.
a "new analysis of larger data sets" showed that the drug, aducanumab, reduced clinical decline in patients with early Alzheimer's disease on multiple measures of drug efficacy. It directly contradicts a decision in March to stop studies of the therapy based on the recommendations of an independent monitoring board that was responsible for protecting patients in the study.
Aducanumab's error sent shock waves far beyond Biogen. It was believed to be the latest in a series of drugs ̵
Biogen's shares were up 43% in the pre-market trade.
Biogen said it conducted a new analysis in consultation with the FDA of a larger dataset from the discontinued studies. The new analysis includes additional data that became available after the previous analysis showed that the study was "meaningless" – that it had no chance of success. Biogen said the new data shows that aducanumab is "pharmacologically and clinically active" and that it reduced the patient's clinical decline based on the results of a study called the Clinical Dementia Rating-Sum of Boxes (CDR-SB), which was the main goal of the study.
“With such a devastating disease affecting tens of millions worldwide, today's announcement is truly encouraging in the fight against Alzheimer's. This is a result of groundbreaking research and is a testimony to Biogen's firm determination to follow science and make it right for patients, said Michel Vounatsos, Biogen's CEO, in a statement. "We are hopeful about the prospect of offering patients the first treatment to reduce the clinical decline of Alzheimer's disease and the potential implications of these results for similar approaches targeting amyloid beta."
& # 39; I have to pinch myself & # 39;
Al Sandrock, Biogen's head of research and development and medical director, said in his first interview about the new results that his team could only find a previous instance where a lawsuit was stopped for futility and then it turned out to be positive.
"I have to pinch because I almost don't believe it yet," Sandrock said. "It's so amazing to get this change from March. But I'm also very, very happy, because … I know people with mild cognitive impairment, and I felt like I'd let go of them all."
In June , when Biogen analyzed the entire dataset, researchers began to realize that Sandrock said a different picture of aducanumab was due to changes Biogen had made in the late-game study, initially the company was concerned about a potential side effect – swelling of the brain – and But later patients were allowed to receive higher doses of the drug.
"In retrospect, the results of the usability analysis were incorrect," Sandrock said. "That's because it was from a smaller data set that looked at patients with less exposure to high-dose aducanumab. "
Sandness, Sandrock said, had the futility analysis done prematurely. It had looked at data based on December last year. But the trials were actually stopped in March. rt two studies, one was positive for its right to high dose. One second still failed, but shows signs in favor of those patients receiving the higher dose of aducanumab.
The situation is very unusual, and Sandrock said it will be up to the FDA to decide whether to approve the drug based on a single positive test. But he said Biogen has met with the FDA twice to discuss the decision, first in June and then again on Monday. He said the FDA had provided preliminary written comments that led to Biogen submitting for approval, and that Biogen proceeded with an announcement without waiting for meeting minutes because there were no surprises during the meeting.
Sandrock has also been sharing the data with external experts. "People have a first skepticism," he said. “Quite enough. But as we begin to show them the data and take them through them, most of them are very happy now they see that there are prospects for the drug to be approved. "