By Jaimy Lee
An Alzheimer’s disease expert said Biogen “absolutely” needs to conduct a third clinical trial for aducanumab, its now controversial and recently resurrected Alzheimer’s disease drug candidate.
The Cambridge, Mass.-based biopharmaceutical company /zigman2/quotes/201531540/composite BIIB -6.11% on Thursday released additional data from a pair of late-stage trials evaluating aducanumab as a treatment for Alzheimer’s patients who have mild cognitive impairment and dementia.
One of those trials—the Emerge study—indicated that a larger group of trial participants who took a high dose of the therapy showed a reduction in clinical decline. The second trial, Engage, failed.
“There were clinical and biomarker signals that showed promise, but the bottom line is we need more data to evaluate the effectiveness,” Dr. Howard Fillit, founding director of the Alzheimer’s Drug Discovery Foundation, said in an email on Friday. “It is clear that Biogen has learned a lot from these two trials and should be able to design a third trial that takes into account the issues that have been raised, particularly the need for patients to be on the highest dose for the longest period of time.”
Biogen and the Japanese drugmaker Eisai /zigman2/quotes/203064480/delayed JP:4523 -3.88% had yanked the trials in March after a futility analysis revealed that the studies’ primary endpoints would not be met, based on an examination of about half the data. Then, in an announcement in October that surprised investors, the company said it had returned to the larger set of data, discussed it with the Food and Drug Administration and would seek regulatory approval.
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Analysts are now questioning whether the science that was presented Thursday is compelling enough for a regulatory application and approval.
“We assign 0% probability to aducanumab being approved,” wrote Raymond James’ Steven Seedhouse wrote Dec. 6. “When you have a conclusion in hand (i.e., ‘some difference in Engage must explain why it was negative’), if you look long enough you will likely find an answer that satisfies you and your chosen statistics.” Wedbush analyst Laura Chico called the data “clear as mud.”
Some, however, are taking a bullish approach. SVP Leerink’s Geoffrey Porges said he anticipates an approval, while Jason McCarthy at Maxim Group thinks there is potential for Biogen to receive a narrow label for aducanumab, perhaps with a required follow-up study.
However, McCarthy also sees the excitement and interest around Biogen’s experimental therapy as a tool to reinvigorate the Alzheimer’s drug development field. Shares of Cassava Sciences /zigman2/quotes/204212356/composite SAVA +5.03% , which also released mid-stage data at the Clinical Trials on Alzheimer’s Disease meeting this week in San Diego, are up 24% on positive data from that study.
“The [Alzheimer’s disease] space has re-emerged back to center stage and that’s a positive for all the players,” he wrote.
Shares of Biogen tumbled 3% during the data presentation on Thursday, before recovering and gaining 5% in afternoon trading. Biogen stock is up 0.63% year-to-date. The S&P 500 /zigman2/quotes/210599714/realtime SPX +0.58% has gained 25%.