Biogen Inc. ( BIIB Quick Quote BIIB - Free Report) announced that an FDA advisory committee voted against aducanumab, its controversial investigational treatment for Alzheimer’s disease.
The biologics license application (BLA) filing for aducanumab included data from the ENGAGE and EMERGE phase III studiesas well as the phase Ib PRIME study in patients with early-stage Alzheimer’s disease.
The Peripheral and Central Nervous System (PCNS) Drugs Advisory Committee voted 8 to 1 with 2 uncertain that data from the EMERGE study on aducanumab (which had met the primary endpoint) does not provide strong evidence to support the efficacy of aducanumab for treating Alzheimer’s when viewed independently, regardless of data from the ENGAGE study (did not meet primary endpoint). The experts voted 10 to 0 with 1 uncertain that EMERGE could be used as primary evidence of efficacy in light of the negative ENGAGE study. The committee also voted against the PRIME study providing supportive evidence of aducanumab’s effectiveness.
In March 2019, Biogen and its partner Eisai had announced the discontinuation of ENGAGE and EMERGE studies as a futility analysis showed that these were unlikely to meet their primary endpoints. In October 2019, surprisingly, Biogen revealed plans to pursue U.S. regulatory approval of aducanumab based on positive results of a new analysis of larger dataset, which became available after the discontinuation of the studies.
The new analysis of the larger dataset showed a different outcome than the one predicted at the time of the futility analysis. The EMERGE study met the primary endpoint, showing that patients treated with a high dose (10mg/kg) of aducanumab experienced a statistically significant reduction in clinical decline of Alzheimer’s disease. The ENGAGE study, however, did not meet the primary endpoint.
Nevertheless, Biogen said that data from a subset of patients in the ENGAGE study who received a higher dose of aducanumab supported the findings from the EMERGE study. In August, the FDA had granted priority review to Biogen’s BLA seeking approval of aducanumab. A decision from the FDA is expected on Mar 7, 2021. On Nov 4, FDA published briefing documents for the advisory committee meeting, which appeared positive and pointed toward a favorable outcome of the meeting, which pushed shares up 40% on Nov 4. The FDA committee’s vote now contradicts the briefing documents.
Trading on Biogen’s stock was halted on Friday, ahead of the advisory committee meeting. Biogen’s shares have risen 10.8% against the
industry’s decrease of 2.3% in the same time frame.
Alzheimer’s, a fatal condition that causes progressive decline in memory, has always been a highly challenging area, and not much progress has been made despite significant investments (both funds and resources). The drugs presently available just treat the symptoms of the disease. Several companies have failed to develop safe and effective treatment options to treat this deadly brain disease. Other than Biogen, several large pharma companies, including
Roche ( RHHBY Quick Quote RHHBY - Free Report) , Amgen ( AMGN Quick Quote AMGN - Free Report) , Novartis ( NVS Quick Quote NVS - Free Report) , Merck, Pfizer, Lilly and AstraZeneca stopped development of their AD candidates in the last few years either due to low possibility of success or safety concerns.
Despite the setbacks, the companies continue to invest heavily in developing AD treatments, given the high commercial potential in this market.
If the FDA approves aducanumab,it will become the first medicine to be approved to reduce the clinical decline associated with this devastating disease. It will also be the first medicine to show that removing amyloid beta (plaque in the brain) results in better clinical outcomes in Alzheimer’s patients. It will bring in huge revenues for Biogen by meaningfully changing the course of Alzheimer’s treatment.
All eyes will now be on the FDA’s decision on Mar 7. The FDA, in its decision, is not bound by the advisory’s committee’s opinion but will definitely take into account the panel’s recommendation, especially while considering such a negative opinion.
Biogen currently carries a Zacks Rank #4 (Sell).
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