Biogen ($BIIB) posted some early-stage data from its Alzheimer’s candidate aducanumab but, despite being headline news around the world, the market shrugged its shoulders.
The data, published in Nature, showed the amyloid-targeting mAb could help reduce amyloid plaque, something believed to clump together in the brains of Alzheimer’s patients.
By removing this plaque, many biotech and pharma researchers believe they can slow down the progression of the disease--although the amyloid thesis has not produced a new drug for the memory-stealing condition.
In the paper, published this week, Biogen said both a preclinical animal model and a Phase Ib, placebo-controlled study in 165 prodromal and mild AD patients both showed that aducanumab reduced amyloid-beta in the brain, and that the reduction was dose-dependent.
Biogen also published some “exploratory results” from the early study which it said showed dose- and time-dependent slowing of clinical decline as measured by the Mini Mental State Examination (MMSE) and the Clinical Dementia Rating scale Sum of Boxes (CDR-SB).
The trial was not however designed to assess whether aducanumab could slow down a patient’s rate of cognitive decline.
Brain scan images showed patients who were given the highest dose of the medicine--10 mg for every kilogram of body weight--were essentially free of amyloid plaques after 12 months of treatment. Although with the higher dose came higher AEs, notably amyloid-related imaging abnormalities (Aria), which can cause serious swelling in the brain.
The terms “game-changer” and even this candidate being “preventative” have been used by U.S. and U.K. newspapers--and even caused an extended gushing piece on the normally level-headed BBC news channel; but investors have been here before, seeing so much hype from an early-stage Alzheimer’s asset that, along with 99% of all candidates over the last decade, have gone on to fail.
The disease affects an estimated 5.4 million Americans and will eventually prove fatal--with its prevalence only expected to rise as the population ages.
Big Pharmas, including Eli Lilly ($LLY), AstraZeneca ($AZN), Johnson & Johnson ($JNJ) and Pfizer ($PFE), have all thrown billions of dollars at this research area, but in return have been beset by failures and setbacks--with some now looking to restrict their spending on an area that yields so little ROI.
Biogen, in fact, ended the day slightly down on the news by 0.7%, but was up marginally in after-hours trading by 0.45%.
And it wouldn’t be the first time the Big Biotech had built up hopes on this very drug, only to see weak results. The drug is also in late-stage testing, but back in 2015, after posting some eyebrow raising data in March, a few months later in July the 6 mg dose of failed to deliver, falling short on MMSE and CDR-SB. Safety issues also raised their heads in this study.
The company is some years off getting the drug on the market, and in its later stage tests it will need to find the sweet spot on dosing to get the level of efficacy balanced against the risks of Aria right if it is to find a path to market.
Biogen has always been seen as the jewel in the biotech crown, but in 2016 this has started to slip, with its CEO George Scangos set to leave and a successor search on the way; troubles in its MS franchise; cutting back on some lupus, immunology and fibrosis research as well as 11% of its staff; and lingering questions over whether its Alzheimer’s program can go the distance when so many others have hit the skids.