Biopharma is a complex, rapidly evolving industry that is highly regulated and closely watched — and that means there is constant news. Here's a closer look at the clinical trials, M&A, cool science and regulations that are driving the industry this week.
In case you missed it
- What Gilead taught pharma about pricing a cure
- Axovant Chairman has no regrets about Alzheimer's failure
- Virtual reality meets marketing
Mergers & analysis
The last few years in dealmaking have been dominated by research collaborations where big pharmas pair up with small biotechs to test their blockbuster immunotherapies with cancer drugs of various mechanisms. These deals typically are not exclusive and have undisclosed deal terms, if money exchanges hands at all.
But Bristol-Myers Squibb Company changed all that this week when on Valentine's Day it said it is cozying up to Nektar Therapeutics with a jaw-dropping $1.85 billion upfront payment to test its checkpoint inhibitors Opdivo (nivolumab) and Yervoy (ipilimumab) with one of Nektar's early-stage compounds. The deal puts an abrupt stop to the free-love combo research deals of the last few years and ups the stakes for other companies looking to find the next great cancer combination therapy.
Roche AG also made an industry-altering move this week, picking up big data company Flatiron Health for $1.9 billion. The deal promises to accelerate the Swiss pharma's use of real-world evidence, a concept that the Food and Drug Administration has expressed it wants to see more of. Real-world evidence could change the way pharma companies get approval for medicines and promises to speed up the time a drug spends in development— potentially allowing companies to take advantage of more patent years and lower drug development costs.
Elsewhere, the vendor side of the equation continues to be reshaped —several deals over the last year or so across the contract research and manufacturing front have changed the landscape. That continued with Charles River Laboratories International Inc. picking up MPI Research Inc. for $800 million.
Clinically relevant
Alzheimer's disease research took several major blows this week, scaring investors and discouraging even the most hopeful. The week began with Axovant's high-profile CEO David Hung — known best for chasing his dreams of finding a cure for the horrible neurodegenerative condition — rapidly, and unexpectedly, exiting the well-financed startup.
Axovant has since begun laying off employees and Chairman Vivek Ramaswamy told an audience at the BIO CEO & Investor conference in New York that the new direction of the company is still undecided. (Although the company confirmed to BioPharma Dive that all affected employees were offered positions elsewhere in the Roivant family of companies and many of them have taken the offers.)
Adding to the carnage, Merck & Co. announced that it is putting the kabosh on another late-stage Alzheimer's trial for its drug verubecestat. The drug failed in mild-to-moderate patients last year, but the company remained hopeful that it could still show promise in even earlier stage patients. Those hopes are now dashed and verubecestat gets added to the pile of failed drugs in the space.
Biogen spooked the market the next day when its CMO off-handedly mentioned at an investor conference that the big biotech has made changes to its late-stage studies for its closely watched Alzheimer's hopeful. Aducanumab is currently the most promising candidate in the pipeline and the vague wording about why Biogen is changing the trial design is lowering expectations that the drug could finally bring a win to the space.
Outside of the neuro field, Pfizer Inc. and Merck KGaA revealed that their checkpoint inhibitor Bavencio (avelumab) failed in the all important non-small cell lung cancer setting. The companies tried to blame the failure on high rates of survival in the docetaxel arm due to patients crossing over to treatment with other PD-1/L1 inhibitors. Yet, that just emphasizes that the treatment effect of Bavencio was too small to overcome the other regimen.
Highly regulated
With all the hits to the Alzheimer's development landscape, the FDA is doing what it can to help boost the chances of getting a disease-modifying drug to market. The agency issued several new guidance documents this week targeted toward improving the development and approval of neurological disease drugs. One said document is specifically focused on the problem of Alzheimer's disease.
"This document describes innovative approaches to studying very early disease before the onset of dementia, including strategies for trials incorporating patients with Alzheimer’s who haven’t experienced any visible impairment (in the form of cognitive or functional deficits), but who may be identified through the use of sensitive cognitive screening, imaging tests, or biomarkers," said Commissioner Scott Gottlieb in a statement.
The use of biomarkers for Alzheimer's would be a major shift. It would allow companies to treat patients before there are signs of disability, allowing them to treat mild cognitive decline before it becomes debilitating.