The pivotal study for SAR302503 nailed the primary endpoint for the proportion of patients with a greater than 35% reduction in the volume of their spleens, which become enlarged as a result of abnormal blood cell production.
The R&D numbers for the top 10 biotechs may only amount to a fraction of what you'll find in Big Pharma. But unlike the giants, which are trying to keep a lid on multibillion-dollar budgets, you'll find a much faster crowd when you turn your gaze to the biotechs. All 10 reported increases in their research spending for last year. And a few of them slammed their foot on the gas pedal.
Altogether the top 10 biotechs spent $11.8 billion on R&D in 2012, according to our research, a hefty 15% average increase over their 2011 performance. Compare that to the stable year-over-year record in Big Pharma, where doing more with the same amount of cash has become an industry mantra.
Two years after leaving the biotech giant Genzyme, Henri Termeer has found increased demand for his expertise and personal resources from a growing crowd of startups in the Boston area and The Netherlands. Termeer, the former chairman and CEO of Genzyme, left the biotech two months after Sanofi acquired it for more than $20 billion in April 2011 to seize control of its lucrative drugs for rare diseases. He was expected to stay involved in biotech, having engineered the growth of Genzyme from a small venture in the early 1980s to a multibillion-dollar force in the industry. Yet not even he could have predicted all the new companies and entrepreneurs he would add to his professional circle.
At the risk of looking a gift horse in the mouth, or more precisely two gift horses, I would like to reconsider the BRAIN initiative that President Obama announced as part of the budget package he sent to Congress on April 2.
I am all for initiative. I am no fan of big government, but government-sponsored large-scale basic research works. In fact it is essential if we are to provide healthcare to a country with an aging population, a shrinking workforce and a growing burden of chronic disease. The task is daunting at best and, without new insights and therapeutic tools, virtually impossible.
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Two more biotech outfits emerged this week as contenders to complete initial public offerings, highlighting the open season for the deals this year. Bluebird bio and PTC Therapeutics has mounted campaigns to go public with pipelines that target the molecular causes of rare genetic diseases.
Shares of XenoPort skidded lower this morning after the biotech announced its lead experimental drug had decisively failed a late-stage study for spasticity triggered by multiple sclerosis. The troubled biopharma company, which has been struggling to spur sales for the approved drug Horizant for restless leg syndrome, immediately opted to bury the program.
In a dealmaking frenzy, cash-rich Elan has agreed to pay up to $693 million for a biotech company focused on rare diseases, picked up a big share of a start-up specialty pharma company based in Dubai, spun out its sole remaining clinical asset into a new company with $90 million in financing and set up an $800 million debt offering to help keep the deals coming.
Effector Therapeutics has rallied marquee investors for a sizable $45 million Series A financing, determined to use discoveries in protein translation to mine for new cancer drugs.
The North Chicago, IL-based drug giant and Belgium's Galapagos have agreed to expand their collaboration on an oral JAK1 inhibitor to include Crohn's disease.
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Perhaps encouraged by the success of its North Carolina neighbor Quintiles, PRA is making moves toward an IPO of its own, filing a confidential draft registration form with the SEC.
An enzyme inhibitor that helps repair DNA damage may provide a new way to boost the effectiveness of radiation therapy for a lethal type of brain cancer.