|Source: Regeneron Pharmaceuticals|
Regeneron: Shots on goal
2011: $529 million
2010: $489 million
As a percentage of revenue: 119%
Research chief: Neil Stahl
Last fall Regeneron Pharmaceuticals ($REGN) scored a big win when it put out the word that the FDA had approved Eylea (aflibercept) for wet, age-related macular degeneration. But instead of focusing on the slight discount Regeneron was planning to use to distinguish it from Roche's Lucentis competition, analysts were wondering how it would do against off-label doses of Avastin.
But Regeneron has always had a hard time when it comes to earning the industry's respect.
As The New York Times' scribe Andrew Pollack noted at the time, Regeneron racked up $1.2 billion in losses as it spent its way through 24 years of R&D, often plagued by setbacks in the clinic. But the regulatory approval registered as a solid achievement for CEO Leonard Schleifer, who holds the title for longest-running chief in the biotech industry.
Over the years, Regeneron has been exposed to quite a bit of skepticism. Just a few days ago its investigators notably failed to persuade an FDA panel to endorse its use of Arcalyst in a lucrative gout market, likely eliminating entre into a disease arena that might have been worth $500 million a year.
And so it goes, one step forward and one step back.
But Regeneron still has a few experimental drugs up its R&D sleeves. Sanofi ($SNY) and Regeneron have been making a case for Zaltrap for treating colorectal cancer, gaining promising survival data in a pivotal late-stage trial of the drug last year. And they have been rewarded with a speedy review process from interested U.S. regulators.
Then there's SAR236553/REGN727--a drug partnered with Sanofi and a potential blockbuster for LDL, if investigators can make a case that it can safely reduce bad cholesterol as they sprint ahead of a pack avid to grab the lead in the field.
Its Phase IIb "MOBILITY" trial showed that the drug sarilumab, in combination with the standard RA treatment methotrexate, made a "significant and clinically meaningful improvement" in RA patients' symptoms compared with those treated with methotrexate alone. And RA symptom improvements were the greatest in patients treated with the highest dose of the sarilumab. Yet the drug--partnered with Sanofi--fell short in a Phase IIb study for a separate inflammatory disease, ankylosing spondylitis.
Regeneron noted that its higher R&D costs in 2011 could be attributed to a bigger head count in the research division and beefed-up research and preclinical work, with a significant focus on its Sanofi antibody pact. Regeneron may still be in the hunt for that one big drug win. But it has a few shots on goal.