Sanofi, Regeneron take the lead in blockbuster PhIII race of PCSK9 drugs

Dr. George Yancopoulos, Regeneron's chief scientific officer

Sanofi ($SNY) and Regeneron ($REGN) have rolled out the first batch of promising Phase III data on their cholesterol drug alirocumab, which handily breezed by Merck's ($MRK) Zetia in lowering bad LDL cholesterol. And the partners say they are on schedule to keep racking up points as they prepare a full portfolio of late-stage data in a high-stakes race to win the first regulatory approval for a PCSK9 inhibitor.

This first late-stage test looked at the effect of taking alirocumab alone and found that it reduced LDL by a mean score of 47% over 6 months compared to 16% for Zetia. Investigators recruited a group of more than 100 patients who could have benefited from statins but weren't able to take the popular treatments available. And most of the patients achieved a significant reduction in LDL on the low, 75 mg dose they started on, with doctors increasing the dose to 150 mg for those who didn't respond at first.

Generic statins will remain the cheap, popular treatment for managing cholesterol. But these companies are investing hundreds of millions of dollars in big Phase III programs with the expectation that they can offer something new and important for people who either can't tolerate statins or don't respond well to them. These are also injected therapies, meaning that they won't come cheap. A high price will potentially net these developers around $3 billion a year for an approved therapy, say some analysts using a common ballpark estimate of peak annual sales. But payers and regulators will also want to see some proof that the drugs will lower the rate of cardiovascular events--the kind of data that won't be quick or easy to gather.

There was a high rate of adverse events in the Phase III study, but nothing that pointed to alirocumab as a particular problem, with 78% in the Zetia group experiencing a problem and 69% in the alirocumab arm reporting trouble. The most common problem was infection, but Zetia proved close to alirocumab with a 39% rate compared to 42% for the experimental group.

In combination with statins, alirocumab was able to reduce LDL by about 70% in Phase II. Investigators, though, will want to present a variety of options to physicians and the massive population of patients who need to lower their LDL.

Sanofi and Regeneron are widely considered leaders in the race to get a PCSK9 drug over the regulatory finish line. To get there by 2015, though, the company will need to keep up a steady track record through a dozen late-stage studies requiring 23,000 patients. Amgen ($AMGN) and Pfizer ($PFE) are also hustling ahead with their own PCSK9 drugs, with other biopharma companies coming up from back in the pack with their own programs.

"There are still millions of people around the globe who have poorly controlled LDL-C," said George Yancopoulos, chief scientific officer at Regeneron. "Three years ago, our Phase I trials generated the first clinical evidence that blocking PCSK9 could markedly lower cholesterol levels in humans. Today, it is very gratifying to be able to report the first Phase III data for this promising potential new class of lipid-lowering agents. It is important to point out that these are just the first of a large amount of data yet to come from our extensive ODYSSEY Phase III program."

- here's the press release

Special Reports: 10 top drugs in biopharma's late-stage pipeline - Alirocumab | Top 15 highest paid biopharma R&D chiefs - George Yancopoulos

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