Sanofi and Roche race to PhIII with drugs aimed at hot PCSK9 gene

Get ready to see one of hottest areas of cholesterol drug research explode with new studies. Sanofi ($SNY) and partner Regeneron ($REGN) have begun enrollment for late-stage trials expected to include 22,000 to test their drug REGN727 that targets the PCSK9 gene to lower cholesterol. And Roche ($RHHBY) is fast on their tail with its own Phase III program for a PCSK9 treatment due to kick off next year, Bloomberg reported.

The companies and others are vying for their piece what could become the next blockbuster market for cardiovascular drugs, with PCSK9-blocking therapies offering a whole different path than statins such as Pfizer's ($PFE) Lipitor to step down levels of bad cholesterol. Yet with many existing anti-cholesterol options, including cheap generics, developers are building comprehensive clinical trial programs for PCSK9 drugs.

Sanofi and Regeneron's late-stage ODYSSEY program, for example, will include 10 clinical trials, including an 18,000-patient study that will look at the impact of the treatment on cardiovascular outcomes. As Reuters reports, the pair aim to hit the market with their PCSK9 contender in 2015, potentially making their treatment the first of its kind to be approved. For Sanofi, which faces generic competition to its top-selling blood thinner Plavix and blood pressure med, Avapro, the PCSK9 drug provides a key addition to its cardiovascular business.

Roche comes to the game in fairly different position. The Swiss drug giant, which currently lacks a big cardio business, seeks its first multibillion-dollar seller in this category. In May the drugmaker wrote off its experimental dalcetrapib after the drug for increasing good cholesterol bombed in a late-stage study. As Bloomberg reports, Roche has kept details about its PCSK9 drug from U.S. unit Genentech under wraps because of the intense competition in the field. Yet that will rapidly change as Roche unveils data on the program and builds its case with docs to enroll their patients in its studies.

Behind Roche's and Sanofi's PCSK9 programs are several more. Amgen ($AMGN) and Pfizer are in mid-stage studies with their own candidates aimed at the hot target. Also, Bristol-Myers Squibb ($BMY), Merck ($MRK) and Alnylam ($ALNY) have research underway in the field.

"These drugs allow us to go where no one has gone before," Steven Nissen, chairman of the Department of Cardiovascular Medicine at the Cleveland Clinic in Ohio, said in an interview with Bloomberg. "There's a tremendous amount of interest among a broad range of makers of pharmaceuticals to get involved."

- here's Sanofi and Regeneron's release
- see Bloomberg's story
- and Reuters' report

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