The drug: REGN727
The disease: Cardiovascular disease
The developers: Sanofi/Regeneron
Peak sales potential: Some estimates start at $2.5 billion, though Deutsche Bank analyst Robyn Karnauskas leads the cheering section with $3 billion to $6 billion in potential sales.

Back in July, Sanofi ($SNY) investigators were ramping up recruiting efforts in search of 40,000 patients for 10 late-stage studies of REGN727, a PCSK9 therapy that has demonstrated a remarkable ability to lower bad LDL cholesterol in patients who are facing a severe cardiovascular risk, can't tolerate statins or suffer from familial hypercholesterolemia. A bit more than half of those patients will be tested for a drop in LDL levels, with the others being scrutinized for cardio outcomes.

At its highest dose, the treatment demonstrated a 68% drop in LDL among patients in one study. On Twitter, REGN727 is the kind of treatment that generates quite a bit of scoffing among some analysts who can't see a treatment muscling aside cheap and effective statins and carving out a blockbuster market. Sanofi and Regeneron ($REGN), on the other hand, see millions of potential patients who could benefit. And that has driven a high-stakes effort to make this the leading therapy among a slate of PCSK9 drugs, one of the hottest new categories in drug development, in the pipeline.

The theory here is quite simple. By interfering with the protein that spurs the body to retain LDL, developers hope to deliver a drug that could rival Lipitor one day. Amgen ($AMGN), Merck ($MRK) and others are hot on their heels.

Under normal circumstances, a big program like this probably couldn't deliver an approval before 2016. But Sanofi officials have said that this drug could clear regulators by late 2015. Speed is critical, as other developers are pushing promising programs of their own.

For more:
Special Report: Regeneron - The Biggest R&D Spenders in Biotech
Big Pharma betting billions in high-stakes hunt for the next Lipitor
Sanofi and Roche race to PhIII with drugs aimed at hot PCSK9 gene
Sanofi/Regeneron add to stockpile of promising PCSK9 LDL drug data


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