The drug: Alirocumab (SAR236553/REGN727)
The disease: LDL cholesterol
The developers: Sanofi, Regeneron
Peak sales potential: The bull case considers $3 billion-plus easily attainable, but there are naysayers.
The scoop: Regeneron ($REGN) and Sanofi ($SNY) were so pumped about the potential for alirocumab as an efficient measure for dialing down high levels of "bad" LDL cholesterol, they started with a slate of 11 Phase III studies last year. And they added another one earlier this year to look at the effect of less frequent dosing.
To be sure, the partners are by no means alone in the field. Quick to see the potential of blocking the protein PCSK9, Amgen ($AMGN) has been hustling along with AMG 145, and Pfizer ($PFE), Novartis ($NVS) and Roche ($RHHBY) have been playing catch-up, fascinated by the market potential of a safe, effective treatment for hypercholesterolemia that could be used to treat statin-resistant patients.
In mid-stage studies, alirocumab cut levels of LDL by up to 68%, depending on the dose. At the top dose, 93.8% of patients achieved LDL-C levels lower than 100 mg/dL compared to 13.3% of patients on placebo. And it won't be long now before the first batch of highly anticipated Phase III data arrives.
To be sure, not everyone is convinced that there's a pot of gold waiting for every PCSK9 drug in the pipeline, considering the generics out there that now dominate the field and the number of competitors in the race. The smart money in pharma circles says they're wrong, though, and Regeneron has been building an excellent reputation for innovation.
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