Sanofi/Regeneron add to stockpile of promising PCSK9 LDL drug data

Over the long Memorial Day, weekend Regeneron Pharmaceuticals ($REGN) and Sanofi ($SNY) added to their stockpile of promising mid-stage data for their PCSK9 drug, which is designed to slash levels of "bad" LDL-cholesterol. Tapped as one of the most promising potential blockbusters in Sanofi's pipeline, REGN727 (or SAR236553) was able to cut the levels of LDL in treatment-resistant patients by anywhere from 28.9% to 67.9%, depending on dosage.

In the placebo arm the average drop was 10.7%. 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 81.3% reached LDL-C levels lower than 70 mg/dL, compared to none receiving a placebo.

Sanofi and Regeneron are teamed up on one of the most advanced PCSK9 therapies in the pipeline, but they are not alone. Amgen ($AMGN) has been playing catch-up with its own program and Alnylam Pharmaceuticals ($ALNY) has an early-stage RNAi approach to the problem. Sanofi/Regeneron plan to start their Phase III program in June. It will face extremely high hurdles on safety, even as some analysts question the overall size of a potential market at a time so many generics are available. Aside from the contrarians, though, others see a big market just treating patients with lethally high levels of LDL or the group which won't respond to the cheap drugs now available.

"There remains a high degree of unmet need in these patients as a large percentage are unable to reach optimal LDL-C goals despite being on maximal lipid-lowering therapy," said Evan A. Stein, M.D., Ph.D., director of the Metabolic and Atherosclerosis Research Center in Cincinnati and principal investigator of the study. "These data suggest that SAR236553/REGN727 may provide a new option, on top of existing therapy."

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- here's the story from Reuters

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