Esperion looks to disrupt the PCSK9 race with new cardio data

Esperion ($ESPR), developing a pill it hopes can eventually cut in on some would-be blockbusters, posted new study results showing that its drug both reduced bad cholesterol and improved inflammation.

In an exploratory Phase II trial, Esperion's ETC-1002 significantly beat out placebo in treating patients with both high cholesterol and high blood pressure. In the 143-patient trial, ETC-1002 met its primary goal by notching a 21% reduction in LDL, or bad, cholesterol from baseline, compared to a 3% increase with placebo.

And the drug came through on its secondary endpoint of reducing high-sensitivity C-reactive protein, or hsCRP, which is a marker of inflammation in coronary disease. ETC-1002 lowered hsCRP 25% from baseline and a whopping 44% compared with placebo. Esperion said the drug had a "neutral" effect on blood pressure, another secondary goal, but the company did not disclose detailed data.

The case for ETC-1002, slated to enter Phase III development in the fourth quarter, is that it can beat cheap statins on LDL reduction and undercut a new class of injected therapies on price. Across its mid-stage development, the drug has consistently reduced bad cholesterol by around 25% from baseline, and while that doesn't measure up to the LDL-lowering effects of so-called PCSK9 inhibitors from Sanofi ($SNY) and Amgen ($AMGN), Esperion's pill will be much cheaper to manufacture than those antibodies.

The U.S.'s first PCSK9 blocker, Sanofi's Regeneron ($REGN)-partnered Praluent, won FDA approval last week based on its ability to lower bad cholesterol in Phase III trials, and the companies plan to charge more than $14,000 a year for the antibody. Esperion and its investors view both of those facts as positives for ETC-1002, as the agency again demonstrated its willingness to approve new cardio drugs without waiting on long-term outcomes data, and Praluent's higher-than-expected sticker price gives the company some material for its future pitches to payers.

But any market prospecting is premature until Esperion can prove ETC-1002's worth in Phase III. The company is planning to kick off a 4,000-subject trial by year's end, testing how well a 180-mg dose of its lead drug can lower cholesterol in a wide variety of patients.

- read the statement

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