Esperion's potential PCSK9 spoiler scores more positive data

Esperion CEO Tim Mayleben

Amid all the fervor over cholesterol-fighting PCSK9 antibodies, Esperion Therapeutics ($ESPR) is touting more positive results for a pill it hopes will play spoiler to those would-be-blockbuster injections.

The drug, ETC-1002, met its primary goal in a Phase IIb study on 134 patients with high LDL, or bad, cholesterol. A combination of Esperion's pill and statins lowered LDL by 17% on the lowest ETC-1002 dose and 24% at the highest, while those taking statins alone posted a 4% reduction. On the safety side, Esperion's drug led to no increase in muscle-related side effects, the company said, and study dropout rates were lower in the treatment arm than in the placebo group.

The results echoed earlier positive mid-stage results for ETC-1002 and burnished the company's ambitions to kick off a Phase III trial later this year. Esperion is planning to enroll about 4,000 patients in a late-stage study to confirm the treatment's effect on LDL cholesterol, marking the next step in its plan to disrupt the blockbuster race for next-generation cardio drugs.

ETC-1002 has repeatedly demonstrated its ability to reduce bad cholesterol, albeit not at the rate of injections from Amgen ($AMGN) and partners Sanofi ($SNY) and Regeneron ($REGN), which can cut LDL by as much as 60%. But, unlike those likely expensive antibodies, Esperion's oral candidate would be positioned as a cheaper, easier solution, CEO Tim Mayleben said at January's JP Morgan Healthcare Conference.

As the CEO sees it, his company's pill would be an ideal second option for the millions of patients who either can't tolerate statins or can't get to healthy LDL levels on the generic drugs. The antibodies, which work by blocking the protein PCSK9, will end up as third-line therapies, he said, subscribing to "the radical belief that patients will prefer an oral therapy over injection."

Express Scripts ($ESRX), emboldened by its recent victories in hepatitis C, has identified the coming crop of PCSK9 treatments as ideal next targets for a price war, figuring it can squeeze some savings by pitting fairly comparable drugs against one another. And Mayleben is well aware of the current landscape in healthcare and what it might mean for Esperion, tweeting Tuesday that "ETC-1002 is patient friendly, physician friendly and payer friendly."

But Esperion will have to come through in Phase III before that conversation can begin. Last month, the company got out from under a partial FDA hold tied to some concerns about ETC-1002's toxicity, worries apparently dispelled by a closer look at preclinical data. Now, thanks to a nearly $100 million public offering late last year, the biotech has the cash to get a late-stage trial rolling, and Mayleben has said Esperion may eventually look to partner up with a larger drugmaker with experience running large cardiovascular trials.

- read the results

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