Esperion gets out from under the FDA and speeds toward Phase III

Esperion CEO Tim Mayleben

Ann Arbor, MI, biotech Esperion Therapeutics ($ESPR) finally resolved the FDA snag standing in the way of its ambitious plans for a new cholesterol drug, clearing the company to begin late-stage trials with its potentially disruptive pill.

The agency has lifted a partial clinical hold that limited Esperion from running studies longer than 6 months, the company said. The FDA had previously expressed concern about the drug's toxicity, but, vetting preclinical data the biotech submitted late last year, regulators have given Esperion the go-ahead to keep moving.

Now the biotech plans to take its oral cholesterol fighter, ETC-1002, into Phase III later this year, CEO Tim Mayleben said. 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 Esperion's plan to shoulder in on the blockbuster race for next-generation cardio drugs.

ETC-1002 is a small-molecule therapy that has successfully slashed levels of LDL, or "bad," cholesterol in clinical trials. So, too, have the blockbuster-in-waiting injections from Amgen ($AMGN) and partners Sanofi ($SNY) and Regeneron ($REGN). But, unlike those likely expensive antibodies, Esperion's candidate is positioned as a cheaper, easier solution, Mayleben said at last month's JP Morgan Healthcare Conference.

As the CEO sees it, his company's pill will 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."

First Esperion will have to come through in Phase III, of course. Thanks to a nearly $100 million public offering late last year, the biotech has enough cash to go it alone, but Mayleben said Esperion may look to partner up with a larger drugmaker with experience running large cardiovascular trials.

Beyond ETC-1002, Esperion has only one other disclosed asset, a preclinical metabolic treatment. That's a reflection of what Mayleben said is an all-hands-on-deck devotion to the top prospect. Esperion is effectively a one-trick pony, he said, but that's "not so bad if you're riding Seabiscuit."

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