Esperion's PCSK9 spoiler clears another FDA roadblock as it heads to Phase III

Esperion ($ESPR), at work on a cholesterol-lowering pill it hopes can disrupt some high-profile new injections, convinced the FDA to remove a partial clinical hold on the highest dose of its drug.

The agency has rescinded its restrictions on testing a 240-mg dose of Esperion's ETC-1002, an oral treatment designed to slash levels of LDL, or bad, cholesterol. The company is already planning to kick off a Phase III trial with a 180-mg version later this year, but the FDA's decision gives Esperion the option of moving forward with a stronger dosage in the future if it so chooses.

The Ann Arbor, MI, company is angling to play spoiler in the coming blockbuster market for new cholesterol treatments, pitching its therapy as a potentially desirable alternative to medicines from Amgen ($AMGN) and partners Sanofi ($SNY) and Regeneron ($REGN) that are expected to hit the market this year. Those antibodies, which work by blocking the protein PCSK9, have charted stellar efficacy in Phase III trials but require regular subcutaneous injections, and Esperion is betting its oral option can eventually take their place among patients not getting what they need from cheap statins.

But Esperion's case--and the case for next-generation cholesterol drugs in general--took a hit last month after a panel of FDA advisers gave the two leading PCSK9 blockers a lukewarm reception. The group of physicians ultimately voted in favor of approving the treatments for patients with dangerously high LDL levels but cautioned against widespread use until long-term results data become available in about two years.

That reticence dampened some of the bullishness around PCSK9 antibodies, which analysts have said could bring in $3 billion a year each. And it trickled down to Esperion, whose shares fell more than 35% after the panel votes, driven down by concerns that ETC-1002 may face an uphill path to approval and market adoption.

But the company remains undaunted. In February, Esperion convinced the FDA to remove a weightier clinical hold, one impeding the company from running trials longer than 6 months, and immediately set out plans to enroll a 4,000-patient Phase III trial. Esperion is slated to hold its requisite end-of-Phase II meeting with the agency next month, and the company expects to start its pivotal program in the fourth quarter.

In Phase IIb data unveiled in March, a combination of Esperion's pill and statins lowered patients' LDL by 17% on the lowest ETC-1002 dose and 24% at the highest, while those taking statins alone posted a 4% reduction. That hardly contends with the effect of PCSK9 treatments, which cut bad cholesterol by as much as 60%, but Esperion believes it can position its product as a considerably cheaper, easier alternative if and when it wins approval.

- read the statement (PDF)