Esperion hits goal in LDL trial, pulls forward NDA target date

A phase 2 trial combining Esperion Therapeutics’ bempedoic acid with the active ingredients in Merck’s Zetia and Pfizer’s Lipitor has met its primary endpoint. The regimen lowered LDL cholesterol by 64%, a bigger drop than the combination of Zetia and Lipitor has achieved in other studies. 

Esperion pointed to the LDL-C reductions seen in IMPROVE-IT, Merck’s trial adding Zetia to statins, to support its belief bempedoic acid affected levels of the lipoprotein. Such cross-trial comparisons are necessary because the phase 2 study of Esperion’s triple combination lacked an active control arm. Instead, the combination was pitted against—and comfortably beat—placebo.  

The data tee Esperion up to initiate further studies of the combination in the first quarter of next year. By then, Esperion will also be closing in on the publication of data from phase 3 trials assessing bempedoic acid as a monotherapy and in combination with Zetia.

Shares in Esperion rose 15% following the release of the data and news of progress in its phase 3 program. Esperion now expects to file NDAs for bempedoic acid and a bempedoic acid-Zetia combination in the first quarter of 2019, three months earlier than previously forecast. The change to the timeline follows speedy progress in the clutch of phase 3 trials now underway. 

Esperion CEO Tim Mayleben thinks his company’s phase 2 data, cash reserves and prospect of phase 3 results next year put it in the box seat as it talks about partnerships with the sorts of "very large, global companies" that can commercialize cardiovascular drugs. Esperion’s hand was strengthened earlier this year by the outcome of discussions with the FDA. 

"I would say that the primary de-risking events have happened," Mayleben said in a conference call with investors. "I would point you back to March of this year, when we reached agreement with the FDA on the regulatory path to approval of the bempedoic acid. And then secondly, in June, when we reached agreement with FDA on the 505(b)(2) pathway, the abbreviated pathway to approval for the combo pill of ezetimibe and bempedoic acid. What we have seen is our discussions really accelerate with that regulatory clarity."