Esperion Therapeutics’ bempedoic acid met its primary endpoint in a phase 3 trial, topline data show. The treatment lowered patients’ LDL cholesterol by 23% from baseline, compared to a 5% increase in LDL observed in patients on placebo.
The 269-patient trial pitted a daily 180-mg dose of bempedoic acid against placebo in patients with atherosclerosis, or patients who are at high risk of atherosclerosis whose cholesterol cannot be controlled with lipid-modifying drugs, such as Merck’s Zetia, or the lowest approved starting dose of a statin.
Patients in the treatment arm also saw a 33% decrease in high-sensitivity C-reactive protein (hsCRP), a biomarker of inflammation linked to heart disease, the company said. While two patients (1.1%) in the treatment arm had elevations in liver function tests, there was no difference in adverse events across the treatment and placebo groups.
“These results suggest that bempedoic acid, with its targeted mechanism of action and convenient, oral, once-daily administration, could be an important new treatment option for a wide range of patients, including those unable to tolerate moderate or high doses of commonly-used statin therapy,” said Christie Ballantyne, M.D., chief of cardiology at Baylor College of Medicine and chairman of Esperion’s Phase 3 Executive Committee.
“The study looks generally fine to us and within our expectation,” wrote Jefferies analysts, who are looking to the readout in May of a one-year phase 3 safety study. “This would be a significant derisking event if all looks fine as long as no major serious liver issues, which we do not foresee,” they said.
The new data comes seven months after Esperion reported that a combo of bempedoic acid with the active ingredients in Merck’s Zetia and Pfizer’s Lipitor lowered LDL by 64%, outperforming results achieved by a Zetia/Lipitor pairing in other studies. This prompted the company to move up its timeline by three months; it now expects to file NDAs for bempedoic acid as a monotherapy and as a combination with Zetia in the first quarter of 2019.