Esperion Eyes Early 2019 to File NDA for Cholesterol-Lowering Therapy

Shares of Ann Arbor, Mich.-based Esperion Therapeutics are up more than 7 percent in premarket trading after the company announced on Sunday that planned to seek regulatory approval for its cholesterol-lowering therapy that hit the mark in a Phase III trial.

Esperion said its bempedoic acid candidate lowered LDL cholesterol 18 percent more than statins alone in patients who were on a maximally-tolerated dose of statins, but were still in need of assistance in lowering LDL cholesterol. The data announced Sunday included four, pivotal, Phase III randomized, double-blind, placebo-controlled studies to evaluate the LDL-C lowering efficacy and safety and tolerability of bempedoic acid 180 mg compared to placebo. The program included high-risk cardiovascular patients, including those with atherosclerotic cardiovascular disease (ASCVD) and/or heterozygous familial hypercholesterolemia (HeFH) patients. At 12 weeks, the bempedoic acid program achieved its primary efficacy endpoints and also hit other key measures, the company said.

Esperion noted that completed Phase II and Phase III studies conducted in almost 4,800 patients, including approximately 3,100 patients treated with bempedoic acid, have produced an additional 20 percent LDL-C lowering when used with maximally tolerated statins, up to 30 percent LDL-C lowering as a monotherapy, 35 percent in combination with ezetimibe when used with maximally tolerated statins, and up to 48 percent LDL-C lowering in combination with ezetimibe as monotherapy.

Cardiovascular disease is a significant health issue in the United States. CV events such as stroke or heart attack affect someone in America every 40 seconds.

Data from the Phase III trial showed that bempedoic acid lowered LDL cholesterol 18 percent to 31 percent versus placebo and in the intent to treat analysis, LDL-C lowering of an additional 17 percent to 28 percent. Additionally, the treatment showed reductions of 19 percent to 33 percent in high-sensitivity C-reactive protein (hsCRP), which Esperion noted is an important marker of the underlying inflammation associated with cardiovascular disease.

Bempedoic acid is a first-in-class ATP Citrate Lyase (ACL) inhibitor. Bempedoic acid reduces cholesterol biosynthesis and lowers LDL-C by up-regulating the LDL receptor. Early and mid-stage trial data presented by Esperion has shown the bempedoic acid has produced clinically relevant LDL-C lowering results of up to 30 percent as monotherapy and an incremental 20+ percent when added to stable statin therapy. Similar to statins, bempedoic acid also reduces hsCRP, a key marker of inflammation associated with cardiovascular disease, the company said.

Esperion said it intends to file a New Drug Application with the U.S. Food and Drug Administration (FDA) in the early months of 2019. The company also intends to file for marketing authorization in Europe during the second quarter of 2019, the company said.

With the potential approval of bempedoic acid, Esperion intends to take on some well-established drugs in the space, including Amgen’s Repatha, a PCSK9 inhibitor.

Esperion said bempedoic acid was observed to be safe and well-tolerated during the Phase III trial, the company noted. The effect of bempedoic acid on cardiovascular morbidity and mortality has not yet been determined. Esperion said it has initiated a global cardiovascular outcomes trial to assess the effects of bempedoic acid on the occurrence of major cardiovascular events in patients with, or at high risk for, cardiovascular disease who are only able to tolerate less than the lowest approved daily starting dose of a statin and considered "statin intolerant."