- Bempedoic acid, an experimental drug from Esperion Therapeutics Inc., significantly lowered bad cholesterol in patients with cardiovascular disease due to clogged arteries, according to new clinical data.
- Over the course of 12 weeks, participants who received Esperion's treatment experienced a 23% reduction from baseline in their low density lipoprotein cholesterol (LDL-C), whereas those on placebo had a 5% increase in their LDL-C levels. Adverse events, including ones that were serious or muscle-related, were similar between the experimental and control groups.
- Despite the positive results, Esperion stock traded down about 5% to $74.28 per share in Wednesday morning trading.
More than a third of U.S. adults and roughly a sixth of U.S. children are obese, and many more are overweight. The trend poses serious health risks too, as research time and again has shown excess fat leads to upticks in cases of diabetes, heart disease and high cholesterol.
Statins, which worked by inhibiting an enzyme involved in the production of cholesterol, were traditionally a go-to treatment for overweight patients. Yet America's growing weight problem has spurred drugmakers to take aim at developing newer and better therapies — though that hasn't exactly panned out.
Another enzyme that interacts with LDL-C, attracted particular interest: PCSK9. Many expected two drugs that inhibit the enzyme, Sanofi SA and Regeneron Pharmaceuticals Inc.'s Praluent (alirocumab) and Amgen Inc.'s Repatha (evolocumab), to become blockbusters.
Though very effective at reducing LDL-C, the drugs haven't gained much traction in the market due, in part, to their high price tags causing pushback from payers.
Esperion is hoping it can succeed where the heavy-hitters in the PCSK9 space seemed unable.
"The study looks generally fine to us and within our expectation and we appreciate the more important study [readouts] in May, which is the Phase 3 one year safety study," Jefferies analyst Michael Yee wrote in a March 7 note. "This would be a significant derisking event if all looks fine as long as no major serious liver issues, which we do not foresee as [the data safety monitoring board] has been reviewing the program and has made no major adjustments — implying no major clinical issues."
In addition to the LDL-C reductions, the latest data showed patients treated with bempedoic acid had significantly reduced levels of a protein tied to the underlying inflammation seen in cardiovascular disease.
Esperion expects to announce topline results from the Phase 2 '039 study, which is evaluating its drug plus monthly PCSK9 therapy. And in May, the company plans to reveal data from two other Phase 3 studies of bempedoic acid. The company has indicated it will file the drug for approval in the U.S. and Europe during the first half of 2019.
Even with those updates, Esperion shares took a considerable hit Wednesday morning. While it's unclear exactly why, Madrigal Pharmaceuticals Inc. fell into a similar situation last month with its cholesterol-lowering candidate — perhaps indicating that cholesterol-focused drugmakers have a high bar to clear before wooing investors.
Esperion estimates there are roughly 12 million to 13 million people with atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia who need additional LDL-C treatments beyond statin therapy.
"Physicians are eagerly awaiting new, once-daily, oral therapies that complement existing oral drugs to provide the LDL-C lowering that their high-risk patients need, the value payers appreciate, and with the convenience and tolerability patients want and deserve," Tim Mayleben<, Esperion CEO, said in a March 7 statement.
"Looking ahead, we expect results from subsequent Phase 3 studies to further confirm that bempedoic acid and the bempedoic acid /ezetimibe combination pill will deliver consistent LDL-C lowering in safe, well-tolerated and convenient once-daily pills that are highly complementary to existing standard-of-care oral LDL-C lowering therapies.