Ann Arbor, Michigan-based Esperion Therapeutics Inc. announced that its bempedoic acid for lowering LDL-C cholesterol had positive results in a Phase III clinical trial.
In the 12-week trial, 269 patients were randomized two to one to receive bempedoic acid or placebo. The patients in the bempedoic acid group received 180 mg/day in addition to now-generic Zetia (ezetimibe). The patients either had atherosclerotic cardiovascular disease (ASCVD) or were at high risk for it, and were not responding well to current lipid-modifying drugs, including ezetimibe and statins.
The trial was conducted at 90 sites in the U.S., Canada and Europe. The drug was seen to be safe and was well-tolerated. The group receiving bempedoic acid had their LDL-C (low density lipoprotein cholesterol) drop by 23 percent from baseline, compared to five percent for the placebo group. The patients on the drug also had a 33 percent decrease in high-sensitivity C-reactive protein (hsCRP), which is a marker related to the underlying inflammation of cardiovascular disease. The patients in the placebo group had a 2 percent increase of hsCRP.
"Many of my patients, including those considered statin intolerant, remain at risk for further cardiovascular disease, including heart attack and stroke, despite the availability of current LDL-C-lowering therapies," said Christie Ballantyne, chairman of Esperion’s Phase III Executive Committee and professor and chief of Cardiology at Baylor College of Medicine in Houston. "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."
Other companies have been working to develop PCSK9 inhibitors, a very potent class of cholesterol-lowering drugs. The two currently on the market are Amgen’s Repatha and Sanofi US and Regeneron Pharmaceuticals, Inc.’s Praluent. Both drugs are very expensive, with Praluent’s wholesale price being $14,600 per year and Repatha’s being $14,100 per year. Statins cost, on average, about $50 per month.
Bempedoic acid is not viewed as potent as PCSK9 inhibitors, but likely would be considerably cheaper.
Michael Yee, an analyst with Jefferies, wrote in a note to clients, "The study looks generally fine to us and within our expectation and we appreciate the more important reads out in May, which is the Phase III one-year safety study."
Company shares increased about 3.9 percent in premarket trading today after the results were released. In the last year, company stocks have risen 149 percent, compared to the S&P 500 SPX, which has gained only 15 percent in the same period.
The study was the first of five. The company hopes to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bempedoic acid and the bempedoic acid/ezetimibe combination pill in the first quarter of 2019. It also expects to submit Marketing Authorization Applications (MAAs) to the European Medicines Agency (EMA) by the second quarter of 2019.
"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," said Tim Mayleben, Esperion’s president and chief executive officer, in a statement. "Looking ahead, we expect results from subsequent Phase III 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."
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