ANN ARBOR — The cholesterol-busting drug developer Esperion Therapeutics Inc. (NASDAQ:ESPR) said its Phase 3 clinical trial of drug candidate bempedoic acid will include patients with high cholesterol who are already taking any and all statin drugs.
The move comes based on positive results from Phase 2 trials adding bempedoic acid to patients already taking the statin drug atorvastatin. Adding bempedoic acid lowered cholesterol a further 22 percent compared to placebo. It also demonstrated an incremental reduction of 35 percent in high-sensitivity C-reactive protein (hsCRP), an important marker of the underlying inflammation associated with cardiovascular disease. Bempedoic acid also appeared to be safe and well-tolerated, with no serious adverse events reported.
“With the positive clinical results of bempedoic acid added to high-dose statins, and following engagement with global regulatory agencies, we are pleased to include in our ongoing global Phase 3 program patients with elevated LDL-C levels inadequately treated with current lipid-modifying therapies, including patients on any statin at any dose,” said Tim Mayleben, president and CEO of Esperion. “Bempedoic acid has demonstrated consistent LDL-C lowering efficacy, safety, and tolerability in completed Phase 1 and Phase 2 studies, including most recently in combination with high-dose statins. We believe that bempedoic acid is well-positioned as a once-daily, oral therapy for patients with hypercholesterolemia taking maximally tolerated statin therapy and/or ezetimibe who require additional LDL-C lowering, including patients considered ‘statin intolerant’. We remain keenly focused on initiating both the bempedoic acid global Phase 3 efficacy program and our cardiovascular outcomes study before year-end.”
Before Dec. 31, Esperion plans to initiate three global pivotal Phase 3 studies in patients with high cholesterol who are inadequately treated with current lipid-modifying therapies such as statins. Included will be people for whom statins have unpleasant side effects, who are termed “statin intolerant.” Results from those studies are expected in 2018, and the company said it plans ot file a New Drug Application with the U.S. Food and Drug Administration and a Marketing Authorization Application to the European Medicines Agency in 2019.
Esperion also announced positive results from its Phase 1, open-label, clinical pharmacology study (1002-037) to assess the PK levels in healthy volunteers receiving single doses of the highest doses of the most commonly prescribed statins — atorvastatin 80 mg, rosuvastatin 40 mg, simvastatin 40 mg and pravastatin 80 mg — when added to steady-state bempedoic acid 180 mg.
The PK profiles demonstrated in 1002-037 were consistent with those seen in previous studies conducted with bempedoic acid, and did not increase with the highest doses of the statins tested in combination with bempedoic acid. Together, with the results of 1002-035, these data support enrollment of patients with hypercholesterolemia on any statin at any dose in the global pivotal Phase 3 CLEAR LDL-C clinical development program.
To listen to a replay of a conference call discussing these developments, visit http://www.esperion.com.
In Phase 1 trials, researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects. In Phase 2 trials, the drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety. And in Phase 3, the drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Bempedoic acid reduces cholesterol biosynthesis and lowers elevated levels of LDL-C by up-regulating the LDL receptor, but with reduced potential for muscle-related side effects. Phase 1 and 2 studies conducted previously in more than 800 patients treated with bempedoic acid have produced clinically relevant LDL-C lowering results of up to 30 percent as monotherapy, approximately 50 percent in combination with ezetimibe, and an incremental 20 to 22 percent when added to stable statin therapy.
In the United States, 78 million people, or more than 20 percent of the population, have elevated LDL-C; an additional 73 million people in Europe and 30 million people in Japan also live with elevated LDL-C. Esperion’s mission is to provide patients and physicians with a new oral therapy to significantly reduce elevated levels of LDL-C in patients inadequately treated with current lipid-modifying therapies.