PLYMOUTH — Esperion Therapeutics’ proposed new cholesterol-busting drug cut the level of low-density lipoprotein, the so-called bad cholesterol, up to 43 percent in a Phase 2 test of diabetic patients.
The privately held biotech company said the study met its goals. Besides the reduction in cholesterol levels, the study showed the compound, now called ETC-1002, was associated with improvements in control of other cardiometabolic risk factors. In addition, ETC-1002 was well tolerated.
The data were presented Thursday in Lake Buena Vista, Fla. at the Arteriosclerosis, Thrombosis, and Vascular Biology (ATVB) 2013 Scientific Sessions. Esperion previously announced topline results from this clinical trial in January.
Esperion is currently evaluating ETC-1002 in multiple Phase 2 clinical trials in more targeted patient populations. These include a Phase 2 trial in patients with high cholesterol who are intolerant to statin therapy and a Phase 2 trial of ETC-1002 in combination with statin therapy in patients with high cholesterol.
“We are encouraged by both the significant efficacy observed, as well as the tolerability results in this trial, especially since there were no reported cases of myalgia,” said Esperion chairman and chief scientific officer Roger S. Newton. “Our clinical program is primarily focused on statin intolerant and resistant patients where tolerability of ETC-1002 is an important factor. We believe that these results show that ETC-1002 has the potential to be developed in broader patient populations with multiple cardiometabolic risk factors, especially in patients who suffer from both hypercholesterolemia and diabetes.”
This randomized, double-blind, placebo-controlled, parallel group, single-site, Phase 2 trial assessed the LDL-C lowering efficacy and safety of ETC-1002 compared with placebo in patients with Type 2 diabetes. A total of 60 patients with a history of Type 2 diabetes, a body mass index of 25-35 and bad cholesterol count of over 100 were randomized to ETC-1002 or placebo for four weeks and treated in an inpatient unit where their diet and lifestyle were controlled.
After two weeks of treatment with 80 mg of ETC-1002, LDL-C was reduced by an average of 32 percent, while after an additional 2 weeks of 120 mg of ETC-1002, LDL-C was reduced by an average of 43 percent, compared with 6 percent and 3 percent reductions, respectively, for those patients treated with placebo. ETC-1002 also significantly reduced non-HDL-C by 30 percent and high sensitivity C-reactive protein and blood pressure compared with placebo. ETC-1002 had neutral effects on other lipids, including triglycerides and HDL-C.
The most common adverse events in the ETC-1002 treated group (experienced by more than 5 percent of trial participants) were headache, hyperglycemia, constipation, arthralgia, dry eye and viral upper respiratory tract infection. The most common adverse events in the placebo-treated group were headache, hyperglycemia, dry eye, viral upper respiratory tract infection, pruritus, abdominal pain, nausea and dizziness. Importantly, no patients discontinued the trial because of myalgia, hypoglycemia or hypotension.
It is estimated that more than 2 million U.S. adults have discontinued statin therapy because of myalgia (muscle pain or weakness). Symptoms of myalgia occur in up to 20 percent of patients on statin therapy in clinical practice.
ETC-1002 is designed to target known lipid and carbohydrate metabolic pathways to lower levels of LDL-C and to avoid many of the side effects associated with existing LDL-C lowering therapies. Phase 2 clinical trials of ETC-1002 conducted to date have demonstrated significant average LDL-C reductions, as well as reductions comparable to statins in levels of hsCRP, a key marker of inflammation associated with cardiovascular disease. In clinical research to date, ETC-1002 has been well tolerated. No serious adverse events have been observed in more than 230 patients treated with ETC-1002.
Unlike some therapies currently in development that, if approved, will require regular injections, ETC-1002 is an oral pill taken once daily, the same way traditional LDL-C lowering therapies are administered. ETC-1002 is targeted for statin-intolerant patients with elevated levels of LDL-C.
More at www.esperion.com.