ANN ARBOR — Esperion Therapeutics Inc. (NASDAQ:ESPR), the Ann Arbor-based pharmaceutical developer, announced positive results from the latest test of its lead drug candidate, bempedoic acid.
The Phase 3 study evaluated the safety, tolerability and efficacy of the drug in lowering low-density lipoprotein cholesterol, the so-called bad cholesterol, in patients whose cholesterol cannot be controlled by current therapies.
The study included 2,230 patients and met the primary endpoint of safety and tolerability and the key efficacy endpoint in terms of cholesterol lowering and reducing biomarkers of inflammation.
“As expected, these new data confirm the long-term safety and tolerability profile of bempedoic acid, even in high cardiovascular risk patients on maximally tolerated statins, especially when looking at muscle-related adverse events,” said Christie M. Ballantyne, M.D., chairman of Esperion’s Phase 3 executive committee and professor and chief of cardiology at Baylor College of Medicine in Houston. “Physicians are in need of additional convenient, once-daily, oral therapies, such as bempedoic acid, to complement currently available, once-daily, oral therapies for patients with ASCVD. These results suggest that bempedoic acid could be an important new oral treatment option for a very broad range of patients.”
In this 52-week study, bempedoic acid was observed to be safe and well-tolerated. There were no clinically relevant differences between the bempedoic acid and placebo groups in the occurrence of adverse events with 78.5 percent and 78.7 percent, respectively; or serious adverse events with 14.5 percent and 14.0 percent, respectively. Discontinuations due to AEs were 10.9 percent and 7.1 percent, respectively for the bempedoic acid and placebo groups; discontinuations due to muscle-related AEs were 2.2 percent and 1.9 percent, respectively in the bempedoic acid and placebo groups.
In the study, 0.54 percent of patients treated with bempedoic acid and 0.13 percent of patients in the placebo group had elevations in liver function tests (ALT/AST) of greater than three times the upper limit of normal, repeated and confirmed.
The cumulative number of patients now treated with bempedoic acid in Phase 2 and Phase 3 clinical trials totals 2,434. Of these, 0.58 percent had elevations in liver function tests greater than three times the upper limit of normal, repeated and confirmed. This rate of elevations in liver function test is consistent with the rate observed in all other previously approved oral LDL-C-lowering therapies, including statins and ezetimibe.
“In this study, the largest in our Phase 3 program, bempedoic acid was observed to be safe and well tolerated over a 52-week period, while providing clinically and statistically significant LDL-cholesterol lowering and reductions in hsCRP when added on to maximally tolerated statin therapy,” said Tim M. Mayleben, president and CEO of Esperion. “In the coming months, results from our three remaining pivotal Phase 3 studies are expected to further validate the safety, efficacy and tolerability profile of bempedoic acid and the bempedoic acid / ezetimibe combination pill, definitively establishing these once-daily oral therapies as convenient and complementary to existing treatments for the 13 million people in the U.S. with ASCVD who live with elevated levels of LDL-cholesterol despite taking maximally-tolerated lipid-modifying therapy and remain at high risk for further cardiovascular disease or events, including heart attack and stroke.”
To listen to a conference call discussing these results, visit the investors and media section of investor.esperion.com.
Esperion plans to submit New Drug Applications (NDAs) to the U.S. Food and Drug Administration (FDA) for bempedoic acid and the bempedoic acid / ezetimibe combination pill for LDL-C-lowering indications no later than the first quarter of 2019. Additionally, Esperion plans to submit Marketing Authorization Applications (MAAs) to the European Medicines Agency (EMA) no later than the second quarter of 2019.
Esperion also announced financial results for the first quarter of 2018. The pre-revenue company announced research and development costs of $40.9 million, up from $35.9 million a year earlier, and a total loss of $46.1 million for the quarter, up from $40.5 million in the same period of 2017. The company said it has cash on hand of $239.6 million as of March 31, down from $273.6 million as of Dec. 31.
Esperion expects full-year 2018 net cash used in operating activities to be approximately $135 to $145 million and its cash and cash equivalents and investment securities to be approximately $130 to $140 million at Dec. 31, 2018. The Company estimates that current cash resources are sufficient to fund operations through the expected approvals of the bempedoic acid / ezetimibe combination pill and bempedoic acid in the first quarter of 2020.