|Esperion CEO Tim Mayleben|
Shares of Esperion Therapeutics ($ESPR) shot up more than 10% after the market closed on Monday--after the biotech tipped off investors that the FDA is not going to require a long-term outcomes study of its cholesterol-lowering drug ahead of a possible approval.
Investors and analysts have been fretting over exactly what the FDA will do with ETC-1002, an LDL-lowering pill that is angling for a share of a blockbuster market many believe lies in wait for a new class of PCSK9 drugs. While the oral ETC-1002 may not be as effective as the PCSK9 drugs, including the newly-approved Praluent from Regeneron ($REGN) and Sanofi ($SNY) as well as a rival Amgen ($AMGN) drug, it also has a shot at grabbing a significant share of the market with a less expensive and more easily managed drug that could do everything many patients in this huge market require.
Overhanging all of these drugs has been a persistent fear that regulators would require a long-running cardiovascular outcomes trial to prove the therapy works as expected in improving patients' health. But according to Esperion, the agency says it's ready to give it a green light for a relatively narrow market--which still amounts to a patient pool of 9 million people--and then hold back on a broader approval until after the CVOT data comes in later.
Noted Esperion: "Based on feedback from the FDA, approval of ETC-1002 in the HeFH (heterozygous familial hypercholesterolemia) and ASCVD (clinical atherosclerotic cardiovascular disease) patient populations will not require the completion of a cardiovascular outcomes trial. The company continues to plan and initiate a CVOT prior to NDA filing to pursue broader label indications related to cardiovascular disease risk reduction."
The regulatory update leaves Esperion prepared to start a Phase III study with 4,000 patients ETC-1002 later this year.
"After an informative and collegial meeting with the FDA, we are pleased that LDL-C remains an accepted clinical surrogate endpoint for the approval of an LDL-C lowering therapy such as ETC-1002 in patients with HeFH and/or patients with ASCVD," said Tim Mayleben, president and chief executive officer of Esperion. "We have a clear regulatory path forward for development and approval of ETC-1002, an oral, once-daily treatment option for these patients that require additional LDL-C lowering."
In a recent Phase II trial, Esperion's ETC-1002 significantly beat out placebo in treating patients with both high cholesterol and high blood pressure. In the 143-patient trial, ETC-1002 met its primary goal by notching a 21% reduction in LDL, or bad, cholesterol from baseline, compared to a 3% increase with placebo.
- here's the release