Dive Brief:
- Esperion Therapeutics said Monday the Food and Drug Administration has agreed that the company's LDL-C lowering drug candidate could be approved before completion of a large cardiovascular outcomes trial, lifting the biotech's stock by over 70%.
- A New Drug Application for the drug, known as bempedoic acid, is planned for sometime in the first half of 2019, assuming successful completion of Phase 3 studies testing the drug as an adjunct to maximally tolerated stating therapy in patients with high cholesterol.
- Esperion's stock had fallen sharply last week in response to weaker-than-expected — but still positive — results from Amgen's cardiovascular outcomes study for its PCSK9 drug Repatha (evolocumab).
Dive Insight:
Esperion's statement Monday seemed to give markets confidence that bempedoic acid's path to market would be shorter than expected.
The company hopes to report top-line results from its Phase 3 safety and efficacy studies in mid-2018, but results from its cardiovascular outcomes trial CLEAR aren't expected until 2022. Confirmation from the FDA that bempedoic acid could be approved after successful completion of the Phase 3 studies in 2018 would shorten Esperion's time to revenue-generation.
That clearly pleased markets, which bid up Esperion's stock substantially Monday. But investors should be careful — the FDA treats discussions with companies as confidential and does not say whether a company's characterization of meetings with the regulator is accurate.
Still, Esperion's statement gives investors a roadmap to hold the company accountable to and adds details on bempedoic acid's route to approval.
The company has beefed up its Phase 3 program, creating a set of three studies testing the drug's efficacy in addition to an ongoing long-term safety and tolerability study. These will cover a broad spread of patients, including those with or without atherosclerotic cardiovascular disease and with or without heterozygous familial hypercholesterolemia.
Bempedoic acid is an ACL inhibitor — a different mechanism of action than Amgen's Repatha or Sanofi and Regeneron's Praluent (alirocumab). But the commercial struggles of those two PCSK9 drugs, which are highly effective in lowering LDL-C, do raise questions about bempedoic acid's potential as a competitor. And while Repatha did not deliver as significant of a risk reduction in major adverse cardiovascular events as hoped for, it now has a proven cardiovascular benefit.
One possible avenue of competition, however, is cost. Repatha and Praluent are expensive, while Esperion's CEO Tim Mayleben has emphasized bempedoic's potential to be cost-effective.