Esperion says FDA okay with approval path for key drug
- 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).
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.
- Esperion Therapeutics Statement
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