A drug from Esperion Therapeutics reduced the risks of heart disease in a large international trial, a result the company hopes could spur wider use of the medicine as an alternative to other cholesterol-lowering treatments.
In December, Esperion said without details that the drug, known as bempedoic acid or Nexletol, had led to a “clinically meaningful” reduction in risk of cardiovascular events, such as heart attacks and strokes, in a study of about 14,000 patients who can’t or won’t take cholesterol-lowering statins because of side effects.
On Saturday, Esperion presented the findings at a medical conference and simultaneously published them in the New England Journal of Medicine. Researchers found Nexletol lowered the risk of those events as a whole by 13% compared to a placebo. The impact was more significant on specific event types, as researchers found relative risk reductions of 23% and 19% in heart attacks and bypass surgeries, respectively.
Esperion believes the results could lead to increased use of Nexletol, which was originally approved by the Food and Drug Administration in 2020 but has struggled to sell. The company expects to submit regulatory filings in the U.S. and Europe in the first half of 2023 to add the reduction in risk of cardiovascular events to its prescribing information, and approvals in the first half of 2024, said CEO Sheldon Koenig in the statement.
In the U.S., the new label would be focused on its effect on heart attacks and heart surgeries, where the drug had its best results, said chief medical officer JoAnne Foody in an interview Sunday.
The company said the expanded approvals could more than double the addressable treatment population for the pill. Estimates vary for those who cannot take statins, from 5 to 30% of the population, she said.
The results of the trial “will and should increase the use of [Nexletol] in patients with established atherosclerotic vascular disease and in those at high risk for vascular disease who are unable or unwilling to take statins,” wrote John Alexander, a cardiologist at the Duke Clinical Research Institute, in an editorial in The New England Journal of Medicine.
Still, Alexander cautioned that it’s “premature” to consider Esperion’s drug as an alternative to statins. It’s also unknown how well the medicine might perform as an add-on to statins, he said. (Foody said the company is testing a combination of the drug with the cholsterol lowering drug ezetimibe and a statin in people who have suffered a heart attack.)
Indeed, investors appear unconvinced by the results. Esperion shares fell by more than 20% on Monday. In a note to clients, Jefferies analyst Dennis Ding noted that the 13% relative risk reduction the company reported “did not seem to meet the 15% benefit bar” Wall Street was looking for.
But Ding noted that the data “still looks strong.” Nexletol’s true benefeits may not have been captured because it faced a tougher comparator than previous heart drug studies, with patients getting the chance to take multiple other types of cholesterol-lowering drugs. The placebo group’s level of LDL-C, or “bad” cholesterol, declined over the course of the trial, unlike other studies where those levels stayed flat or increased, he wrote.
“That said, these are nuances with the data and may take time for investors to fully appreciate,” Ding wrote.
Esperion would receive a $300 million payment from Daiichi Sankyo if European regulators expand Nexletol’s label. It could get up to another $140 million from Japanese partner Otsuka Pharmaceutical for other regulatory milestones including an expanded U.S. label.