A small biotech company has a shot at shaking up a market roosted by giants, moving toward approval with a pill it believes can lower bad cholesterol at a discount to other medicines, reports Stat News. According to Esperion Therapeutics, a combination of its once-a-day treatment and a maximum dose of statin lowered LDL cholesterol 18% more than statins alone after 12 weeks.
The results come from the last of five successful trials on Esperion’s drug, called bempedoic acid. The company plans to submit all of its data to the US Food and Drug Administration in the early months of 2019.
The report says too cardiologists, the most important finding of the latest bempedoic acid trial related to safety. In the 779-patient study, Esperion’s drug was indistinguishable from placebo when it came to side effects and deaths. That’s important because, in an earlier trial involving more than 2,200 subjects, more patients getting bempedoic acid died than those getting placebo. The difference wasn’t big enough to rule out random chance, and none of the deaths was blamed on the drug, but it was enough to stoke concern that the FDA might think twice about approving Esperion’s treatment.
“From my perspective, I think the noise around those imbalances should not be overestimated in importance,” said Dr Steven Nissen, a cardiologist at the Cleveland Clinic who has served as unpaid investigator of bempedoic acid. “I certainly don’t think it’s a regulatory issue at this point.”
The report says Esperion’s drug is meant for patients who have had a cardiovascular event, like a heart attack or a stroke, and who are getting as large a dose of statins as they can handle. The drug is widely expected to win FDA approval and hit the market in 2020, and that’s when the company will find out whether its Goldilocks plan can turn bempedoic acid into a commercial success.
As it stands, the vast majority of at-risk patients get statins, which have long since gone generic and are available for pennies a day for those with insurance. If bad LDL cholesterol stays high, doctors prescribe the now-generic Zetia. And in extreme cases, they reach for injected treatments from Amgen and partners Regeneron Pharmaceuticals and Sanofi, drugs that block a protein called PCSK9.
The report says Esperion, which plans to set a $3,500 list price for bempedoic acid, believes it can play interloper. The list prices of PCSK9 treatments, which currently exceed $14,000 a year, have limited their use in the three years since they hit the market. Esperion’s drug doesn’t lower LDL as well as PCSK9 blockers, which can roughly halve bad cholesterol, but it would be cheaper and wouldn’t require an injection.
But cracking the market may not be so simple. Earlier this year, Sanofi and Regeneron struck a deal with Express Scripts in which they’ll discount their drugs by more than 50 percent in exchange for an easier path to patients. And, last week, Amgen made an unprecedented move to slash the list price of its treatment to $5,850 a year by 2020. According to the report, that could create a difficult market dynamic for Esperion. Zetia prescriptions, like statins, are practically free for patients with insurance, and each has an established effect on preventing heart attacks and strokes in the long term. The PCSK9 treatments have demonstrated dramatic long-term benefits of their own, and their impending price decreases could put them within reach of more patients than ever before.
Bempedoic acid has established that it can reduce LDL cholesterol, but its effects on cardiovascular health won’t be known until a 13,000-patient trial reads out in 2022. That could leave Esperion struggling to make its case in a crowded market.
“I’m still not totally convinced that there’s an obvious place for this drug,” said Dr Ethan Weiss, a cardiologist at the University of California – San Francisco Medical Centre, who wasn’t involved in the study. “We have a good drug with modest efficacy in statins, and then two drugs with significant efficacy in PCSK9. If you’re a marketer, I don’t know where you aim your gun at this one.”
The report says Esperion believes bempedoic acid will have plenty of room to compete. Despite the established benefits of PCSK9 therapy, only about 20,000 people are getting the drugs, Esperion CEO Tim Mayleben said. And that suggests there’s pent-up demand for an alternative.
“There are 13m people in the US that have elevated LDL cholesterol and have had a cardiovascular event and need more cholesterol lowering,” Mayleben said. “The fact that the price of PCSK9s is being lowered, that’s great. But it’s still almost twice the list price of our drug.”Stat News report