Esperion $ESPR would like to have back its rep for owning a cholesterol drug designed to hit a sweet spot in the market — without any unsavory safety issues that could persuade regulators to require an expensive and lengthy cardio outcomes study ahead of a marketing approval.
To that end, CEO Tim Mayleben is rolling out a fresh battery of data from their third pivotal study of bempedoic acid. And they want you to believe as they do: That the consistent ability to cut LDL — in this case by demonstrating a familiar 23% drop, with a 25% lowering in high-sensitivity C-reactive protein — tied in to no new fatalities (whew) and what they insist is a clean safety profile should be widely applauded.
Whether they can achieve their goal in a market that has sharpened its knives for biotech stocks and kept on the lookout for any weaknesses, though, is still open to question. But they’re going to try.
“We’re trying to provide more context than we did earlier this month,” CEO Tim Mayleben told me in a preview of today’s numbers. Mayleben has seen his stock price gyrate before on the way an individual data point is interpreted, but now he has reported data on 4,000 patients all together — 1,500 who have been on the drug for more than a year.
“That’s what regulators will look at, the pooled data,” says the CEO. “When you look at it overall, you see a very balanced safety and tolerability profile.” And he still isn’t worried about the prospect of head-to-head competition with the PCSK9s.
Part of that pooled data that will get close scrutiny today is a fatality rate of 0.5 percent in the drug arm and 0.2 percent for the placebo group.
“I would say that with respect to pricing, one of the things we have seen constantly is confusion, misunderstandings about the net price,” the CEO adds. Once you factor in the usual discounts expected in this market, he says, you can expect a net price of less than half the $5,000 (or so) that the PCSK9s will go for. And for millions of patients who are not controlled on statins and don’t need a dramatic cut in LDL, he believes he’ll have a clearly defined market offering a big advantage to payers.
The team at Esperion has good reason to push back after their stock was pummeled earlier this month in the aftermath of its last Phase III readout, when analysts and investors recoiled from an imbalance of deaths in the study and asked some sharply pointed questions about the future of a drug that has a projected price just under what Regeneron and Sanofi recently set as their low mark for an approved PCSK9 rival that everyone agrees is much, much better at lowering LDL, with rivals on the market and in the pipeline that could do just as well — or better.
That prospect of lowering prices on the PCSK9 class could crush a player like Esperion, a sentiment that helped trigger a 30% landslide in the share price.
Despite the absence of deaths, the bempedoic acid arm did see a 6% rate of serious adverse events, compared to 3.6% for the placebo group. Discontinuations were 18.4% for the drug arm versus 11.7% for the placebo — but the study investigator ruled out any connection between the drug and the SAEs.
Ulrich Laufs, a member of Esperion’s Phase III executive committee and director of the department of cardiology at Leipzig University, called the data “amazingly consistent” and added that “the medical community is in need of a new oral therapy which is effective, well tolerated and convenient for this complex patient population who may have run out of other options.”
Esperion has two more Phase III readouts, this summer and then in September. No later than Q1, says Mayleben, they’ll have the NDA complete. And then regulators can have the last say on efficacy and safety.
In the meantime, the investors are still seeing more red than green in the updates from Esperion. The stock was down 3% by mid-day Wednesday.
Image: Tim Mayleben
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