Esperion $ESPR is rolling out its first positive data set from a slate of pivotal late-stage studies of its LDL-lowering drug bempedoic acid this morning — one of the most closely followed programs in the business.
Facing a skeptical audience of payers and rivals — which have already shown they can do much, much better with the same target — Esperion posted a 28% spread against a placebo, with a 23% drop in LDL-c for the drug plus ezetimibe against a 5% increase for patients in the placebo group.
Investigators also tracked another plus for statin intolerant patients, with a 32% drop in high-sensitivity C-reactive protein, a marker for damaging inflammation which can have serious longterm consequences for cardio health.
“In both cases,” CEO Tim Mayleben tells me as he set the stage for today’s announcement, “this study exceeded expectations.”
But is that enough?
It’s all statistically significant, giving supportive analysts the data they were looking for to celebrate the first of 5 Phase III studies due out by the fall, setting up a marketing pitch to the FDA in Q1 2019. Based on the data, there are good reasons to believe that the biotech can come through with more positive efficacy data and a clean bill of health on liver tox.
But there’s a lot more involved here than demonstrating that a new drug can beat out a placebo. That’s been done before. Esperion has to convince everyone that its new LDL lowering pill can succeed where other drugs — injectables — with much better results have fared poorly thus far. In the cardio field, as Merck demonstrated with its recent decision to jettison a CETP therapy in the wake of a win against a placebo, positive data is no guarantee of success. It also has to be commercially viable, able to convince the market that this drug can break into blockbuster territory.
Bempedoic acid in different combinations has also scored higher in mid-stage studies. But Esperion designed this first, critical study to add their drug after a round of ezetimibe, mimicking the real world environment where doctors will be looking to follow up on statin resistance after first trying a generic at a very, very low cost.
Mayleben has already broken the cardinal rule of drug development, naming a price of about $9 to $10 a day — or in the $3,500 per year range — well ahead of a launch.
That just doesn’t happen in biopharma, where the virtual law of drug marketing requires that the price be kept under lockdown until after a marketing OK comes through for a new drug — especially if developers know perfectly well what they will sell it for.
The Esperion CEO, though, can’t play by those rules. He has to steer a course around PCSK9 drugs from Regeneron/Sanofi and Amgen, which easily outscore his therapy on lowering LDL. Mayleben’s target will be a wide range of patients who need to see a big drop in LDL, but don’t necessarily require the axe PCSK9 brings to the table — and who don’t want any part of the $14,000 annual price tag attached that has spurred payers to throw up a complex set of hurdles to prevent widespread use.
That requires a willingness to upset some apple carts along the way. Sticking with the industry standard here won’t help the roll out he’s planning, with some analysts setting peak sales estimates deep into blockbuster terrain.
“Why should this not be the way things are?” asks the CEO.
He cited the outcry that met Gilead when they priced their pioneering hep C cure at $84,000 as an example of what can go wrong.
“The issue was not just the price, but it was above expectations,” he notes. Flagging the price and starting that discussion well ahead of a launch just makes sense.
Mayleben adds: “I don’t think I realized that we were setting any sort of precedent with what we were doing.”
Esperion, though, doesn’t have the luxury of a breakthrough drug, in the same way that the PCSK9s or Sovaldi were able to enjoy. To compete will take some disruptive rule breaking.
And Mayleben plans to do just that.
There are 4 more pivotal studies to come. And Esperion is bringing on a chief commercial officer soon to start laying the foundation for a commercial wing. At some point, Mayleben isn’t saying when, the company also wants to bring in commercial partners to help roll out a mass market effort in early 2020. And then they’ll still have to wait for cardio outcomes data in 2022.
The 2018 data season has begun for Esperion, and there’s virtually no wiggle room in the course the biotech has to steer now.
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