- Esperion Therapeutics already planned to file its cholesterol-lowering drug for approval in a couple years, but a newly announced, late-stage trial now also makes it possible for a second treatment from the clinical-stage company to hit regulators' desks around that timeframe.
- The Phase 3 study will assess that treatment — a combination of ezetimibe, the active ingredient in Merck's Zetia, with Esperion's bempedoic acid — in patients with high cholesterol, including those who are at increased risk from other disease or genetic factors. The smaller drugmaker expects the investigation to kick off during the fourth quarter and conclude by the end of 2018. If all goes well, U.S. and European regulatory submissions for the pairing would come in the first half of 2019.
- That timeline closely mirrors the one set for bempedoic acid monotherapy, which is being tested across a trio of trials. Esperion bullishly predicts the franchise surrounding its drug could reach "mega-blockbuster status" in cumulative sales, if it wins approval.
Pharmaceutical companies haven't had an easy time getting their newer cholesterol treatments to unseat statins. The PCSK9 drug class, which encompasses Amgen's Repatha (evolocumab) and Sanofi and Regeneron's Praluent (alirocumab), is trying, but faces formidable resistance from payers over the biologics' hefty cost.
Esperion is confident, however, that its treatments won't be stonewalled in the same way. For one, bempedoic acid is similar to statins in that it prevents cholesterol production, rather than PCSK9 medicines which degrade low-density lipoprotein (LDL) cholesterol, commonly known as the "bad" cholesterol. Regarding the combination therapy, ezetimibe has been on markets for years and garnered strong revenues.
Sales of Merck's Zetia and Vytorin (ezetimibe and simvastatin), for instance, hit $3.70 billion in 2016, and Esperion estimates the franchise raked in more than $50 billion over the course of its life.
Generic ezetimibe launched last December, and Vytorin lost patent protection in April. While that's bad news for Merck's bottom lines, Esperion executives noted in a June 26 investor call that the precedent set by the drugs will benefit uptake for its own products.
"We're not predicting that the combo bempedoic acid franchise is going to generate over $50 billion in cumulative revenue," Marianne Andreach, Esperion's head of strategic marketing and product planning, said during the call.
"We're in a much different environment today, with more generics and certainly more access challenges. However, I want you to have the perspective today that the potential is there for the combo bempedoic acid franchise to achieve mega blockbuster status," she said.
Bempedoic acid and the combination therapy target patients who receive maximally tolerated background statin therapy, which Esperion's CEO Tim Mayleben estimated is about 8.5 million people in both the U.S. and Europe. The new late-stage study of the combo will enroll about 350 patients with hypercholesterolemia as well as those who are at high risk of hardening of the arteries or those that have a genetic risk for an ultra high cholesterol disease known as heterozygous familial hypercholesterolemia (HeFH).
Touting their candidates' similar efficacy profiles to statins, as well as a once-daily, oral administration, Esperion executives said they also have faith insurers will look fondly on the franchise.
"Payers are influenced by professional societies, and the much-discussed LDL cholesterol guidelines currently in development are expected to drive LDL cholesterol goals even lower in the future, further increasing the demand for new, highly effective, convenient, cost-effective therapies," Andreach said.
"Payers have previously supported branded ezetimibe and we expect that they will continue to support generic ezetimibe even more, providing the combo with an on-ramp to readily identifiable, accessible and rapidly growing patient segments."