With FDA Hold Lifted, Esperion Cholesterol Drug Heads to Phase 3

In 1998, Xconomist Roger Newton co-founded a company called Esperion Therapeutics and drove the development of atorvastatin (Lipitor), a blockbuster cholesterol-lowering statin drug that is reportedly the most widely prescribed medicine in the world.

After ushering in the age of statins, Newton eventually sold Esperion to Pfizer for $1.3 billion in 2008. The deal represented one of the most successful startup exits in Michigan’s history.

Newton hung on to some of the original intellectual property used to create atorvastatin, so he decided to reboot Esperion (NASDAQ: ESPR) in 2008 to continue his work developing cholesterol drugs. With the announcement last week that the FDA will let clinical trials go forward, Esperion plans to soon begin Phase 3 trials of ETC-1002, its lead drug candidate—and Newton may be one step closer to getting a second, groundbreaking therapy to market.

ETC-1002, says Esperion CEO Tim Mayleben, was discovered by the original Esperion team. The once-a-day pill lowers LDL cholesterol without the muscle-weakening side effects that atorvastatin has on about 10 percent of patients, he says. The FDA placed on a partial clinical hold on the drug in 2009, requiring that carcinogenicity studies be completed before beginning a clinical trial longer than six months.

After completing two-year carcinogenicity studies in mid-2014, Esperion turned in its data to the FDA in early January and the federal agency removed the hold by month’s end. The company says it’s not uncommon for small molecule drugs to have to undergo carcinogenicity studies as part of its journey to marketplace.

Mayleben says the company had always targeted 2015 to begin the Phase 3 trials, and that will happen as planned, most likely in the fourth quarter.

Should the trial prove that ETC-1002 is effective and safe, it could provide a new option for patients for whom the side effects of statins are too great. Mayleben estimates there are 3 million such patients in the U.S. alone, representing a multi-billion-dollar market. “The irony is that Roger helped develop the most successful statin, and now he’s trying to help the patients who can’t tolerate it,” Mayleben says.  If atorvastatin was lightning in a bottle, Mayleben says, ETC-1002 is “double lightning in a bottle—it’s a huge market opportunity.”

Esperion has almost finished a Phase 2b study of ETC-1002, and is currently testing what happens when the drug is given to patients who are already on a low-dose statin. “We think there are a lot of patients like that out there, and we want to see how our treatment works with the typical protocol,” he adds. It’s expected that Esperion will have data from Phase 2b to report over the summer. The company plans to present the complete results of its studies of ETC-1002 so far at the American College of Cardiology conference in San Diego on March 14.

Mayleben says that the last year or so has also brought the band back together, so to speak: two-thirds of Esperion’s employees also worked on the atorvastatin project. “From the top of the organization to the bottom, most of the company and the entire development team have worked together previously,” he says. “The team is very experienced in this area of therapeutics.”

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