Hearts & Minds Blog
Going with Your GutAugust 30, 2013
Sometimes in science, you have to go with your gut. Years ago, my gut instinct told me that a drug I was working on would be successful, even though it was still early in development and several effective competitors were already on the market. I believed in this drug so much that I begged the company multiple times not to discontinue the program, and pleaded with them for the necessary funds to continue development. I wrote memos to the business people at Warner-Lambert saying that if they licensed this drug away, they would regret it for the rest of their lives.
I put my credibility on the line–and I turned out to be right. The drug that I worked so hard to save eventually became Lipitor (atorvastatin calcium)–the world’s best-selling statin and best-selling drug, which has helped millions of people lower their low density lipoprotein cholesterol (LDL-C) levels.
I’m not the kind of person who is happy to sit back and relax. There are always new medical problems to solve, especially with cardiovascular disease. That’s why I’m back leading a team at the new Esperion to develop a new therapy for the millions of people with elevated LDL-C (also known as hypercholesterolemia) who aren’t able to tolerate statins.
Our efforts are focused on a compound called ETC-1002, which is a first-in-class therapy designed to significantly reduce levels of high LDL-C and improve other cardiometabolic risk factors without many of the side effects associated with statin use. ETC-1002 works by inhibiting one enzyme and activating another that the body uses to synthesize cholesterol in the liver. By influencing these enzymes, ETC-1002 has the potential to not only lower LDL-C, but also reduce blood pressure, blood glucose, inflammation and weight gain. ETC-1002 also doesn’t appear to accumulate in the blood and muscles, as statins can in some people, causing them pain and weakness.
Having a promising therapy in development doesn’t mean that you’ll be successful in the marketplace. At times I feel like Sisyphus, the mythological Greek character who is forced to repeatedly push a boulder up a hill, only to have it roll down to the bottom again. Just as I had to fight to keep Lipitor on track, our team has had to overcome several hurdles to push forward the development of ETC-1002.
The biggest challenge was getting back the rights to ETC-1002 from Pfizer, which acquired the first Esperion in 2004 in part because of the promise our HDL therapies had shown in early clinical studies. However, a few years later, Pfizer decided to shift its research and development efforts away from cardiovascular disease, and, as a result, Esperion’s programs were no longer relevant.
In 2007, I made the decision to try to re-start Esperion–and to re-acquire the Esperion name and, most importantly, the rights to ETC-1002 and another program. I believed very strongly in the potential of ETC-1002 even though it was still very early in development. After nearly a year of negotiations, I successfully acquired ETC-1002 and the Esperion name, and several of the original Esperion colleagues are back working with me. I can’t express how excited I am to be working with them and the rest of the team to develop a therapy that could potentially help millions of people lower their LDL-C and reduce their risk of cardiovascular disease–still the nation’s (and the world’s) number one killer!
Right now, Esperion is the only company in mid-stage clinical development with a once-daily, oral small molecule to lower LDL-C levels in individuals who can’t tolerate statins. While I can’t predict the future, my gut–and our clinical research to date–tells me that ETC-1002 will be successful in lowering LDL-C in patients with hypercholesterolemia–not only for those who can’t tolerate statins, but also for those who are considered to be statin resistant. These are individuals who need additional LDL-C lowering to achieve their LDL-C goal despite already being on a statin.
What we are doing isn’t easy. While I could have retired several years ago, I like challenges, and more importantly, I like to finish what I have started. I also love what I do and have spent my entire career working to help save lives. I’m not ready to give up on that goal. We still have a lot of work to do. That boulder isn’t over the hill just yet!