Hearts & Minds Blog
Resilient and Relentlessly Focused in 2017January 19, 2017
Happy New Year!
We welcomed 2017 with tremendous momentum as we advanced our bempedoic acid (ETC-1002) pivotal Phase 3 LDL cholesterol lowering development program – including steadily reducing the overall program risk. The Esperion team is relentless and resilient, and we remain focused on patients and their physicians waiting on new, non-statin, complementary, cost-effective, convenient, oral, once-daily LDL-C lowering therapies. They provide us the motivation to do what we do best.
First, I want to start with an update on one such patient: my wife, Dawn. As I wrote in August [Raison d’etre], Dawn has very high LDL cholesterol – 193 mg/dL – and is unable to tolerate statins despite her valiant efforts to do so. She’s tried all four of the most commonly prescribed statins, and experienced severe muscle pain and weakness within days of starting each drug. Dawn worked with several different doctors this year, including two cardiologists and two internists, and in November, finally met with a lipidologist.
After extensive review and testing, Dawn was diagnosed with a genetic disorder – she is one of approximately 600,000 people in the U.S. that have heterozygous familial hypercholesterolemia (HeFH) – resulting in genetically (and dangerously) high LDL cholesterol levels. HeFH leaves her at much greater risk for cardiovascular disease (CVD). Recall that she had acute heart problems in July – and cardiovascular disease runs in her family. This family history became even more evident in November 2016 when Dawn’s dad had two heart attacks in a matter of days and required a stent in his main coronary artery.
Understanding Dawn’s increased CVD risk, her lipidologist prescribed one of the new injectable PCSK9i therapies. After going through the extensive prior-authorization process with our insurance company, Dawn’s prescription was approved for an initial three-month period. We received her prescription via overnight delivery and after reading the comprehensive instruction sheet, I injected Dawn on a Saturday morning. The great news is that Dawn’s LDL cholesterol levels dropped by 60% within days! However, during this same period Dawn experienced flu-like symptoms and the now-familiar muscle pain and weakness. This went on for several more days. Nevertheless, two weeks later Dawn asked me to inject her again – she was willing to try it one more time. Within days the flu-like symptoms and the muscle pain and weakness returned and lasted for several agonizing days. This was very disappointing to Dawn, to me and to her physician.
Dawn wants to keep her LDL cholesterol level down, especially after what her dad recently experienced, but she wants to live a “normal” life, too. The medicine that reduces her LDL cholesterol is great at doing that – but the “cost” to Dawn is not being able to live a normal life. Her life is now filled with daily pain, spates of depression and lethargy. She did not take another injection during the month of December and we find ourselves once again looking for alternatives as her LDL cholesterol levels – and cardiovascular disease risk – creep back up. Stay tuned, as we remain hopeful and Dawn remains strong on her journey to find an effective – and tolerable – therapy. I’ll update you again on her progress later in the year when we know more.
Now, I want to turn to anticipated highlights you can expect from the Esperion team throughout 2017. We expect this to be a rewarding and transformational year for Esperion and the development of bempedoic acid. Throughout 2017 we expect to:
- Complete enrollment in a few weeks in our global pivotal Phase 3 long-term safety study, and later this year complete enrollment in our recently-initiated three global pivotal Phase 3 LDL cholesterol lowering efficacy studies totaling more than 3, 200 patients;
- Begin enrolling patients this month who completed the Phase 3 long-term safety study into an open-label extension study, which will further expand our robust patient safety database;
- Continue to rapidly enroll patients considered “statin intolerant” into our recently-initiated CLEAR Outcomes cardiovascular outcomes trial;
- Begin to prepare submissions for an LDL cholesterol lowering indication filing, targeted for 1H 2019;
- Initiate and report top-line results from a Phase 2 “Triplet Oral Therapy” study combining 180 mg of bempedoic acid + 10 mg of ezetimibe + 20 mg of atorvastatin, and;
- Advance our potent non-statin, convenient, complementary, cost-effective, oral, once-daily combination pill – BA+EZ (180 mg of bempedoic acid + 10 mg of ezetimibe) – into a global pivotal Phase 3 program following an agreement with regulators on the strategy and timing for development.
Thank you for your continued interest in Esperion and bempedoic acid clinical development. I look forward to providing updates on our progress throughout 2017!