Hearts & Minds Blog
Important Bempedoic Acid-Based Franchise Progress UpdatesAugust 19, 2017
Last week was exciting for us at Esperion as we reported on tremendous progress achieved in enrolling patients for our bempedoic acid global pivotal Phase 3 program, as well as highly positive Phase 2 results from the combo plus statin study. The Lipid Management Team has been “heads down” making critical advancements for the bempedoic acid franchise both in the clinic and in conversations with regulators that will undoubtedly propel us to a successful second half of the year.
First, as you may recall, we made significant progress on the regulatory front with the FDA’s confirmation of the abbreviated 505(b)(2) regulatory pathway to approval for the bempedoic acid / ezetimibe combination pill for an LDL-cholesterol lowering indication. The complementary, non-statin combo pill lowers LDL-cholesterol by almost 50%, significantly reduces a key marker of inflammation associated with cardiovascular disease known as high-sensitivity C-reactive protein (hsCRP), while potentially lowering the occurrence of muscle-related side effects.
Second – and perhaps most exciting – we just announced remarkably positive top-line results from the Phase 2 study 1002-038 evaluating the bempedoic acid / ezetimibe combination pill plus atorvastatin, which demonstrated:
- Robust LDL-cholesterol lowering of 64%, which was consistent across patients treated with a rapid onset of LDL-cholesterol lowering as early as three weeks and with over 90% of patients achieving 50% or greater LDL-cholesterol lowering or a 70 mg/dL LDL-cholesterol level;
- Clinically relevant hsCRP reduction of 48%; and
- Observed to be safe and well-tolerated
These results further validate the power of once-daily, oral bempedoic acid-based products in combination with maximally-tolerated statin therapy to achieve optimal LDL-cholesterol lowering for patients with hypercholesterolemia. Importantly, these data emphasize bempedoic acid’s potential as the only therapy, other than statins, to consistently show significant reductions in both LDL-cholesterol and hsCRP, an important marker of underlying inflammation associated with cardiovascular disease.
The outcome of this study is valuable in helping to deepen the understanding for the potential of our lead product – the once-daily, oral bempedoic acid / ezetimibe combination pill – so that we can confidently design, initiate and complete additional studies to round out the profile of our oral combination therapies. Plans for additional non-registrational studies will be announced by Q1 2018 and will continue to explore the market dynamics of combinations of convenient, cost-effective, once-daily, oral LDL-cholesterol lowering therapies. We expect these studies to provide physicians with a deeper understanding of how best to treat their patients with the power of oral combination therapies with maximally-tolerated statin therapy, while achieving optimal tolerability that has been so elusive with high-intensity statin doses. It is also expected that payers will appreciate the cost-effectiveness of additional oral combinations with robust LDL-cholesterol potential in an economically constrained healthcare environment.
And finally, the team has made tremendous progress toward the enrollment of patients in our global pivotal Phase 3 LDL-cholesterol lowering efficacy studies. Not only are we are on track to complete patient enrollment in the third quarter of this year, but we are eager to report top-line results from the entire Phase 3 program in the second and third quarters of next year. Our team’s efforts have put us in a position to accelerate the timing for submission of our New Drug Applications (NDAs) for both the bempedoic acid / ezetimibe combination pill and bempedoic acid, and we now expect to submit the NDAs by the first quarter of 2019 – that is three months earlier than we originally guided, and I could not be more pleased with this outcome!
We’re energized by all this exciting news, and by the important progress our Lipid Management Team has made in recent months. We look forward to continuing to accelerate the development of our bempedoic acid franchise to potentially address the needs of patients who are living with elevated LDL-C despite taking maximally tolerated statin therapy. Our goal is to demonstrate the value of our franchise to physicians and payers who are seeking the flexibility to utilize multiple cost-effective and convenient treatment options to extend the time before patients need to move to expensive, injectable LDL-cholesterol lowering therapies.
Next up: we are heading to Barcelona, Spain next week for the European Society of Cardiology Congress (ESC). We look forward to hearing more about updates in the field, including the full results from REVEAL and CANTOS. Stay tuned for updates from us on key takeaways from ESC, the largest gathering of cardiovascular health experts in the world!