Study 1 (1002-040) Pivotal Phase 3 Long-Term Safety Study Top-Line Results May 2, 2018 1 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Safe Harbor Forward-Looking Statements These slides and the accompanying oral presentation contain forward-looking statements and information. The use of words such as may, might, will, should, expect, plan, anticipate, believe, estimate, project, intend, future, potential, or continue, and other similar expressions are intended to identify forward looking statements. For example, all statements we make regarding the regulatory approval pathway for the bempedoic acid / ezetimibe combination pill and bempedoic acid and the therapeutic potential of, clinical development plan for, the bempedoic acid / ezetimibe combination pill and bempedoic acid, including Esperion's timing, designs, plans and announcement of results regarding its global pivotal Phase 3 clinical development program for bempedoic acid and the bempedoic acid / ezetimibe combination pill, Esperion's timing and plans for submission of NDAs to the FDA and MAAs to the EMA and Esperion's expectations for the market for therapies to lower LDL-C, including the market adoption of bempedoic acid and the bempedoic acid / ezetimibe combination pill, if approved, are forward looking. All forward-looking statements are based on estimates and assumptions by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that we expected. These statements are also subject to a number of material risks and uncertainties, including but not limited to, delays or failures in Esperion s studies, that positive results from a clinical study of bempedoic acid may not be sufficient for FDA approval or necessarily be predictive of the results of future or ongoing clinical studies, that existing cash resources may be used more quickly than anticipated, and the risks detailed in Esperion's filings with the Securities and Exchange Commission. Any forward-looking statement speaks only as of the date on which it was made. Esperion disclaims any obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. 2 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Bempedoic Acid Franchise Development Program Updates May 2, 2018 3 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
The Lipid Management Team: Addressing a Global Problem Cardiovascular Disease is the #1 Cause of Death Globally Esperion is focused on developing LDL-C lowering therapies to address the unmet needs of patients with atherosclerotic cardiovascular disease (ASCVD), or who are at a high risk for ASCVD, with hypercholesterolemia who continue to have elevated levels of LDL-C despite the use of maximally-tolerated statins and ezetimibe Patients Not Adequately Treated with Currently Available Therapies Phase 3 Study TOTAL STATIN ADD-ON (ASCVD & HeFH) Study 1 & 2 8.6 million 8.4 million 4 6 million 21 23 million ASCVD, HeFH & High Risk for CVD (NO STATIN BACKGROUND) Study 3 & 4 3.5 million 3.3 million 0.8 million 7.6 million TOTAL 12.1 million 11.7 million 4.8 6.8 million 28.6 30.6 million Source: AHA Heart Disease, Stroke and Research Statistics At-A-Glance 2016 4 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Number of Patients in Need of Additional LDL-C Lowering* 12-13M ASCVD and/or HeFH Patients with Elevated LDL-C Bempedoic Acid Franchise Addresses Most Patients Not at LDL-C Treatment Goal 13M 12M 11M 10M 9M 9.5 Million 84% (8M) of these patients will get below 70mg/dL with BA or BA+EZ Patients achieving goals on either BA or BA+EZ Bempedoic acid patients not at goal PCSK9i 8M 7M 100 130 mg/dl 6M 5M 4M 3M 2M 1M 70 100 mg/dl 1.8 Million 92% (1.7M) of these patients will get below 100mg/dL with BA or BA+EZ 1.4 Million 14% (200k) of these patients will get below 100mg/dL with BA+EZ Maximally Tolerated Statin Therapy** +/- Ezetimibe Moderate Risk (70 130 mg/dl) High Risk (130 160 mg/dl) Very High Risk (>160 mg/dl) *Excludes Low CVD Risk patients because, by definition, they do not need additional LDL-C lowering **Includes patients only able to tolerate less than the approved daily starting dose of a statin (considered statin intolerant) 5 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Bempedoic Acid and Bempedoic Acid / Ezetimibe Combination Pill to Address Factors Increasingly Critical in LDL-C Treatment Choices Updated treatment goals from ACC/AHA by Q1 2019 will result in more patients eligible for LDL-C lowering therapy Updated ACC/AHA goals increase demand for complementary, accessible, cost-effective, oral LDL-C lowering therapies to help patients achieve the new treatment goals Payers drawn to cost-effective, convenient, once-daily, oral LDL-C lowering therapies BEMPEDOIC ACID Clinically and Statistically Significant LDL-C Lowering and Reductions in hscrp, Observed to be Safe and Well-Tolerated Targeted Mechanism of Action Complementary and Convenient Oncedaily, Oral Treatment Option Increasing utilization of generic ezetimibe will drive combination pill adoption 6 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Key Upcoming Milestones Results from Three Remaining Phase 3 Studies Expected This Year; NDA Submissions No Later Than Q1 2019 March Study 4 (1002-048) BA Ph 3 TLRs August 1002FDC-053 BA / EZ Combo Pill Ph 3 TLRs March 2018 Phase 2 BA PCSK9 Add-on TLRs Q1 2018 Announce Combination + Statin Future Development Plans 2018 May Study 1 (1002-040) BA Ph 3 TLRs September Study 2 (1002-047) BA Ph 3 TLRs Q1 2019 LDL-C Lowering Indication NDA Submissions Q2 2019 LDL-C Lowering Indication MAA Submissions 1H 2020 LDL-C Lowering Anticipated Commercial Launch 2022 CLEAR Outcomes CVOT TLRs 2018 Study 3 (1002-046) BA Ph 3 TLRs 2019 2020 2022 7 Copyright 2018 Esperion. All Rights Reserved Do Not Copy Or Distribute
Bempedoic Acid Phase 3 Program for LDL-C Lowering Pivotal Phase 3 Top-line Results Began Reporting in March LDL-C Lowering Indication (Total N~=4,000): Comparable in Design and Scale to PCSK9i Programs Study 1; Long-Term, N=2,230 TLRs Announced May 2 52 weeks safety / 12 weeks LDL-C ASCVD and/or HeFH Statin Add-On OLE N=1,462 (Fully Enrolled) 1.5 yrs safety Study 2; High Risk, N=779 TLRs Expected in September 12 weeks LDL-C / 52 weeks safety ASCVD and/or HeFH/1 0 ; Low, Very Low or No Statin Background Therapy* Study 3; SI, N=345 TLRs Expected in May Study 4; SI, +EZ, N=269 TLRs announced March 7 12 weeks LDL-C / 12 weeks safety 12 weeks LDL-C / 24 weeks safety ASCVD and/or HeFH/1 0 ; FDC Added to Statin Study 053; N=382 (Fully Enrolled) TLRs Expected in August 12 weeks LDL-C / 12 weeks safety *Studies are being conducted to obtain an indication for use in patients on no background statin therapy in Europe 8 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Clinical Development and Regulatory Strategy Bempedoic Acid / Ezetimibe Combination Pill and Bempedoic Acid Global Clinical Development Programs to Support Target Label(s) Bempedoic Acid / Ezetimibe Combo Pill LDL-C Lowering NDA Submission (No later than Q1 2019) Bempedoic Acid LDL-C Lowering NDA Submission (No later than Q1 2019) CV RR Submission (2022) LDL-C Lowering Program Initial LDL-C Lowering label in U.S. and Europe (like pre-cvot PCSK9i labels in 2015) CLEAR Outcomes CVOT CV Risk Reduction Label in U.S. and Europe (Note: breadth of LDL-C lowering label and CV RR label will broaden similar to post-cvot PCSK9i CV RR label 2017) 9 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Study 1 (1002-040) Phase 3 Long-Term Safety Study Background Statins and ezetimibe are the standard of care for the treatment of patients with hypercholesterolemia Approximately 90% of patients on LDL-C lowering therapy are taking a low- (12%), moderate- (63%) or high-intensity (25%) statin* The goal of Study 1 was to evaluate the long-term safety and tolerability of bempedoic acid added-on to maximally tolerated statin therapy in high-risk patients with hypercholesterolemia Older, high-risk patients with advanced cardiovascular disease ASCVD and/or HeFH Patients on maximally tolerated statin therapy, including 50% on high-intensity statins and 95% on moderate- or high-intensity statins 52-week study duration the gold standard to evaluate the safety and tolerability of a chronic therapy * Symphony Health Solutions, 2017 Data 10 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Study 1 (1002-040) Pivotal Phase 3 Long-Term Safety Study Top-Line Results May 2, 2018
Study 1 (1002-040) Long-Term Safety Study Study Design 2230 patients at high CV risk (ASCVD and/or HeFH) with LDL-C 70 mg/dl on stable background lipid-modifying therapy, including maximally tolerated statin therapy Bempedoic acid 180 mg (n=1488) Placebo (n=742) 52-Week Treatment* *Following the 52-week treatment period, patients enrolled in the OLE (N=1,462) or were followed for an additional 30 days for safety (N=768) Primary objective: evaluate the long-term safety and tolerability of bempedoic acid over 52 weeks Overview of adverse events, serious adverse events, discontinuations due to adverse events, fatal adverse events, muscle-related adverse events and safety labs (LFTs) Secondary objective (primary efficacy): change in LDL-C at week 12 12 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Study 1 (1002-040) Long-Term Safety Study Demographics & Baseline Characteristics Mean ± SD a Mean ± SD a Demographics Gender: % Male (M/F) Bempedoic Acid N=1,488 Placebo N=742 Age: years 65.8 ± 9.1 66.8 ± 8.6 73.9% (1099/389) 71.3% (529/213) Race, White: % (N) 95.6% (1423) 96.5% (716) Ethnicity, Hispanic or Latino: % (N) 1.6% (24) 1.5% (11) Baseline Characteristics BMI: kg/m 2 29.7 ± 4.9 29.4 ± 4.9 History of ASCVD Only: % (N) 95.1% (1415) 95.3% (707) HeFH (with or without ASCVD): % (N) 4.9% (73) 4.7% (35) Diabetes: % (N) 28.6% (425) 28.6% (212) Hypertension: % (N) 78.9% (1174) 80.1% (594) LDL-C (mg/dl) Bempedoic Acid N=1,488 Placebo N=742 LDL-C: mg/dl 103.6 ± 29.1 102.3 ± 30.0 Lipid Parameters and Risk Markers non-hdl-c: mg/dl 130.9 ± 33.7 129.4 ± 33.9 Total Cholesterol: mg/dl 179.7 ± 35.1 178.6 ± 35.6 apob: mg/dl 88.5 ± 21.6 86.8 ± 21.8 hscrp: mg/l b 1.49 1.51 HDL-C: mg/dl 48.7 ± 11.9 49.3 ± 11.5 Triglycerides: mg/dl 141.8 ± 67.3 139.6 ± 64.1 Baseline Statin Intensity % (N) Low 6.7% (100) 6.5% (48) Moderate 43.4% (646) 43.7% (324) High 49.9% (742) 49.9% (370) a Unless otherwise indicated b Median Values Full Analysis Set Population 13 Copyright 2018 Esperion. All Rights Reserved Do Not Copy Or Distribute
Study 1 (1002-040) Long-Term Safety Study Safety & Tolerability Overview of Adverse Events and Discontinuations Treatment Emergent Adverse Events (AEs) Overview of AEs in All Patients (patient incidence) Bempedoic Acid N=1,487 % (Number) of Patients Placebo N=742 Any AE(s) 78.5% (1167) 78.7% (584) Serious AE(s) 14.5% (216) 14.0% (104) Discontinuation due to AE(s) 10.9% (162) 7.1% (53) Fatal Adverse Events Unrelated to Study Treatment 0.9% (13) 0.3% (2) Safety Analysis Set Population 14 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Study 1 (1002-040) Long-Term Safety Study Safety & Tolerability Overview of Muscle-Related Adverse Events Note: All patients on background statin therapy with ~95% on moderate- or high-intensity statins Potential Muscle AEs* Overview of Potential Muscle AEs in All Patients (patient incidence) Bempedoic Acid N=1,487 % (Number) of Patients Placebo N=742 Any Potential Muscle AE(s)* 13.1% (195) 10.1% (75) Discontinuation of Study Medication due to Potential Muscle AEs* 2.2% (32) 1.9% (14) All Potential Muscle AEs by MedDRA Preferred Term* Myalgia 6.0% (89) 6.1% (45) Muscle spasms 4.2% (62) 2.7% (20) Pain in extremity 3.4% (50) 2.2% (16) Muscular weakness 0.6% (9) 0.5% (4) Safety Analysis Set Population *Includes all muscular disorders defined in the SAP as: muscle spasms, myalgia, muscular weakness, myoglobin blood increased, myoglobin blood present, myoglobin urine present, myoglobinaemia, myoglobinuria, myopathy, myopathy toxic, necrotizing myositis, pain in extremity, rhabdomyolysis 15 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Bempedoic Acid Integrated Safety LFT Elevations are Rare Overview of Liver Function Tests (AST/ALT) - % (number) of Patients LFT Increases (Repeated and Confirmed) Phase 3 Study 1 (040) Completed Integrated Data 1 Total Ongoing Phase 3 Program 3 Bempedoic Acid N=1,487 Placebo 2 N=742 Bempedoic Acid N=2,434 Placebo 2 N=1,227 Bempedoic Acid N=2,423 Placebo 2 N=1,119 ALT/AST > 3 x ULN 0.54% (8*) 0.13% (1) 0.58% (14*) 0.08% (1) ~0.62% (15*) 0.09% (1) 1 Completed Phase 2/Phase 3 data includes 1002-003, -005, -006, -007, -008, -009, -014, -035, -038, -039, -048 (Study 4) and -040 (Study 1) 2 Placebo treatment group includes patients with no background therapy; low, moderate, or high intensity statins; and/or ezetimibe; or PCSK9i 3 Based upon blinded data review as of March 2018 for Study 3 and Study 2 subject to variability until the end of the studies and as data are cleaned; assumes all elevations in bempedoic acid-treated patients for Studies 2 and 3; includes final data from Study 1 and Study 4 *Two patients from -040 who rolled over in to the -050 (OLE Study) who reported an initial LFT increase were found not to meet the definition of repeated and confirmed LFT elevations. Incorporating these data, the rate of LFT increases for -040 would be 0.40%; Completed Integrated Data would be 0.49%; Total Ongoing Phase 3 Program would be 0.54%. Overview of Liver Function Tests for Statins and Ezetimibe 4 (AST/ALT) Dose Atorvastatin Rosuvastatin Simvastatin Ezetimibe Vytorin Atorva Placebo Rosuva Placebo Simva Placebo Eze Placebo Vytorin Placebo 10mg 0.2% - 1.1% 0.5% - - 0.5% 0.3% 1.7% - 20mg 0.2% - 1.1% 0.5% - - N/A N/A 1.7% - 40mg 0.6% - 1.1% 0.5% 0.9% - N/A N/A 1.7% - 80mg 2.3% - N/A N/A 2.1% - N/A N/A 2.6% - 4 Data collected from FDA approved package inserts for each drug. Note that all reported Liver Function Test increases occurred within 12 weeks of initiating therapy. 16 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Study 1 (1002-040) Long-Term Safety Study Safety & Tolerability Overview of Adverse Events Leading to Discontinuation of Study Medication > 1% Incidence Treatment Emergent AEs Overview of AEs in All Patients (patient incidence) % (Number) of Patients Bempedoic Acid N=1,487 Placebo N=742 Discontinuation of Study Medication due to AE(s) 10.9% (162) 7.1% (53) AEs Leading to Discontinuation of Study Medication by System Organ Class Musculoskeletal and connective tissue disorders 2.8% (42) 2.3% (17) Gastrointestinal disorders 1.7% (26) 0.7% (5) Changes in lab values and clinical observations* 1.2% (18) 0.3% (2) Nervous system disorders 1.1% (17) 1.8% (13) *Labeled as Investigations in MedDRA Safety Analysis Set Population 17 Copyright 2018 Esperion. All Rights Reserved Do Not Copy Or Distribute
Study 1 Efficacy
Study 1 (1002-040) Long-Term Safety Study Efficacy Percent Change from Baseline after 12 Weeks 19 Copyright 2018 Esperion. All Rights Reserved Do Not Copy Or Distribute
Study 1 (1002-040) Long-Term Safety Study Efficacy Percent Change from Baseline after 12 Weeks 20 Copyright 2018 Esperion. All Rights Reserved Do Not Copy Or Distribute
Study 1 Top-Line Data Highlights Bempedoic Acid 180mg Added to Maximally Tolerated Statins (50% on High-Intensity Statins) Observed to be safe and well-tolerated in more than 2,200 high-risk patients with ASCVD and/or HeFH on stable background lipid-modifying therapy, including maximally-tolerated statins Comparable to placebo with similar frequency of AEs, SAEs, muscle-related AEs and discontinuations LFT elevations were low overall and consistent with previous studies Significantly lowered LDL-C by 20% vs. placebo (p<0.001) (on-treatment) Consistent lowering of LDL-C regardless of statin intensity; sustained over time Significantly reduced hscrp by 22% (p<0.001) Significantly lowered non-hdl-c, total cholesterol and apob (p<0.001) 21 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Study 1 (1002-040) Conclusions Bempedoic Acid Could be an Important New Oral Treatment Option In the largest and longest duration of our Phase 3 studies, bempedoic acid was observed to be safe and well tolerated, and provided clinically and statistically significant LDL-C lowering and reductions in hscrp when added on to maximally tolerated statins (including high-intensity statins). Bempedoic acid could be an important, new, convenient, once-daily, oral treatment option that complements the LDL-C lowering efficacy of available LDL-C lowering therapies, including statins, ezetimibe and PCSK9i. In the coming months, results from three remaining pivotal Phase 3 studies are expected to further validate the efficacy, safety and tolerability profile of bempedoic acid and the bempedoic acid / ezetimibe combination pill as convenient, once-daily, oral therapies that are complementary to existing standard of care LDL-C lowering therapies. 22 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Q & A 23 COPYRIGHT 2018 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE
Key Upcoming Milestones Results from Three Remaining Phase 3 Studies Expected This Year; NDA Submissions No Later Than Q1 2019 March Study 4 (1002-048) BA Ph 3 TLRs August 1002FDC-053 BA / EZ Combo Pill Ph 3 TLRs March 2018 Phase 2 BA PCSK9 Add-on TLRs Q1 2018 Announce Combination + Statin Future Development Plans 2018 May Study 1 (1002-040) BA Ph 3 TLRs September Study 2 (1002-047) BA Ph 3 TLRs Q1 2019 LDL-C Lowering Indication NDA Submissions Q2 2019 LDL-C Lowering Indication MAA Submissions 1H 2020 LDL-C Lowering Anticipated Commercial Launch 2022 CLEAR Outcomes CVOT TLRs 2018 Study 3 (1002-046) BA Ph 3 TLRs 2019 2020 2022 24 Copyright 2018 Esperion. All Rights Reserved Do Not Copy Or Distribute