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Study 3 (1002-046)

Pivotal Phase 3 Efficacy Study


Top-Line Results

May 23, 2018


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 or EMA 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.
Bempedoic Acid Franchise
Development Program Updates

May 23, 2018


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 8.6 8.4 4–6 21 – 23


Study 1 & 2
(ASCVD & HeFH) million million million million
ASCVD, HeFH & High Risk
3.5 3.3 0.8 7.6
for CVD Study 3 & 4
million million million million
(No Statin Background)
12.1 11.7 4.8 – 6.8 28.6 – 30.6
TOTAL
million million million million

Source: AHA Heart Disease, Stroke and Research Statistics At-A-Glance 2016

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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 9.5 Million
Number of Patients in Need of

84% (8M) of these patients will get Patients achieving goals on either BA or BA+EZ
Additional LDL-C Lowering*

11M below 70mg/dL with BA or BA+EZ


Bempedoic acid patients not at goal
10M
PCSK9i
9M
8M
7M
100 – 130 mg/dL
6M
5M
4M
1.8 Million 1.4 Million
3M 70 – 100 mg/dL 92% (1.7M) of these patients will get
below 100mg/dL with BA or BA+EZ 14% (200k) of these patients will get
2M
below 100mg/dL with BA+EZ
1M

Maximally Tolerated Statin Therapy** +/- Ezetimibe


Moderate Risk High Risk Very High Risk
(70 – 130 mg/dL) (130 – 160 mg/dL) (>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)

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Bempedoic Acid Franchise Target Patient Populations
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*

12 – 13 million patients in the U.S. can’t/don’t achieve their LDL-C lowering goal with statins and/or ezetimibe alone.
Approximately four out of five patients require less than 50% additional LDL-C lowering to reach their treatment goal. Most
patients can’t/won’t access expensive, inconvenient, injectable PCSK9i therapies.

The goal of our Phase 3 Program is to evaluate the efficacy and long-term safety and tolerability of bempedoic acid
added-on to maximally tolerated LDL-C lowering therapies in high-risk patients with hypercholesterolemia
(LDL-C > 100 mg/dL) in two patient populations:

1 2
High-risk patients on maximally tolerated doses of
statins who require additional LDL-C lowering High-risk patients who are considered statin intolerant

(~8.5 – 9 million patients) (~3.5 - 4 million patients)

* Symphony Health Solutions, 2017 Data

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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
BEMPEDOIC ACID
Updated ACC/AHA goals increase demand for complementary, Clinically and Statistically Significant LDL-C
accessible, cost-effective, oral LDL-C lowering therapies to Lowering and Reductions in hsCRP ,
help patients achieve the new treatment goals Observed to be Safe and Well-Tolerated

Targeted
Payers drawn to cost-effective, convenient, once-daily, oral Mechanism of Action
LDL-C lowering therapies
Complementary and Convenient Once-
daily, Oral Treatment Option

Increasing utilization of generic ezetimibe will drive


combination pill adoption

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Key Upcoming Milestones
Results from Three Remaining Phase 3 Studies Expected This Year; NDA Submissions No Later Than Q1 2019

March August
Study 4 1002FDC-053
(1002-048) BA / EZ Combo Pill
BA Ph 3 TLRs Ph 3 TLRs

Q1 2019

Q1 2018
2018 LDL-C Lowering
Indication NDA
Announce Submissions 1H 2020
March 2018
Phase 2
Combination +
Statin Future
May September Q2 2019
LDL-C Lowering
Anticipated
2022
CLEAR
BA PCSK9 Development Study 1 Study 2 LDL-C Lowering
Indication MAA
Commercial Outcomes
Add-on TLRs Plans Launch CVOT TLRs
(1002-040) (1002-047) Submissions
BA Ph 3 TLRs BA Ph 3 TLRs

2018 Study 3 2019 2020 2022


(1002-046)
BA Ph 3 TLRs

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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
52 weeks safety /
Study 1; Long-Term, N=2,230| TLRs Announced May 2
12 weeks LDL-C

ASCVD
and/or
HeFH OLE N=1,462| (Fully Enrolled) 1.5 yrs safety
Statin Add-On

12 weeks LDL-C /
Study 2; High Risk, N=779| (Fully Enrolled) TLRs Expected in September
52 weeks safety

Study 3; SI, N=345| TLRs Announced May 23 12 weeks LDL-C / 24 weeks safety
ASCVD and/or HeFH/10;
Low, Very Low or No Statin
Background Therapy* Study 4; SI, +EZ, N=269
TLRs Announced March 7
12 weeks LDL-C / 12 weeks safety

ASCVD and/or HeFH/10; Study 053; N=382| (Fully


FDC Added to Statin 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

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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) CV RR
Submission
(2022)
Bempedoic Acid LDL-C
Lowering NDA Submission
(No later than Q1 2019)

LDL-C Lowering Program  CLEAR Outcomes CVOT 


Adjunct to diet and maximally tolerated statin therapy CV Risk Reduction Label in U.S. and Europe
for the treatment of adults with ASCVD and/or HeFH (Note: breadth of LDL-C lowering label and CV RR label will
who require additional lowering of LDL-C broaden similar to post-CVOT PCSK9i CV RR label 2017)

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Study 3 (1002-046) Phase 3 Efficacy Study
Study Design

Patients Considered Statin Intolerant

345 high risk statin intolerant Bempedoic acid 180 mg (n=234)


patients with ASCVD or no
evidence of ASCVD*
LDL-C screening criteria: Placebo (n=111)
No evidence of ASCVD: ≥ 130 mg/dL
ASCVD/HeFH: ≥ 100 mg/dL
24-Week Treatment

• Primary Objective:
• 12-week LDL-C lowering
• Secondary Objectives:
• 24-week LDL-C lowering
• 12-week hsCRP, non-HDL-C, total cholesterol and apoB
• Safety and tolerability
*Patients with no evidence of ASCVD must as a minimum have a history of requiring lipid-modifying therapy based on local guidelines (AHA/ACC or Canadian
Cardiovascular Society). ASCVD includes CAD, symptomatic PAD, and/or cerebrovascular atherosclerotic disease based on 2013 ACC/AHA and/or HeFH

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1002-046 (Study 3) Phase 3 Efficacy Study
Demographics & Baseline Characteristics: Full Analysis Set

Bempedoic Acid Placebo Total Across Study


N=234 N=111 N=345
Demographics
Age: years 65.2 ± 9.7 65.1 ± 9.2 65.2 ± 9.5
Gender: % Female (M/F) 56.8% (133F/101M) 55% (61F/45M) 56% (194F/151)
Race
White: % (N) 90% (211) 87% (96) 89% (307)
Baseline Characteristics
BMI: kg/m2 30.1 ± 5.8 30.6 ± 5.2 30.29 ± 5.6
No evidence of ASCVD: % (N) 61.5% (144) 60% (67) 61.2% (211)
ASCVD and/or HeFH : % (N) 38.5% (90) 40% (44) 38.8% (134)
Diabetes: % (N) 27% (63) 23% (26) 25.8% (89)
Hypertension: % (N) 68% (158) 68% (75) 67.5% (233)
Lipid modifying therapy use at baseline (Statin*): % (N) 7.7% (18) 9.9% (11) 8.4% (29)
Lipid modifying therapy use at baseline (Non-statin): % (N) 35.5% (83) 29.7% (33) 33.6% (116)
No lipid modifying therapy at baseline: % (N) 56.8% (133) 60.4 % (67) 58.0% (200)
* All statins were used at less than the lowest approved daily starting dose

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1002-046 (Study 3) Phase 3 Efficacy Study
Baseline Lipids and hsCRP: Full Analysis Set

Mean ± SD
Total Across
Bempedoic Acid Placebo Study
N=234 N=111 N=345
Primary Efficacy Endpoint
LDL-C: mg/dL 158 ± 40.4 156 ± 38.8 158 ± 39.9
Secondary Efficacy Endpoints
non-HDL-C: mg/dL 193 ± 45.1 191 ± 43.8 193 ± 44.6
Total Cholesterol: mg/dL 246 ± 47.3 241 ± 44.3 244 ± 46.3
apoB: mg/dL 141 ± 31.6 142 ± 30.4 141 ± 31.2
2.9 2.8 2.9
hsCRP: mg/L*
(1.34, 5.29) (1.21, 5.15) (1.29, 5.15)
Other Endpoints
HDL-C: mg/dL 52 ± 14.5 50 ± 14.4 51.6 ± 14.5
156.5 164.0 159.5
Triglycerides: mg/dL*
(114.5, 219) (120.0, 225.5) (117.0, 222.5)

*Median Values (Q1, Q3)

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1002-046 (Study 3) Phase 3 Efficacy Study
LDL-C Percent Change from Baseline at 12 weeks:

An absolute LDL- C reduction of 43 mg/dL was


observed in patients that remained on treatment An absolute LDL- C reduction of 39 mg/dL was
LDL-C On-Treatment Analysis observed in this full analysis population (ITT)
LDL-C Full Analysis Set
LS Mean % Change from Baseline

N=101 N=204

LS Mean % Change from Baseline


N=111 N=234
0

-2% -24% 0
placebo- -1% -22%
-10 corrected
-23% placebo-
p<0.001 -10 corrected
-26%
p<0.001

-20 -20
Placebo
Placebo
Bempedoic Acid
-30 Bempedoic Acid -30

*On treatment population includes patients who were on treatment at the visit analyzed.

Baseline LDL-C 157 ± 40 158 ± 41 Baseline LDL-C 156 ± 39 159 ± 40


(mean ± SD, mg/dL) (mean ± SD, mg/dL)

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1002-046 (Study 3) Phase 3 Efficacy Study
hsCRP Percent Change from Baseline after 12 weeks of dosing of bempedoic acid 180mg

hsCRP
N=106 N=231
10
Median % Change from Baseline

3%
0
Placebo

-10 -25%

-20 Bempedoic Acid

p<0.001
-30

Baseline hsCRP 2.8 2.9


p-value based on Wilcoxon Rank Sum Test
(median; Q1, Q3 mg/L) (1.21, 5.15) (1.34, 5.29)

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1002-046 (Study 3) Phase 3 Efficacy Study – Safety & Tolerability
Overview of Adverse Events

% (Number) of Patients
Treatment Emergent Bempedoic acid Placebo
Adverse Events (AEs) N=234 N=111
Overview of AEs in All Patients (patient incidence)
Any AE(s) 64.1% (150) 56.8% (63)
Serious AE(s)* 6.0% (14) 3.6% (4)
Discontinuation due to AE(s) 18.4% (43) 11.7% (13)
Fatal Adverse Events – Unrelated to Study Treatment 0 0
Safety Population
*No SAE reported as related to study medication

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1002-046 (Study 3) Phase 3 Efficacy Study – Safety & Tolerability
Overview of Muscle-Related Adverse Events

% (Number) of Patients
Bempedoic acid Placebo
Potential Muscle AESI
N=234 N=111
Overview of Potential Muscle AESIs in All Patients (patient incidence)
Any Potential Muscle AESI(s)* 12.8% (30) 16.2% (18)
Discontinuation of Study Drug due to Potential Muscle
7.7% (18) 6.3% (7)
AESI(s)*
All Potential Muscle AESIs by MedDRA Preferred Term*
Myalgia 4.7% (11) 7.2% (8)
Muscular weakness 0.4% (1) 1.8% (2)
Muscle Spasms 4.3% (10) 4.5% (5)
Pain in extremity 5.6% (13) 3.6% (4)

*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

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Study 3 Top-Line Data Highlights
Bempedoic Acid 180mg in Patients Considered Statin Intolerant

Significantly lowered LDL-C


by 26% (43 mg/dL) at 12 and
24 weeks (on treatment)
Significantly lowered
Significantly reduced
• Significantly lowered LDL-C by non-HDL-C, total
23% (39 mg/dL) (ITT)
hsCRP by 25%
cholesterol and apoB
• Confirms Phase 2 results
• Confirms design and power
calculations for CVOT

Observed to be safe and well-tolerated in this study of


high-risk patients considered statin intolerant
No fatalities and no increases in muscle-related AEs
LFT elevations were low overall and consistent with previous studies

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Study 3 (1002-046) – Conclusions
Bempedoic Acid Could Be an Important New Oral Treatment Option

In this Phase 3 study, bempedoic acid and provided clinically relevant and statistically significant
LDL-C lowering and reductions in hsCRP in high-risk statin intolerant patients, and was observed to
be safe and well-tolerated.

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 our 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.

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Cumulative Phase 2 / Phase 3 Results

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Phase 3 Program (Study 4, Study 1, Study 3)
Comparison of Demographics & Baseline Characteristics Across Studies
Study 4 Study 1 Study 3
N=269 N=2230 N=345
Demographics
Age: years 63.8 ± 10.9 66.1 ± 9.0 65.2 ± 9.5
61.3% 24% 56%
Gender: % Female (M/F)
(165F/104M) (602F/1628M) (194F/151 M)
Baseline Characteristics
BMI: kg/m2 29.8 ± 5.1 29.6 ± 4.9 30.29 ± 5.6
No evidence of ASCVD: % (N) 75.8% (204) 0 61.2% (211)
ASCVD and/or HeFH : % (N) 24.2% (65) 100% (2230) 38.8% (134)
Diabetes: % (N) 19.3% (51) 28.6% (637) 25.8% (89)
Hypertension: % (N) 60.2% (162) 79.3% (1768) 67.5% (233)
Statin use (Less than starting dose): % (N) 0 0 8.4% (29)
Statin use (low intensity): % (N) 31.2% (84) 6.6% (148) 0
Statin use (moderate intensity): % (N) 0 43.5% (970) 0
Statin use (high intensity): % (N) 0 49.9% (1112) 0
100% 8.0%
Lipid modifying therapy use (Non-statin): % (N) 33.6% (116)
(ezetimibe-269) (ezetimibe—178)
No lipid modifying therapy: % (N) -- 0 58.0% (200)

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Phase 3 Program (Study 4, Study 1, Study 3)
Comparison of Baseline Lipids and hsCRP Across Studies

Mean ± SD
Study 4 Study 1 Study 3
N=269 N=2230 N=345
Primary Efficacy Endpoint
LDL-C: mg/dL 127.6 ± 29.8 103 ± 29.4 158 ± 39.9
Secondary Efficacy Endpoints
non-HDL-C: mg/dL 159 ± 34.9 130 ± 33.7 193 ± 44.6
Total Cholesterol: mg/dL 215 ± 36.0 179 ± 35.3 244 ± 46.3
apoB: mg/dL 121 ± 25.7 88 ± 21.6 141 ± 31.2
hsCRP: mg/L* 3.6 1.5 2.9
Other Endpoints
HDL-C: mg/dL 56 ± 18.1 49 ± 11.7 51.6 ± 14.5
Triglycerides: mg/dL* 159.2 125.0 159.5

*Median Values

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Phase 2 / Phase 3 Program - Cumulative Efficacy (Completed Studies)
Overview of LDL-C and hsCRP Reductions
• Among the completed phase 2 clinical trials:
• Bempedoic acid 180 mg monotherapy produced LDL-C lowering of 30%
• Bempedoic acid 180 mg/ezetimibe 10 mg combination monotherapy produced LDL-C lowering of 50%
• Bempedoic acid 180 mg on top of background statins produced LDL-C lowering of 20-24%

• Among the three completed phase 3 clinical trials*:


• In study 1 (patients with ASCVD on MTD statin), bempedoic acid 180 mg lowered LDL-C by 20%
• In study 3 (statin intolerant patients, 8% on very low dose statin) bempedoic acid lowered LDL-C by
26%
• In study 4 (statin intolerant patients, 30% on low dose statin), bempedoic acid lowered LDL-C by 28%

• Among all of the phase 2 and 3 trials, the reductions in hsCRP have ranged from 22-40% across
all patient populations
*Reported topline results

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Phase 2/Phase 3 Program - Cumulative Safety & Tolerability (Completed Studies)
Overview of Adverse Events and Discontinuations

% (Number) of Patients

Treatment Emergent Bempedoic Acid Placebo/Active Control


Adverse Events (AEs) N=2668 N=1338

Overview of AEs in All Patients (patient incidence)


Any AE(s) 67.8% (1809) 66.1% (884)
Serious AE(s)* 9.2% (245) 8.9% (119)
Discontinuation of Study Drug due to AE(s) 9.5% (253) 6.9% (92)
Fatal Adverse Events – Unrelated to Study Treatment 0.5% (14) 0.2% (2)
Safety Analysis Set Population
*No SAE reported as related to study medication
Includes data from Phase 2 (003, 005, 006, 007, 008,, 009, 014, 035, 038, 039) and Phase 3 (Study 1, Study 3, Study 4)

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Bempedoic Acid Integrated Safety
LFT Elevations are Rare
Overview of Liver Function Tests (AST/ALT) - % (number) of Patients
Phase 3 – Study 3 (046) Completed Integrated Data1 Total Ongoing Phase 3 Program3
LFT Increases
(Repeated and Confirmed) Bempedoic Acid Placebo2 Bempedoic Acid Placebo2 Bempedoic Acid Placebo2
N=234 N=111 N=2,668 N=1,338 N=2,657 N=1,230
ALT/AST > 3 x ULN 0.43% (1) 0% (0) 0.56% (15*) 0.15% (2) 0.56% (15*) 0.16% (2)
1 Phase 2/Phase 3 integrated data includes all completed studies to date
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 Study 2; includes

final data from Study 1, Study 3 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 Ezetimibe4 (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% -
4Data collected from FDA approved package inserts for each drug. Note that all reported Liver Function Test increases occurred within 12 weeks of initiating therapy.

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Phase 2/Phase 3 Program – Cumulative Safety & Tolerability (Completed Studies)
Overview of Muscle-Related Adverse Events

% (Number) of Patients

Potential Muscle TEAEs Bempedoic Acid Placebo/Active Control


N=2668 N=1338
Overview of Potential Muscle TEAEs in All Patients (patient incidence)
Any Potential Muscle AE(s) 10.9% (290) 9.0% (112)
Discontinuation of study drug due to potential muscle AEs 2.0% (54) 2.3% (31)
Muscle AEs by MedDRA Preferred Term
Myalgia 4.6% (122) 4.7% (58)
Muscular weakness 0.6% (16) 0.7% (9)
Muscle spasm 3.7% (98) 2.8% (35)
Pain in extremity 2.8% (76) 1.9% (23)
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, myoglobinemia,
myoglinuria, myopathy, myopathy toxic, necrotizing myositis, pain in extremity, rhabdomyolysis
Includes data from Phase 2 (003, 005, 006, 007, 008,, 009, 014, 035, 038, 039) and Phase 3 (Study 1, Study 3, Study 4)

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CVOT Powering Assumptions

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CLEAR Outcomes Powering Assumptions: Insights from Study 3

• Study 3 enrolled a very similar patient population to CLEAR Outcomes: statin intolerant, taking no or
very low doses of statins, ASCVD or at high risk for ASCVD, higher baseline LDL-C than other studies.
• In a CVOT with a baseline LDL-C of ~135 mg/dL, the LDL-C lowering in Study 3 in statin intolerant
patients would be expected to produce an absolute LDL-C reduction of ~35 mg/dL.
• Based on the Cholesterol Treatment Trialists’ meta-analysis of major statin CVOTs, this reduction in LDL-C
would be expected to translate to a ~20% reduction in major CV events.
• The CTT also referenced length of treatment as a factor in the magnitude of CV risk reduction;
therefore the expected ~4 year follow up in CLEAR Outcomes (similar to statin CVOTs; longer than
PCSK9 CVOTs) helps support the expected relative risk reduction.
• Based on the expected unit reduction in LDL-C in CLEAR Outcome based on our findings in Study 3, we
are confident that bempedoic acid will show a statistically significant and clinically relevant reduction
in CV risk.

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Cumulative Phase 2 / Phase 3 Results – Conclusions
Bempedoic Acid Could Be an Important New Oral Treatment Option

Across the Phase 2 and Phase 3 programs, bempedoic acid provided clinically relevant and
statistically significant LDL-C lowering and reductions in hsCRP in high-risk patients, including those
on background statin therapy and those unable to tolerate statins and considered statin intolerant,
and was observed to be safe and well-tolerated.

Bempedoic acid can 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 our two 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.
Q&A

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