Bempedoic Acid / Eze.mibe Combina.on Development & Regulatory Update. June 26, 2017

Transcription

1 Bempedoic Acid / Eze.mibe Combina.on Development & Regulatory Update June 26, 2017

2 Safe Harbor Forward-Looking Statements These slides and the accompanying oral presentaoon contain forward-looking statements and informaoon. The use of words such as may, might, will, should, expect, plan, anocipate, believe, esomate, project, intend, future, potenoal, or cononue, and other similar expressions are intended to idenofy forward looking statements. For example, all statements we make regarding the inioaoon, Oming, progress and results of our preclinical and clinical studies and our research and development programs, our ability to advance ETC-1002 into, and successfully complete, clinical studies, and the Oming or likelihood of regulatory filings and approvals are forward looking. All forward-looking statements are based on esomates and assumpoons by our management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are subject to risks and uncertainoes 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 uncertainoes that are described in the preliminary prospectus supplement and the accompanying prospectus. Any forwardlooking statement speaks only as of the date on which it was made. Neither we, nor our affiliates, advisors, or representaoves, including the underwriters of this offering, undertake any obligaoon to publicly update or revise any forward-looking statement, whether as a result of new informaoon, future events or otherwise, except as required by law. 2 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

3 Bempedoic Acid / Eze.mibe Combina.on LDL-C Lowering ( ) LS Mean % Change LDL-C % Change Over Time Bempedoic Acid 180mg EzeOmibe 10 mg Bempedoic Acid 180mg + EzeDmibe 10mg Weeks on Treatment Thompson et al. J Clin Lipidol 2016;10: COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

4 Bempedoic Acid / Eze.mibe Combina.on Overview Convenient, complementary, cost-effecove, non-staon, once-daily, oral LDL-C lowering therapy Efficacy is comparable to injectable PCSK9i (~50% LDL-C lowering) plus differenoated hscrp reducoon Profile: Once-daily, oral combinaoon pill ~50% LDL-C lowering 26% hscrp reducoon PotenOal for lower occurrence of muscle-related side effects No effects on HDL-C, TGs, blood pressure, or blood glucose Safe and well-tolerated Agreement with FDA on global pivotal Phase 3 bridging study and abbreviated 505(b)(2) regulatory pathway to approval Patent filings in process Bempedoic Acid (top), CombinaOon (bolom) *Thompson PD et al. J Clin Lipidol: 2016;10: ; ezeomibe US patent(s) expire April COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

5 Development and Regulatory Strategy Bempedoic Acid / Eze<mibe Combina<on and Bempedoic Acid Global Clinical Development Programs to Support Target Label(s) Bempedoic Acid / EzeDmbe Combo LDL-C Lowering Submission (1H 2019) Bempedoic Acid LDL-C Lowering Submission (1H 2019) CV RR Submission (2022) LDL-C Lowering Program è IniOal LDL-C Lowering Label in U.S. and Europe (like PCSK9i labels) CLEAR Outcomes CVOT è CV Risk ReducOon Label in U.S. and Europe (Note: breadth of LDL-C lowering label will likely depend on PCSK9i CV label) 5 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

6 Bempedoic Acid / Eze.mibe Combina.on Phase 3 Study Bempedoic acid / ezeomibe combinaoon global pivotal Phase 3 Program to be conducted concurrently with bempedoic acid global pivotal Phase 3 program Phase 3 bridging study designed to enroll up to 350 paoents with ASCVD and/or HeFH, including high CV-risk primary prevenoon paoents, whose LDL-C is not adequately controlled Evaluate the safety and efficacy of bempedoic acid 180 mg & ezeomibe 10 mg, a single once-daily, oral pill Phase 3 bridging study expected to inioate by the fourth quarter of 2017 with top-line results expected by the end of COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

7 Bempedoic Acid / Eze.mibe Combina.on Complementary Non-Sta<n Mechanisms Of Ac<on (MOAs) Bempedoic Acid Inhibits ATP Citrate Lyase (ACL) AcOve in liver cells Acts in the same cholesterol biosynthesis pathway as staons Upregulates LDL receptors EzeDmibe Inhibits NPC1L1 (sterol transporter) Primary InhibiOon of gastrointesonal cholesterol absorpoon Secondary: Upregulates LDL receptors 7 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

8 U.S. and Europe Proposed Indica.ons for Both the Combina.on and Bempedoic Acid at Launch U.S. IndicaDon LDL-C Lowering Bempedoic acid (and/or combinadon) is indicated as an adjunct to diet and: Maximally tolerated staon therapy for treatment of adults with ASCVD and/or HeFH who require addioonal LDL-C lowering CV: The effect of bempedoic acid on cardiovascular morbidity and mortality has not been determined Europe IndicaDon LDL-C Lowering Bempedoic acid (and/or combinadon) is indicated in primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet: in combinaoon with a staon or staon with other lipid lowering therapies in paoents unable to reach LDL-C goals with the maximum tolerated dose of a staon or, alone or in combina<on with other lipid-lowering therapies in pa<ents who are sta<n intolerant, or for whom a sta<n is contraindicated CV: The effect of bempedoic acid on cardiovascular morbidity and mortality has not been determined 8 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

9 Bempedoic Acid / Eze.mibe Combina.on Overview Convenient, complementary, cost-effecove, non-staon, once-daily, oral LDL-C lowering therapy Efficacy is comparable to injectable PCSK9i (~50% LDL-C lowering) plus differenoated hscrp reducoon Profile: Once-daily, oral combinaoon pill ~50% LDL-C lowering 26% hscrp reducoon PotenOal for lower occurrence of muscle-related side effects No effects on HDL-C, TGs, blood pressure, or blood glucose Safe and well-tolerated Agreement with FDA on global pivotal Phase 3 bridging study and abbreviated 505(b)(2) regulatory pathway to approval Patent filings in process Bempedoic Acid (top), CombinaOon (bolom) *Thompson PD et al. J Clin Lipidol: 2016;10: ; ezeomibe US patent(s) expire April COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

10 Bempedoic Acid / Eze.mibe Combina.on Poten.al The Ze<a/Vytorin Non-Sta<n, Oral Precedent ZeOa/Vytorin franchise differenoated, oral, paroally non-staon LDL-C lowering therapies (e.g., staon-sparing ) Over $50 billion cumulaove revenue The bempedoic acid / ezeomibe combinaoon key posiooning factors: ~50% LDL-C lowering; plus significant hscrp reducoon NON-STATIN LDL-C lowering, complementary MOAs; well-tolerated with potenoal for lower occurrence of muscle-related side effects PaDent convenience: once-daily, oral dosing Physician convenience: complementary to SOC oral therapies, two disonct efficacy profiles, and limited step-edits/prior-authorizaoons Payer convenience: limited formulary management effort due to cost-effecove pricing 10 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

11 Bempedoic Acid / Eze.mibe Combina.on Poten.al The Ze<a(eze<mibe) and Vytorin Precedent $6,000.0 ZeDa and Vytorin Annual Sales Sales in millions $5,000.0 $4,000.0 $3,000.0 $2,000.0 $1,000.0 $ Vytorin ZeOa Source: SEC Filings, EvaluatePharma 11 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

12 Key Upcoming Milestones BEMPEDOIC ACID / EZETIMIBE COMBINATION June 2017 Announce Combination Development & Regulatory Strategy Q Phase 2 Study Combo+Statin Top-Line Results Q Phase 3 Combination Bridging Study Initiation End of 2018 Phase 3 Combination Bridging Study Top-Line Results 1H 2019 LDL-C Lowering Indication NDA/MAA Submissions 1H 2020 LDL-C Lowering Anticipated Approval Q Initiate Phase 2 PCSK9i Study BEMPEDOIC ACID 2H 2017 Publication of ACLi Mendelian Randomization Results 2H 2017 Complete Enrollment in Phase 3 LDL-C Efficacy Studies (Studies 2 4) Q Phase 3 Long-term Safety Study (Study 1) Top-Line Results Mid-2018 Phase 3 LDL-C Efficacy Studies (Studies 2 4) Top-Line Results 1H 2019 LDL-C Lowering Indication NDA/MAA Submissions 1H 2020 LDL-C Lowering Anticipated Approval 2022 Cardiovascular Disease Risk Reduction Indication NDA/ MAA Submissions 12 COPYRIGHT 2017 ESPERION. ALL RIGHTS RESERVED DO NOT COPY OR DISTRIBUTE

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