deutsche conference final · market opportunity $21b prevalence 2.3m source: market size...

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Deutsche Bank 43rd Annual Health Care Conference May 8, 2018 C HANGING THE C OURSE OF H UMAN H EALTH T HROUGH B OLD P URSUITS IN S CIENCE C HANGING THE C OURSE OF H UMAN H EALTH T HROUGH B OLD P URSUITS IN S CIENCE

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Page 1: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Deutsche Bank 43rd Annual Health Care Conference

May 8, 2018

CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD

PURSUITS IN SCIENCE

CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD

PURSUITS IN SCIENCE

Page 2: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Forward-Looking Statements and Adjusted Financial Information

2

This presentation contains forward-looking statements, which are generally statements that are not historical facts.Forward-looking statements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,”“plans,” “will,” “outlook,” “targets” and similar expressions. Forward-looking statements are based on management’scurrent plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertakeno obligation to update any forward-looking statement in light of new information or future events, except as otherwiserequired by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult topredict and are generally beyond our control. Actual results or outcomes may differ materially from those implied bythe forward-looking statements as a result of the impact of a number of factors, many of which are discussed in moredetail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.

In addition to unaudited financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures. Further information relevant to the interpretation of adjusted financial measures, and reconciliations of these adjusted financial measures to the most comparable GAAP measures, may be found in the Appendix and on our website at www.Celgene.com in the “Investor Relations” section.

Page 3: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Our Mission and Vision

3

Celgene is building a preeminent global biopharmaceutical company focusedon the discovery, development and commercialization of innovative therapies for patients with cancer, immune-inflammatory, and other unmet medical needs

Page 4: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Deploying a Strategy to Grow Through 2020 and Beyond

ExecuteDeliveringon 2020

AcceleratePositioning toGrow Beyond

2020

ExpandCreating

SustainableGrowth

4

Page 5: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Strong Volume-Driven Growth Expected in 2018

5

14Y/Y Growth

%+%

EPS Y/Y growth*

~56%Operating Margin*

~$9.5B~ $14.8B

TotalRevenue

Tax Rate*

~17%

~ $8.45Diluted EPS*

~$2.0B

Total Revenue

~$1.5B ~$1.0B

*Adjusted financial measure which includes the impact of our acquisition of Juno Therapeutics Inc., which is expected to be dilutive to adjusted diluted EPS in 2018 by approximately $0.50.

~13.6%

Page 6: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

MultipleMyeloma

Accelerating Diversification by Advancing Medicinesto Transform the Treatment of Diseases

6

Market Opportunity$27BIncidence60K

HEMATOLOGY & ONCOLOGYNon-Hodgkin Lymphoma

Market Opportunity$17BIncidence90K

MyeloidDiseases

Market Opportunity$5BIncidence65K

Psoriasis / Psoriatic Arthritis

Market Opportunity$26BPrevalence5M

MultipleSclerosis

Market Opportunity$23BPrevalence650K

INFLAMMATION & IMMUNOLOGYInflammatory Bowel Disease

Market Opportunity$21BPrevalence2.3M

Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease Landscape and Forecast; Epidemiology is for 2018 from Decision Resources Disease Landscape and Forecast, Kantar Health CancerMPact database and Putnam Associates

Page 7: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Innovative Therapies Are Transforming Multiple Myeloma

7

+3 months

OS increase

Median Overall Survival(months)

Source: Drawid A et al. Impact of Novel Therapies on Multiple Myeloma – Current and Future Outcomes. Poster presented at the 20th Congress of the European Haematology Association; Vienna, Austria, June 11–14, 2015

Note: Historical median OS for MM patients based on analysis of the National Cancer Institute Surveillance, Epidemiology, and End Results Program; Predictive analysis of OS was modelled using data from clinical trials, cancer registries and insight from key opinion leaders.

Current Therapies

IMiD® Agents Proteasome Inhibitors Monoclonal Antibodies Novel Triplets

Immunotherapy

Future Therapies

+29months

Predicted OS increase

2932

43

72

1990 1995 2000 2005 2010 2015 2020

+11 months

OS increase

Page 8: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Targeting BCMA:A Potentially Disruptive Approach to Myeloma Therapy

*partnered program with bluebird bio**partnered program with Sutro Biopharma 8

T Cell Engager Antibody CC-93269 – phase I trial initiated

2

1 CAR T Therapy bb2121* – pivotal KarMMa™ trial initiated bb21217* – phase I trial initiated JCARH125 – phase I trial initiated

3 Antibody Drug Conjugate BCMA ADC**– IND planned in early 2019

CAR TCells

TUMOR

3. Antibody Drug Conjugate

BCMA

TCells

2. T Cell Engager Antibody

BCMA

1. CAR T Therapy

BCMA

TCR

Cytotoxicgranules

Cytotoxicgranules

1 2

3

Page 9: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Targeting High Unmet Needs in NHL with Multiple Mechanisms

Note: REVLIMID® is not approved for follicular or diffuse Large B-cell non-Hodgkin lymphomaCDx: companion diagnostic 9

Diffuse Large B-cell Lymphoma Follicular Lymphoma

Indu

ctio

n

ABC UC GCB

1st

Rel

apse

SCT Eligible SCT Ineligible Relapsed / Refractory2nd

Rel

apse

+ Double Refractory

CC-122

REVLIMID®

AUGMENT™

CC-122CDx

Liso-cel

REVLIMID®

ROBUST®

CC-486

Liso-cel

CD19 CAR TLiso-cel (JCAR017) Potential best-in-class profile Pivotal program in DLBCL under way

EpigeneticsCC-486 Combo with R-CHOP in DLBCL at ASH ‘17 Additional POC trial planned in 2018

Key MechanismsProtein Homeostasis

REVLIMID®

AUGMENT™ data expected in 2018 ROBUST® data (event-driven)

CC-122 Initiate pivotal program in 2018

Next-gen CELMoD® Agents

Page 10: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Liso-cel – Emerging Favorable Profile in R/R DLBCL

Data include: JCAR017 CORE R/R DLBCL Phase I for both DL1S and DL2S groups (safety n=67; efficacy n=65 data cut-off October 9, 2017, ASH 2017); YESCARTA™ Phase II (n=101, ASCO 2017); and, KYMRIAH™ Phase II (safety n=99, efficacy n=46 ASH 2017). Data presented to show potential profile of JCAR017, which is subject to ongoing investigation, within context of other CAR T treatments. Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. The most common adverse events across all patients treated in JCAR017 the study (n=91) other than CRS and NT that occurred at ≥25% included neutropenia (49%), anemia (38%), fatigue (37%), thrombocytopenia (29%), nausea (27%), and diarrhea (25%). Grade 1/2 events for CRS and NT were 34% and 6%, respectively.

42%36%

30%

Liso-cel YESCARTA KYMRIAHComplete Response PR

Response Rates at 6 Months Cytokine Release Syndrome

23%

13%

1%

KYMRIAH

YESCARTA

Liso-cel

Grade 1/2 Grade 3/4

Neurotoxicity

12%

28%

15%

KYMRIAH

YESCARTA

Liso-cel

Grade 1/2 Grade 3/4

SafetyEfficacy

Grade 1/2 Grade 3/4

Grade 1/2 Grade 3/4™™

Page 11: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Improving the Standard of Care Across Myeloid Diseases

IDHIFA®

Approved for R/R IDH2 mutant AML

1st Line AMLHigher-Risk MDSLower-Risk, RBC TD MDS 2nd Line AML

Luspatercept Ph III COMMANDS™

trial in front-line MDS Ph III MEDALIST™

trial in ESA refractory

REVLIMID®

Approved for del5q MDS

CC-486 Ph III QUAZAR®

trial in low-risk MDS

VIDAZA®

Approved for higher-risk MDS and elderly 1st-line AML

CC-486 PH III QUAZAR®

data in AML maintenance

11

Luspatercept

Erythroid maturation agent (EMA) targeting severe chronic anemias

Ph II myelofibrosis trial initiated Ph III data expected in mid-18 Initiate Ph III trial in H1:18Ph III data expected in mid-18

Page 12: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Expected Data Presentations at ASCO 2018Expected Data Presentations at ASCO 2018

12

Ph III OPTIMISMM® trial with POMALYST® in combination with bortezomib and dexamethasone (PVd) in 2nd line+ MM

Ph III RELEVANCE® trial with REVLIMID® in combination with rituximab (R²) in patients with previously untreated FL

Ph III IMpower131* trial with ABRAXANE® in combination with atezolizumab in squamous NSCLC

Updated data from Ph I trial with bb2121 in RRMM Updated data from pivotal TRANSCEND U.S. trial with liso-

cel (JCAR017) in relapsed or refractory DLBCL

* IMpower131 is a Genentech, a member of the Roche Group, sponsored clinical trial.

Page 13: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

R&D Engine

Building a Pipeline of Next-Generation Growth Drivers

13

Centers ofExcellence

Partner Network

Epigenetics

Immuno-Oncology

Inflammation & Immunology

Protein Homeostasis

Neuroscience & Imaging

Internal Innovation EngineAdvancing 8 New Programs into the Clinic

Next-Gen CELMoD®

Ph I for MM

BET inhibitor Ph I for solid

tumors

Mat2A inhibitor Ph I for solid

tumors

BCMA T cell engager

Ph I for MM

CC-92480

CC-90010 AG-270

CC-93269

BCMA CAR T Ph I for MM

CD3xCD33 bispecific

Ph I for AML

Anti-LIF1 MAb Ph I for solid tumors

GLP-1R modulator Targeted for NASH

bb21217

MSC-1 RPC8844

GEM333

Inflammation & Immunology Immuno-OncologyProtein Homeostasis Epigenetics

Page 14: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

BGB-A317 Accelerates Our Solid Tumor I/O Strategy

14

Reverse effector T cell exhaustion Anti-ICOS (JTX-2011) CELMoD® agent (CC-122)

Block regulatory T cells mediated immune suppression Anti-ICOS (JTX-2011) Anti-TIGIT (OMP-313-M32)

Stimulate Th17/Tc17 anti-tumor immunity ROR agonist (LYC-55716)

Activate the innate immune system TLR-8 agonist (motolimod)

Block immune suppressive cytokines Anti-LIF (MSC-1)

Induce macrophages to destroy tumor cells Anti-CD47 (CC-90002)

Tregcell

Teffectorcell

Tc17 /Th17 cell

NK

MDSC

Macrophages

Potentially differentiated anti-PD-1 MAbbased on engineered Fc region: potential to enhance anti-tumor activity by

preventing binding to Fc gamma receptor bind to unique PD-1 epitopes with high affinity

Evaluated in over 600 patients to date Demonstrated activity in a range of solid

tumors with an acceptable safety profile to support continued development Pivotal program plan initiated:

Hepatocellular carcinoma Non-small cell lung cancerEsophageal cancer

– Gastric cancer

Exploring combinatorial potential with emerging I/O agents

BGB-A317 (tislelizumab) Celgene’s Early Clinical Stage I/O Assets

Celgene has an exclusive option to license JTX-2011, OMP-313-M32, and an option to acquire LYC-55716

Page 15: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Building Leadership in a New Disease: Myelofibrosis (MF)

15

Myelofibrosis

Market Opportunity$4BIncidence17K

Fedratinib – A Potential Blockbuster in MF

Addressing Unmet Needs in MF

Source: Market size projection is for 2022 from Evaluate Pharma, December 2017. Epidemiology from Titmarsh, G.J. et.al., American Journal of Hematology, 2014Physician Interviews; ClearView Analysis, September, 2017, n=35

Highly selective JAK2 kinase inhibitor Myelofibrosis clinical program completed to date:

− Ph III trial in treatment-naïve patients − Ph II trial in patients resistant or intolerant to Jakafi®

NDA submission for myelofibrosis planned in 2018 Accelerate ongoing myelofibrosis program with luspatercept, in addition to

early Protein Homeostasis and Epigenetic programs

~60%

~20%

~20%

Currently Receiving Jakafi®First-line, Low Platelet CountJakafi® Failures

Page 16: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Leading the Way in Cellular Immunotherapy

State-of-the-Art Manufacturing

CAR T technology: recognizes proteins on the

surface of cancer cells

TCR Technology: recognizes intracellular tumor-specific proteins

Broad and Deep

Pipeline

BCMA CAR T: bb2121*, bb21217*, JCARH125 CD19 CAR T: JCAR017 CD22: JCAR018 L1CAM: JCAR023 MUC16/ IL-12: JCAR020 ROR-1: JCAR024 LeY WT1: JCAR016

Celgene’s Cellular Immunotherapy Portfolio

16

Innovative Cellular Engineering

*partnered program with bluebird bio

Page 17: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Delivering Industry-Leading Growth Through 2020…

Late-stage Pipeline with Potential to Add Over $16B in Incremental Peak Revenue Through 2030

17

…And Positioned to Grow Beyond 2020

Expected to Launch Ten Potential Blockbusters

Adj. Diluted EPS

Revenue

~19%CAGR

14.5%CAGR

Note: CAGR calculation is from 2017 measurements to the midpoint of the 2020 total revenue range$16 billion represents sum of individual potential peak sales for each illustrated product, which may not coincide.

$1B >$2BL E G E N D

Current Estimate of Peak Sales Potential:

Page 18: Deutsche Conference FINAL · Market Opportunity $21B Prevalence 2.3M Source: Market size projections are for 2022 from Evaluate Pharma, December 2017 and Decision Resources Disease

Deutsche Bank 43rd Annual Health Care Conference

May 8, 2018

CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD

PURSUITS IN SCIENCE

CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD

PURSUITS IN SCIENCE