Celgene I&I Overview

Celgene I&I OverviewCelgene I&I Overview

Scott Smith Senior VP, Global Head of I&I,

Forward Looking Statements and Adjusted Financial Information

This presentation contains forward-looking statements, which are generally statements that are nothistorical facts. Forward-looking statements can be identified by the words “expects,” “anticipates,”“believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similar expressions. Forward-looking, , , p , , p gstatements are based on management’s current plans, estimates, assumptions and projections, andspeak only as of the date they are made. We undertake no obligation to update any forward-lookingstatement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict andare generally beyond our control Actual results or outcomes may differ materially from those impliedare generally beyond our control. Actual results or outcomes may differ materially from those impliedby the forward-looking statements as a result of the impact of a number of factors, many of whichare discussed in more detail in our Annual Report on Form 10-K and our other reports filed with theSecurities and Exchange Commission.

In addition to financial information prepared in accordance with U.S. GAAP, this presentation alsocontains adjusted financial measures that we believe provide investors and management withsupplemental information relating to operating performance and trends that facilitate comparisonsbetween periods and with respect to projected information. These adjusted financial measures arenon-GAAP and should be considered in addition to but not as a substitute for the informationnon GAAP and should be considered in addition to, but not as a substitute for, the informationprepared in accordance with U.S. GAAP. We typically exclude certain GAAP items thatmanagement does not believe affect our basic operations and that do not meet the GAAP definitionof unusual or non-recurring items. Other companies may define these measures in different ways.Further information relevant to the interpretation of adjusted financial measures, and reconciliations

f th dj t d fi i l t th t bl GAAP b f d

2

of these adjusted financial measures to the most comparable GAAP measures, may be found onour website at www.Celgene.com in the “Investor Relations” section.

Agenda

• Welcome / Introduction Scott Smith

• PALACE Program Efficacy Chris Edwards MD

• PALACE Program Safety, Behçet’s Artie Kavanaugh MD

• Q&A

• Apremilast Positioning Mark Krestonp g

• Launch Readiness Terrie Curran

• Conclusion Scott SmithConclusion Scott Smith

• Q&A

3

Building I&I Through Executing Successful Business Strategy

Target Target significant markets with high unmet1 Target Markets

Target significant markets with high unmet medical need1

Clinical Development

Conduct leading edge clinical development programs2

Launch Capabilities Build world class global launch capabilities3

Accelerate Portfolio

Accelerate I&I portfolio by developing pipeline through internal and external opportunities

4

4

Strong Execution with Multiple Positive Phase III Studies

20102010 20112011 20122012 20132013Q1Q1 Q2Q2 Q3Q3 Q4Q4 Q1Q1 Q2Q2 Q3Q3 Q4Q4 Q1Q1 Q2Q2 Q3Q3 Q4Q4 Q1Q1 Q2Q2 Q3Q3 Q4Q4

PALACE 1 PALACE 1 (n=504)(n=504)PALACE 1 PALACE 1 (n=504)(n=504)

PALACE 2PALACE 2 (n=488)(n=488)PALACE 2PALACE 2 (n=488)(n=488)



ESTEEM 1 (n=844)ESTEEM 1 (n=844)ESTEEM 1 (n=844)ESTEEM 1 (n=844)


Behçet'sBehçet's--001 (n=111001 (n=111))Behçet'sBehçet's--001 (n=111001 (n=111))

ESTEEM 2 (n=413)ESTEEM 2 (n=413)ESTEEM 2 (n=413)ESTEEM 2 (n=413)

Approximately 3,500 patients in registration program

5

Significant Phase III Data Enhancing Profile

• PALACE Program (1-3) – PALACE 2 52-week Results (Oral)PALACE 2 52 week Results (Oral)– PALACE 3 52-week Update– Pooled Safety– Pooled Enthesitis/Dactylitis (Oral)– Pooled Laboratory Datay– Pooled HAQ-DI– Pooled TJC and SJC

• PALACE 4 DMARD Naïve 52-weekresults (Late Breaking)

• BCT -001 (Oral Plenary)

• CC-220 ( 2 Posters and Oral)– Moving to Phase II in 2014

6

Apremilast, an Oral Phosphodiesterase p , p4 Inhibitor, in Patients With Psoriatic

Arthritis and Current Skin Involvement: Results of a Phase 3, Randomized,

Controlled Trial (PALACE 3)Christopher J. Edwards, MD, FRCP

University Hospital SouthamptonSouthampton, UK

Charles Birbara1; Francisco J. Blanco2; Jeffrey Crowley3; ChiaChi Hu4; Randall M. Stevens4; Christopher J. Edwards5

1University of Massachusetts Medical School, Worcester, MA; 2INIBIC-Hospital Universitario A C ñ G li i S i 3B k fi ld D t l B k fi ld CA 4C l C tiCoruña, Galicia, Spain; 3Bakersfield Dermatology, Bakersfield, CA; 4Celgene Corporation,

Warren, NJ; 5University Hospital Southampton, Southampton, UK

Presented at: the 2013 ACR/ARHP Annual Meeting; October 26 -30, 2013; San Diego, CA. Abstract 311

7

PsA: Clinical Presentation

Back involvement

S

DIP involvement

Skin Involv

In the majority of patients, cutaneous lesions precede the

onset of joint pain

Nail psoriasis

vement

Peripheral involvement

E th th

Dactylitis

Enthesopathy

DIP: Distal interphalangeal

8

Summary of the ApremilastPALACE Programg

Study PALACE 1 PALACE 2 PALACE 3 PALACE 4 Patient Moderate to Moderate to Moderate to DMARD naïvePatient population (Active PsA)*

Moderate to severe psoriatic

arthritis


arthritis


arthritis withskin lesions

DMARD naïve moderate to

severe psoriatic arthritis

Prior treatments

Stratified by previous

DMARD and


DMARD and


DMARD and

None

biologic use/failure



Concurrent use with

Allowed Allowed Allowed No use with DMARDs Patients randomized

504 484 505 527randomized

9*Most patients had moderate to severe PsA

PALACE: Study Design

Baseline Week 4 Week 16 Week 24 Week 52 5 Years

Active Treatment Phase/Long-term Safety PhasePlacebo-Controlled

Phase

Apremilast 30 mg BID O

e§

Week –4 Apremilast 30 mg BIDApremilast 30 mg BID

*SCREEN Placebo

AN

DO

MIZ

E*1:

1:1

Es

cape

rand

omiz

e‡

RA

Early

Re-

r

Apremilast 20 mg BID Apremilast 20 mg BID Apremilast 20 mg BID

Stratify by DMARD use

(PALACE 1‒3)

Primary End Point: ACR20 at Week 16

and BSA ≥3% (PALACE 3)

*All doses were titrated over the first week of treatment.§Placebo patients whose swollen and tender joint counts had not improved by at least 20% at week 16 were required to be re-randomized to apremilast20 mg or 30 mg BID. ‡At week 24, all remaining placebo patients were re-randomized to apremilast 20 mg BID or 30 mg BID.DMARD=disease-modifying antirheumatic drug; BSA=body surface area; ACR20=20% improvement in modified American College of Rheumatology response criteria.

10

PALACE 3: ACR20/50/70 Responses Over 52-Weeks

Patients Receiving Apremilast From BaselineData as Observed

ACR20

Apremilast 20 mg BIDApremilast 30 mg BID

evin

g an

ns

e (%

)Pa

tient

s A

chie

AC

R R

espo

n

ACR50

10%

16 24 40 52

ACR70

Weeks

16 24 40 52

Edwards et al, ACR 2013. Abstract 311

11

PALACE 3: 52-Week Key Secondary Endpoints: SJC/TJC and DAS-28 (CRP)

M di % Ch i SJC/TJC

p ( )

% A hi i R i i


Median % Change in SJC/TJC Over 52 Weeks

50

% Achieving Remission

20

30

40

ts A

chie

ving

(CR

P) <

2.6

(%)

30%

-70%

-79%

0

10

20

Patie

ntD

AS-

28 (

SJC and TJC

Apremilast 20 mg BID, n 162 146 127 122

Apremilast 30 mg BID, n 154 147 131 127

DAS-28 <2.6

Apremilast 20 mg BID, n/m 30/158 40/143 40/126 34/121

Apremilast 30 mg BID, n/m 31/154 33/145 39/131 38/127

16 24 40 52

Weeks

0

Edwards et al, ACR 2013. Abstract 311

12

Apremilast 20 mg BID Apremilast 30 mg BID

PALACE 3: 52-Week Key Secondary Endpoints: Skin Response and Disabilityp p y

M Ch i HAQ DIPASI 50 d PASI 75 R


Mean Change in HAQ-DI

)

PASI-50

PASI-50 and PASI-75 Response

55%

an C

hang

e m

Bas

elin

e

ents

Ach

ievi

ngI R

espo

nse

(%)

PASI-7539%

Mea

Fro

Patie

PASI

-0.35%

HAQ-DIApremilast 20 mg BID, n 161 146 126 122Apremilast 30 mg BID, n 153 147 132 127

16 24 40 52

Weeks

016 24 52

Weeks

0

PASI-50Apremilast 20 mg BID, n/m 31/84 33/75 31/63Apremilast 30 mg BID, n/m 37/78 36/74 35/64

PASI 75PASI-75Apremilast 20 mg BID, n/m 19/84 21/75 18/63Apremilast 30 mg BID, n/m 19/78 23/74 25/64

Edwards et al, ACR 2013 . Abstract 311

13


Psoriatic Arthritis: Clinical Presentation of Enthesitis and Dactylitisy

Enthesopathy Dactylitis

ACR Slide Collection on the Rheumatic Diseases. 3rd ed. 1994.

14

PALACE 1-3: Change in Enthesitis and Dactylitis Over 52-WeeksyPatients Receiving Apremilast From Baseline

Data as ObservedE th iti D t liti

ES

Weeks0 16 24 52

Enthesitis Dactylitis

0 16 24 52Weeks

ange

in M

ASE Apremilast 20 mg BID

Apremilast 30 mg BID

ent C

hang

e s

Cou

nt

Med

ian

% C

ha

Med

ian

Perc

ein

Dac

tylit

i

Apremilast 20 mg BID, n 291 264 215 197 188 157

‒100%‒66.7%

15

MASES=Maastricht Ankylosing Spondylitis Entheses Score.Note: A decrease in MASES or dactylitis count indicates symptom improvement.

Apremilast 30 mg BID, n 298 288 254 202 196 170

PALACE 1, 2 and 3Gladman et al ACR 2013 Abstract 311

PALACE 1-3: Primary Endpoint: ACR20 Responses at Week 16p

Per-Protocol Population, NRI

‡

pons

e (%

)

‡§

*

‡

*§

ving

AC

R R

esp *

atie

nts

Ach

iev

P

PALACE 1 PALACE 2 PALACE 3

168 163 161 154 159 151 164 163 159n=

16

*P<0.05, §P<0.005, ‡P≤0.0001 vs. placeboACR20=20% improvement in modified American College of Rheumatology response criteria; NRI=non-responder imputation.

Placebo Apremilast 20 mg BID Apremilast 30 mg BID

PALACE 1‒3: ACR20 Over 52-Weeks

80

ACR20 at Week 52Data as Observed

60

70

80PALACE 1 PALACE 2 PALACE 3

)

63%

40

50

60

s A

chie

ving

es

pons

e (%

)

55%53%

20

30

40

Patie

nts

AC

R20

Re

0

10

20

0

17

Weeks16 24 40 52

PALACE 1, 2 and 3Apremilast 30 mg BID arm

Apremilast in the Treatment of DMARD N ï P i i A h i iDMARD-Naïve Psoriatic Arthritis

Patients: Results of a Phase 3 RandomizedRandomized,

Controlled Trial (PALACE 4)Alvin F WellsAlvin F. Wells

Rheumatology and Immunotherapy CenterFranklin, WI

Alvin F. Wells1; Christopher J. Edwards2; Adewale O. Adebajo3; Alan J. Kivitz4; Paul Bird5; Kamal Shah6; ChiaChi Hu6; Randall M. Stevens6; Jacob A. Aelion7

Late Breaking Abstracts

18

Tuesday, October 29, 2013 2:30-4:00 PMSan Diego Convention Center: 6 A

Presentation Number: L4

PALACE 4: ACR20, ACR50, and ACR70 Scores at Week 52

Week 52 Data in Patients Originally Randomized to ApremilastData as observed


n 131 138n 131 138

ACR20 (% responders) 53 59

ACR50 (% responders) 27 32ACR50 (% responders) 27 32

ACR70 (% responders) 14 18

• The primary end point was ACR20 at Week 16– Placebo: 16.9%– Apremilast 20 mg BID: 29.2% (P=0.0076 vs. placebo)– Apremilast 30 mg BID: 32.3% (P=0.0011 vs. placebo)

19 PALACE 4Wells et al, ACR 2013 Late Breaking Abstract # L4

PALACE: Summary

• In 4 Phase 3 trials, apremilast demonstrated clinically meaningful activity in the treatment of PsA includingmeaningful activity in the treatment of PsA, including signs and symptoms and physical functionality over 52-weeks

• Across various manifestations of PsA– Swollen / Tender Joints, Enthesitis, Dactylitis, Psoriasis

• Across the spectrum of patient populations – DMARD Naïve to Biologic Failure

A t bl f t fil d ll ll• Acceptable safety profile and was generally well tolerated

20

Considerations of Psoriatic Arthritis Therapy Selection

Arthur Kavanaugh, MDUniversity of California San DiegoUniversity of California, San Diego

21

Considerations of Psoriatic Arthritis Therapy Selectionpy

• Clinical Efficacy• Co-morbidity• Complicationsp• Convenience

Cost• Cost

22

PALACE 1‒3: ACR20 Over 52-Weeks

80

ACR20 at Week 52Data as Observed

60

70

80PALACE 1 PALACE 2 PALACE 3

)

63%

40

50

s A

chie

ving

es

pons

e (%

)

55%53%

20

30

Patie

nts

AC

R20

Re

0

10

23

Weeks

16 24 40 52

PALACE 1, 2 and 3Apremilast 30 mg BID arm

Comorbidities in PsA Patients

Psoriasis patients• Psychosocial burden• Reactive depression

Ocular inflammation(iritis/uveitis/episcleritis)

• Higher suicidal ideation• Alcoholism

IBD

Metabolic Syndrome• Hyperlipidemia• Hypertension PALACE 1, 2, & 3 Patient Population • Insulin resistant • Diabetes • Obesity Higher risk of cardiovascular

disease

• BMI >30 ~45% of pts• Hypertension ~40%• Hypercholesterolemia ~15%• Hyperlipidemia ~9%• Type 2 diabetes ~7.5%disease

24

PALACE 1-3: Pooled Safety: Overall Safety Profiley

Placebo-Controlled Phase(Weeks 0 to 24)*

Apremilast-Exposure Period(Weeks 0 to ≥52)§

Patients

Placebo(n=495)

APR 20 mg BID(n=720)


n (%)EAIR/100

Patient-Years n (%)EAIR/100


Patient-Years

1 AE 235 (47 5) 200 7 524 (72 8) 163 5 534 (74 1) 174 0≥1 AE 235 (47.5) 200.7 524 (72.8) 163.5 534 (74.1) 174.0

≥1 SAE 19 (3.8) 11.5 49 (6.8) 6.5 52 (7.2) 7.0

≥1 severe AE 19 (3.8) 11.5 48 (6.7) 6.5 60 (8.3) 8.2

AE leading to drug withdrawal

21 (4.2) 12.5 54 (7.5) 7.1 60 (8.3) 7.9

Death 0 (0.0) 0.0 1‡ (0.1) 0.1 0 (0.0) 0.0

*Includes all data through week 16 for patients initially assigned to placebo who escaped and all data through week 24 for all other patients

25

Includes all data through week 16 for patients initially assigned to placebo who escaped, and all data through week 24 for all other patients. §Includes all patients who received ≥1 dose of apremilast, regardless of when they were randomized.‡Multiorgan failure not suspected by the investigator to be treatment-related.

EAIR=exposure-adjusted incidence rate.Mease et al, ACR 2013 Abstract # 310

PALACE 1, 2 and 3

PALACE 1-3: Most Common Adverse Events

Placebo-Controlled Phase(Weeks 0 to 24)*


Patients

Placebo(n=495)



n (%)EAIR/100



Patient-Years

14 (2 8) 8 5 88 (12 2) 12 8 118 (16 4) 17 7Diarrhea 14 (2.8) 8.5 88 (12.2) 12.8 118 (16.4) 17.7

Nausea 23 (4.6) 14.0 71 (9.9) 9.9 111 (15.4) 16.2

Headache 23 (4.6) 14.1 65 (9.0) 9.1 80 (11.1) 11.3

URTI 15 (3.0) 9.1 82 (11.4) 11.6 67 (9.3) 9.3

Nasopharyngitis 13 (2.6) 7.8 57 (7.9) 7.9 50 (6.9) 6.8

*Includes all data through week 16 for patients initially assigned to placebo who escaped and all data through week 24 for all other patientsIncludes all data through week 16 for patients initially assigned to placebo who escaped, and all data through week 24 for all other patients. §Includes all patients who received ≥1 dose of apremilast, regardless of when they were randomized.‡Multiorgan failure not suspected by the investigator to be treatment-related.

26


PALACE 1-3: Diarrhea

18%

20%

N=1441 Patients in Apremilast-Exposure Period(Weeks 0 to ≥52)Severe

Mild/Moderate

6 pts

12%

14%

16%

18%

14.3%

6%

8%

10%

12%

200 pts 37 pts 22 pts0%

2%

4%

6%

1.5%2.6%

p p 22 pts0%Diarrhea Any Grade Used OTC Anti-Diarrheals Discontinuations due to

Diarrrhea– Occurred most often in the first 2 weeks of exposure to apremilast and resolved by week 4

(fi t t d i it) R d d i id ft th fi t th f d i

*

27

(first study visit). Reduced incidence seen after the first month of dosing– >80% patients that reported diarrhea, did not use OTC Anti-Diarrheal Medications – Most cases were self-limited and did not recur despite continued treatment with apremilast

*1 serious adverse event in patient with C. Difficile

PALACE 1-3: MACE, Malignancy, and Infection Rates

PlaceboPatient-Years=168.2

EAIR/100 Patient-

APR 20 mg BIDPatient-Years=766.4

EAIR/100 Patient-

APR 30 mg BIDPatient-Years=769.0

EAIR/100 Patient-

Apremilast TotalPatient-Years=1,535.4

EAIR/100 Patient-Years Years Years Years

MACE 0.0 0.3 0.1 0.2Potential MACE 0.6 0.5 0.5 0.5

Malignancy events

Hematologic 0.0 0.1 0.0 0.1Skin (excluding melanoma) 1.2 0.4 0.5 0.5

Solid (including melanoma) 0.6 0.3 0.1 0.2

Infection events

Non-opportunistic serious 0 6 0 4 0 5 0 5Non opportunistic serious infection 0.6 0.4 0.5 0.5

Systemic opportunistic infection 0.6 0.0 0.0 0.0

EAIR per 100 patient ears is 100 times the n mber (n) of patients reporting the e ent di ided b patient ears ( p to the first e ent start date for patients reporting

28

EAIR per 100 patient-years is 100 times the number (n) of patients reporting the event divided by patient-years (up to the first event start date for patients reporting the event). EAIR per 100 patient-years for major adverse cardiac events (MACE), serious infection, including systemic opportunistic infection, and malignancies were comparable to placebo.

PALACE 1, 2 and 3


PALACE 1-3: Select Marked Abnormalities in Clinical Chemistry Laboratory Parameters

Placebo-Controlled Phase

(Weeks 0 to 24)*



Marked Abnormalities‡

( )

Placebo(n=495)

Apremilast Apremilast

20 mg BID(n=720)

30 mg BID(n=721)

20 mg BID(n=720)

30 mg BID(n=721)

ALT >3X ULN 1/492 (0 2) 5/710 (0 7) 9/709 (1 3) 8/713 (1 1) 9/713 (1 3)ALT >3X ULN 1/492 (0.2) 5/710 (0.7) 9/709 (1.3) 8/713 (1.1) 9/713 (1.3)

Creatinine >1.7X ULN 1/492 (0.2) 1/710 (0.1) 1/709 (0.1) 1/713 (0.1) 1/713 (0.1)

Leukocytes <1.5 X 109/L 0/489 (0.0) 0/700 (0.0) 0/706 (0.0) 0/712 (0.0) 0/713 (0.0)

Neutrophils <1 X 109/L 2/488 (0.4) 1/698 (0.1) 1/705 (0.1) 4/712 (0.6) 4/713 (0.6)

Platelets <75 X 109/L 0/487 (0.0) 0/700 (0.0) 0/705 (0.0) 1/712 (0.1) 0/713 (0.0)

Hemoglobin, male <10.5 g/dL female <8 5 g/dL 1/489 (0.2) 3/700 (0.4) 3/706 (0.4) 5/712 (0.7) 6/713 (0.8)g/dL, female <8.5 g/dL ( ) ( ) ( ) ( ) ( )

• Marked abnormalities were infrequent and transient, resolving without intervention or discontinuation, and did not recur despite continuation of study drug

29

*Includes all data through week 16 for patients initially assigned to placebo who escaped, and all data through week 24 for all other patients. §Includes all patients who received ≥1 dose of apremilast, regardless of when they were randomized.‡Represents the number of patients with ≥1 occurrence of the abnormality at any time point/number of patients with ≥1 post-baseline value.Mease et al, ACR 2013 Abstract # 348

PALACE 1, 2 and 3

Apremilast for the Treatment of Behçet’s Syndrome: A Phase IIBehçet s Syndrome: A Phase II

Randomized, Placebo-Controlled, Double-Blind StudyDouble Blind Study

Gulen Hatemi1; Melike Melikoglu1; Recep Tunc2; Cengiz Korkmaz3; Banu Turgut Ozturk2; Cem Mat1; Peter A. Merkel4; Kenneth T. Calamia5;

Ziqi Liu6; Lilia Pineda6; Randall M. Stevens6; Hasan Yazici1; Yusuf Yazici7

1Istanbul University Cerrahpasa Medical School, Istanbul, Turkey; 2Selçuk University, Konya, Turkey; 3Eskişehir Osmangazi University, Eskişehir, Turkey; 4Boston University School of Medicine, Boston,

MA, USA; 5Mayo Clinic College of Medicine, Jacksonville, FL, USA; 6Celgene Corporation, Warren, NJ, USA; 7New York University School of Medicine New York NY USA

30

Hatemi et al, ACR 2013 Abstract # 761

USA; New York University School of Medicine, New York, NY, USA.

Primary End Point: Mean Number of Oral Ulcers at Week 12

3.5

Intent-to-Treat Population, LOCF (N=111)

2.1 (2.58)2.5

3.0

3.5

Ora

l Ulc

ers Placebo crossover to

apremilastApremilast discontinuation

0.5 (1.03)*1.0

1.5

2.0

n N

umbe

r of

wk0 wk2 wk4 wk6 wk8 wk10 wk12 wk14 wk16 wk18 wk20 wk22 wk24 wk26 wk28Placebo 2.9 1.7 1.9 1.9 1.6 1.4 2.1 0.4 0.4 0.6 0.5 0.3 0.4 1.3 1.6

0.0

0.5

Mea

n

30 mg BID 2.7 0.3 0.7 0.5 0.5 0.7 0.5 0.6 0.6 0.6 0.7 0.2 0.6 1.9 1.7

• 71% Complete Response, 89% Partial Response

31

*P<0.0001 vs. placebo.

Hatemi et al, ACR 2013 Abstract # 761

Q & AQ & A

32

I&I Global MarketingMark Kreston

VP I&I Gl b l M k tiVP, I&I Global Marketing

The Multinational Assessment of Psoriasisand Psoriatic Arthritis (MAPP) Survey

Conducted in 2012 by Celgene and overseen by an academic steeringcommittee of dermatology and rheumatology experts from around the world

3,426 PATIENTS WERE SURVEYED FROM 139,948 SCREENED HOUSEHOLDS IN7 COUNTRIES IN NORTH AMERICA AND EUROPE

34

Symptoms Contributing to Disease Severity

Psoriasis Patients (%) PsA Patients (%)Psoriasis Patients (%)

Bleeding, 3%

PsA Patients (%)

Lack of Sleep

Bleeding, 1%

Other, 9%

Itching, 38%Pain/Swelling of Joints, 5%

Lack of Sleep, 3%

gOther, 13%

Itching, 18%

Location/Size,

Lack of Sleep, 7%

, %

Location/SizeScales 11%

Flaking, 10%

, ,10%

Scales,5%Pain/Swelling of

Joints, 45%

Location/Size, 17%

Scales, 11%

Flaking, 5%

35

Lebwohl M, et al. Poster presented at the 22nd annual meeting of the European Academy of Dermatology and Venereology, 2013.

Medical Care and Treatment of PsA and Psoriasis

No Rx Topical only Oral +/- topical Oral + biologic Biologic +/- topical

2

8 55

14

80

100

8

55 52

31

19

40

60

Patie

nts

(%)

59% of PsA

32 3728

20

40P

patients are on No Rx or Topicals only

0 Moderate Psoriasisn=393

Severe Psoriasisn=166

PsA patientsn=712

Body Surface Area

36

Lebwohl M, et al. Poster presented at the 22nd annual meeting of the European Academy of Dermatology and Venereology, 2013.

Body Surface AreaData does not include use of phototherapy as monotherapy for psoriasis.

Patients Report an Overwhelming Desirefor Improved Therapies

54%of psoriasispatients and

57%of psoriasispatients and85%p

52%of psoriatic

arthritispatients said

biologic

p

45%of psoriatic

arthritispatients

discontinued

85%of Patients

Reported a Needfor Betterbiologic

therapy wasburdensome

discontinuedtheir biologic

therapyTherapies

37

Desired Attributes in PsA and PsO therapy

Efficacy SafetyStrong and durable

efficacy acrossmultiple disease

A unique safetyprofile with no known apparent risk ofmultiple disease

manifestationsapparent risk of malignancy or serious infection, and no exacerbation of comorbidities

Ease of UseA convenient treatment regimen

with oral dosing and lack of screening / lab monitoring

requirements

38

Apremilast Profile Has High Preference for Both Rheumatologists and Patients

Question: For your next 100 active Psoriatic Arthritis patients, to what percentage would you prescribe each option?

Question: If your physician provided you with the opportunity to SWITCH or ADD any one of the following products, which one, if any, would you choose?

36% 35%33%

30%

26%

n = 100 n = 31

16%

11% 10%

3%

11%

Stay with current therapy

Apremilast Methotrexate Humira Enbrel

39

Source: Research conducted between March 25th and April 15th 2013

Apremilast Enbrel Humira Methotrexate

Rheumatologists Plan To Use Apremilast Before Biologics

Question: Of your next 100 patients on Apremilast, what percent of patients would you distribute to each of the following points in the therapy sequence?

~66% of patients would be pre-biologic

12% 54% 34%12% 54% 34%

0% 50% 100%

Before 1st DMARD DMARD Experienced After ≥ 1 biologic

40

Before 1st DMARD DMARD Experienced After ≥ 1 biologic

Source: National Analysts, n=150 U.S. rheumatologists, Review of Apremilast PALACE 1-3 24 week Target Product Profile Q3 2013

PsA Launch ReadinessTerrie Curran

S CVP – US Commercial

Building a Global BrandApremilast PsA Strategic Imperatives

Increase rheumatologists’ appreciation of the unique characteristicsof PsA and the major unmet market need11

Build a strong brand that will transform the way physicians treat psoriatic disease22

Generate awareness of apremilast among patients anddrive brand requests33 q

Ensure access and successful trial and adoption44

Establish Celgene I&I as acommitted and trusted new partner55

42

committed and trusted new partner

Launch Leadership Team Actively Managing Critical Launch Activities

Ensuring Accurate Demand Forecasting/ Securing Favorable Hiring and EffectivelyDemand Forecasting/ Readiness for Early

Launch

Securing Favorable Market Access On-Boarding the

Salesforce

• Pressure testing multiple scenarios and developing mitigation plans

• Identifying potential

• Both Regional Medical Liaisons and market access team expanded and in field

• Key Leadership in place:

– RSDs and DSMs

• Field Force recruitment Identifying potential actions that can be implemented at risk to expedite supply availability

• Conducting research with payer and Specialty Pharmacy for input in to the US Market Access strategy

on track:

– Recruiting Rheumatology Sales Force

strategy

• Initial discussions with payers

– On-board Jan 2014

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“Aggressive Customizers” and “High Biologic Users”Are Priority Segments

% of Rheums % of TRx Summary

C Hi h t TR l“Aggressive Customizers” 32% 50% • Highest TRx volume • Earlier adopters• Use broad range of drugs• Ready to embrace new optionsReady to embrace new options

“High Biologic Users ” 23% 25% • Efficacy-focused MDs who readily use biologics

• Least concerned re risks associated w/ biologicsassociated w/ biologics

“Low Volume/Conservative”

18% 3% • Desire to defer biologics• Slow Adopters

“C ti S iti t t d f t / i k“CautiousCost Conscious”

27% 22% • Sensitive to cost and safety/risk profile of products

• Slow adopters

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*% of TRx = Segment share of sum of DMARD Market TRx and Biologic Market TRx.

Patient Support Programs Are Critical Drivers of Pharmaceutical Adoption and Patient Adherence

Program Type

Brand

Starter Kits Sampling CouponsCustomer Cost

Savings –Annual OOP

Patient Assistance

Brand

• Rebates• Trial Offer $60 YES

• Trial Offer$• Rebates

• Loyalty Card$60 YES

• Support card • Trial Offer• Rebates $0 YESRebates• Brand Loyalty

Card

$0 YES

• Cost Support• Co-pay $60 YES

Reduction Card

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Sources: Campbell Alliance Analysis, September 2013; Brand and manufacturer websites, accessed September 2013.Notes: OOP=out-of-pocket; Trial Offer= represents a temporary free or discounted offer, to encourage patients to purchase products; Copay Reduction/Savings Card is a card provided to a patient that sets a maximum co-payment for a specific transaction or time period.

Building Towards a World Class Launch

• Secured US commercial organization: sales management, marketing, operations, market access & training

• Secured additional payer and contracting capability WorldSecured additional payer and contracting capability. payer profiling has commenced

• RML structure expanded

• Accelerating all key US branding activities

Class LaunchQ1.14

Accelerating all key US branding activities

• Building premium patient and physician support offering to simplify and expedite access

• Finalizing pricing and payer contracting strategyFinalizing pricing and payer contracting strategy

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SummaryScott Smith

S G f &Senior VP, Global Head of I&I

PALACE 1-4: ACR 20 Response Through Week 52 Observed Data 30 mg BID

80Primary End-Point

50

60

70a y d o t

63%59%55%53%

30

40

50 53%

10

20

0week 0 week 16 week 24 week 40 week 52

PALACE 1 PALACE 2 PALACE 3 PALACE4

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ACR 20 Results at 1 Year

ACR20 at ~1 year

80Data as Observed

55 53

6359

6460

67

60

70

CR

20

48

30

40

50

s ac

hiev

ing

AC

10

20

30

% o

f pat

ient

s

0APR 30mg

OralPALACE 1

APR 30mgOral

PALACE 2

APR 30 mgOral

PALACE 3

APR 30 mgOral

PALACE 4

Enbrel25mg SC

Stelara90mg SC

PSUMMIT2

Stelara90mg SC

PSUMMIT1

Simponi 50mg SC

*

Data from individual studies

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Source: Mease et al J Rheumatol 2006; Ritchlin ACR 2013; McInnes Lancet 2013; Kavanaugh et al Arthritis Rheum 2012.

Data from individual studies

Phase III Safety Profile Differentiated

Apremilast* DMARDs Biologics

Serious infection

TB and opportunistic infection

Increased frequency of ymalignancies

Requirements for monitoring

Organ toxicity

Pregnancy Category C/B

GI tolerability

Competitive Advantage Competitive Disadvantage

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p g p g

*Based on Phase III 52 week data vs. MTX and biologic PIs

Apremilast POSTURE Trial in Ankylosing Spondylitis

• Fully enrolled months ahead of scheduleschedule

• More than 2 million patients in the US and EU

• No oral disease-modifying treatment options

• Top Line data expected H1:14• Top-Line data expected H1:14

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I&I Pipeline Projects Continue To Advance

Preclinical Phase I Phase II Phase III Regulatory Filing& Approval

APREMILASTPsoriatic Arthritis

Psoriasis

Ankylosing Spondylitis

Behçet’s Disease

Crohn’s Disease

Ulcerative Colitis

Atopic Dermatitis

SOTATERCEPT (ACE-011)SOTATERCEPT (ACE-011)Renal Anemia / Bone

POMALIDOMIDESystemic Sclerosis (POC)

CC 292CC-292SLE/B-Cell mediated diseases (POC)

CC-220Systemic Sclerosis / SLE / Sarcoidosis

CC 90001CC-90001IPF / Liver Fibrosis

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I&I Pipeline Projects Continue To Advance

Preclinical Phase I Phase II Phase III Regulatory Filing& Approval

APREMILASTPsoriatic Arthritis

Psoriasis

Ankylosing Spondylitis

Behçet’s Disease

PsO NDA and combined PsA/PsO MAA

Crohn’s Disease

Ulcerative Colitis

Atopic Dermatitis

SOTATERCEPT (ACE-011)

PsA/PsO MAA on track Q4:13

SOTATERCEPT (ACE-011)Renal Anemia / Bone

POMALIDOMIDESystemic Sclerosis (POC)

CC 292CC-292SLE/B-Cell mediated diseases (POC)

CC-220Systemic Sclerosis / SLE / Sarcoidosis

CC 90001CC-90001IPF / Liver Fibrosis

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I&I Has Transformational Potential for Patients and for Celgene

Apremilast

• 52 week data for apremilast supports i ifi t t it lti l

Potential to createa new cornerstone

significant opportunity across multiple segments of the market

• Submissions progressing on schedule

for treatment ofpsoriatic diseaseas well as other

serious immunologic

• Preparations for Q1:14 Launch ongoing

• Commercial build-out on track, hiring talented, experienced leadership in Sales, g

diseases, p p ,

Marketing and Medical Affairs

• ACR is a significant meeting in advance of the US PsA launch

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Celgene I&I Overview

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