October 27, 2016€¦ · 2016-10-27 · Q&A Scott Smith, President, Global I&I Jackie Fouse, ... 2016 adjusted diluted EPS raised from $5.70-$5.75 to $5.88-$5.92 2016 REVLIMID®

Q3 2016 Conference CallOctober 27, 2016

Q3 2016 Earnings Call

Mark Alles, Chief Executive Officer

Peter Kellogg, Chief Financial Officer

Michael Pehl, President, Hematology & Oncology

Q&A

Scott Smith, President, Global I&I

Jackie Fouse, President & Chief Operating Officer

2

Forward Looking Statements and Adjusted Financial Information

This presentation contains forward-looking statements, which are generally statements that are not historical facts. Forward-lookingstatements can be identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates,” “plans,” “will,” “outlook” and similarexpressions. Forward-looking statements are based on management’s current plans, estimates, assumptions and projections, and speakonly as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information orfuture events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which aredifficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, many of which are discussed in more detail in our Annual Report onForm 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 financialmeasures. Further information relevant to the interpretation of adjusted financial measures, and reconciliations of these adjusted financialmeasures 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.

3

Mark Alles

Q3 2016: Exceptional Results; Advancing Transformational Opportunities

5

Exceptional Q3:16 performance driven by increasing demand and duration for key products Achieving meaningful leverage while advancing next-generation growth drivers Updating 2016 guidance

Delivering Outstanding ResultsDelivering Outstanding Results

Key data supports transformative potential of late-stage assets Continued geographic expansion and market access driving global growth Expect to achieve high-end of the 2017 net product sales and adjusted EPS targets

Accelerating Near-Term GrowthAccelerating Near-Term Growth

Filed 8 investigational new drug applications year-to-date Investing in and advancing high-potential next generation therapies Well-positioned to deliver strong growth through 2020 and beyond

Driving Innovation & Long-Term Growth Driving Innovation & Long-Term Growth

Peter Kellogg

Strong product growth across all franchises Continue to invest in R&D to advance the pipeline

Exceptional Q3 2016 Operating Results

7

Q3:16 year-over-year net product sales grew 28% and adjusted diluted EPS grew 28% Adjusted operating margins improved by 80 bps

Financial HighlightsFinancial Highlights

Excellent Performance on Operating MetricsExcellent Performance on Operating Metrics

2016 adjusted diluted EPS raised from $5.70-$5.75 to $5.88-$5.92 2016 REVLIMID® net product sales and total net product sales guidance updated 2017 REVLIMID® net product sales, total net product sales and adjusted diluted EPS targets updated

2016 Guidance and 2017 Targets Updated2016 Guidance and 2017 Targets Updated

Total Net Product Sales

Q3:14 Q3:15 Q3:16

$1,957

$2,313

$2,969

8

$ M

illio

ns ↑19% ↑18% ↑28%

$0

$500

$1,000

$1,500

$2,000

$2,500

$3,000

Q3:15 Volume Price Fx /Hedge

Q3:16

↑28.4%↓0.7%↑24.6% ↑4.5%

Contribution to Q3:16 Total Net Product Sales Growth

$ M

illio

ns

Total Net Product Sales

Footnote: Growth Rates = Growth vs. Prior Year Period

Adjusted Diluted Earnings Per Share

$0.97

$1.23

$1.58

Q3:14 Q3:15 Q3:16

9

↑24% ↑27%

Dol

lars

Per

Sha

re

↑28%

Q3:15 Oper. Income

OIE Tax Rate

Share Count

Q3:16

Dol

lars

Per

Sha

re

$1.58$0.40$1.23 ($0.03) $0.04

Contribution to Q3:16 Adjusted Diluted EPS

($0.06)

Adjusted Diluted EPS

Footnote: Growth Rates = Growth vs. Prior Year Period

Q3:16 ∆ vs.Q3:15

YTD:16 ∆ vs.YTD:15

Product Gross Margin 96.6% ↑100 bps ↑80 bps

R&D expenses% of revenue

$644M21.6% ↑70 bps ↑150 bps

SG&A expenses% of revenue

$591M19.8% ↓50 bps ↓260 bps

Operating Margin 55.3% ↑80 bps ↑200 bps

Effective Tax Rate 15.4% ↑170 bps ↑70 bps

Key P&L Line Items (Adjusted)

10

Cash and Marketable Securities

• Cash flow from operations was approximately $723M during Q3:16• In Q3:16, purchased $273M of shares; YTD purchased $2.0B

$4.9B remaining under stock repurchase program at 9/30/16• Six strategic business development transactions YTD 2016

Upfront research and development expense of approximately $1.3B

11

(in Billions) 9/30/16 12/31/15

Cash and Marketable Securities $6.87 $6.55

Previous 2016 Guidance Updated 2016 Guidance

REVLIMID® ~$6.8B ~$7B

Total Net Product Sales ~$11B ~$11.2B

Adjusted Diluted EPS $5.70-$5.75 $5.88-$5.92

Updating 2016 Guidance and 2017 Targets

12

Previous 2017 Targets Updated 2017 Targets

REVLIMID® ~$8B >$8B

Total Net Product Sales $12.7B - $13.0B High end of the range

Adjusted Diluted EPS $6.75 - $7.00 High end of the range

Michael Pehl

Q3 2016 Hematology & Oncology Franchise Results

14

– Q3:16 net sales growth of +24% Y/Y; +8% Q/Q– Worldwide record performance for REVLIMID® and POMALYST®/IMNOVID®

Strong Net Product Sales and Franchise Operating MomentumStrong Net Product Sales and Franchise Operating Momentum

– REVLIMID® NDMM share increasing globally; Reimbursement secured in key markets– Increasing utilization of REVLIMID®- and POMALYST®-based triplets; Driving share and duration– ABRAXANE® maintains demand in U.S. while growing outside the U.S. for pancreatic cancer

2016 Product Growth Drivers On-Track2016 Product Growth Drivers On-Track

– NDA submission of enasidenib (AG-221) in RR AML by year-end– CC-122, durvalumab and luspatercept programs advancing– Acquisition of EngMab AG uniquely positions Celgene to pursue BCMA development in MM and NHL

Long-term Growth Drivers AdvanceLong-term Growth Drivers Advance

Q3 2016 REVLIMID® Net Sales Summary

15

$633 $760 $895$1,153

$457$540

$558

$738

Q3:13 Q3:14 Q3:15 Q3:16

US ROW

$1,090

$1,300$1,453

$1,891Current Results & Future Growth DriversCurrent Results & Future Growth Drivers

• Q3:16 net sales $1,891M; +30% Y/Y; +11% Q/Q• 2016 Growth Drivers

REVLIMID® strong duration and share trends globally NDMM launch momentum in U.S. and EU launch

countries Reimbursement in Italy; France expected in Q1:17

Russian tender in Q3:16

• Future Growth Drivers– Triplet combinations continue to become standard of care– REVLIMID® maintenance post-ASCT submitted in EU;

U.S. submitted in Q3:16, PDUFA date of Feb 24, 2017– Myeloma XI data expected at ASH– Updated NCCN guidelines and regulatory submissions in

combination with novel agents

Net Sales ($M)

Q1 2015 Q2 2015 Q3 2015 Q4 2015 Q1 2016 Q2 2016 Q3 2016 Q4 2016

Global REVLIMID® Multiple Myeloma Momentum and Opportunity Continues to Grow

Vd control arms from ENDEAVOR/PANORAMA


ASH 2015 and SWOG data release


Updated mSMART & NCCN Guidelines


Rd approval in ndMM

Rd approval in ndMM

Phase III Rd+X readouts and FDA approvals


Rd+X clinical experience, later line market expansion

REVLIMID® re-challenge



16

Rd+Dara POLLUX, REVLIMID®

Post-ASCT Maintenance US & EU Submissions

Rd+Dara POLLUX, REVLIMID®

Post-ASCT Maintenance US & EU Submissions

Q1 2015 Q2 2015 Q3 2015 Q4 2015 Q1 2016 Q2 2016 Q3 2016 Q4 2016

Global REVLIMID® Multiple Myeloma Momentum and Opportunity Continues to Grow




Rd approval in ndMM




17

REVLIMID® Post-ASCT Maintenance US & EU

Submissions

• Myeloma XI & Final MM-020 OS at ASH 2016

• NCCN Guidelines update• Regulatory submissions

with novel agents

• Myeloma XI & Final MM-020 OS at ASH 2016

• NCCN Guidelines update• Regulatory submissions

with novel agents

Q3 2016 POMALYST®/IMNOVID® Net Sales Summary

18

$77$118

$150$203$13

$63

$107

$138

Q3:13 Q3:14 Q3:15 Q3:16

US ROW

$90

$181

$257

$341Current Results & Future Growth DriversCurrent Results & Future Growth Drivers

• Q3:16 net sales $341M; +33% Y/Y; +7% Q/Q• Global launch continues

– Strong volume and duration trends– All time record TRx and NRx in U.S. in Q3:16

• 2016 Growth Drivers Market share continues to increase in reimbursed

markets in Europe Duration increasing across geographies

• Future Growth Drivers IMNOVID® EU approval for renal impairment data Updated NCCN guidelines and regulatory submissions in

combination with novel agents Enrollment of OPTIMISMM® (N=544) phase III

combination study with bortezomib expected to complete in H1:17

Net Sales ($M)

Q3 2016 ABRAXANE® Net Sales Summary

19

$132 $151 $145 $144

$38

$61 $85 $89

Q3:13 Q3:14 Q3:15 Q3:16

US ROW

$170

$212$230 $233

Current Results & Future Growth DriversCurrent Results & Future Growth Drivers• Q3:16 net sales $233M; +1% Y/Y; -6% Q/Q • 2016 Drivers

ABRAXANE® is a standard of care in the U.S. for metastatic pancreatic cancer

Increasing market share in Europe especially in metastatic pancreatic cancer

U.S. demand in breast and lung cancer stabilized• Future Growth Drivers

Data from abound® program at IASLC/WCLC Data from I/O combinations expected beginning in 2017 Phase III adjuvant pancreatic trial (apact®) data

expected in 2017

Net Sales ($M)

Q4 2016 Key Data Presentations

December:• Data from the abound® program with ABRAXANE® in NSCLC• Phase III Myeloma IX: REVLIMID® as induction and maintenance in

NDMM following ASCT• Final OS from MM-020• REMARC and CONTINUUM®

• Interim data from MAGNIFYTM with REVLIMID® in indolent lymphoma• CC-122 in combination with obinutuzumab in NHL• Phase II tnAcity® trial: ABRAXANE® in TNBC

November:• Phase I trial with marizomib in combination with bevacizumab in

recurrent glioblastoma*

20

* In collaboration with Triphase Accelerator Corporation

Luspatercept pivotal studies in MDS and beta-thalassemia advancing Significant progress with protein homeostasis and I/O pipeline; CC-122 and CC-90009 trials enrolling EngMab AG lead molecule EM901 highly complementary to our MM portfolio

Q3 and FY2016 Outstanding Progress; Accelerating Momentum

21

REVLIMID® as a backbone of therapy across all lines of therapy REVLIMID® net product sales guidance for 2016 now ~$7B POMALYST®/IMNOVID® sales strong; ABRAXANE® stable

REVLIMID® maintenance post-ASCT filed in the U.S.; Decisions for U.S. and EU expected in 2017 POMALYST®/IMNOVID® U.S. and EU labels updated with renal impairment data NDA submission of enasidenib (AG-221) in RR AML in the U.S. by YE16

Early- and Mid-Stage Programs AdvanceEarly- and Mid-Stage Programs Advance

Robust Results from Key Products

On-Track with Key Regulatory Activities

Scott Smith

Q3 2016 I&I Franchise Results

23

– Revenue continued to accelerate through Q3:16– Strong TRx trajectory relative to recent market entrants– Strengthening position in psoriasis and PsA as prescriber adoption, market share, and persistency continue to increase

Accelerating Sales Performance and Momentum for OTEZLA®Accelerating Sales Performance and Momentum for OTEZLA®

– Access and reimbursement in 20 countries to-date– Preparing for launch in major European markets and Japan– Advancing regulatory submissions in Eastern Europe, Asia and Latin America

Expanding OTEZLA®’s Global FootprintExpanding OTEZLA®’s Global Footprint

– Executing registration program for GED-0301 in Crohn’s disease; Phase Ib clinical & endoscopy outcomes at UEGW– GED-0301 UC phase II enrollment complete– Executing pivotal programs for ozanimod in MS and UC– Ozanimod Crohn’s disease phase II enrollment complete– CC-220 phase IIa showed positive trend across multiple measures of SLE disease activity, compared to placebo

Advancing Development of I&I PipelineAdvancing Development of I&I Pipeline

• Q3:16 net sales $275M; +98% Y/Y, +14% Q/Q growth

• Increasing OTEZLA® adoption in the U.S. amidst seasonal market declines in prescribing

• Access gains enabling launches in several key EMEA markets

• Achieved reimbursement in the UK and France, launching in Q4:16

• Longer-term data enhancing clinical profile

Advancing Revenue Growth Drivers for OTEZLA®

24

Current Results & Future Growth Drivers

$167 $175$217

$245

$16 $21

$25

$30

Q4:15 Q1:16 Q2:16 Q3:16US ROW

$183 $196

$242

$275

Net Sales ($M)

14% 10%

43%41%

8%7%

35% 42%

Aug-15 Jul-16

Biologics TopicalsOral Systemics No tx in prior 6 months

0%

5%

10%

15%

20%

25%

30%

35%

40%

ENBREL STELARA HUMIRACOSENTYX OTEZLA

Market Dynamics Supporting Positive US Launch Performance

25

• Systemic treatment market share continues to grow, following on sustained leadership in new-to-brand share, for both PsA and psoriasis

• Maintaining pre-biologic source of business, despite new market entrants

• Continuing to see favorable treatment retention

Note: Symphony data is subject to restatement; source of business calculated for new-to-brand patientsSource: Symphony Prescriber-level data through week ending 23 September 2016; SHS PTD claims data. Sept ‘16 feed for month ending July ‘16

OTEZLA®

22.4%

90%

Pre

-bio

logi

c

Psoriasis Market Share Psoriasis Source of Business (Based on therapy prior to OTEZLA® in the last 6 months)

Advancing Development of GED-0301

26

Objective: To explore signs of endoscopic improvement and clinical activity in a difficult-to-treat, moderate-to-severe Crohn’s patient population with confirmed endoscopic inflammation at baseline, including but not limited to:

• Both TNF-naïve and prior TNF-exposed patient population

• Prior surgeries• Proximal and distal disease

All patients had significant clinical and endoscopic disease at entry and spanned a broader geography

CD-001 Study OverviewCD-001 Study Overview

UC-002: UC POC (n=41)

UC-002: UC POC (n=41)

CD-002: 52 Week Pivotal Ph III (n=1,064)

CD-002: 52 Week Pivotal Ph III (n=1,064)

CD-003: 12 Week Pivotal Ph III (n=798)

CD-003: 12 Week Pivotal Ph III (n=798)

2015 2016 2017 2018 2019

Approval

CD-005: PD

(n=20)

CD-005: PD

(n=20)

CD-004: Long Term Extension (n~1,400)

CD-004: Long Term Extension (n~1,400)

CD-001: Exploratory Ph Ib

(n=63)

CD-001: Exploratory Ph Ib

(n=63)IGON-1*

Ph II POC (n=166)IGON-1*

Ph II POC (n=166)

201420132012

* Placebo-controlled phase II proof-of-concept trial

Emerging GED-0301 Profile to Provide Differentiated Benefit/Risk

27Evidence demonstrated in IGON-1 trial to-dateLegend: To be assessed in future trials

Clinical Response and Remission


Once-dailyOral DosingOnce-daily

Oral Dosing

Negligible Systemic Exposure


Endoscopic ImprovementEndoscopic

Improvement

Efficacy in TNF-exposed PatientsEfficacy in TNF-

exposed Patients

Rapid Onset of Clinical EfficacyRapid Onset of

Clinical Efficacy

Efficacy in Left-sided Disease


Long-term Endoscopic Remission


GED-0301AEs and SAEs

Similar to PlaceboAEs and SAEs

Similar to Placebo

IGON Program Findings

Emerging GED-0301 Profile to Provide Differentiated Benefit/Risk

28Evidence demonstrated in IGON-1 and CD-001 trials to-dateLegend: To be assessed in future trials



Once-dailyOral DosingOnce-daily

Oral Dosing



Endoscopic ImprovementEndoscopic

Improvement

Efficacy in TNF-exposed PatientsEfficacy in TNF-

exposed Patients

Rapid Onset of Clinical EfficacyRapid Onset of

Clinical Efficacy





GED-0301AEs and SAEs

Similar to PlaceboAEs and SAEs

Similar to Placebo

Updated GED-0301 Program Findings

Q4 2016 Key Data Presentations

November:• OTEZLA® 4-year efficacy and safety in psoriatic arthritis

• OTEZLA® pooled 3-year safety in psoriatic arthritis from PALACE 1-3

October:• GED-0301 exploratory phase Ib in Crohn’s disease• RPC4046 proof of concept phase II in eosinophilic esophagitis• Ozanimod TOUCHSTONE (phase II) open-label extension efficacy and

safety in ulcerative colitis• OTEZLA® pooled 3-year results from ESTEEM 1 and 2 in psoriasis and

PALACE 1-3 in psoriatic arthritis• OTEZLA® phase IIb in Japanese patients with psoriasis• CC-220 phase II in systemic lupus erythematosus

December:• GED-0301 exploratory phase Ib in Crohn’s disease

29

Maximizing I&I Portfolio Value

30

Continue strong operating momentum for OTEZLA® in the U.S. Accelerate uptake in early launch markets Advance launch preparations for multiple major European markets and Japan Expand eligible patient base across geographies and indications

Maximizing the OTEZLA® Opportunity

Advance ozanimod phase III trial in ulcerative colitis Complete ozanimod phase II trial in Crohn’s disease Progress enrollment of GED-0301 pivotal trials in Crohn’s disease Complete GED-0301 phase II trial in UC

Moving Ozanimod and GED-0301 Forward

Complete CC-220 phase IIb trial in cutaneous lupus Initiate CC-90001 idiopathic pulmonary fibrosis phase II trial

Advancing Development of the I&I Pipeline

Jackie Fouse

2016 Anticipated Milestones

Regulatory Submissions/Decisions Submit REVLIMID® in U.S. and EU for maintenance post-ASCT Submit POMALYST® renal impairment data in US and EUX Submit ABRAXANE® for early-stage breast cancer in EU Submit OTEZLA® for PSOR in Japan CHMP opinion on REVLIMID® for MCL

Trial Enrollment Complete enrollment in AUGMENT® – REVLIMID® in RR FL Complete enrollment in apact® – ABRAXANE® in adjuvant PanC Complete enrollment in RELIEF® – OTEZLA® in Behçet’s disease Complete enrollment in Ph II trial of CC-486+pembrolizumab in NSCLC Initiate enrollment in Ph I trial of BCMA CART in RRMM Initiate enrollment in FUSION™ program with durvalumab in NDMM,

RRMM, NHL, MDS/AML

Trial Initiations Initiate pivotal trial with CC-122 in NHL Initiate Ph III trial with OTEZLA® in AD Initiate second Ph III trial with GED-0301 Initiate Ph III trial with RPC4046 in EoE

Financial Performance Total Net Product Sales ~$11.0B1 Total Net Product Sales ~$11.2B2

Net REVLIMID® sales ~$6.8B1 Net REVLIMID® sales ~$7.0B2

Adj. operating margin ~54.0%1

Adj. EPS $5.70-$5.751 Adj EPS $5.88 to $5.922

32

Clinical DataX Ph III REMARC – REVLIMID® in DLCBL maintenance Ph III CONTINUUM® – REVLIMID® in CLL maintenanceX Ph III ETNA – ABRAXANE® in neoadjuvant BC Ph III POSTURE® – long-term radiographic data of OTEZLA® in AS Ph III PSA-006 – OTEZLA® in biologic-naïve PsA Ph II CC-122 in NHLX Ph II motolimod (VTX-2337) in SCCHN and ovarian cancer Ph II portion of tnAcity® – ABRAXANE® in TNBC Ph II OTEZLA® in AD ✓ and UC Ph II CC-220 in SLE Ph II RPC4046 in EoE Pharmacokinetic comparability study – OTEZLA® once-daily formulation

R&ED File at least 8 IND’s Advance at least 2 compounds to mid-to-late stage development

1. July 2016 2. Updated October 2016

Pipeline Acceleration Through YE 2018

33

Phase III Data 1919

>20>20

15-1715-17

Phase II and POC Data

New INDs

Driving Sustainable High Growth

News flow from medical meetings on commercial products and pipeline

Strong commercial momentum

On-track for 2017 and 2020 targets

34

Q&A

Appendix

Celgene Pipeline

37

Celgene Pipeline

38

Celgene Pipeline

39

Celgene Pipeline

40

REVLIMID® Multiple Myeloma Late Stage Programs

Patient Population Maintenance Post-VMP induction

Trial NameMM-026ARUMM

Phase III

Target Enrollment 350

Design

2:1 randomizationInduction with Melphalan/prednisone/bortezomib (VMP)

for 6-9 cyclesArm A: REVLIMID® (10mg) d 1-21

for 28-day cycleArm B: Placebo d 1-21 for 28-day cycle

Primary Endpoint Progression Free Survival

Status Trial enrolling

41

REVLIMID® Multiple Myeloma Late Stage Programs

Patient Population Induction and Maintenance in ASCT EligibleTrial Name MYELOMA XI

Phase III

Target Enrollment 3,970

Design

Arm A: Cyclophosphamide (500mg) d1,8,15; THALOMID® (100mg d1-21 then 200mg daily), Dexamethasone (40mg) d1-4, 12-15 for minimum of 4 21-day cycle

Arm B: REVLIMID® (25mg) d 1-21, Cyclophosphamde (500mg) d1,8, dexamethasone (40mg) d1-4,12-15 for minimum of 4 28-day cycles

Arm C: Cyclophosphamde (500mg) d1,8, Carfilzomib (20 mg/m2) d 1,2 cycle 1 then (36 mg/m2) d 1,2,8,9,15,16, REVLIMID® (25mg) d1-21, Dexamethasone (40mg) d 1-4,8,9,15,16 for 4 21-day cycles

Patients with no change, progressive disease, PR or MR randomized toArm A: Bortezomib (1.3mg/m2) d 1,4,8,11, Cyclophosphamide (500mg) d 1,8,15, Dexamethasone (20mg) d 1,2,4,5,8,9,11,12 for

max of 8 21-day cyclesArm B: No treatmentAll patients go to SCT

After SCT randomization to:Arm A: REVLIMID® (10mg) d 1-21 for 28-day cycle to disease progression

Arm B: No maintenance

Primary Endpoint Overall Survival and Progression Free Survival

Status Data expected at ASH 2016

42

POMALYST®/IMNOVID® Multiple Myeloma Late Stage Programs

Patient Population RRMM

Trial NameMM-007

OPTIMISMM®

Phase III


Design

Arm A: POMALYST®/IMNOVID® (4mg), bortezomib (1.3 mg/m2 IV) and low-dose dexamethasone to disease

progressionArm B: Bortezomib (1.3 mg/m2 IV) and low-dose

dexamethasone to disease progression


Status Trial enrolling; Data in 2018E

43

MDS/AML/MF Late Stage Programs

Patient Population Low risk/INT-1 transfusion-dependent MDS Post induction AML Maintenance

MoleculeCC-486

(Oral Azacitidine)CC-486

(oral azacitidine)

Trial Name AZA-MDS-003 CC-486-AML-001

Phase III III

Target Enrollment 386 460

DesignArm A: CC-486 (150mg or 200mg)

Arm B: PlaceboArm A: CC-486 (150mg or 200mg)

Arm B: Best Supportive Care

Primary Endpoint RBC-transfusion independence for more than 12 weeks Overall Survival

Status Trial enrolling Trial enrolling

44


Patient Population Anemia in to Very Low-, Low-, or Intermediate-Risk MDS

Red Blood Cell Transfusion Dependent Beta-Thalassemia

Molecule Luspatercept Luspatercept

Trial Name MEDALISTTM BELIEVETM

Phase III III


DesignArm A: Luspatercept (Starting dose of 1.0 mg/kg

subcutaneous injection every 3 weeksArm B: Placebo (Subcutaneous injection every 3 weeks)

Arm A: Luspatercept (1mg/kg plus Best Supportive CareArm B: Placebo plus Best Supportive Care

Primary Endpoint Red Blood Cell Transfusion Independence (RBC-TI) ≥ 8 weeks

Proportion of subjects with hematological improvement from Week 13 to Week 24 compared to 12-week prior to

randomizationHematological improvement from Week 13 to Week 24

compared to the 12-week.

Status Trial enrolling Trial enrolling

45


Patient Population IDH2 Mutant AML

Molecule Enasidenib (AG-221, CC-90007)

Trial Name IDHENTIFYTM

Phase III


Design Arm A: Enasidenib (100 mg daily , 28-day cycle) + Best supportive care

Arm B: Best supportive care

Primary Endpoint Overall survival

Status Trial enrolling

46

REVLIMID® Chronic Lymphocytic Leukemia Late Stage Program

Patient Population Maintenance in 2nd Line CLL

Trial NameCLL-002

CONTINUUM®

Phase III


DesignArm A: REVLIMID® (starting dosage 2.5mg/day escalated to

10mg/day) until disease progression - 28-day cycleArm B: Placebo

Primary Endpoint Overall Survival and ProgressionFree Survival

StatusEnrollment complete

Data expected at ASH 2016

47

REVLIMID® Lymphoma Late Stage Programs

Patient Population Maintenance in Patients with DLBCL responding to R-CHOP to induction therapy Newly Diagnosed Follicular Lymphoma

Trial Name REMARC RELEVANCE®

Phase III III

Target Enrollment 621 1,000

DesignArm A: REVLIMID® D1-21 of 28-day

cycle for 24 monthsArm B: Placebo D1-21 of 28-day

cycle for 24 months

Arm A: REVLIMID® (starting dose 20mg) D2-22 for up to 18 28-day cycles and Rituximab (starting

dose 375 mg/m2) weekly for up to 12 28-day cyclesArm B: Physician’s choice of rituximab-CHOP, rituximab-CVP

or rituximab-bendamustine

Primary Endpoint Progression Free Survival Complete Response Rate and Progression Free Survival

StatusPrimary endpoint met

Data expected at ASH 2016Enrollment complete

Data in 2017E

48


Patient Population Relapsed or Refractory Follicular Lymphoma Untreated Activated B-Cell DLBCL

Trial NameAUGMENTTM

NHL-007ROBUST®

DLC-002

Phase III III


Design

Arm A: REVLIMID® (10-20mg) D1-21 / Rituximab 375 mg/m2 weekly for cycle 1 then D 1 of cycles 2-5 for 5

28-day cyclesArm B: Placebo D1-21, / Rituximab 375 mg/m2 weeklyfor cycle 1 then D 1 of cycles 2-5 for 5 28-day cycles

Arm a: REVLIMID® (15mg) D1-14/+ R-CHOP21 for 6 21-day cycles

Arm B: Placebo + R-CHOP21 for 6 cycles

Primary Endpoint Progression Free Survival Progression Free Survival

StatusTrial enrollingData in 2017E

Trial enrollingData in 2019E

49


Patient Population Relapsed or Refractory Indolent Lymphoma

Trial NameMAGNIFYTM

NHL-008

Phase III


Design

Arm A: REVLIMID® (10-20mg) D1-21 / Rituximab 375 mg/m2 weekly for cycle 1 then D 1 of cycles 3, 5, 7,9 and 11 for 12 28-day cycles followed by REVLIMID® (10mg) D1-21 / Rituximab 375 mg/m2 D 1 of cycles 13, 15,

17,19, 21, 23, 25, 27 and 29 for 18 28-day cycles followed by REVLIMID® (10mg) D 1-21 until disease progression – 28 day cycle

Arm B: REVLIMID® (10-20mg) D1-21 / Rituximab 375 mg/m2 weekly for cycle 1 then D 1 of cycles 3, 5, 7,9 and 11 for 12 28-day cycles followed by REVLIMID® (10mg) D1-21 / Rituximab 375 mg/m2 D 1 of cycles 13, 15,

17,19, 21, 23, 25, 27 and 29 for 18 28-day cycles


StatusTrial enrollingData in 2020E

50

ABRAXANE® Solid Tumor Late Stage Programs

Patient Population Maintenance After Induction in Squamous Non-Small Cell Lung Cancer

Adjuvant Therapy in Surgically Resected Pancreatic Cancer

Trial Name NSCL-003PANC-003

apact®

Phase III III


Design

Induction: ABRAXANE® (100 mg/m2) D 1, 8,and 15 / Carboplatin (6 mg min/mL) D 1 for

4 21-day cyclesMaintenance:

Arm A: ABRAXANE® (100 mg/m2) D 1 and 8 plus BSC until disease progression –

21-day cycleArm B: BSC until disease progression

Arm A: ABRAXANE® (125 mg/m2) / Gemcitabine (1000 mg/m2) D 1, 8 and 15 for 6 28-day cycles

Arm B: Gemcitabine (1000 mg/m2) D 1, 8 and 15 for 6 28-day cycles.

Primary Endpoint Progression Free Survival Disease Free Survival

Status Trial enrollingEnrollment complete

Data in 2017E

51

ABRAXANE® Solid Tumor Late Stage Programs

Patient Population First-Line Triple Negative Metastatic Breast Cancer First Line Stage IIIB / IV Squamous NSCLC

Trial NametnAcity®

ABI-007-MBC-001NSCL-003

abound.sqm®

Phase II/III III

Target Enrollment 240/550 540

Design

Phase IIArm A: ABRAXANE® 1(25mg/m2) / Gemcitabine (1000 mg/m2) D 1

and 8 – 21-day cycleArm B: ABRAXANE® (125mg/m2) / Carboplatin AUC 2 IV, D 1 and

8 – 21-day cycleArm C: Gemcitabine (1000 mg/m2) / Carboplatin AUC 2 IV, D 1

and 8 – 21-day cyclePhase III

Arm 1: Selected phase II ABRAXANE® armArm 2: Gemcitabine (1000 mg/m2) / Carboplatin AUC 2 IV, D 1

and 8 – 21-day cycle

Arm A: Induction – ABRAXANE® (100 mg/m2) D 1, 8 and 15 / Carboplatin (6 mg/min/ml) D 1 of a 21-day cycle;

Maintenance – ABRAXANE® (100 mg/m) D 1 and 8 of a 21-day cycle or Best supportive care

Arm B: Induction – ABRAXANE® (100 mg/m2) D 1, 8 and 15 / Carboplatin (6 mg/min/ml) D 1 of a 21-day cycle;

Maintenance – Best supportive care

Primary Endpoint Progression Free Survival Progression Free Survival

StatusTrial enrolling;

Phase II data expected at SABCS 2016Trial enrollingData in 2017E

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I&I Late Stage Programs

Patient Population Untreated Moderate-to-SevereLate Stage Psoriatic Arthritis Active Behçet’s Disease

Molecule OTEZLA® OTEZLA®

Trial Name PSA-006BCT-002RELIEFTM

Phase III III


DesignArm A: OTEZLA® single agent (30mg)

twice dailyArm B: Placebo

Arm A; Placebo for 12 weeks followed by 30mg OTEZLA® twice daily for 52-weeksArm B: 30mg OTEZLA® twice daily for 64

weeks

Primary Endpoint ACR 20 at Week 16Area under the curve (AUC) for the number of oral ulcers from baseline through week

12

StatusPrimary endpoint met

Data at an major medical congress expected

Trial enrollingData in 2017E

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Patient Population Active Crohn’s Disease Moderate to Severe Ulcerative Colitis

Molecule GED-0301 Ozanimod

Trial Name CD-002 TRUE NORTH

Phase III III

Target Enrollment 1,064 900

Design

Arm A: GED-301 160mg daily 12 weeks/GED-301 40mg daily 40 weeks

Arm B: GED-301 160mg daily 12 weeks/GED-301 40mg daily 4 weeks on/4 weeks off 40 weeks

Arm C: GED-301 160mg daily 12 weeks/GED-301 160mg daily 4 weeks on/4 weeks off 40 weeks

Arm A: Ozanimod 1mg (daily for induction and maintenance)

Arm B: Placebo (induction and maintenance)

Primary Endpoint Clinical remission defined by Crohn's Disease Activity Index (CDAI)

Clinical remission assessed by Mayo component sub-scores at week 10

Clinical remission assessed by Mayo component sub-scores at week 52

StatusEnrolling

Data in 2018EEnrolling

Data in 2018E

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Patient Population Relapsing Multiple Sclerosis Relapsing Multiple Sclerosis

Molecule Ozanimod Ozanimod

Trial Name SUNBEAM RADIANCE

Phase III II/III


DesignArm A: Ozanimod (0.5mg) daily/placebo IM weeklyArm B: Ozanimod (1mg) daily/placebo IM weekly

Arm C: Oral placebo daily/Beta-interferon IM weekly

Phase IIArm A: Ozanimod (0.5mg) dailyArm B: Ozanimod (1mg) daily

Arm C: Placebo dailyPhase III

Arm A: Ozanimod (0.5mg) daily/placebo IM weeklyArm B: Ozanimod (1mg) daily/placebo IM weekly

Arm C: Oral placebo daily/Beta-interferon IM weekly

Primary Endpoint Annualized relapse rate at month 12 Annualized relapse rate at month 24

StatusEnrollment complete

Data expected in H1:17EEnrollment complete

Data expected in H1:17E

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Reconciliation Tables


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Use of Non-GAAP Financial Measures

In addition to financial information prepared in accordance with U.S. GAAP, this document also contains certain non-GAAP financial measures based on management’s view of performance including:

• Adjusted research and development expense• Adjusted selling, general and administrative expense• Adjusted operating margin• Adjusted net income• Adjusted earnings per share

Management uses such measures internally for planning and forecasting purposes and to measure the performance of the Company. We believe these adjusted financial measures provide useful and meaningful information to us and investors because they enhance investors’ understanding of the continuing operating performance of our business and facilitate the comparison of performance between past and future periods. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. When preparing these supplemental non-GAAP financial measures we typically exclude certain GAAP items that management does not consider to be normal, recurring, cash operating expenses but that may not meet the definition of unusual or non-recurring items. Other companies may define these measures in different ways. The following categories of items are excluded from adjusted financial results:

Acquisition and Divestiture-Related Costs: We exclude the impact of certain amounts recorded in connection with business combinations and divestitures from our adjusted financial results that are either non-cash or not normal, recurring operating expenses due to their nature, variability of amounts, and lack of predictability as to occurrence and/or timing. These amounts may include non-cash items such as the amortization of acquired intangible assets, amortization of purchase accounting adjustments to inventories, intangible asset impairment charges and expense or income related to changes in the estimated fair value measurement of contingent consideration. We also exclude transaction and certain other cash costs associated with business acquisitions and divestitures that are not normal recurring operating expenses, including severance costs which are not part of a formal restructuring program.


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Share-based Compensation Expense: We exclude share-based compensation from our adjusted financial results because share-based compensation expense, which is non-cash, fluctuates from period to period based on factors that are not within our control, such as our stock price on the dates share-based grants are issued.

Collaboration-related Upfront Expenses: We exclude collaboration-related upfront expenses from our adjusted financial results because we do not consider them to be normal, recurring operating expenses due to their nature, variability of amounts, and lack of predictability as to occurrence and/or timing. Upfront payments to collaboration partners are made at the commencement of a relationship anticipated to continue for a multi-year period and provide us with intellectual property rights, option rights and other rights with respect to particular programs. The variability of amounts and lack of predictability of collaboration-related upfront expenses makes the identification of trends in our ongoing research and development activities more difficult. We believe the presentation of adjusted research and development, which does not include collaboration-related upfront expenses, provides useful and meaningful information about our ongoing research and development activities by enhancing investors’ understanding of our normal, recurring operating research and development expenses and facilitates comparisons between periods and with respect to projected performance. All expenses incurred subsequent to the initiation of the collaboration arrangement, such as research and development cost-sharing expenses/reimbursements and milestone payments up to the point of regulatory approval are considered to be normal, recurring operating expenses and are included in our adjusted financial results.

Research and Development Asset Acquisition Expense: We exclude costs associated with acquiring rights to pre-commercial compounds because we do not consider such costs to be normal, recurring operating expenses due to their nature, variability of amounts, and lack of predictability as to occurrence and/or timing. Research and development asset acquisition expenses includes expenses to acquire rights to pre-commercial compounds from a collaboration partner when there will be no further participation from the collaboration partner or other parties. The variability of amounts and lack of predictability of research and development asset acquisition expenses makes the identification of trends in our ongoing research and development activities more difficult. We believe the presentation of adjusted research and development, which does not include research and development asset acquisition expenses, provides useful and meaningful information about our ongoing research and development activities by enhancing investors’ understanding of our normal, recurring operating research and development expenses and facilitates comparisons between periods and with respect to projected performance.

Restructuring Costs: We exclude costs associated with restructuring initiatives from our adjusted financial results. These costs include amounts associated with facilities to be closed, employee separation costs and costs to move operations from one location to another. We do not frequently undertake restructuring initiatives and therefore do not consider such costs to be normal, recurring operating expenses.


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Certain Other Items: We exclude certain other significant items that may occur occasionally and are not normal, recurring, cash operating expenses from our adjusted financial results. Such items are evaluated on an individual basis based on both the quantitative and the qualitative aspect of their nature and generally represent items that, either as a result of their nature or magnitude, we would not anticipate occurring as part of our normal business on a regular basis. While not all-inclusive, examples of certain other significant items excluded from adjusted financial results would be: expenses for significant litigation-related loss contingency accruals and expenses to settle other disputed matters.

Estimated Tax Impact From Above Adjustments: We exclude the net income tax impact of the non-tax adjustments described above from our adjusted financial results. The net income tax impact of the non-tax adjustments includes the impact on both current and deferred income taxes and is based on the taxability of the adjustment under local tax law and the statutory tax rate in the tax jurisdiction where the adjustment was incurred.

Non-Operating Tax Adjustments: We exclude the net income tax impact of certain other significant income tax items, which are not associated with our normal, recurring operations (“Non-Operating Tax Items”), from our adjusted financial results. Non-Operating Tax Items include items which may occur occasionally and are not normal, recurring operating expenses (or benefits), including adjustments related to acquisitions, divestitures, collaborations, certain adjustments to the amount of unrecognized tax benefits related to prior year tax positions, and other similar items.

Long-Term Targets

A reconciliation of long-term adjusted financial targets to the most comparable GAAP measures cannot be provided because we are unable to forecast with reasonable certainty many of the items necessary to calculate such comparable GAAP measures, including share-basedcompensation expense, collaboration-related upfront expense, research and development asset acquisition expense, acquisition-related expenses, fair value adjustments to contingent consideration, the ultimate outcome of legal proceedings and unusual gains and losses, as well as unforeseen events, risks and developments. These items are uncertain, depend on various factors, and could be material to our results computed in accordance with GAAP. We believe the inherent uncertainties in reconciling our long-term non-GAAP measures to the most comparable GAAP measures would make the forecasted comparable GAAP measures nearly impossible to predict with reasonable certainty and therefore inherently unreliable.

See the attached Reconciliations of GAAP to Adjusted Net Income for explanations of the amounts excluded and included to arrive at the adjusted measures for the three- and nine-month periods ended September 30, 2016 and 2015, and for the projected amounts for the year ending December 31, 2016.


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61


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Explanation of adjustments:(1) Exclude share-based compensation expense totaling $147.7 for the three-month period ended September 30, 2016 and $149.9 for the three-month

period ended September 30, 2015. Exclude share-based compensation expense totaling $451.7 for the nine-month period ended September 30, 2016 and $426.4 for the nine-month period ended September 30, 2015.

(2) Exclude upfront payment expense for research and development collaboration arrangements.(3) Exclude research and development asset acquisition expenses for EngMab AG.(4) Exclude loss contingency accrual expense related to a contractual dispute.(5) Exclude amortization of intangible assets acquired in the acquisitions of Pharmion Corp., Gloucester Pharmaceuticals, Inc. (Gloucester), Abraxis

BioScience Inc. (Abraxis), Celgene Avilomics Research, Inc. (Avila), and Quanticel Pharmaceuticals, Inc. (Quanticel). The excluded amortization expense for the nine-month period ended September 30, 2016 includes $83.1 million related to the impairment of an intangible asset acquired in the Avila acquisition.

(6) Exclude changes in the fair value of contingent consideration related to the acquisitions of Gloucester, Abraxis, Avila, Nogra Pharma Limited and Quanticel.

(7) Exclude equity compensation and other fees and costs related to the acquisition of Receptos, Inc.(8) Exclude restructuring charges related to our relocation of certain operations into our two Summit, NJ locations as well as costs associated

with certain headcount reductions.(9) Exclude the estimated tax impact from the above adjustments.(10) Exclude other non-operating tax expense items. The adjustment for the three- and nine-month periods ended September 30, 2015 related primarily to

the impact of accelerating the full year tax expense on a gain on the sale of an equity investment into the second quarter of 2015, which was the period in which the gain occurred, and the impact of a one-time tax expense incurred as a result of our global mix of funding sources for certain upfront collaboration payments.

(11) Adjusted diluted net income per share for the three-month period ended September 30, 2015 was determined using diluted weighted average shares of 823.6.


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Documents

October 27, 2016€¦ · 2016-10-27 · Q&A Scott Smith, President, Global I&I Jackie Fouse, ... 2016 adjusted diluted EPS raised from $5.70-$5.75 to $5.88-$5.92 2016 REVLIMID®