q3 2018 conference call - s24.q4cdn.com · 10/25/2018 · q3 2018 conference call mark alles,...
TRANSCRIPT
Q3 2018 Conference CallOctober 25, 2018
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q3 2018 Conference Call
Mark Alles, Chairman & Chief Executive Officer
Nadim Ahmed, President, Hematology & Oncology
Jay Backstrom, MD, Chief Medical Officer
Q&A
Terrie Curran, President, I&I
David Elkins, Chief Financial Officer
2
Forward-Looking Statements and Adjusted Financial Information
3
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 containsadjusted financial measures. Further information relevant to the interpretation of adjusted financial measures, andreconciliations of these adjusted financial measures to the most comparable GAAP measures, may be found in theAppendix and on our website at www.Celgene.com in the “Investor Relations” section.
Mark AllesChairman & Chief Executive Officer
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q3 2018: Strong Performance and Pipeline Momentum
Delivering Excellent Operating Results− Outstanding year-over-year top- and bottom-line growth − Raising 2018 financial guidance and reaffirming 2020 outlook
Accelerating Inline, Late-Stage Products and Early Pipeline− More than 10 positive Ph III trials across portfolio year-to-date− Advancing 5 late-stage assets expected to launch by 2020 – ozanimod, fedratinib,
luspatercept, liso-cel and bb2121− R&D engine progressing; 5 INDs filed for high-potential new medicines year-to-date
Strengthening the Organization− Establishing dedicated global clinical drug development function− Alise Reicin, MD, appointed as President, Global Clinical Development− Aligns with mission to discover, develop and commercialize innovative medicines
5
David ElkinsChief Financial Officer
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q3 2018 Financial Highlights
7
Strong Operating Results Driven by Volume− Net product sales grew 18% Y/Y; Adjusted diluted EPS $2.29 (+20% Y/Y)− 15 of the 18 percentage points of net product sales growth from volume
Strategic and Balanced Capital Deployment to Support Future Growth− On track to invest ~$3.6B in R&D in FY2018− Reaffirm 2020 outlook: $19B-$20B total revenue and >$12.50 adjusted EPS
2018 Total Revenue, OTEZLA® Net Sales and Adj. Diluted EPS Guidance Raised− Total revenue raised from ~$15.0B to ~$15.2B− OTEZLA® net sales raised from ~$1.5B to ~$1.6B− Adjusted diluted EPS raised from the range of $8.70-$8.75 to the range of $8.75-$8.80
Q3 2018 Total Net Product Sales
Q3:16 Q3:17 Q3:18
$ M
illio
ns ↑28% ↑11% ↑18%
$0
$500
$1,000
$1,500
$2,000
$2,500
$3,000
$3,500
$4,000
Q3:17 Volume Price FX/Hedge Q3:18
↑18.5%↓0.5%↑14.7% ↑4.3%
Contribution to Q3:18 Total Net Product Sales Growth
$ M
illio
ns
Total Net Product Sales
8
$2,969$3,283
$3,890
Growth Rates = Growth vs. Prior Year Period
Q3 2018 Adjusted Diluted Earnings Per Share
Q3:16 Q3:17 Q3:18
↑28% ↑21%
Dol
lars
Per
Sha
re
↑20%
Q3:17 Oper. Income
OIE Tax Rate
Share Count
Q3:18
Dol
lars
Per
Sha
re
$2.29$0.201$1.91 $0.001 $0.27($0.09)1
9
Contribution to Q3:18 Adjusted Diluted EPSAdjusted Diluted EPS
$1.58
$1.91
$2.29
Growth Rates = Growth vs. Prior Year Period1. Includes net impact of Juno Therapeutics and Impact Biomedicines dilution.
Key P&L Line Items (Adjusted)
Q3:18 ∆ vs.Q3:17
∆ vs.Q2:18
Product Gross Margin 96.2% ↓ 40 bps ↓ 70 bps
R&D Expense% of revenue
$948M24.4% ↑ 320 bps ↓ 50 bps
SG&A Expense% of revenue
$642M16.5% ↑ 60 bps ↓ 110 bps
Operating Margin 55.3% ↓ 420 bps ↑ 80 bps
Effective Tax Rate 16.7% ↑ 20 bps ↓ 10 bps
10
Capital Allocation in Q3 2018Capital Allocation in Q3 2018
Cash flow from operations was approximately $1.9B
Settlement of the Accelerated Share Repurchase (ASR), which commenced in Q2; Repurchased approximately 24 million shares under the ASR
(in $ Billions) 12/31/17 9/30/18
Cash, Cash Equivalents, Marketable Debt Securities and Publicly-Traded Equity Securities
$12.04 $4.38
11
Updated 2018 Guidance
Previous Updated
Total Revenue ~$15.0B ~$15.2B
REVLIMID® Net Sales ~$9.7B Unchanged
POMALYST®/IMNOVID® Net Sales ~$2.0B Unchanged
OTEZLA® Net Sales ~$1.5B ~$1.6B
ABRAXANE® Net Sales ~$1.0B Unchanged
Adjusted Operating Margin ~56.0% ~55.5%
Adjusted Tax Rate ~17% Unchanged
Adjusted Diluted EPS $8.70-$8.75 $8.75-$8.80
Weighted Average Diluted Shares ~735M Unchanged
12
Terrie CurranPresident, I&I
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q3 2018 I&I Franchise Results
Robust Worldwide Growth for OTEZLA®
– Increasing commercial TRx demand drove Q3:18 net product sales growth of +40% Y/Y– U.S. contracting strategy continues to contribute to topline results– Solid ex-U.S. performance driven by share gains in key markets
14
Enhancing the OTEZLA® Profile– Positive Ph III data for STYLETM in scalp psoriasis– Submitted Behҫet’s disease sNDA in the U.S.– Advancing multiple lifecycle development opportunities
Building a Diversified Portfolio of I&I Assets– Ozanimod RMS regulatory filings in U.S. and EU on track for Q1:19– Positive Ph II 52-week data for HEROESTM trial evaluating RPC4046 in EoE– Advancing development of early- and mid-stage pipeline assets
Net Sales ($M)
$244 $250
$348
$31 $58
$84
Q3:15 Q3:16 Q3:17 Q3:18U.S. ROW
$275$308
$10
$129
$139
Q3 2018 OTEZLA® Net Sales Summary
15
Current Results & Potential Future Growth Drivers
• Q3:18 net sales $432M, +40% Y/Y
– U.S. net sales $348M, +39% Y/Y– International net sales $84M, +45% Y/Y
• Continued growth of OTEZLA® as utilization increases worldwide– Moderate script growth in Q3 due to expected
seasonality– Some inventory build at the channel level– Continued benefit from improved access– Key U.S. performance metrics are on track
• Leadership position in new-to-brand patients• Psoriasis source of business >85% pre-biologic patients
– Strong performance continues in France and Japan
$432
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention
15
16
Multiple Future Growth Drivers for OTEZLA® and Ozanimod
OTEZLA®
• Ph III STYLETM study in scalp psoriasis
• Behҫet’s disease indication
• Ph III ADVANCETM study in mild to moderate plaque psoriasis
• Ph IIIb DISCREETTM study in genital psoriasis
• Ph III SPROUTTM study in pediatric moderate to severe plaque psoriasis
• Ph IV study in moderate psoriatic arthritis
Ozanimod• Ph III YELLOWSTONETM study in Crohn’s disease initiated
• Ph III TRUE NORTHTM enrollment in UC on track for completion in mid-2019
Nadim AhmedPresident, Hematology & Oncology
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q3 2018 Hematology & Oncology Franchise ResultsQ3 2018 Hematology & Oncology Franchise Results
Strong Net Product Sales and Operating Momentum– Q3:18 net product sales: $3.5B, +16% Y/Y
– Sales performance driven by strong demand across geographies and brands
Growth Drivers Delivering− REVLIMID® continues to grow across geographies with NSCT and post-ASCT maintenance adoption
− POMALYST®/IMNOVID® growth continues through gains in market share and duration
− Positive Phase III AUGMENTTM data with R2 in RR indolent lymphoma expected at ASH
− Positive Phase III data for ABRAXANE®/IO combination regimens in NSCLC and TNBC at ESMO
Advancement and Expansion of Innovative Pipeline− Significant data on pipeline expected at ASH:
− Ph III data on luspatercept (MEDALISTTM and BELIEVETM)
− Data on CAR T: Liso-cel (JCAR017) in CLL, bb21217 in RRMM and JCARH125 in RRMM
− bb2121 clinical program in earlier lines advancing
− Fedratinib U.S. regulatory submission on track for end 2018 18
$150$203
$268$357$107
$138
$149
$156
Q3:15 Q3:16 Q3:17 Q3:18U.S. ROW
$895 $1,154 $1,361$1,667
$558
$738$720
$782
Q3:15 Q3:16 Q3:17 Q3:18U.S. ROW
Current Results & Potential Future Growth Drivers
Q3 2018 IMiD® Net Sales Summary Q3 2018 IMiD® Net Sales Summary
• REVLIMID® Q3:18 net sales $2,449M, +18% Y/Y• POMALYST® Q3:18 net sales $513M, +23% Y/Y• Strong growth with contribution from increased
market share, duration and triplet combinations• Clinical development and potential future growth
drivers:– REVLIMID®
• Positive Ph III data (ECOG E3A06) in smoldering MM• REVLIMID®-based triplet regimens in NDMM Ph III data
readouts expected in NDMM• Ph III ROBUST® study in 1st line ABC-subtype diffuse
large B-cell lymphoma (event driven)
– POMALYST®
• Newer triplet regimens expected to increase share and duration
19
REVLIMID® Net Sales ($M)
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
$1,453
$1,892$2,081
$2,449
POMALYST® Net Sales ($M)
$257
$341
$417
$513
Current Results & Potential Future Growth Drivers
Q3 2018 ABRAXANE® Net Sales SummaryQ3 2018 ABRAXANE® Net Sales Summary
$145 $144 $149 $174
$84 $89 $102$114
Q3:15 Q3:16 Q3:17 Q3:18
U.S. ROW
$288
$229$251
$233
Net Sales ($M)
20
• Q3:18 net sales $288M, 15% Y/Y (demand +5% Y/Y)• Potential future growth drivers: Ph III apact® trial of ABRAXANE® in adjuvant pancreatic
cancer data (event driven)
Positive Ph III IO combination trials at ESMO 2018 (TECENTRIQ®+ABRAXANE®):
• IMpassion130* 1st line TNBC
• IMpower131* 1st line squamous NSCLC
• IMpower130* 1st line non-squamous NSCLC
– Ph III IO combination trials published in NEJM:
• IMpassion130* 1st line TNBC (TECENTRIQ®+ABRAXANE®)
• KEYNOTE-407§ 1st line squamous NSCLC
*IMpower130, IMpower131 and IMpassion130 are Genentech, a member of the Roche Group, sponsored clinical trials§ KEYNOTE-407 is a Merck-sponsored trial
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
*Luspatercept in collaboration with Acceleron Pharma. bb2121 in collaboration with bluebird bio
Showcasing Our Next-generation Pipeline at ASHShowcasing Our Next-generation Pipeline at ASH
21
• Ph III MEDALISTTM data with luspatercept in MDS*
• Ph III BELIEVETM data with luspatercept in beta-thalassemia*
• Ph I/II data with liso-cel in RR CLL
• Ph I data with bb21217 in RRMM*
• Ph I/II data with JCARH125 in RRMM
• Ph III AUGMENTTM data with R2 in RR indolent lymphoma
Jay Backstrom, MDChief Medical Officer
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Ozanimod RMS NDA & MAA Submissions On Track for Q1:19
23
Data Required for Submission
Non-clinical bridging studies
Utilizing existing PK/PD data
Additional human clinical efficacy and safety studies not needed
Additional Data to Optimize Label
Drug-drug interaction trials in healthy volunteers; Trials are not required for submission
Data have the potential to enhance the opportunity across the broadest patient population
– U.S. approval now expected in RR DLBCL in mid-2020– Data from Ph I CLL trial to be presented at ASH 2018
– MEDALISTTM and BELIEVETM to be presented at ASH– U.S. and EU regulatory submissions expected in H1:19
– U.S. NDA and EU MAA submissions planned for Q1:19– TRUE NORTHTM UC trial enrollment targeted for completion in mid-2019
– U.S. NDA submission in myelofibrosis expected by YE18– EU MAA submission planned in 2019
– Clinical program in earlier lines advancing– U.S. approval expected in highly refractory MM in 2020; KarMMaTM to complete
enrollment by YE18
5 New Late-Stage Products Expected to Launch Through 2020
24
Luspatercept
Liso-cel
Ozanimod
Fedratinib
bb2121
Q3 2018 Conference CallOctober 25, 2018
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q&A
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Use of Non-GAAP Financial Measures and
Reconciliation Tables
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Use of Non-GAAP Financial Measures
28
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 measuresbased 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 believethese adjusted financial measures provide useful and meaningful information to us and investors because they enhance investors’ understanding ofthe continuing operating performance of our business and facilitate the comparison of performance between past and future periods. Theseadjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information preparedin accordance with U.S. GAAP. When preparing these supplemental non-GAAP financial measures we typically exclude certain GAAP items thatmanagement does not consider to be normal, recurring cash operating expenses but that may not meet the definition of unusual or non-recurringitems. Other companies may define these measures in different ways. The following categories of items are excluded from adjusted financialresults:
Acquisition and Divestiture-Related Costs: We exclude the impact of certain amounts recorded in connection with business combinations anddivestitures from our adjusted financial results that are either non-cash or not normal, recurring operating expenses due to their nature, variabilityof amounts, and lack of predictability as to occurrence and/or timing. These amounts may include non-cash items such as the amortization ofacquired intangible assets, amortization of purchase accounting adjustments to inventories, intangible asset impairment charges and expense orincome related to changes in the estimated fair value measurement of contingent consideration and success payments. We also exclude transactionand certain other cash costs associated with business acquisitions and divestitures that are not normal, recurring operating expenses, includingseverance costs which are not part of a formal restructuring program.
Use of Non-GAAP Financial Measures
29
Share-Based Compensation Expense: We exclude share-based compensation from our adjusted financial results because share-based compensationexpense, 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 datesshare-based grants are issued.
Collaboration-Related Upfront Expenses: We exclude collaboration-related upfront expenses from our adjusted financial results because we do notconsider them to be normal, recurring operating expenses due to their nature, variability of amounts, and lack of predictability as to occurrenceand/or timing. Upfront payments to collaboration partners are made at the commencement of a relationship anticipated to continue for a multi-yearperiod and provide us with intellectual property rights, option rights and other rights with respect to particular programs. The variability of amountsand lack of predictability of collaboration-related upfront expenses makes the identification of trends in our ongoing research and developmentactivities more difficult. We believe the presentation of adjusted research and development, which does not include collaboration-related upfrontexpenses, 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 respectto projected performance. All expenses incurred subsequent to the initiation of the collaboration arrangement, such as research and developmentcost-sharing expenses/reimbursements and milestone payments up to the point of regulatory approval are considered to be normal, recurringoperating 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 becausewe do not consider such costs to be normal, recurring operating expenses due to their nature, variability of amounts, and lack of predictability as tooccurrence and/or timing. Research and development asset acquisition expenses includes expenses to acquire rights to pre-commercial compoundsfrom a collaboration partner when there will be no further participation from the collaboration partner or other parties. The variability of amountsand lack of predictability of research and development asset acquisition expenses makes the identification of trends in our ongoing research anddevelopment activities more difficult. We believe the presentation of adjusted research and development, which does not include research anddevelopment asset acquisition expenses, provides useful and meaningful information about our ongoing research and development activities byenhancing investors’ understanding of our normal, recurring operating research and development expenses and facilitates comparisons betweenperiods and with respect to projected performance.
Use of Non-GAAP Financial Measures
30
Restructuring Costs: We exclude costs associated with restructuring initiatives from our adjusted financial results. These costs include amountsassociated with facilities to be closed, employee separation costs and costs to move operations from one location to another. We do not frequentlyundertake restructuring initiatives and therefore do not consider such costs to be normal, recurring operating expenses.
Certain Other Items: We exclude certain other significant items that may occur occasionally and are not normal, recurring cash operating expensesfrom our adjusted financial results. Such items are evaluated on an individual basis based on both the quantitative and the qualitative aspect of theirnature and generally represent items that, either as a result of their nature or magnitude, we would not anticipate occurring as part of our normalbusiness on a regular basis. While not all-inclusive, examples of certain other significant items excluded from adjusted financial results would be:significant litigation-related loss contingency accruals and expenses to settle other disputed matters and, effective for fiscal year 2018, changes inthe fair value of our equity securities upon the adoption of ASU 2016-01 (Financial Instruments-Overall: Recognition and Measurement ofFinancial Assets and Financial Liabilities).
Estimated Tax Impact From Above Adjustments: We exclude the net income tax impact of the non-tax adjustments described above from ouradjusted financial results. The net income tax impact of the non-tax adjustments includes the impact on both current and deferred income taxes andis 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 withour normal, recurring operations (“Non-Operating Tax Items”), from our adjusted financial results. Non-Operating Tax Items include items whichmay 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, the impact of tax reformlegislation commonly referred to as the Tax Cuts and Jobs Act (2017 Tax Act), and other similar items. We also exclude excess tax benefits and taxdeficiencies that arise upon vesting or exercise of share-based payments recognized as income tax benefits or expenses due to their nature,variability of amounts, and lack of predictability as to occurrence and/or timing.
See the attached Reconciliations of GAAP to Adjusted Net Income for explanations of the amounts excluded and included to arrive at the adjustedmeasures for the three- and nine-month periods ended September 30, 2018 and 2017, and for the projected amounts for the twelve-month periodending December 31, 2018.
Reconciliation Tables
31
Reconciliation Tables
32
Reconciliation Tables
33
Appendix
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
CHANGING THE COURSE OFHUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
1. Original guidance provided on January 2018 did not include the impact of our acquisition of Juno, which was expected to be dilutive to adjusted diluted EPS by approximately $0.50.2. Updated guidance May 2018 3. Updated guidance July 2018.4. Updated guidance October 2018
2018 Milestones2018 Milestones
Financial Performance Total revenue $14.4B-$14.8B1 Total revenue ~$15.2B4
REVLIMID® net sales ~$9.4B1 REVLIMID® net sales ~$9.7B3
POMALYST® net sales ~$1.9B1 POMALYST® net sales ~$2.0B2
OTEZLA® net sales ~$1.5B1 OTEZLA® net sales ~$1.6B4
ABRAXANE® net sales ~$1.0B1
Adj. operating margin ~60%1 Adj. operating margin ~55.5%4
Adj. diluted EPS $8.70 to $8.901 Adj. diluted EPS $8.75-$8.804
Clinical DataPh III AUGMENTTM – REVLIMID® in R/R FL
Ph III ROBUST ® – REVLIMID® in 1st line ABC-subtype DLBCL- event driven Ph III apact® – ABRAXANE® in adjuvant PanC – event driven
Ph III OPTIMISMM® trial – POMALYST® in 2nd line MM Ph III OTEZLA® in scalp PSOR
Ph III QUAZAR® AML-001 – CC-486 in AML maintenance- event drivenPh III MEDALISTTM – Luspatercept in RS+ MDSPh III BELIEVETM – Luspatercept in beta-thalassemiaPh II OTEZLA ® in UC Trial Enrollment
Complete enrollment in Ph III TRUE NORTHTM – Ozanimod in UC– Ph III TRUE NORTHTM Moved to mid-2019
Complete enrollment in pivotal KarMMaTM trial – bb2121 in RRMM Complete enrollment in TRANSCEND WORLD – Liso-cel in DLBCL
Regulatory Submissions/DecisionsSubmit sNDA for RVd in NDMM Submitted in EU
Submit NDA for Fedratinib in myelofibrosisFDA decision on Ozanimod in RMSFDA decision on OTEZLA ® once-daily formulationSubmit sNDA for OTEZLA® in Behçet’s diseaseSubmit a Marketing Authorization Application (MAA) for Ozanimod in RMS
Trial Initiations Initiate the pivotal program with CC-122 in NHL
Initiate Ph III with OTEZLA® in UC Initiate the pivotal program with Tislelizumab in NSCLC Initiate Ph III with OTEZLA® in mild to moderate PSOR
Initiate Ph III trial with bb2121 in 3rd line+ MMInitiate Ph III trial with Liso-cel in TE 2nd line DLBCL Initiate Ph III COMMANDSTM with Luspatercept in 1st line, lower-risk MDS
Initiate Ph III trial with Ozanimod in SPMS
R&EDFile at least 5 IND’s
35
X
X
X
X
X
REVLIMID®
Del 5q MDS
VIDAZA®
MDS, AML
IDHIFA®
IDH2 RRAML
CC-486 MDS, AML
LuspaterceptMDS, Beta-thalassemia
CC-90009RRAML
FT-1101MDS, AML
MyeloidDisease
10MarketPh I
L E G E N D
REVLIMID®
MCL
ISTODAX®
PTCL, CTCL
REVLIMID®
NHL
CC-122NHL, CLL
Liso-celNHL
CC-486NHL
Lymphoma& Leukemia
10
LuspaterceptMF
CC-90002NHL
Advancing a High Quality Pipeline with Significant Potential
Celgene has an exclusive option to license and/or option to acquire: JTX-2011, Etigilimab, FT-4101, FT-1101, AG-270, and MSC-1
CC-90010NHL
bb21217RRMM
REVLIMID®
NDMM, RRMM
POMALYST®
RRMM
THALOMID®
NDMM, RRMM
CC-220RRMM
bb2121RRMM
CC-92480RRMM
MultipleMyeloma
9
MarizomibGBM
CC-122HCC
CC-90002Solid TumorsCC-90011
Solid Tumors
ABRAXANE®
PanC, NSCLC, mBC
SolidTumors
11EtigilimabSolid Tumors
JTX-2011Solid Tumors
CC-90010Solid Tumors
TislelizumabSolid Tumors
FedratinibMF
FT-1101NHLCC-93269
RRMM
JCARH125RRMM
MSC-1Solid Tumors
Liso-celCLL
GEM333AML
AG-270Solid Tumors
OzanimodUC
Inflammation& Immunology
11
OzanimodMS
OTEZLA®
PSOR, PSA
OTEZLA®
Behçet’s, Scalp PSOR
RPC4046EoE
CC-220SLE
CC-90001 IPF
CC-90006PSOR
FT-4101NASH
OzanimodCD
CC-99677I&I
36
Return On Invested Capital (ROIC): Focused on Efficient Growth
0.0%
5.0%
10.0%
15.0%
20.0%
25.0%
30.0%
35.0%
$0.0
$5.0
$10.0
$15.0
$20.0
$25.0
2009 2010 2011 2012 2013 2014 2015 2016 2017 Q3:18 (TTM)
Capital Base Excluding Cash* Capital Base ROIC Excluding Cash* ROIC
Aver
age
Inve
sted
Cap
ital
$ B
illio
n
ROIC
*For purposes of this calculation, cash includes cash and cash equivalents and marketable debt securities and publicly-traded equity securities.
Footnote: Financial performance is based on GAAP operating income adjusted to reflect amortization of certain charges excluded from 2008 calculation and tax impact. Calculation for 2015 includes expenses driven by the Juno Therapeutics and AstraZeneca collaborations and expenses incurred in connection with the acquisition of Receptos as well as the impact of the August 2015 debt issuance on the capital base. Calculation for 2018 includes expenses incurred in connection with the acquisition of Juno as well as the impact of the February 2018 debt issuance on the capital base. Refer to reconciliation tables for complete calculation methodology. Calculation revised in 2015 for all prior periods to reflect amortization of certain charges excluded from 2008 calculation.
37
Multiple Myeloma Late-Stage/Pivotal Programs
Patient Population RRMM RRMM RRMM
Molecule POMALYST®/IMNOVID® bb2121 bb2121
Trial NameMM-007
OPTIMISMM®
BB2121-MM-001KarMMaTM
BB2121-MM-003KarMMa-3TM
Phase III II III
Target Enrollment 559 140 381
Design
Arm A: POMALYST®/IMNOVID®
(4mg) + bortezomib (1.3 mg/m2 IV) + low-dose dexamethasone to disease
progressionArm B: Bortezomib (1.3 mg/m2 IV) + low-dose dexamethasone to disease
progression
bb2121 autologous CAR T cells (infused at a dose ranging from 15 -45 x 107 CAR T cells after receiving
lymphodepleting chemotherapy)
Arm A: bb2121 autologous CAR T cells (infused at a dose ranging from
15 - 45 x 107 CAR T cells after receiving lymphodepleting
chemotherapy)Arm B: Physicians’ choice
Primary Endpoint Progression Free Survival ORR PFS
Status
Primary endpoint metData presented at ASCO 2018
Submitted in EU; Japan submission planned
Trial enrolling Not yet enrolling
38
MDS/AML/MF Late-Stage/Pivotal 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 217 472
DesignArm A: CC-486 (300mg daily D1-21 of a
28-D cycle) + best supportive careArm B: Placebo + best supportive care
Arm A: CC-486 (300mg D1-14 of 28-D cycle)
Arm B: Best supportive care
Primary Endpoint RBC-transfusion independence formore than 12 weeks Overall Survival
Status Trial enrollingEnrollment complete
Data expected in 2018 (event driven)
39
MDS/AML/MF Late-Stage/Pivotal Programs
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
Target Enrollment 229 335
DesignArm A: Luspatercept (starting dose of 1.0 mg/kg
subcutaneous injection every 3 weeks)Arm B: Placebo (subcutaneous injection every 3
weeks)
Arm A: Luspatercept (1 mg/kg) + best supportive care
Arm B: Placebo + 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 randomization
StatusPrimary endpoint met
Data expected at ASH 2018Regulatory submissions planned in H1:19
Primary endpoint metData expected at ASH 2018
Regulatory submissions planned in H1:19
40
MDS/AML/MF Late-Stage/Pivotal Programs
Patient Population ESA Naïve Very Low, Low or Intermediate Risk MDS IDH2 Mutant RR AML
Molecule Luspatercept IDHIFA®
Trial Name COMMANDSTM IDHENTIFYTM
Phase III III
Target Enrollment 350 316
DesignArm A: Luspatercept (1.0 mg/kg SC every 3 weeks)
Arm B: Epoetin alfa (450 IU/kg SC weekly)
Arm A: IDHIFA® (100 mg daily, 28-D cycle) + best supportive care
Arm B: Best supportive care
Primary Endpoint Red blood cell transfusion independence at 24 weeks Overall Survival
Status Not yet recruiting Trial enrolling
41
Lymphoma Late-Stage/Pivotal Programs
Patient Population Relapsed or Refractory Follicular Lymphoma
Newly Diagnosed Follicular Lymphoma
Untreated Activated B-Cell DLBCL
Molecule REVLIMID® REVLIMID® REVLIMID®
Trial NameAUGMENTTM
NHL-007RELEVANCE®
ROBUST®
DLC-002
Phase III III III
Target Enrollment 358 1,031 570
Design
Arm A: REVLIMID® (10-20mg, D1-21) + rituximab (375 mg/m2 weekly for cycle 1 then D1 of cycles 2-5 for 5 28-D cycles)
Arm B: Placebo (D1-21) + rituximab (375 mg/m2 weekly for cycle 1 then D1
of cycles 2-5 for 5 28-D cycles)
Arm A: REVLIMID® (starting dose 20mg, D2-22 for up to 18 D cycles) + rituximab (starting dose 375 mg/m2
weekly for up to 12 28-D cycles)Arm B: Physician’s choice of
Rituximab-CHOP, Rituximab-CVP or Rituximab-bendamustine
Arm A: REVLIMID® (15mg, D1-14) + R-CHOP21 (6 21-D cycles)
Arm B: Placebo + R-CHOP21 (6 21-D cycles)
Primary Endpoint Progression Free Survival Complete Response Rate and Progression Free Survival Progression Free Survival
Status
Primary endpoint metData to be presented at ASH 2018Regulatory submissions planned in
Q1:19
Trial did not achieve superiority in co-primary endpoints
Data presented at ASCO 2018
Data expected in 2018 (event driven trial
42
Lymphoma Late-Stage/Pivotal Programs
Patient Population Relapsed or Refractory Indolent Lymphoma Relapsed or Refractory B-cell NHL
Molecule REVLIMID®Liso-cel
(lisocabtagene maraleucel; JCAR017)
Trial NameMAGNIFYTM
NHL-008TRANSCEND-NHL-001
Phase III I
Target Enrollment 500 274
Design
Arm A: REVLIMID® (10-20mg, D1-21) + rituximab (375 mg/m2
weekly for cycle 1 then D1 of cycles 3, 5,7,9 and 11 for 12 28-D cycles) followed by REVLIMID® (10mg, D1-21) + rituximab (375 mg/m2 D1 of cycles 13,15,17,19,21,23,25,27 and 29 for 18 28-D
cycles) followed by REVLIMID® (10mg, D1-21 until disease progression, 28 D cycle)
Arm B: REVLIMID® (10-20mg, D1-21) + rituximab (375 mg/m2
weekly for cycle 1 then D1 of cycles 3, 5,7,9 and 11 for 12 28-D cycles) followed by REVLIMID® (10mg, D1-21) + rituximab (375 mg/m2 D1 of cycles 13,15,17,19,21,23,25,27 and 29 for 18 28-D
cycles)
Arm A: JCAR017 single-dose scheduleArm B: JCAR017 2-dose schedule
Primary Endpoint Progression Free Survival Objective Response Rate; Safety
StatusTrial enrolling
Data expected in 2020Enrollment complete
43
Lymphoma Late-Stage/Pivotal Programs
Patient Population
Aggressive Relapsed orRefractory B-Cell Lymphoma
MoleculeLiso-cel
(lisocabtagene maraleucel; JCAR017)
Trial Name TRANSCEND WORLD
Phase II
Target Enrollment 124
DesignArm A: JCAR017 (1 x 108 positive
transfected viable T cells on D 1; 2 to 7 days after completion of lymphodepleting
chemotherapy).
Primary Endpoint Overall Response Rate
Status Trial enrolling
44
Solid Tumor Late-Stage/Pivotal Programs
Patient Population Adjuvant Therapy in Surgically Resected Pancreatic Cancer Newly Diagnosed Glioblastoma
Molecule ABRAXANE® Marizomib
Trial NamePANC-003
apact®EORTC-BTG-1709
Phase III III
Target Enrollment 866 750
Design
Arm A: ABRAXANE® (125 mg/m2); Gemcitabine (1000 mg/m2) D1,8,15 for 6 28-
D cyclesArm B: Gemcitabine (1000 mg/m2) D1,8,15
for 6 28-D cycles
Arm A: Radiotherapy + temozolomide + marizomib followed by adjuvant
temozolomide + marizomib
Arm B: Radiotherapy + temozolomide followed by adjuvant temozolomide
Primary Endpoint Disease Free Survival Overall Survival
StatusEnrollment complete
Data expected in 2018 (event driven)Trial enrolling
45
I&I Late-Stage/Pivotal Programs
Patient Population Active Behçet’s Disease Scalp Psoriasis
Molecule OTEZLA® OTEZLA®
Trial NameBCT-002RELIEF®
SPSO-001STYLETM
Phase III III
Target Enrollment 208 300
DesignArm A: Placebo (for 12 weeks) followed by OTEZLA® (30mg twice daily for 52 weeks)Arm B: OTEZLA® (30mg twice daily for 64
weeks)
Arm A: Placebo (for 16 weeks) followed by OTEZLA® (30mg twice daily for 16 weeks)
Arm B: Placebo (for 32 weeks)
Primary Endpoint Area under the curve (AUC) for the number of oral ulcers from baseline through week 12
Proportion of subjects with ScPGA score of clear (0) or almost clear (1) with at least a 2-
point reduction from baseline at Week 16
Status
Met primary endpointData presented at AAD 2018
sNDA submitted; Additional regulatory submissions planned
Met primary endpoint
46
I&I Late Stage Programs
Patient Population Moderate to Severe Ulcerative Colitis
Moderately to Severely Active Crohn's Disease
Moderately to Severely Active Crohn's Disease
Molecule Ozanimod Ozanimod Ozanimod
Trial Name TRUE NORTHTM RPC01-3201 RPC01-3202
Phase III III III
Target Enrollment 900 600 600
DesignArm A: Ozanimod (1mg daily) for
induction and maintenanceArm B: Placebo induction and
maintenance
Arm A: Ozanimod (0.92 mg daily) with a 7-D dose escalation
Arm B: Placebo
Arm A: Ozanimod (0.92 mg daily) with a 7-D dose escalation
Arm B: Placebo
Primary EndpointClinical remission assessed by Mayo component sub-scores at week 10
Clinical remission assessed by Mayo component sub-scores at week 52
Proportion of subjects with a CDAI score < 150 at Week 12
Proportion of subjects with a CDAI score < 150 at Week 12
Status Enrollment expected to complete by mid-2019 Trial enrolling Trial enrolling
47
I&I Late Stage Programs
Patient Population
Maintenance for Moderately to Severely Active Crohn's Disease
Relapsing Multiple Sclerosis Relapsing Multiple Sclerosis
Molecule Ozanimod Ozanimod Ozanimod
Trial Name RPC01-3203 SUNBEAMTM RADIANCETM
Phase III III III
Target Enrollment 485 ~1,300 ~1,300
DesignArm A: Ozanimod (0.92 mg daily
for 40 weeks)Arm B: Placebo (daily for 40
weeks)
Arm A: Ozanimod (0.5mg daily) + placebo IM weekly
Arm B: Ozanimod (1mg daily) + placebo IM weekly
Arm C: Placebo (daily) + beta-interferon IM weekly
Arm A: Ozanimod (0.5mg daily) + placebo IM weekly
Arm B: Ozanimod (1mg daily) + placebo IM weekly
Arm C: Placebo (daily) + beta-interferon IM weekly
Primary Endpoint
Proportion of subjects with a CDAI score of < 150 at week 40
Proportion of subjects with a (SES-CD) score decrease from baseline of ≥ 50% at week 40
Annualized relapse rate at month 12 Annualized relapse rate at month 24
Status Trial not yet enrolling
Data presented at ECTRIMS 2017 and AAN 2018
NDA expected to be resubmitted in Q1:19; MAA expected to be submitted in
Q1:19
Data presented at ECTRIMS 2017 and AAN 2018
NDA expected to be resubmitted in Q1:19; MAA expected to be submitted in Q1:19
48