Q1 2018 Conference Call...May 04, 2018 · Q1 2018 Conference Call Mark Alles, Chairman & Chief...
Transcript of Q1 2018 Conference Call...May 04, 2018 · Q1 2018 Conference Call Mark Alles, Chairman & Chief...
Q1 2018 Conference CallMay 4, 2018
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Q1 2018 Conference Call
Mark Alles, Chairman & Chief Executive Officer
Jay Backstrom, MD, Chief Medical Officer
Nadim Ahmed, President, Hematology & Oncology
Q&A
Terrie Curran, President, I&I
Peter Kellogg, 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 contains adjusted financial measures. Further information relevant to the interpretation of adjusted financial measures, and reconciliations of these adjusted financial measures to the most comparable GAAP measures, may be found in the Appendix and on our website at www.Celgene.com in the “Investor Relations” section.
Mark AllesChairman & Chief Executive Officer
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Q1 2018: Creating Long-Term ValueQ1 2018: Creating Long-Term Value
Delivering Exceptional Financial Results− Strong performance across the portfolio and geographies
− Growth driven primarily by volume and favorable market dynamics
Advancing the Late-Stage Pipeline− Defined regulatory path forward for ozanimod in relapsing multiple sclerosis in U.S. and Europe
− Significant clinical and regulatory inflection points over next 12-18 months
Strengthening the Organization− Adding critical expertise to Board of Directors− Aligned management organization to enhance communication, accountability and focus
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Peter KelloggChief Financial Officer
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Q1 2018 Financial HighlightsQ1 2018 Financial Highlights
Exceptional Operating Results − Net product sales grew 20% Y/Y− Adjusted diluted EPS $2.10 (+26% Y/Y) excluding dilution from Juno acquisition;
$2.05 (+23% Y/Y) with dilution
2018 Revenue Guidance Raised; Adjusted EPS Updated for Acquisition− Total revenue raised to the higher end of the range of $14.4B-$14.8B; REVLIMID® net sales raised
from ~$9.4B to ~$9.5B; POMALYST® net sales raised from ~$1.9B to ~$2B − 2018 adjusted diluted EPS raised to ~$8.95 excluding acquisition dilution;
~$8.45 inclusive of Juno dilution7
Strong Product Performance− Strong year-over-year product sales growth across the portfolio− 15 of the 20 percentage points of net sales growth from volume
Strategic and Balanced Capital Deployment to Support Future Growth− Completed Impact Biomedicines and Juno Therapeutics acquisitions− $2.7B in shares repurchased in Q1:18
Q1 2018 Total Net Product Sales
Q1:16 Q1:17 Q1:18
$ M
illio
ns ↑21% ↑18% ↑20%
$0
$500
$1,000
$1,500
$2,000
$2,500
$3,000
$3,500
$4,000
Q1:17 Volume Price FX/Hedge Q1:18
↑19.6%↑0.2%↑15.5% ↑3.9%
Contribution to Q1:18 Total Net Product Sales Growth
$ M
illio
ns
Total Net Product Sales
Growth Rates = Growth vs. Prior Year Period8
$2,495
$2,952
$3,531
Q1 2018 Adjusted Diluted Earnings Per Share
Q1:16 Q1:17 Q1:18
↑23% ↑27%
Dol
lars
Per
Sha
re
↑23%
Q1:17 Oper. Income
OIE Tax Rate
Share Count
Q1:18
Dol
lars
Per
Sha
re
$2.05$0.34$1.67 ($0.02) $0.11($0.05)
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Contribution to Q1:18 Adjusted Diluted EPSAdjusted Diluted EPS
$1.32
$1.67
$2.05
Growth Rates = Growth vs. Prior Year Period
As previously disclosed, during the third quarter of 2017, we adopted ASU 2017-12 with an initial application date of January 1, 2017. Prior to the adoption of ASU 2017-12, we recognized all changes in the fair value of the excluded component of a hedge in Other income net in the Consolidated Statements of Income under a mark-to-market approach. Pursuant to the provisions of ASU 2017-12, we no longer recognize the adjustments to the fair value of the excluded component in Other income net but we instead recognize the initial value of the excluded component using an amortization approach over the life of the hedging instrument. As such, our results for the quarterly period ended March 31, 2017 have been recast to reflect the adoption of ASU 2017-12.
Key P&L Line Items (Adjusted)
Q1:18 ∆ vs.Q1:17
∆ vs.Q4:17
Product Gross Margin 96.4% - ↓ 40 bps
R&D Expenses% of revenue
$694M19.6% ↓ 50 bps ↓ 240 bps
SG&A Expenses% of revenue
$671M19.0% ↑ 80 bps ↓ 70 bps
Operating Margin 57.9% ↓ 20 bps ↑ 280 bps
Effective Tax Rate 17.3% ↑ 80 bps ↑ 330 bps
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Capital Allocation in Q1 2018Capital Allocation in Q1 2018
Cash flow from operations was approximately $(325)M– Primarily impacted by $1.1B upfront cash payment to acquire Impact Biomedicines
Deployed $10B of cash for two strategic acquisitions: Impact Biomedicines and Juno Therapeutics
Issued aggregate of $4.5B in unsecured notes
Purchased $2.7B of shares– Authorized additional $5B; $3.1B remaining under stock repurchase program as of 3/31/18
(in $ Billions) 12/31/17 3/31/18
Cash, Cash Equivalents, Marketable Debt Securities and Publicly-Traded Equity Securities
$12.04 $4.74
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2018 Guidance
Previous*Updated w/o Dilution from
Juno
Updated w/ Dilution from
Juno
Total Revenue $14.4B-$14.8B ~$14.8B ~$14.8B
REVLIMID® Net Sales ~$9.4B ~$9.5B ~$9.5B
POMALYST®/IMNOVID® Net Sales ~$1.9B ~$2.0B ~$2.0B
OTEZLA® Net Sales ~$1.5B Unchanged Unchanged
ABRAXANE® Net Sales ~$1.0B Unchanged Unchanged
Adjusted Operating Margin ~60.0% Unchanged ~56.0%
Adjusted Tax Rate ~18% ~17.5% ~17%
Adjusted Diluted EPS $8.70-$8.90 ~$8.95 ~$8.45
Weighted Average Diluted Shares ~775M ~755M ~755M12
* Previous 2018 guidance did not include the impact of our acquisition of Juno, which was expected to be dilutive to adjusted diluted EPS by approximately $0.50.
Terrie CurranPresident, I&I
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Q1 2018 I&I Franchise Results
Solid OTEZLA® Net Product Sales and Operating Momentum – Q1:18 sales growth +46% Y/Y and volume growth +46% Y/Y– Sales performance driven by market share gains in the U.S. and international markets
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Executing on Path Forward for Ozanimod in MS– U.S. NDA resubmission expected in Q1:19– European MAA submission expected in Q1:19
Portfolio Optimization and Expansion Underway– Executing multiple OTEZLA® lifecycle opportunities– Progressing phase III trial enrollment with ozanimod in UC– Advancing development of early- and mid-stage pipeline assets– Discontinued GED-0301 development in UC
Net Sales ($M)
$175 $199
$276
$21 $43
$77
Q1:15 Q1:16 Q1:17 Q1:18
U.S. ROW
$60
$196
$242
Q1 2018 OTEZLA® Net Sales Summary
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Current Results & Potential Future Growth Drivers
• Q1:18 sales $353M, +46% Y/Y; Volume growth +46% Y/Y
• International sales +79% Y/Y
• Accelerating OTEZLA® growth with strong volume gains as demand and pull-through continues to improve
– U.S. TRx gains supported by improved access and further market expansion
– Increasing adoption in key ex-U.S. markets• Potential future growth drivers
– Completed enrollment in scalp psoriasis Ph III– Development of new indications, QD formulation
and label enhancement opportunities to expand OTEZLA® clinical profile and utilization
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention; volume measured in 30-day equivalent units
$353
19% 16% 16% 16% 16% 16% 15% 13% 13% 12% 11% 10% 12%
10%10% 11% 11% 11% 12% 11% 11% 10% 9% 9% 10% 10%
49% 51% 51% 50% 50% 49% 51% 51% 52% 52% 53% 53% 52%
22% 23% 23% 23% 23% 23% 23% 24% 25% 27% 27% 27% 26%
0%
20%
40%
60%
80%
100%
Jan'17 Feb'17 Mar'17 Apr'17 May'17 Jun'17 Jul'17 Aug'17 Sep'17 Oct'17 Nov'17 Dec'17 Jan'18
Biologics Oral Systemics Topicals No Treatment
Expanding the U.S. Pre-Biologic Market Opportunity
16Source: SHS claims data through January 2018, last updated 5 April 2018, 60 day grace period; Symphony prescriber-level data through 30 March 2018
OTEZLA® Plaque Psoriasis Source of Business (Most Intense Treatment – 12 month lookback, 3-month rolling view)
OTEZLA®
21.9%
0%
5%
10%
15%
20%
25%
30%
35%
40%
ENBREL STELARA HUMIRA COSENTYX
OTEZLA TALTZ TREMFYA
Psoriasis Market Share(Branded Market Basket)
Japan: Psoriasis Market Share(Full Market Basket)France: Launch-Aligned New Patient Starts
In France and Japan, OTEZLA® Patient Uptake Continuing to Accelerate
Source: Gers; IMS JPM 17
3% 11% 15%
June 2017 September 2017 December 2017
Other
Biologics
TIGASON
NEORAL
Topicals
OTEZLA0
500
1,000
1,500
2,000
2,500
3,000
3,500
4,000
4,500
5,000
5,500
m1 m2 m3 m4 m5 m6 m7 m8 m9 m10 m11 m12 m13 m14 m15
Cosentyx Stelara Taltz OtezlaCOSENTYX STELARA TALTZ OTEZLA
New
Pat
ient
Sta
rts
2018 I&I Franchise Outlook
Expand OTEZLA® Opportunity in Dermatology and Rheumatology Realize pre-biologic market opportunity for appropriate patients Data expected from Ph III trial in scalp psoriasis; Ph III in mild to moderate PSOR expected to initiate FDA decision on once-daily formulation; U.S. and Japan NDA submissions for Behçet’s disease
Maximize Ozanimod Opportunity in MS Incorporate supplemental nonclinical and clinical pharmacology data into NDA for resubmission Prepare U.S. and Europe registration dossiers
Advance Future Growth Drivers Advance Ph II development of CC-220 in SLE and CC-90001 in IPF Multiple assets entering early-stage clinical development
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Evolve IBD Development Portfolio Complete enrollment of ozanimod Ph III TRUE NORTHTM trial in moderate to severe UC
expected by mid-2019 Begin Ph III trial initiation activities for OTEZLA® in mild to moderate UC
Jay Backstrom, MDChief Medical Officer
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0.3500.241 0.181
0.000
0.100
0.200
0.300
0.400
0.500
0.600
1
Adj
uste
d A
RR
(±95
% C
I)
0.276 0.218 0.1720
0.1
0.2
0.3
0.4
0.5
0.6
1
Adj
uste
d A
RR
(±95
% C
I)
Ozanimod Regulatory Filings Supported by Clinical Efficacy & Safety Data from Two Phase III Trials in Patients with RMS
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SUNBEAMTM – Over 1 year
448 451 447n
31% reductionP=0.0013
48% reductionP<0.0001
IFN β-1a 30 µg
(n= 885)
Ozanimod 0.5 mg
(n = 892)
Ozanimod 1.0 mg
(n = 882)
Any AE701
(79.2%)585
(65.6%)592
(67.1%)
Serious AE39
(4.4%)47
(5.3%)41
(4.6%)AE Leading to Study Drug Discontinuation
34(3.8%)
21(2.4%)
26 (2.9%)
Death on study** 01
(0.1%)0RADIANCETM PART B – Over 2 years
SUNBEAMTM and RADIANCETM PART B PooledSummary of Adverse Events
21% reductionP=0.0167 38% reduction
P<0.0001
441 439 433n
**Drowning, unrelated to treatment, on study day 637
• No subjects had a 2nd degree or higher AV block• Infection risk with ozanimod was comparable to
treatment with IFN β-1a (Avonex®) • Ozanimod resulted in low levels of liver enzyme
elevations
IFN β-1a Ozanimod 1.0 mgOzanimod 0.5 mg
Data from AAN 2018: Cree, et. al. #006 and Kappos, et.al, #005Based on the Poisson regression model, adjusted for region (Eastern Europe vs rest of world), age at baseline, and baseline number of gadolinium-enhancing lesions. Natural log transformation of time on study included as an offset term. ARR, annualized relapse rate; CI, confidence interval; IFN β-1a, interferon β-1a; ITT, intent-to-treat.
IFN β-1a Ozanimod 1.0 mgOzanimod 0.5 mg
U.S. & EU Ozanimod Regulatory Submissions Expected in Q1:19
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Ozanimod and CC-112273 Profile
• Ozanimod is metabolized in humans to form one major active metabolite (CC-112273) and other minor active metabolites
• Characteristics of CC-112273 include:• Accounts for the majority of the total
activity of ozanimod in humans• Minor metabolite in animal species• Structurally similar to ozanimod• Similar potency and selectivity to
ozanimod for S1P1 and S1P5 • Tmax of 6-10 hours • Half-life of 10-13 days
Regulatory Path Forward
Following the Type A meeting with FDA in early April, our resubmission plan includes:– Bridging non-clinical studies– Utilizing existing PK/PD data
Additional human clinical efficacy and safety studies not needed
U.S. NDA resubmission expected in Q1:19
EU MAA submission expected in Q1:19
Nadim AhmedPresident, Hematology & Oncology
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Q1 2018 Hematology & Oncology Franchise ResultsQ1 2018 Hematology & Oncology Franchise Results
Strong Net Product Sales and Operating Momentum into 2018− Q1:18 net product sales: $3.2B, +17% Y/Y− Sales performance driven by strong demand across geographies and brands
Growth Drivers On Track− REVLIMID® continues to grow across geographies with NSCT and post-ASCT maintenance launches− POMALYST®/IMNOVID® growth continues through gains in market share and duration− Ph III data expected on luspatercept in MDS and beta-thalassemia, REVLIMID® in R/R FL,
ABRAXANE® in adjuvant pancreatic cancer
Advancement and Expansion of Innovative Pipeline− Advancing cellular immunotherapy leadership; Updated data on liso-cel (JCAR017) and bb2121
expected at ASCO. Completed enrollment in pivotal liso-cel TRANSCEND U.S. trial− Expansion of myeloid portfolio through the fedratinib acquisition− Advanced new MM assets JCARH125 (BCMA CAR T), CC-93269 (BCMA T cell engager) and CC-92480
(new CELMoD® compound) into the clinic
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$811$997
$1,234$1,487
$532
$577
$650
$747
Q1:15 Q1:16 Q1:17 Q1:18
U.S. ROW
Current Results & Potential Future Growth Drivers
Q1 2018 REVLIMID® Net Sales Summary Q1 2018 REVLIMID® Net Sales Summary
• Q1:18 sales $2,234M, +19% Y/Y• Strong front line launch momentum; market
share continues to grow ex-U.S.– REVLIMID® NSCT share growth continues ex-U.S.– Duration continues to grow globally
– Post-ASCT maintenance adoption in early launch markets
• Adoption of triplet combinations leading to increased duration and continuous treatment
• Potential future growth drivers Ph III AUGMENTTM study in relapsed indolent lymphoma
expected to read out in 2018
Ph III ROBUST® study in 1st line diffuse large B-cell lymphoma (event-driven)
REVLIMID®-based triplet regimen Ph III data readouts expected in NDMM during 2018
24
Net Sales ($M)
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
$1,343
$1,574
$1,884
$2,234
Current Results & Potential Future Growth Drivers
Q1 2018 POMALYST®/IMNOVID® Net Sales SummaryQ1 2018 POMALYST®/IMNOVID® Net Sales Summary
$129$171
$216
$300$70
$103
$148
$153
Q1:15 Q1:16 Q1:17 Q1:18
U.S. ROW
$274
$453
• Q1:18 sales $453M, +24% Y/Y• POMALYST®/IMNOVID® growth continues
– Strong momentum in the U.S. continues for POMALYST®
in combination with daratumumab and dex in RRMM– Share and duration trends increasing through the use of
triplet regimens • Potential future growth drivers
Ph III OPTIMISMM® trial of PVd in 2nd line+ MM (oral presentation at ASCO)
Newer triplet regimens expected to increase share and duration for POMALYST®/IMNOVID®
Net Sales ($M)
25Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
$364
$199
Current Results & Potential Future Growth Drivers
Q1 2018 ABRAXANE® Net Sales SummaryQ1 2018 ABRAXANE® Net Sales Summary
$159 $144 $142 $159
$64 $81 $94$103
Q1:15 Q1:16 Q1:17 Q1:18
U.S. ROW
$223 $225$236
$262
Net Sales ($M)
26
• Q1:18 sales $262M, +11% Y/Y• Positive Q1 growth included impact of buying
patterns• Potential future growth drivers
Ph III apact® of ABRAXANE® in adjuvant pancreatic cancer data readout expected in 2018
Ph III I/O combination trials reporting out in 2018:IMpower131* squamous NSCLC IMpower130* non-squamous NSCLC IMpassion130* triple negative breast cancer
*IMpower130, IMpower131 and IMpassion130 are Genentech, a member of the Roche Group, sponsored clinical trials.Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
Expected Data Presentations at ASCO 2018Expected Data Presentations at ASCO 2018
27
Ph III OPTIMISMM® trial with POMALYST® in combination with bortezomib and dexamethasone (PVd) in 2nd line+ MM
Ph III RELEVANCE® trial with REVLIMID® in combination with rituximab (R²) in patients with previously untreated FL
Ph III IMpower131* trial with ABRAXANE® in combination with atezolizumab in squamous NSCLC
Updated data from Ph I trial with bb2121 in RRMM Updated data from pivotal TRANSCEND U.S. trial with liso-
cel (JCAR017) in relapsed or refractory DLBCL
* IMpower131 is a Genentech, a member of the Roche Group, sponsored clinical trial.
2018 Hematology & Oncology Franchise Outlook2018 Hematology & Oncology Franchise Outlook
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Commercial Portfolio On Track to Deliver Robust Results in 2018− Strong momentum continues for REVLIMID® and POMALYST®/IMNOVID®
− REVLIMID® positioned for continued growth in NSCT and post-ASCT maintenance settings − POMALYST®/IMNOVID® share and duration growth continues in RRMM
Positioned for Near-Term Growth with Upcoming Milestones− Ph III readout for AUGMENTTM (REVLIMID® in R/R FL)− Ph III readout for MEDALISTTM (luspatercept in MDS) and BELIEVETM (luspatercept in
beta-thalassemia)− Ph III readout for apact® (ABRAXANE® in adjuvant PanC)− Fedratinib U.S. NDA submission
Mid- and Late-Stage Pipeline Advancing− Advancing MM programs through two major campaigns: BCMA (bb2121, CC-93269), CELMoD®
compounds (CC-220, CC-92480) − Advancing leadership in lymphoma: liso-cel (JCAR017), CC-122− Additional pivotal programs advancing: tislelizumab (BGB-A317), marizomib, luspatercept
Q1 2018 Conference CallMay 4, 2018
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Use of Non-GAAP Financial Measures and
Reconciliation Tables
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Use of Non-GAAP Financial Measures
31
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 ofperformance 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 measuresprovide useful and meaningful information to us and investors because they enhance investors’ understanding of the continuing operating performance of our business andfacilitate the comparison of performance between past and future periods. These adjusted financial measures are non-GAAP measures and should be considered in addition to, butnot as a substitute for, the information prepared in accordance with U.S. GAAP. When preparing these supplemental non-GAAP financial measures we typically exclude certainGAAP 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. Othercompanies 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 adjustedfinancial 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/ortiming. 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 and success payments. We alsoexclude transaction and certain other cash costs associated with business acquisitions and divestitures that are not normal recurring operating expenses, including severance costswhich are not part of a formal restructuring program.
Use of Non-GAAP Financial Measures
32
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 aremade 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 withrespect to particular programs. The variability of amounts and lack of predictability of collaboration-related upfront expenses makes the identification of trends in our ongoingresearch 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 operatingresearch and development expenses and facilitates comparisons between periods and with respect to projected performance. All expenses incurred subsequent to the initiation of thecollaboration arrangement, such as research and development cost-sharing expenses/reimbursements and milestone payments up to the point of regulatory approval are considered tobe 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.
Use of Non-GAAP Financial Measures
33
Certain Other Items: We exclude certain other significant items that may occur occasionally and are not normal, recurring, cash operating expenses from our adjustedfinancial 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 certainother significant items excluded from adjusted financial results would be: significant litigation-related loss contingency accruals and expenses to settle other disputed mattersand, effective for fiscal year 2018, changes in the fair value of our equity securities upon the adoption of ASU 2016-01 (Financial Instruments-Overall: Recognition andMeasurement of Financial 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 our adjusted financial results. The netincome 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 taxlaw 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, recurringoperations (“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 taxbenefits related to prior year tax positions, the impact of tax reform legislation commonly referred to as the Tax Cuts and Jobs Act (2017 Tax Act), and other similar items. Wealso exclude excess tax benefits and tax deficiencies that arise upon vesting or exercise of share-based payments recognized as income tax benefits or expenses due to theirnature, 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 adjusted measures for the three-month periods ended March 31, 2018 and 2017, and for the projected amounts for the twelve-month period ending December 31, 2018.
Reconciliation Tables
34
2018
2017
*
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1,84
9
Ope
ratin
g in
com
e21
8
1,
113
Inte
rest
and
inve
stm
ent i
ncom
e, n
et13
15
In
tere
st (e
xpen
se)
(166
)
(127
)
Oth
er in
com
e, n
et96
5
13
Inco
me
befo
re in
com
e ta
xes
1,03
0
1,01
4
Inco
me
tax
prov
ision
18
4
82
Net
inco
me
846
$
932
$
Net
inco
me
per c
omm
on s
hare
:B
asic
1.13
$
1.20
$
Dilu
ted
1.10
$
1.15
$
Wei
ghte
d av
erag
e sh
ares
:B
asic
748.
3
779.
0
Dilu
ted
768.
3
811.
2
As
Rep
orte
dA
s R
evise
dN
et p
rodu
ct s
ales
2,95
0$
2,95
2$
Oth
er in
com
e, n
et26
13
In
com
e ta
x pr
ovisi
on84
82
N
et in
com
e94
1
93
2
D
ilute
d ne
t inc
ome
per c
omm
on s
hare
1.16
$
1.15
$
Mar
ch 3
1,D
ecem
ber 3
1,20
1820
17B
alan
ce s
heet
item
s:
Cas
h, c
ash
equi
vale
nts,
debt
sec
uriti
es a
vaila
ble-
for-
sale
and
equ
ity
inv
estm
ents
with
read
ily d
eter
min
able
fair
valu
es4,
740
$
12
,042
$
Tota
l ass
ets
34,5
56
30
,141
Long
-term
deb
t, in
clud
ing
curr
ent p
ortio
n20
,271
15,8
38
To
tal s
tock
hold
ers'
equi
ty5,
172
6,
921
Cel
gene
Cor
pora
tion
and
Subs
idia
ries
Con
dens
ed C
onso
lidat
ed S
tate
men
ts o
f Inc
ome
(Una
udite
d)(In
mill
ions
, exc
ept p
er s
hare
dat
a)
Mar
ch 3
1,Th
ree-
Mon
th P
erio
ds E
nded
* D
urin
g th
e th
ird q
uarte
r of 2
017,
we
adop
ted
ASU
201
7-12
with
an
initi
al a
pplic
atio
n da
te o
f Jan
uary
1, 2
017.
Prio
r to
the
adop
tion
of A
SU 2
017-
12,
we
reco
gnize
d al
l cha
nges
in th
e fa
ir va
lue
of th
e ex
clud
ed c
ompo
nent
of a
hed
ge in
Oth
er in
com
e, n
et in
the
Con
solid
ated
Sta
tem
ents
of I
ncom
e un
der a
mar
k-to
-mar
ket a
ppro
ach.
Pur
suan
t to
the
prov
ision
s of
ASU
201
7-12
, we
no lo
nger
reco
gnize
the
adju
stm
ents
to th
e fa
ir va
lue
of th
e ex
clud
ed c
ompo
nent
in O
ther
inco
me,
net
but
we
inst
ead
reco
gnize
the
initi
al v
alue
of t
he e
xclu
ded
com
pone
nt u
sing
an a
mor
tizat
ion
appr
oach
ove
r th
e lif
e of
the
hedg
ing
inst
rum
ent.
The
resu
lts fo
r the
qua
rterly
per
iod
ende
d M
arch
31,
201
7 ha
ve b
een
reca
st to
refle
ct th
e ad
optio
n of
ASU
201
7-12
. The
thre
e-m
onth
per
iod
ende
d M
arch
31,
201
7 in
clud
es th
e fo
llow
ing
imm
ater
ial r
evisi
ons
to p
revi
ously
issu
ed fi
nanc
ial r
esul
ts:
Thre
e-M
onth
Per
iod
Ende
dM
arch
31,
201
7
Reconciliation Tables
35
2018
2017
*
Net
inco
me
- GA
AP
846
$
932
$
Bef
ore
tax
adju
stm
ents
:C
ost o
f goo
ds s
old
(exc
ludi
ng a
mor
tizat
ion
of a
cqui
red
inta
ngib
le a
sset
s):
Shar
e-ba
sed
com
pens
atio
n ex
pens
e (1
)9
7
Res
earc
h an
d de
velo
pmen
t:Sh
are-
base
d co
mpe
nsat
ion
expe
nse
(1)
199
65
C
olla
bora
tion-
rela
ted
upfr
ont e
xpen
se(2
)24
5
10
Res
earc
h an
d de
velo
pmen
t ass
et a
cqui
sitio
n ex
pens
e(3
)1,
125
32
5
A
djus
tmen
t to
clin
ical
tria
l and
dev
elop
men
t act
ivity
win
d-do
wn
char
ge(4
)(6
0)
-
Sellin
g, g
ener
al a
nd a
dmin
istra
tive:
Shar
e-ba
sed
com
pens
atio
n ex
pens
e(1
)19
3
81
Am
ortiz
atio
n of
acq
uire
d in
tang
ible
ass
ets
(5)
87
82
Acq
uisit
ion
rela
ted
char
ges
and
rest
ruct
urin
g, n
et:
Cha
nge
in fa
ir va
lue
of c
ontin
gent
con
sider
atio
n an
d su
cces
s pa
ymen
ts(6
)(3
0)
39
Acq
uisit
ion
rela
ted
char
ges
(7)
61
-
Oth
er in
com
e, n
et:
Cha
nges
in fa
ir va
lue
of e
quity
inve
stm
ents
(8)
(959
)
-
Inco
me
tax
prov
ision
:Es
timat
ed ta
x im
pact
from
abo
ve a
djus
tmen
ts(9
)(1
33)
(1
11)
N
on-o
pera
ting
tax
adju
stm
ents
(10)
(11)
(75)
Net
inco
me
- A
djus
ted
1,57
2$
1,35
5$
Net
inco
me
per c
omm
on s
hare
- A
djus
ted
Bas
ic2.
10$
1.
74$
D
ilute
d2.
05$
1.
67$
Expl
anat
ion
of a
djus
tmen
ts:
(1)
Excl
ude
shar
e-ba
sed
com
pens
atio
n ex
pens
e to
talin
g $4
01, i
nclu
ding
$25
0 re
late
d to
Jun
o Th
erap
eutic
s, In
c. (J
uno)
, for
the
thre
e-m
onth
per
iod
end
thr
ee-m
onth
per
iod
ende
d M
arch
31,
201
7.(2
)Ex
clud
e up
fron
t pay
men
t exp
ense
for r
esea
rch
and
deve
lopm
ent c
olla
bora
tion
arra
ngem
ents
.(3
)Ex
clud
e re
sear
ch a
nd d
evel
opm
ent a
sset
acq
uisit
ion
expe
nses
.(4
)Ex
clud
e ad
just
men
t of c
linic
al tr
ial a
nd d
evel
opm
ent a
ctiv
ity w
ind-
dow
n ch
arge
ass
ocia
ted
with
the
disc
ontin
uanc
e of
GED
-030
1 cl
inic
al tr
ials
in C
(5)
Excl
ude
amor
tizat
ion
of in
tang
ible
ass
ets
acqu
ired
in th
e ac
quisi
tions
of P
harm
ion
Cor
p., G
louc
este
r Pha
rmac
eutic
als,
Inc.
(Glo
uces
ter)
, Abr
axis
B A
vilo
mic
s R
esea
rch,
Inc.
(Avi
la),
Qua
ntic
el P
harm
aceu
tical
s, In
c. (Q
uant
icel
) and
Jun
o.(6
)Ex
clud
e ch
ange
s in
the
fair
valu
e of
con
tinge
nt c
onsid
erat
ion
rela
ted
to th
e ac
quisi
tions
of G
louc
este
r, A
brax
is, A
vila
, Nog
ra P
harm
a Li
mite
d (N
o c
hang
es in
the
fair
valu
e of
Jun
o's
succ
ess
paym
ents
. (7
)Ex
clud
e ac
quisi
tion
cost
s re
late
d to
Jun
o.(8
)Ex
clud
e ch
ange
s in
the
fair
valu
e of
equ
ity in
vest
men
ts d
ue to
the
adop
tion
of A
SU 2
016-
01 (F
inan
cial
Inst
rum
ents
-Ove
rall:
Rec
ogni
tion
and
Mea
Fin
anci
al L
iabi
litie
s).
(9)
Excl
ude
the
estim
ated
tax
impa
ct o
f the
abo
ve a
djus
tmen
ts.
(10)
Excl
ude
othe
r non
-ope
ratin
g ta
x ex
pens
e ite
ms.
The
adj
ustm
ent f
or th
e th
ree-
mon
th p
erio
ds e
nded
Mar
ch 3
1, 2
018
and
Mar
ch 3
1, 2
017
is to
exc
l
and
$75,
resp
ectiv
ely,
reco
rded
in th
e In
com
e Ta
x Pr
ovisi
on a
s pe
r ASU
201
6-09
(Com
pens
atio
n-St
ock
Com
pens
atio
n).
As
Rep
orte
dA
s R
evise
dN
et in
com
e - G
AA
P94
1$
93
2$
N
et in
com
e - A
djus
ted
1,36
4
1,35
5
Dilu
ted
net i
ncom
e pe
r com
mon
sha
re -
Adj
uste
d1.
68$
1.
67$
Cel
gene
Cor
pora
tion
and
Subs
idia
ries
Rec
onci
liatio
n of
GA
AP
to A
djus
ted
Net
Inco
me
(In m
illio
ns, e
xcep
t per
sha
re d
ata)
Thre
e-M
onth
Per
iods
End
ed
* D
urin
g th
e th
ird q
uarte
r of 2
017,
we
adop
ted
ASU
201
7-12
with
an
initi
al a
pplic
atio
n da
te o
f Jan
uary
1, 2
017.
Prio
r to
the
adop
tion
of A
SU 2
017-
12,
we
reco
gnize
d al
l cha
nges
in th
e fa
ir va
lue
of th
e ex
clud
ed c
ompo
nent
of a
hed
ge in
Oth
er in
com
e, n
et in
the
Con
solid
ated
Sta
tem
ents
of I
ncom
e un
der a
mar
k-to
-mar
ket a
ppro
ach.
Pur
suan
t to
the
prov
ision
s of
ASU
201
7-12
, we
no lo
nger
reco
gnize
the
adju
stm
ents
to th
e fa
ir va
lue
of th
e ex
clud
ed c
ompo
nent
in O
ther
inco
me,
net
but
we
inst
ead
reco
gnize
the
initi
al v
alue
of t
he e
xclu
ded
com
pone
nt u
sing
an a
mor
tizat
ion
appr
oach
ove
r th
e lif
e of
the
hedg
ing
inst
rum
ent.
The
resu
lts fo
r the
qua
rterly
per
iod
ende
d M
arch
31,
201
7 ha
ve b
een
reca
st to
refle
ct th
e ad
optio
n of
ASU
201
7-12
. The
thre
e-m
onth
per
iod
ende
d M
arch
31,
201
7 in
clud
es th
e fo
llow
ing
imm
ater
ial r
evisi
ons
to p
revi
ously
issu
ed fi
nanc
ial r
esul
ts:
Thre
e-M
onth
Per
iod
Ende
dM
arch
31,
201
7
Mar
ch 3
1,
Reconciliation Tables
36
Upd
ated
w
ithou
t Dilu
tion
from
Jun
o
Upd
ated
with
D
ilutio
n fr
om
Juno
Proj
ecte
d ne
t inc
ome
- GA
AP
(1)
5,55
6$
4,76
7$
Bef
ore
tax
adju
stm
ents
:C
ost o
f goo
ds s
old
(exc
ludi
ng a
mor
tizat
ion
of a
cqui
red
inta
ngib
le a
sset
s):
Shar
e-ba
sed
com
pens
atio
n ex
pens
e 30
30
Res
earc
h an
d de
velo
pmen
t:Sh
are-
base
d co
mpe
nsat
ion
expe
nse
269
524
Col
labo
ratio
n-re
late
d up
fron
t exp
ense
257
257
Res
earc
h an
d de
velo
pmen
t ass
et a
cqui
sitio
n ex
pens
e1,
125
1,
125
A
djus
tmen
t to
clin
ical
tria
l and
dev
elop
men
t act
ivity
win
d-do
wn
char
ge(6
0)
(6
0)
Sellin
g, g
ener
al a
nd a
dmin
istra
tive:
Shar
e-ba
sed
com
pens
atio
n ex
pens
e 34
7
51
1
Am
ortiz
atio
n of
acq
uire
d in
tang
ible
ass
ets
257
319
Acq
uisit
ion
rela
ted
char
ges
and
rest
ruct
urin
g, n
et:
Cha
nge
in fa
ir va
lue
of c
ontin
gent
con
sider
atio
n an
d su
cces
s pa
ymen
ts(3
0)
(1
6)
A
cqui
sitio
n re
late
d ch
arge
s-
61
Oth
er in
com
e, n
et:
Cha
nges
in fa
ir va
lue
of e
quity
inve
stm
ents
(950
)
(950
)
Inco
me
tax
prov
ision
:Es
timat
ed ta
x im
pact
from
abo
ve a
djus
tmen
ts(3
3)
(1
77)
N
on-o
pera
ting
tax
adju
stm
ents
(11)
(11)
Proj
ecte
d ne
t inc
ome
- Adj
uste
d6,
757
$
6,
380
$
Proj
ecte
d ne
t inc
ome
per d
ilute
d co
mm
on s
hare
- G
AA
P~
7.36
$
~6.
31$
Proj
ecte
d ne
t inc
ome
per d
ilute
d co
mm
on s
hare
- A
djus
ted
~8.
95$
~
8.45
$
Proj
ecte
d w
eigh
ted
aver
age
dilu
ted
shar
es75
5.0
75
5.0
(1)
Our
pro
ject
ed 2
018
earn
ings
do
not i
nclu
de th
e ef
fect
of a
ny b
usin
ess
com
bina
tions
, col
labo
ratio
n ag
reem
ents
, ass
et
acqu
isitio
ns, a
sset
impa
irmen
ts, l
itiga
tion-
rela
ted
loss
con
tinge
ncy
accr
uals,
cha
nges
in th
e fa
ir va
lue
of o
ur C
VR
s iss
ued
as
part
of th
e ac
quisi
tion
of A
brax
is, c
hang
es in
the
fair
valu
e of
equ
ity in
vest
men
ts a
s pe
r ASU
201
6-01
(Fin
anci
al In
stru
men
ts-
Ove
rall:
Rec
ogni
tion
and
Mea
sure
men
t of F
inan
cial
Ass
ets
and
Fina
ncia
l Lia
bilit
ies)
or n
on-o
pera
ting
tax
adju
stm
ents
that
m
ay o
ccur
afte
r the
day
prio
r to
the
date
of t
his
pres
s re
leas
e.
Cel
gene
Cor
pora
tion
and
Subs
idia
ries
Rec
onci
liatio
n of
Ful
l-Yea
r 20
18 P
roje
cted
GA
AP
to A
djus
ted
Net
Inco
me
(In m
illio
ns, e
xcep
t per
sha
re d
ata)
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
2018 Milestones2018 Milestones
Financial Performance Total revenue $14.4B-$14.8B1 Total revenue ~$14.8B2
REVLIMID® net sales ~$9.4B1 REVLIMID® net sales ~$9.5B2
POMALYST® net sales ~$1.9B1 POMALYST® net sales ~$2.0B2
OTEZLA® net sales ~$1.5B1
ABRAXANE® net sales ~$1B1
Adj. operating margin ~60%1 Adj. operating margin ~56%2
Adj. diluted EPS $8.70 to $8.901 Adj. diluted EPS ~$8.452
Clinical Data Ph III AUGMENTTM – REVLIMID® in R/R FL Ph III ROBUST ® – REVLIMID® in 1st Line ABC-subtype DLBCL Ph III apact® – ABRAXANE® in adjuvant PanC
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 Ph III MEDALISTTM – Luspatercept in RS+ MDS Ph III BELIEVETM – Luspatercept in beta-thalassemia
Ph II OTEZLA ® in UC Trial EnrollmentComplete 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 enrolment in TRANSCEND WORLD – JCAR017 in DLBCL
Regulatory Submissions/Decisions Submit sNDA for RVd in NDMM Submit NDA for Fedratinib in myelofibrosis
FDA decision on Ozanimod in RMS FDA decision on OTEZLA ® once-daily formulation Submit sNDA for OTEZLA® in Behçet’s disease
Submit 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 BGB-A317 in NSCLC Initiate Ph III with OTEZLA® in mild-to-moderate PSOR Initiate Ph III trial with bb2121 in 3rd Line+ MM Initiate Ph III trial with JCAR017 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&ED File at least 5 IND’s
38
X
X
X
REVLIMID®
Del 5q MDS
VIDAZA®
MDS, AML
IDHIFA®
IDH2 RRAML
CC-486 MDS, AML Luspatercept
MDS, Beta-thalassemia
CC-90002AML
CC-90009RRAML
FT-1101MDS, AML
MyeloidDisease
11
Inflammation& Immunology
12
OzanimodMS
OTEZLA®
PSOR, PSA
OTEZLA®
Behçet’s, Scalp PSOR
RPC-4046EoE
OTEZLA®
UC
CC-220SLE
CC-90001 IPF
CC-90006PSOR
FT-4101NASH
LYC-30937UC, PSOR
MarketPh I
L E G E N D
REVLIMID®
MCL
ISTODAX®
PTCL, CTCL
REVLIMID®
NHL
CC-122NHL, CLL
JCAR017NHL
CC-486NHL
Lymphoma& Leukemia
10
LuspaterceptMF
CC-90002NHL
Advancing a High Quality Pipeline with Significant Potential
Celgene has an exclusive option to license JTX-2011, navicixizumab, OMP-313M32, FT-1101, FT-4101, AG-881 and an option to acquire LYC-55716, LYC-30937and 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 Tumors
CC-90011Solid Tumors
ABRAXANE®
PanC, NSCLC, mBC
AG-881Glioma
SolidTumors
14OMP-313M32
Solid Tumors
navicixizumabSolid Tumors
LYC-55716Solid Tumors
JTX-2011Solid Tumors
CC-90010Solid Tumors
BGB-A317Solid Tumors
OzanimodUC
OzanimodCD
FedratinibMF
FT-1101NHLCC-93269
RRMM
JCARH125RRMM MSC-1
Solid Tumors
JCAR017CLL
GEM333AML
AG-270Solid Tumors
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 Q1:18 (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. 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. 40
Multiple Myeloma Late-Stage/Pivotal Programs
Patient Population RRMM RRMM
Molecule POMALYST®/IMNOVID® bb2121
Trial NameMM-007
OPTIMISMM®
BB2121-MM-001KarMMaTM
Phase III II
Target Enrollment 559 94
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 -30 x 107 CAR T cells after receiving
lymphodepleting chemotherapy)
Primary Endpoint Progression Free Survival ORR
StatusPrimary endpoint met
Data to be presented at ASCO 2018
Trial enrolling
41
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 for more than 12 weeks Overall Survival
Status Trial enrollingEnrollment complete
Data expected in 2018
42
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
StatusEnrollment complete
Data expected in mid-2018Enrollment complete
Data expected in mid-2018
43
MDS/AML/MF Late-Stage/Pivotal Programs
Patient Population IDH2 Mutant AML
Molecule IDHIFA®
Trial Name IDHENTIFYTM
Phase III
Target Enrollment 316
DesignArm A: IDHIFA® (100 mg daily, 28-D cycle) + best
supportive careArm B: Best supportive care
Primary Endpoint Overall Survival
Status Trial enrolling
44
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-day cycles)
Arm B: Placebo (D1-21) + rituximab (375 mg/m2 weekly for cycle 1 then D1
of cycles 2-5 for 5 28-day cycles)
Arm A: REVLIMID® (starting dose 20mg,
D2-22 for up to 18 28-day cycles) + rituximab (starting dose 375 mg/m2
weekly for up to 12 28-day cycles)Arm B: Physician’s choice of
Rituximab-CHOP, Rituximab-CVP or Rituximab-bendamustine
Arm a: REVLIMID® (15mg, D1-14) + R-CHOP21 (6 21-day cycles)Arm B: Placebo + R-CHOP21
(6 21-day cycles)
Primary Endpoint Progression Free Survival Complete Response Rate and Progression Free Survival Progression Free Survival
StatusEnrollment complete
Data in H1:18E
Trial did not achieve superiority in co-primary endpoints
Data to be presented at ASCO 2018
Trial enrollingEvent-driven trial
45
Lymphoma Late-Stage/Pivotal Programs
Patient Population Relapsed or Refractory Indolent Lymphoma TRANSCEND
Molecule REVLIMID®JCAR017
(lisocabtagene maraleucel; liso-cel)
Trial NameMAGNIFYTM
NHL-008TRANSCENDTM
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-day 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-day cycles) followed by REVLIMID® (10mg, D1-21 until disease
progression, 28 day 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-day 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-day 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
46
Lymphoma Late-Stage/Pivotal Programs
Patient Population Aggressive Relapsed or Refractory B-Cell Lymphoma
MoleculeJCAR017
(lisocabtagene maraleucel; liso-cel)
Trial Name TRANSCEND WORLDTM
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 not yet enrolling
47
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-
day cyclesArm B: Gemcitabine (1000 mg/m2) D1,8,15
for 6 28-day 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 2018Trial not yet enrolling
48
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
StatusMet primary endpoint
Data presented at AAD 2018Regulatory submissions planned
Trial enrolling
49
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-day dose escalation
Arm B: Placebo
Arm A: Ozanimod (0.92 mg daily) with a 7-day 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
50
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
51