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Q4 and Full Year 2018 Conference Call
January 31, 2019
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q4:18 Conference Call
Mark Alles, Chairman & Chief Executive Officer
Nadim Ahmed, President, Hematology & Oncology
Jay Backstrom, MD, Chief Medical Officer
Q&A
David Elkins, Chief Financial Officer
2
Terrie Curran, President, Inflammation & Immunology
3
Important Information For Investors And Stockholders
This communication does not constitute an offer to sell or the solicitation of an offer to buy any securities or a solicitation of any vote or approval. It does not constitute a prospectus
or prospectus equivalent document. No offering of securities shall be made except by means of a prospectus meeting the requirements of Section 10 of the U.S. Securities Act of
1933, as amended.
In connection with the proposed transaction between Bristol-Myers Squibb Company (“Bristol-Myers Squibb”) and Celgene Corporation (“Celgene”), Bristol-Myers Squibb and
Celgene will file relevant materials with the Securities and Exchange Commission (the “SEC”), including a Bristol-Myers Squibb registration statement on Form S-4 that will include
a joint proxy statement of Bristol-Myers Squibb and Celgene that also constitutes a prospectus of Bristol-Myers Squibb, and a definitive joint proxy statement/prospectus will be
mailed to stockholders of Bristol-Myers Squibb and Celgene. INVESTORS AND SECURITY HOLDERS OF BRISTOL-MYERS SQUIBB AND CELGENE ARE URGED TO READ
THE JOINT PROXY STATEMENT/PROSPECTUS AND OTHER DOCUMENTS THAT WILL BE FILED WITH THE SEC CAREFULLY AND IN THEIR ENTIRETY WHEN THEY
BECOME AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION. Investors and security holders will be able to obtain free copies of the registration
statement and the joint proxy statement/prospectus (when available) and other documents filed with the SEC by Bristol-Myers Squibb or Celgene through the website maintained
by the SEC at http://www.sec.gov. Copies of the documents filed with the SEC by Bristol-Myers Squibb will be available free of charge on Bristol-Myers Squibb’s internet website
at http://www.bms.com under the tab, “Investors” and under the heading “Financial Reporting” and subheading “SEC Filings” or by contacting Bristol-Myers Squibb’s Investor
Relations Department through https://www.bms.com/investors/investor-contacts.html. Copies of the documents filed with the SEC by Celgene will be available free of charge on
Celgene’s internet website at http://www.celgene.com under the tab “Investors” and under the heading “Financial Information” and subheading “SEC Filings” or by contacting
Celgene’s Investor Relations Department at ir@celgene.com.
Certain Information Regarding Participants
Bristol-Myers Squibb, Celgene, and their respective directors and executive officers may be considered participants in the solicitation of proxies in connection with the proposed
transaction. Information about the directors and executive officers of Bristol-Myers Squibb is set forth in its Annual Report on Form 10-K for the year ended December 31, 2017,
which was filed with the SEC on February 13, 2018, its proxy statement for its 2018 annual meeting of stockholders, which was filed with the SEC on March 22, 2018, and its
Current Report on Form 8-K, which was filed with the SEC on August 28, 2018. Information about the directors and executive officers of Celgene is set forth in its Annual Report on
Form 10-K for the year ended December 31, 2017, which was filed with the SEC on February 7, 2018, its proxy statement for its 2018 annual meeting of stockholders, which was
filed with the SEC on April 30, 2018, and its Current Reports on Form 8-K, which were filed with the SEC on June 1, 2018, June 19, 2018 and November 2, 2018. Other information
regarding the participants in the proxy solicitations and a description of their direct and indirect interests, by security holdings or otherwise, will be contained in the joint proxy
statement/prospectus and other relevant materials to be filed with the SEC regarding the proposed transaction when they become available. You may obtain these documents
(when they become available) free of charge through the website maintained by the SEC at http://www.sec.gov and from Investor Relations at Bristol-Myers Squibb or Celgene as
described above.
Forward-Looking Statements and Adjusted Financial Information
4
This communication contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. You can generally identify forward-looking
statements by the use of forward-looking terminology such as “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “explore,” “evaluate,” “intend,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “seek,” “should,” or “will,” or the negative
thereof or other variations thereon or comparable terminology. These forward-looking statements are only predictions and involve known and unknown risks and uncertainties, many of which are beyond Bristol-Myers Squibb’s and Celgene’s control.
Statements in this communication regarding Bristol-Myers Squibb, Celgene and the combined company that are forward-looking, including projections as to the anticipated benefits of the proposed transaction, the impact of the proposed transaction on
Bristol-Myers Squibb’s and Celgene’s business and future financial and operating results, the amount and timing of synergies from the proposed transaction, the terms and scope of the expected financing for the proposed transaction, the aggregate
amount of indebtedness of the combined company following the closing of the proposed transaction, expectations regarding cash flow generation, accretion to non-GAAP earnings per share, capital structure, debt repayment, adjusted leverage ratio and
credit ratings following the closing of the proposed transaction, Bristol-Myers Squibb’s ability and intent to conduct a share repurchase program and declare future dividend payments, the combined company’s pipeline, intellectual property protection and
R&D spend, the timing and probability of a payment pursuant to the contingent value right consideration, and the closing date for the proposed transaction, are based on management’s estimates, assumptions and projections, and are subject to
significant uncertainties and other factors, many of which are beyond Bristol-Myers Squibb’s and Celgene’s control. These factors include, among other things, effects of the continuing implementation of governmental laws and regulations related to
Medicare, Medicaid, Medicaid managed care organizations and entities under the Public Health Service 340B program, pharmaceutical rebates and reimbursement, market factors, competitive product development and approvals, pricing controls and
pressures (including changes in rules and practices of managed care groups and institutional and governmental purchasers), economic conditions such as interest rate and currency exchange rate fluctuations, judicial decisions, claims and concerns that
may arise regarding the safety and efficacy of in-line products and product candidates, changes to wholesaler inventory levels, variability in data provided by third parties, changes in, and interpretation of, governmental regulations and legislation
affecting domestic or foreign operations, including tax obligations, changes to business or tax planning strategies, difficulties and delays in product development, manufacturing or sales including any potential future recalls, patent positions and the
ultimate outcome of any litigation matter. These factors also include the combined company’s ability to execute successfully its strategic plans, including its business development strategy, the expiration of patents or data protection on certain products,
including assumptions about the combined company’s ability to retain patent exclusivity of certain products, the impact and result of governmental investigations, the combined company’s ability to obtain necessary regulatory approvals or obtaining these
without delay, the risk that the combined company’s products prove to be commercially successful or that contractual milestones will be achieved. Similarly, there are uncertainties relating to a number of other important factors, including: results of clinical
trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA and other regulatory authorities, investigational review boards
at clinical trial sites and publication review bodies; the ability to enroll patients in planned clinical trials; unplanned cash requirements and expenditures; competitive factors; the ability to obtain, maintain and enforce patent and other intellectual property
protection for any product candidates; the ability to maintain key collaborations; and general economic and market conditions. Additional information concerning these risks, uncertainties and assumptions can be found in Bristol-Myers Squibb’s and
Celgene’s respective filings with the SEC, including the risk factors discussed in Bristol-Myers Squibb’s and Celgene’s most recent Annual Reports on Form 10-K, as updated by their Quarterly Reports on Form 10-Q and future filings with the SEC.
It should also be noted that projected financial information for the combined businesses of Bristol-Myers Squibb and Celgene is based on management’s estimates, assumptions and projections and has not been prepared in conformance with the
applicable accounting requirements of Regulation S-X relating to pro forma financial information, and the required pro forma adjustments have not been applied and are not reflected therein. None of this information should be considered in isolation from,
or as a substitute for, the historical financial statements of Bristol-Myers Squibb or Celgene. Important risk factors could cause actual future results and other future events to differ materially from those currently estimated by management, including, but
not limited to, the risks that: a condition to the closing of the proposed acquisition may not be satisfied; a regulatory approval that may be required for the proposed acquisition is delayed, is not obtained or is obtained subject to conditions that are not
anticipated; Bristol-Myers Squibb is unable to achieve the synergies and value creation contemplated by the proposed acquisition; Bristol-Myers Squibb is unable to promptly and effectively integrate Celgene’s businesses; management’s time and
attention is diverted on transaction related issues; disruption from the transaction makes it more difficult to maintain business, contractual and operational relationships; the credit ratings of the combined company declines following the proposed
acquisition; legal proceedings are instituted against Bristol-Myers Squibb, Celgene or the combined company; Bristol-Myers Squibb, Celgene or the combined company is unable to retain key personnel; and the announcement or the consummation of
the proposed acquisition has a negative effect on the market price of the capital stock of Bristol-Myers Squibb and Celgene or on Bristol-Myers Squibb’s and Celgene’s operating results.
No assurances can be given that any of the events anticipated by the forward-looking statements will transpire or occur, or if any of them do occur, what impact they will have on the results of operations, financial condition or cash flows of Bristol-Myers
Squibb or Celgene. Should any risks and uncertainties develop into actual events, these developments could have a material adverse effect on the proposed transaction and/or Bristol-Myers Squibb or Celgene, Bristol-Myers Squibb’s ability to
successfully complete the proposed transaction and/or realize the expected benefits from the proposed transaction. You are cautioned not to rely on Bristol-Myers Squibb’s and Celgene’s forward-looking statements. These forward-looking statements
are and will be based upon management’s then-current views and assumptions regarding future events and operating performance, and are applicable only as of the dates of such statements. Neither Bristol-Myers Squibb nor Celgene assumes any duty
to update or revise forward-looking statements, whether as a result of new information, future events or otherwise, as of any future date.
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
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
FY:18 Strong Operating Results & Pipeline Momentum
Delivering Excellent Operating Results
− Exceeded 2018 top- and bottom-line guidance; 2019 guidance of total revenue of
$17.0B-$17.2B and adjusted diluted earnings per share (EPS) of $10.60-$10.80
− Reaffirming 2020 outlook: total revenue of $19-$20B & adj. diluted EPS of >$12.50
Accelerating Inline and Pipeline Assets
− Five late-stage assets on-track for U.S. launches expected through 2020 –
ozanimod, fedratinib, luspatercept, liso-cel and bb2121
− Increasingly productive early R&D engine, including 7 novel INDs filed in 2018
Announced Acquisition by Bristol-Myers Squibb
− Creates a leading biopharma company while enhancing global leadership and
core competencies in high-value therapeutic areas, including oncology and I&I
− Recognizes and unlocks significant value for Celgene shareholders 6
▪ Two companies with one mission – discover, develop and deliver the most innovative medicines to patients with unmet medical needs across the continuum of care
▪ Recognizes and unlocks significant value for Celgene shareholders
– Delivers immediate and substantial cash value
– Provides meaningful participation in the combined company’s future growth
– Additional cash via dividends and potential contingent value right (CVR)
▪ Enhances global leadership and core competencies in high-value therapeutic categories across small molecules, biologics and cell therapies
▪ Accelerates research and development programs for sustainable long-term growth
▪ Combined company has the capabilities and financial strength to continue investing in external research partners
▪ Builds on the skills, dedication and passion of talented employees
Bristol-Myers Squibb: The Right Transaction for Celgene
7
David ElkinsChief Financial Officer
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q4:18 & FY:18 Financial Highlights
2018 Operating Results
− Q4:18 Y/Y net product sales grew 16% to $4.0B and adjusted diluted EPS grew 20% to $2.39
− 2018 Y/Y net product sales grew 18% to $15.3B and adjusted diluted EPS grew 19% to $8.87
2019 Guidance Based on Continued Operating Momentum
− Volume driven sales growth from REVLIMID®, POMALYST®, ABRAXANE ® and OTEZLA®
− Continued investment in key R&D programs while driving meaningful SG&A leverage
9
Strong Execution on Operating Metrics
− Strong product growth across the portfolio and geographies
− Significant R&D investments to support planned regulatory submissions
Strategic and Balanced Capital Deployment
− Invested ~$9.7B in acquisitions and over $3.5B in internal programs for FY 2018
− ~$6B in cash and marketable securities as of December 31, 2018
Q4:18 Total Net Product Sales
Q4:16 Q4:17 Q4:18
$ M
illio
ns
↑17% ↑17%↑16%
$0
$500
$1,000
$1,500
$2,000
$2,500
$3,000
$3,500
$4,000
$4,500
Q4:17 Volume Price Fx /Hedge
Q4:18
↑16.0%↓0.5%↑15.2% ↑1.3%
Contribution to Q4:18 Total Net Product Sales Growth
$ M
illio
ns
Total Net Product Sales
Footnote: Growth Rates = Growth vs. Prior Year Period10
$2,977
$3,479
$4,036
FY:18 Total Net Product Sales
2016 2017 2018
$ M
illio
ns
↑22% ↑16% ↑18%
$0
$2,000
$4,000
$6,000
$8,000
$10,000
$12,000
$14,000
$16,000
2017 Volume Price Fx /Hedge
2018
↑17.7%↓0.4%↑15.2% ↑2.9%
Contribution to 2018 Total Net Product Sales Growth
$ M
illio
ns
Total Net Product Sales
11Footnote: Growth Rates = Growth vs. Prior Year Period
$11,185
$12,973
$15,265
Q4:18 Adjusted Diluted Earnings Per Share
Q4:16 Q4:17 Q4:18
↑36% ↑24%
Do
llars
Per
Sh
are
↑20%
Q4:17 Oper. Income
OIE Tax Rate
Share Count
Q4:18
Do
llars
Per
Sh
are
$2.39$0.30$2.00 ($0.04) $0.25($0.12)
12
Contribution to Q4:18 Adjusted Diluted EPSAdjusted Diluted EPS
Footnote: Growth Rates = Growth vs. Prior Year Period
$1.61
$2.00
$2.39
FY:18 Adjusted Diluted Earnings Per Share
2016 2017 2018
↑26% ↑25%
Do
llars
Per
Sh
are
↑19%
Do
llars
Per
Sh
are
$8.87$0.97$7.44 ($0.06) $0.81($0.29)
13
Contribution to 2018 Adjusted Diluted EPSAdjusted Diluted EPS
Footnote: Growth Rates = Growth vs. Prior Year Period
$5.94
$8.87
$7.44
2017 Oper. Income
OIE Tax Rate Share Count
2018
Key P&L Line Items (Adjusted)
Q4:18∆ vs.
Q4:17FY2018
∆ vs.
FY2017
Product Gross Margin 96.0% ↓ ~80 bps 96.4% ↓ ~30 bps
R&D Expenses
% of revenue$919M22.8%
↑ ~80 bps$3,509M
23.0%↑ ~190 bps
SG&A Expenses
% of revenue$762M18.9%
↓ ~ 80 bps$2,747M
18.0%↑ ~50 bps
Operating Margin 54.4% ↓ ~70 bps 55.5% ↓ 250 bps
Effective Tax Rate 15.4% ↑~140 bps 16.5% ↑ 60 bps
14
Capital Allocation
▪ Cash flow from operations was approximately $5.2B during 20181
▪ In 2018, invested ~$9.7B in new acquisitions2
▪ In 2018, purchased ~$6.1B of shares
($ in Billions) 12/31/18 12/31/17
Cash, Cash Equivalents, Marketable
Debt Securities and Publicly-Traded
Equity Securities
$6.04 $12.04
151 Includes $1.1B of cash outflow related to the acquisition of Impact Biomedicines 2 Comprised of $8.6B and $1.1B for Juno Therapeutics and Impact Biomedicines, respectively
2019 Guidance
2019 Guidance∆ vs.
2018
Total Revenue $17.0B-$17.2B ↑ ~12%1
REVLIMID® Net Product Sales ~$10.8B ↑ ~12%
POMALYST®/IMNOVID® Net Product Sales ~$2.4B ↑ ~18%
OTEZLA® Net Product Sales ~$1.9B ↑ ~18%
ABRAXANE® Net Product Sales ~$1.1B ↑ ~4%
Adjusted Operating Margin ~57.5% ↑ ~200 bps
Adjusted Tax Rate ~17.0% ↑~ 50 bps
Adjusted Diluted EPS $10.60-$10.80 ↑ ~21%1
Weighted Average Diluted Shares ~715M ↓~20M
161. Using mid-point of the range
Nadim AhmedPresident, Hematology & Oncology
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q4:18 & FY:18 Hematology & Oncology Franchise Results
Strong Net Product Sales and Operating Momentum– Net product sales: Q4:18 - $3.6B, +15% Y/Y; FY:18 - $13.7B +17% 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
− REVLIMID® + rituximab (R2) dossiers for R/R indolent lymphoma subtypes submitted in the U.S. and EU
− ABRAXANE IO combination U.S. approvals in 1L mTNBC and 1L metastatic non-squamous NSCLC expected in 2019
Advancement and Expansion of Innovative Pipeline− Luspatercept: Ph III MEDALIST™ trial (MDS) and Ph III BELIEVE™ trial (beta-thalassemia) presented at ASH 2018
− Liso-cel (JCAR017): Initial data from the Ph I/II TRANSCEND™ R/R CLL trial presented at ASH 2018
− BCMA campaign:
− bb2121: KarMMa™ pivotal trial in RRMM completed enrollment in Q4:18
− bb21217 and JCARH125 data presented at ASH 2018
− Fedratinib U.S. NDA submitted in myelofibrosis
− Launch planning initiated for anticipated near-term approvals
18
$170$219
$283
$393$124
$159
$159
$174
Q4:15 Q4:16 Q4:17 Q4:18U.S. ROW
$957$1,187
$1,473$1,729
$604
$621
$715
$820
Q4:15 Q4:16 Q4:17 Q4:18U.S. ROW
Current Results & Potential Future Growth Drivers
Q4:18 & FY:18 IMiD® Net Sales Summary
• REVLIMID® - Q4:18 net sales ~$2.5B, +16% Y/Y;
FY:18 net sales ~$9.7B, +18% Y/Y
• POMALYST® - Q4:18 net sales $567M, +28% Y/Y;
FY:18 net sales ~$2.0B, +26% Y/Y
• Strong growth with contribution from increased market
share, duration and triplet combination adoption
• Clinical development and potential future growth drivers:
– REVLIMID®
• REVLIMID®-based triplet regimens in NDMM
(RVd, Rd-daratumumab)
• Anticipate R2 approval in R/R indolent lymphoma subtypes
• Ph III ROBUST® study in 1st line ABC-subtype diffuse large B-cell
lymphoma (event-driven)
– POMALYST®
• Newer triplet regimens expected to increase share and duration
• Anticipate PVd approval in EU and Japan
19
REVLIMID® Net Sales ($M)
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
$1,561
$1,808
$2,188
$2,549
POMALYST® Net Sales ($M)
$294
$378
$442
$567
Current Results & Potential Future Growth Drivers
Q4:18 & FY:18 ABRAXANE® Net Sales Summary
$179 $172 $155
$178
$91 $94 $96
$91
Q4:15 Q4:16 Q4:17 Q4:18
U.S. ROW
$269$270$251
$266
ABRAXANE® Net Sales ($M)
20
• Q4:18 net sales $269M, +7% Y/Y;
• FY:18 net sales ~$1.1B, +7% Y/Y
• Potential future growth drivers:
− Ph III apact® trial of ABRAXANE® in adjuvant pancreatic cancer data (event-driven)
− First approved IO combination regimen for 1L metastatic squamous NSCLC:
− Pembrolizumab + ABRAXANE® approved by FDA on October 30, 20181
− Upcoming PDUFA dates for IO combinations:
− Atezolizumab + ABRAXANE® in 1L metastatic TNBC (March 12, 2019) 2
− Atezolizumab + ABRAXANE® in 1L metastatic non-squamous NSCLC (Sept 2, 2019) 2
1 Pembrolizumab + ABRAXANE® is a Merck & Co. approval2 Atezolizumab + ABRAXANE® are Genentech, a member of the Roche Group, action dates
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
Terrie CurranPresident, Inflammation & Immunology
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q4:18 & FY:18 I&I Franchise Results
22
Continued Positive Momentum for OTEZLA®
– Achieved updated 2018 revenue guidance
– Strong demand driving significant growth across geographies
– Favorable access positions in the U.S., key EU markets, and Japan
Advancing the OTEZLA® Lifecycle Development Plan
– Submitted Behҫet’s disease sNDA in the U.S. and JNDA in Japan
– Positive Ph III STYLETM data in scalp psoriasis
– Initiated additional Ph III studies in new and complementary patient populations
Progressing Future I&I Growth Drivers
– Ozanimod RMS/RRMS regulatory filings in U.S. and EU on track for Q1:19
– Enrolling Ph III trials for ozanimod in ulcerative colitis (UC) and Crohn’s disease
– Positive Ph II 52-week data for RPC4046 in eosinophilic esophagitis (EoE)
– Completion of enrollment in Ph II trial of CC-220 in SLE expected in 2019
0%
10%
20%
30%
40%
ENBREL STELARA HUMIRA COSENTYXOTEZLA TALTZ Acitretin MethotrexateCyclosporine SILIQ TREMFYA
$167
$268 $303 $360 $16
$37
$68
$88
Q4:15 Q4:16 Q4:17 Q4:18U.S. ROW
Q4:18 & FY:18 OTEZLA® Performance and Future Growth Drivers
23
Current Results & Potential Future Growth Drivers
• Q4:18 net sales $448M, +21% Y/Y
• FY:18 net sales ~$1.6B, +26% Y/Y
• Continued robust volume-based growth
– Maintaining U.S. new-to-brand leadership despite increasingly competitive market
– Achieved dynamic market leadership in France; continue to drive fastest post-launch uptake in Japan
• Potential future growth drivers for OTEZLA®
– Behҫet’s disease1: EMA submission H1:19, anticipating H2:19 approvals in US (July 21 PDUFA date) and Japan
– Scalp psoriasis label enhancement: sNDA submission expected in Q2:19
– Ph III/IIIb trials in pediatric, genital, and mild to moderate plaque psoriasis
OTEZLA® Net Sales ($M)
1Proposed indication: treatment of adult patients with oral ulcers associated with Behҫet’s disease2Source: SHS claims data through Nov 2018, last updated Jan 17,2019. includes patients on bridge and PAP for OTEZLA.Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention
OTEZLA
35.3%
OTEZLA® New to Brand Share in Psoriasis2
(Normalized Patient Equivalents)
$183
$305
$371
$448
Jay Backstrom, MDChief Medical Officer
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
0.3500.241
0.1810.000
0.100
0.200
0.300
0.400
0.500
0.600
1
Ad
juste
d A
RR
(±95%
CI)
0.276 0.218 0.1720
0.1
0.2
0.3
0.4
0.5
0.6
1
Ad
juste
d A
RR
(±95%
CI)
Ozanimod Regulatory Filings Supported by Clinical Efficacy & Safety Data from Two Phase III Trials in Patients with RMS
25
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%)0
RADIANCETM PART B – Over 2 years
SUNBEAMTM and RADIANCETM PART B Pooled
Summary of Adverse Events
21% reductionP=0.0167
38% reductionP<0.0001
441 439 433n
**Drowning, unrelated to treatment, on study day 637
• No subjects had a 2nd degree or higher AV block
• Rate of infection 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, #005
Based 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
Fedratinib
Luspatercept
Liso-cel
bb2121
Ozanimod: Advancing a Next-Generation S1P Modulator for RMS and IBD
26
Ozanimod • Potentially differentiated risk-benefit profile in RMS
• U.S. NDA and EU MAA submissions for RMS/RRMS on track for Q1:19
− Non-clinical bridging studies, PK/PD and DDI trials completed
• Ph III TRUE NORTH™ ulcerative colitis trial enrollment targeted for completion in H1:19
• Ph III YELLOWSTONE™ Crohn’s disease program enrolling
Ozanimod
Luspatercept
Liso-cel
bb2121
Fedratinib: Building Leadership in Myelofibrosis
27
Fedratinib
• Highly selective JAK2 inhibitor
• Studied in ruxolitinib-naïve and ruxolitinib-exposed patients with myelofibrosis
• NDA submitted for myelofibrosis; U.S. approval expected by year-end 2019
• EU MAA submission planned in H1:19
• FREEDOM/FREEDOM-2 myelofibrosis trials advancing
• Ph I/II combination trial with luspatercept planned
Liso-cel
bb2121
Ozanimod
Fedratinib
Luspatercept: A Potential Platform Molecule for Chronic Anemias
28
Luspatercept
• First-in-class erythroid maturation agent
• U.S. and EU regulatory submissions for RS+ MDS and transfusion-dependent beta-thalassemia on-track for H1:19
• Broad development strategy
– Ph III COMMANDS trial in ESA-naïve MDS
– Randomized Ph II BEYOND trial in non-transfusion dependent beta-thalassemia
– Ph II myelofibrosis data expected in H2:19
In collaboration with Acceleron Pharma
Luspatercept
bb2121
Ozanimod
Fedratinib
Liso-cel: Harnessing Immunotherapy in NHL and CLL
29
Liso-cel
• Potential best-in-class CD19 CAR T profile
• BLA submission expected in H2:19; U.S. approval expected in mid-2020
• Early Ph I/II data in R/R CLL (BTK failures) compelling; Pivotal Ph II trial initiating
• Clinical trials in earlier lines of DLBCL underway
− Ph III TRANSFORM in 2nd line transplant eligible
− Ph II PILOT in 2nd line non-transplant eligible
Liso-cel
Luspatercept
Ozanimod
Fedratinib
bb2121: Potential to Redefine the Treatment of Multiple Myeloma
30
bb2121
• Potential first-in-class BCMA CAR T for multiple myeloma
• Pivotal KarMMa™ trial enrollment completed
• U.S. approval in highly refractory MM expected in H2:20
• Clinical program in earlier lines of MM advancing
− Ph III KarMMa™ 3 in 3rd line+
− Ph II KarMMa™ 2 in 2nd line
− Ph II NDMM trial planned in H2:19
Program in collaboration with bluebird bio
Q4 and Full Year 2018 Conference Call
January 31, 2019
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Q&A
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Use of Non-GAAP Financial Measures and
Reconciliation Tables
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Use of Non-GAAP Financial Measures
34
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 and success payments. 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.
Use of Non-GAAP Financial Measures
35
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 multiyear
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.
Use of Non-GAAP Financial Measures
36
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.
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: significant litigation-related
loss contingency accruals and expenses to settle other disputed matters and, 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 and Measurement 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 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, the impact of tax reform legislation commonly referred
to as the Tax Cuts and Jobs Act (2017 Tax Act), and other similar items. We also 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 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 adjusted
measures for the three- and twelve-month periods ended December 31, 2018 and 2017, and for the projected amounts for the twelve-month period ending
December 31, 2019.
Reconciliation Tables
37
Reconciliation Tables
38
Reconciliation Tables
39
Appendix
CHANGING THE COURSE OF
HUMAN HEALTH THROUGH BOLD
PURSUITS IN SCIENCE
Advancing a High Quality Pipeline with Significant Potential
OzanimodS1P1/5 agonist
UC
I & I
11
OzanimodS1P1/5 agonist
MS
RPC-4046Anti-IL-13
EoE
CC-220CELMoD
SLE
CC-90001 JNK inhibitor
IPF
CC-90006Anti-PD-1
PSOR
OzanimodS1P1/5 agonist
CD
CC-99677MK2 inhibitor
I&I
CC-486 DNMT inhibitor
AML
LuspaterceptTGFβ inhibitor
MDS, Beta-thalassemia
CC-90009CELMoDR/RAML
FT-1101BET inhibitorMDS, AML
Myeloid
Disease
10
Liso-celCD19 CAR T
R/R NHL
CC-486DNMT inhibitor
NHL
NHL & CLL
10
LuspaterceptTGFβ inhibitor
MF
CC-90002Anti-CD47
NHL
CC-90010BET inhibitor
NHLbb21217
BCMA CAR TRRMMCC-220
CELMoDRRMM
bb2121BCMA CAR T
RRMM
CC-92480CELMoDRRMM
Multiple
Myeloma
9Marizomib
Proteasome inhibitorGBM
CC-90011LSD1 inhibitorSolid Tumors
Solid
Tumors
9
EtigilimabAnti-TIGIT
Solid Tumors
JTX-2011ICOS agonistSolid Tumors
CC-90010BET inhibitorSolid Tumors
TislelizumabAnti-PD-1
Solid Tumors
FedratinibJAK2 kinase inhibitor
MF
CC-93269BCMA TCE
RRMM
JCARH125BCMA CAR T
RRMM
MSC-1Anti-LIF
Solid tumors
Liso-celCD19 CAR T
R/R CLL
GEM333CD3xCD33
AML
AG-270Mat2A inhibitorSolid tumors
TRPH-222CD22 ADC
NHL
CC-92252IL-2 mutein
I&I
AG-270Mat2A inhibitor
NHL
L E G E N D
Celgene has an exclusive option to license and/or option to acquire: TRPH-222,JTX-2011, Etigilimab, AG-270, and MSC-141
REVLIMID®
iMiDNDMM, RRMM
POMALYST®
iMiDRRMM
THALOMID®
iMiDNDMM, RRMM
REVLIMID®
iMiDMCL
ISTODAX®
HDAC inhibitorPTCL, CTCL
REVLIMID®
iMiDR/R NHL
ABRAXANE®
nab-paclitaxelPanC, NSCLC, mBC
REVLIMID®
iMiDDel 5q MDS
VIDAZA®
DNMT inhibitorMDS, AML
IDHIFA®
IDH2 inhibitorIDH2 R/RAML
OTEZLA®
PDE4 inhibitorPSOR, PSA
OTEZLA®
PDE4 inhibitorBehçet’s, Scalp PSOR
MarketPh I
2019 Milestones
Financial Performance❑ Total revenue $17.0B-$17.2B
❑ REVLIMID® net sales ~$10.8B
❑ POMALYST® net sales ~$2.4B
❑ OTEZLA® net sales ~$1.9B
❑ ABRAXANE® net sales ~$1.1B
❑ Adj. operating margin ~57.5%
❑ Adj. diluted EPS $10.60 to $10.80
Maximize Commercial Assets
REVLIMID
❑ FDA decision on sNDA for REVLIMID® - AUGMENTTM in R/R iNHL
❑ EMA CHMP decision on sNDA for RVd in NDMM
❑ Ph III ROBUST ® data – REVLIMID® in 1st line ABC-subtype DLBCL (event-driven)
POMALYST®/ IMNOVID®
❑ EMA CHMP decision on sNDA for PVd in RRMM
❑ Japan PMDA decision on sNDA for PVd in RRMM
OTEZLA®
❑ FDA decision on sNDA for OTEZLA® in Behҫet’s disease
❑ Japan PMDA decision on sNDA for OTEZLA® in Behҫet’s disease
❑ Submit sNDA for OTEZLA® in moderate to severe scalp psoriasis
ABRAXANE®
❑ Ph III apact® data- ABRAXANE® in adjuvant PanC (event-driven) 42
Milestones Expected for Key Pivotal Assets
Ozanimod
❑ Submit NDA to FDA for ozanimod in RMS
❑ Submit MAA to EU for ozanimod in RRMS
❑ Complete enrollment of Ph III TRUE NORTHTM in ulcerative colitis
Fedratinib
❑ FDA decision on NDA for fedratinib in myelofibrosis
❑ Submit EU MAA for Fedratinib in myelofibrosis
❑ Initiate Ph I/II combination trial with luspatercept
Luspatercept
❑ Submit U.S. and EU regulatory applications for MEDALISTTM and BELIEVETM
❑ Ph II myelofibrosis data in H2:2019
Liso-cel
❑ Pivotal TRANSCEND™ data in R/R DLBCL
❑ Submit U.S. BLA in R/R DLBCL
Initiate the pivotal Ph II trial in R/R chronic lymphocytic leukemia (CLL)
bb2121
❑ Pivotal KarMMa™ data in RRMM
❑ Initiate Ph II trial in NDMM
Research & Early Development Pipeline
❑ File at least 5 INDs or CTAs for novel assets
✓
Multiple Myeloma Late-Stage/Pivotal Programs
Patient Population
RRMM RRMM RRMM
Molecule POMALYST®/IMNOVID® bb2121 bb2121
Trial NameMM-007
OPTIMISMM®
BB2121-MM-001
KarMMaTM
BB2121-MM-003
KarMMa-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
progression
Arm 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 met
Data presented at ASCO 2018
Submitted in EU and Japan
Enrollment complete Not yet enrolling
43
MDS/AML/MF Late-Stage/Pivotal Programs
Patient PopulationLow risk/INT-1 transfusion-
dependent MDSPost 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 216 472
Design
Arm A: CC-486 (300mg daily D1-21 of a 28-D cycle) + best supportive care
Arm B: Placebo + best supportive care
Arm A: CC-486 (300mg D1-14 of 28-D cycle)
Arm B: Best supportive care
Primary EndpointRBC-transfusion independence for
more than 8 weeksOverall Survival
StatusEnrollment complete
Data expected in 2Q2019
Enrollment complete
Data expected in 1H2019 (event driven)
44
MDS/AML/MF Late-Stage/Pivotal Programs
Patient PopulationAnemia in to Very Low-, Low-, or
Intermediate-Risk MDSRed Blood Cell Transfusion Dependent
Beta-Thalassemia
Molecule Luspatercept Luspatercept
Trial Name MEDALISTTM BELIEVETM
Phase III III
Target Enrollment 229 335
Design
Arm 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 EndpointRed 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
Status
Primary endpoint met
Data presented at ASH 2018
Regulatory submissions planned in H1:19
Primary endpoint met
Data presented at ASH 2018
Regulatory submissions planned in H1:19
45
MDS/AML/MF Late-Stage/Pivotal Programs
Patient PopulationESA Naïve Very Low, Low or Intermediate
Risk MDSIDH2 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 Trial enrolling Trial enrolling
46
Lymphoma Late-Stage/Pivotal Programs
Patient PopulationRelapsed 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 SurvivalComplete Response Rate and
Progression Free SurvivalProgression Free Survival
Status
Primary endpoint met
Submitted in US & EU
Submission in Japan planned for 1Q2019
Trial did not achieve superiority in co-primary endpoints
Data presented at ASCO 2018
Data expected in 2019 (event driven trial
47
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 schedule
Arm B: JCAR017 2-dose schedule
Primary Endpoint Progression Free Survival Objective Response Rate; Safety
StatusTrial enrolling
Data expected in 2020
Enrollment complete
Submission expected for 2H:2019
48
Lymphoma Late-Stage/Pivotal Programs
Patient PopulationAggressive Relapsed or Refractory B-Cell
LymphomaRelapsed or Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma
MoleculeLiso-cel
(lisocabtagene maraleucel; JCAR017)
Liso-cel
(lisocabtagene maraleucel; JCAR017)
Trial Name TRANSCEND WORLDTRANSCEND-CLL-004
(017004)
Phase II I/II
Target Enrollment 124 400
DesignArm A: JCAR017 (1 x 108 positive transfected viable T cells
on D 1; 2 to 7 days after completion of lymphodepleting chemotherapy).
Phase I
JCAR017 +/- ibrutinib
Phase II
Experimental: JCAR017 monotherapy at recommended dose (RD)
Active comparator: standard of care
Primary Endpoint Overall Response RatePhase I – Safety and RD of JCAR017 +\- ibrutinib
Phase II – PFS with JCAR017 monotherapy
Status Trial enrolling Enrollment initiated in the Pivotal cohort
49
Lymphoma Late-Stage/Pivotal Programs
Patient PopulationAggressive Relapsed or Refractory B-Cell non-
Hodgkin LymphomasRelapsed or Refractory Angioimmunoblastic T
Cell Lymphoma
MoleculeLiso-cel
(lisocabtagene maraleucel; JCAR017)
CC-486
(Oral Azacitidine)
Trial NameTRANSFORM
(JCAR017-BCM-003)OA-CL-LYM-LYSARC-13134
Phase III III
Target Enrollment 182 86
Design
Arm A: standard of care (SOC) per investigator’s decision
Arm B: lymphodepleting chemotherapy followed by JCAR017 infusion
Experimental Arm
Oral azacitidine 200 mg or 300 mg QD x 14 days of 28-day cycle
Control Arm
Investigator’s choice including the following:
Romidepsin: 14 mg/m2 on D 1, 8, and 15 of 28-day cycle
or
Gemcitabine: 1000 mg/m2 on days 1, 8, and 15 of 28-day cycle
Primary Endpoint Event-free Survival (EFS) Progression Free Survival
Status Trial enrolling Trial enrolling50
Solid Tumor Late-Stage/Pivotal Programs
Patient PopulationAdjuvant Therapy in Surgically
Resected Pancreatic CancerNewly Diagnosed Glioblastoma
Newly Diagnosed Stage III non-Small Cell Lung Cancer
Molecule ABRAXANE® Marizomib Tislelizumab (BGB-A317)
Trial NamePANC-003
apact®EORTC-BTG-1709
BGB-A317-NSCL-001
RATIONALE001
Phase III III III
Target Enrollment 866 750 840
Design
Arm A: ABRAXANE® (125 mg/m2); Gemcitabine (1000 mg/m2) D1,8,15 for 6
28-D cycles
Arm 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
Arm 1: tislelizumab + concurrent chemoradiotherapy (cCRT) followed by
tislelizumab monotherapy
Arm 2: placebo + cCRT followed by tislelizumab monotherapy
Arm 3: placebo + cCRT followed by placebo monotherapy
Primary Endpoint Disease Free Survival Overall Survival Progression Free Survival
StatusEnrollment complete
Data expected in 2019 (event driven)Trial enrolling Trial enrolling
51
I&I Late-Stage/Pivotal Programs
Patient Population Active Behçet’s Disease Scalp PsoriasisMild to Moderate Plaque
Psoriasis
Molecule OTEZLA® OTEZLA® OTEZLA®
Trial NameBCT-002
RELIEF®
SPSO-001
STYLETM
PSOR-022
ADVANCETM
Phase III III III
Target Enrollment 208 300 574
Design
Arm 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)
Arm A: OTEZLA® 30 mg BID for 16 weeks
Arm B: Placebo BID for 16 weeks
Primary EndpointArea 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
Proportion of subjects with an sPGAscore of clear (0) or almost clear (1) and
with at least a 2- point reduction from baseline at Week 16.
Status
Met primary endpoint
Data presented at AAD 2018
sNDA submitted; Additional regulatory submissions planned
Met primary endpoint Trial not yet enrolling
52
I&I Late Stage Programs
Patient PopulationModerate to Severe Ulcerative
ColitisModerately to Severely Active
Crohn's DiseaseModerately 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
Design
Arm A: Ozanimod (1mg daily) for induction and maintenance
Arm 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 Endpoint
Clinical 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
StatusEnrollment expected to complete by
mid-2019Trial enrolling Trial enrolling
53
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
Design
Arm 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
54
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