Q4 and Full Year 2018 Conference Call - CHANGING THE COURSE OF HUMAN HEALTH THROUGH BOLD PURSUITS IN SCIENCE - Bristol-Myers ...
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C HANGING THE C OURSE OF
H UMAN H EALTH T HROUGH B OLD
P URSUITS IN S CIENCE
Q4 and Full Year 2018
Conference Call
January 31, 2019
Q4:18 Conference Call
Mark Alles, Chairman & Chief Executive Officer
David Elkins, Chief Financial Officer
Nadim Ahmed, President, Hematology & Oncology
Terrie Curran, President, Inflammation & Immunology
Jay Backstrom, MD, Chief Medical Officer
Q&A
2
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.
3
Forward-Looking Statements and Adjusted Financial Information
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.
4
C HANGING THE C OURSE OF
H UMAN H EALTH T HROUGH B OLD
P URSUITS IN S CIENCE
Mark Alles
Chairman & Chief Executive Officer
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
Bristol-Myers Squibb: The Right Transaction for Celgene
▪ 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
7
C HANGING THE C OURSE OF
H UMAN H EALTH T HROUGH B OLD
P URSUITS IN S CIENCE
David Elkins
Chief Financial OfficerQ4:18 & 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
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
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
9Q4:18 Total Net Product Sales
Total Net Product Sales Contribution to Q4:18 Total Net Product Sales Growth
$4,036 $4,500 ↑15.2% ↑1.3% ↓0.5% ↑16.0%
$4,000
$3,479
$3,500
$2,977
$3,000
$2,500
↑16%
$ Millions
$ Millions
↑17% ↑17% $2,000
$1,500
$1,000
$500
$0
Q4:16 Q4:17 Q4:18 Q4:17 Volume Price Fx / Q4:18
Hedge
10
Footnote: Growth Rates = Growth vs. Prior Year PeriodFY:18 Total Net Product Sales
Total Net Product Sales Contribution to 2018 Total Net Product Sales Growth
$15,265 $16,000 ↑15.2% ↑2.9% ↓0.4% ↑17.7%
$12,973 $14,000
$11,185 $12,000
$10,000
$8,000
$ Millions
$ Millions
↑22% ↑16% ↑18%
$6,000
$4,000
$2,000
$0
2016 2017 2018 2017 Volume Price Fx / 2018
Hedge
11
Footnote: Growth Rates = Growth vs. Prior Year PeriodQ4:18 Adjusted Diluted Earnings Per Share
Adjusted Diluted EPS Contribution to Q4:18 Adjusted Diluted EPS
$2.00 $0.30 ($0.12) ($0.04) $0.25 $2.39
$2.39
$2.00
$1.61
Dollars Per Share
Dollars Per Share
↑36% ↑24% ↑20%
Q4:16 Q4:17 Q4:18 Q4:17 Oper. OIE Tax Share Q4:18
Income Rate Count
12
Footnote: Growth Rates = Growth vs. Prior Year PeriodFY:18 Adjusted Diluted Earnings Per Share
Adjusted Diluted EPS Contribution to 2018 Adjusted Diluted EPS
$8.87 $7.44 $0.97 ($0.29) ($0.06) $0.81 $8.87
$7.44
$5.94
Dollars Per Share
Dollars Per Share
↑26% ↑25% ↑19%
2016 2017 2018 2017 Oper. OIE Tax Rate Share 2018
Income Count
13
Footnote: Growth Rates = Growth vs. Prior Year PeriodKey P&L Line Items (Adjusted)
∆ vs. ∆ vs.
Q4:18 FY2018
Q4:17 FY2017
Product Gross Margin 96.0% ↓ ~80 bps 96.4% ↓ ~30 bps
R&D Expenses $919M $3,509M
↑ ~80 bps ↑ ~190 bps
% of revenue 22.8% 23.0%
SG&A Expenses $762M $2,747M
↓ ~ 80 bps ↑ ~50 bps
% of revenue 18.9% 18.0%
Operating Margin 54.4% ↓ ~70 bps 55.5% ↓ 250 bps
Effective Tax Rate 15.4% ↑~140 bps 16.5% ↑ 60 bps
14Capital Allocation
($ in Billions) 12/31/18 12/31/17
Cash, Cash Equivalents, Marketable
Debt Securities and Publicly-Traded $6.04 $12.04
Equity Securities
▪ Cash flow from operations was approximately $5.2B during 2018 1
▪ In 2018, invested ~$9.7B in new acquisitions2
▪ In 2018, purchased ~$6.1B of shares
1 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
152019 Guidance
∆ vs.
2019 Guidance
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
1. Using mid-point of the range
16C HANGING THE C OURSE OF
H UMAN H EALTH T HROUGH B OLD
P URSUITS IN S CIENCE
Nadim Ahmed
President, Hematology & OncologyQ4:18 & 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 - $13.7B +17% Y/Y
– Sales performance driven by strong demand across geographies and brands
Growth Drivers Delivering
− REVLIMID® continues to grow across geographies with NSCT and post-ASCT maintenance adoption
− POMALYST®/IMNOVID® growth continues through gains in market share and duration
− REVLIMID® + rituximab (R2) dossiers for R/R indolent lymphoma subtypes submitted in the U.S. and EU
− ABRAXANE IO combination U.S. approvals in 1L mTNBC and 1L metastatic non-squamous NSCLC expected in 2019
Advancement and Expansion of Innovative Pipeline
− Luspatercept: Ph III MEDALIST™ trial (MDS) and Ph III BELIEVE™ trial (beta-thalassemia) presented at ASH 2018
− Liso-cel (JCAR017): Initial data from the Ph I/II TRANSCEND™ R/R CLL trial presented at ASH 2018
− BCMA campaign:
− bb2121: KarMMa™ pivotal trial in RRMM completed enrollment in Q4:18
− bb21217 and JCARH125 data presented at ASH 2018
− Fedratinib U.S. NDA submitted in myelofibrosis 18
− Launch planning initiated for anticipated near-term approvalsQ4:18 & FY:18 IMiD® Net Sales Summary
REVLIMID® Net Sales ($M)
$2,549
Current Results & Potential Future Growth Drivers $2,188
$820
$1,808
• REVLIMID® - Q4:18 net sales ~$2.5B, +16% Y/Y; $1,561
$715
FY:18 net sales ~$9.7B, +18% Y/Y $621
$604
• POMALYST® - Q4:18 net sales $567M, +28% Y/Y;
FY:18 net sales ~$2.0B, +26% Y/Y $1,729
$1,473
$1,187
• Strong growth with contribution from increased market $957
share, duration and triplet combination adoption
Q4:15 Q4:16 Q4:17 Q4:18
• Clinical development and potential future growth drivers: U.S. ROW
– REVLIMID® POMALYST® Net Sales ($M) $567
• REVLIMID®-based triplet regimens in NDMM
(RVd, Rd-daratumumab) $442 $174
• Anticipate R2 approval in R/R indolent lymphoma subtypes $378
$159
• Ph III ROBUST® study in 1st line ABC-subtype diffuse large B-cell $294
lymphoma (event-driven) $159
$124
– POMALYST® $393
• Newer triplet regimens expected to increase share and duration $283
• Anticipate PVd approval in EU and Japan $170
$219
Q4:15 Q4:16 Q4:17 Q4:18 19
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.
U.S. ROWQ4:18 & FY:18 ABRAXANE® Net Sales Summary
Current Results & Potential Future Growth Drivers ABRAXANE® Net Sales ($M)
$270 $266 $269
$251
• Q4:18 net sales $269M, +7% Y/Y;
• FY:18 net sales ~$1.1B, +7% Y/Y
$91 $94 $91
• Potential future growth drivers: $96
− 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 $179 $172 $178
$155
− 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 Q4:15 Q4:16 Q4:17 Q4:18
U.S. ROW
1 Pembrolizumab + ABRAXANE® is a Merck & Co. approval
2 Atezolizumab + ABRAXANE® are Genentech, a member of the Roche Group, action dates
20
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding convention.C HANGING THE C OURSE OF
H UMAN H EALTH T HROUGH B OLD
P URSUITS IN S CIENCE
Terrie Curran
President, Inflammation & ImmunologyQ4:18 & FY:18 I&I Franchise Results
Continued Positive Momentum for OTEZLA®
– Achieved updated 2018 revenue guidance
– Strong demand driving significant growth across geographies
– Favorable access positions in the U.S., key EU markets, and Japan
Advancing the OTEZLA® Lifecycle Development Plan
– Submitted Behҫet’s disease sNDA in the U.S. and JNDA in Japan
– Positive Ph III STYLETM data in scalp psoriasis
– Initiated additional Ph III studies in new and complementary patient populations
Progressing Future I&I Growth Drivers
– Ozanimod RMS/RRMS regulatory filings in U.S. and EU on track for Q1:19
– Enrolling Ph III trials for ozanimod in ulcerative colitis (UC) and Crohn’s disease
– Positive Ph II 52-week data for RPC4046 in eosinophilic esophagitis (EoE)
22
– Completion of enrollment in Ph II trial of CC-220 in SLE expected in 2019Q4:18 & FY:18 OTEZLA® Performance and Future Growth Drivers
OTEZLA® Net Sales ($M) $448
$371 $88
Current Results & Potential Future Growth Drivers
$305 $68
$37
• Q4:18 net sales $448M, +21% Y/Y $183
$16 $360
• FY:18 net sales ~$1.6B, +26% Y/Y $268 $303
$167
• Continued robust volume-based growth
– Maintaining U.S. new-to-brand leadership despite
increasingly competitive market Q4:15 Q4:16 Q4:17 Q4:18
– Achieved dynamic market leadership in France; continue U.S. ROW
to drive fastest post-launch uptake in Japan
OTEZLA® New to Brand Share in Psoriasis2
• Potential future growth drivers for OTEZLA® (Normalized Patient Equivalents)
OTEZLA
– Behҫet’s disease1:
EMA submission H1:19, anticipating 40% 35.3%
H2:19 approvals in US (July 21 PDUFA date) and Japan
30%
– Scalp psoriasis label enhancement: sNDA submission
expected in Q2:19 20%
– Ph III/IIIb trials in pediatric, genital, and mild to moderate
10%
plaque psoriasis
0%
23
ENBREL STELARA HUMIRA COSENTYX
1Proposed indication: treatment of adult patients with oral ulcers associated with Behҫet’s disease OTEZLA TALTZ Acitretin Methotrexate
2Source: SHS claims data through Nov 2018, last updated Jan 17,2019. includes patients on bridge and PAP for OTEZLA. Cyclosporine SILIQ TREMFYA
Certain prior year amounts have been rounded +/- $1M to conform to the current year rounding conventionC HANGING THE C OURSE OF
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Jay Backstrom, MD
Chief Medical OfficerOzanimod Regulatory Filings Supported by Clinical Efficacy &
Safety Data from Two Phase III Trials in Patients with RMS
SUNBEAMTM – Over 1 year
0.600
SUNBEAMTM and RADIANCETM PART B Pooled
Adjusted ARR (±95% CI)
0.500 Summary of Adverse Events
0.400 31% reduction 48% reduction IFN β-1a Ozanimod Ozanimod
P=0.0013 POzanimod:
Advancing a Next-Generation S1P Modulator for RMS and IBD
Ozanimod • Potentially differentiated risk-benefit profile in RMS
• U.S. NDA and EU MAA submissions for RMS/RRMS on
Fedratinib
track for Q1:19
− Non-clinical bridging studies, PK/PD and DDI trials completed
Luspatercept
• Ph III TRUE NORTH™ ulcerative colitis trial enrollment
Liso-cel targeted for completion in H1:19
• Ph III YELLOWSTONE™ Crohn’s disease program enrolling
bb2121
26Fedratinib:
Building Leadership in Myelofibrosis
Ozanimod • Highly selective JAK2 inhibitor
• Studied in ruxolitinib-naïve and ruxolitinib-exposed patients
Fedratinib with myelofibrosis
• NDA submitted for myelofibrosis; U.S. approval expected
Luspatercept by year-end 2019
• EU MAA submission planned in H1:19
Liso-cel
• FREEDOM/FREEDOM-2 myelofibrosis trials advancing
bb2121 • Ph I/II combination trial with luspatercept planned
27Luspatercept:
A Potential Platform Molecule for Chronic Anemias
Ozanimod • First-in-class erythroid maturation agent
• U.S. and EU regulatory submissions for RS+ MDS and
Fedratinib
transfusion-dependent beta-thalassemia on-track for H1:19
Luspatercept • Broad development strategy
– Ph III COMMANDS trial in ESA-naïve MDS
Liso-cel – Randomized Ph II BEYOND trial in non-transfusion dependent
beta-thalassemia
bb2121 – Ph II myelofibrosis data expected in H2:19
28
In collaboration with Acceleron PharmaLiso-cel:
Harnessing Immunotherapy in NHL and CLL
Ozanimod • Potential best-in-class CD19 CAR T profile
• BLA submission expected in H2:19; U.S. approval expected in
Fedratinib mid-2020
• Early Ph I/II data in R/R CLL (BTK failures) compelling;
Luspatercept Pivotal Ph II trial initiating
• Clinical trials in earlier lines of DLBCL underway
Liso-cel
− Ph III TRANSFORM in 2nd line transplant eligible
− Ph II PILOT in 2nd line non-transplant eligible
bb2121
29bb2121:
Potential to Redefine the Treatment of Multiple Myeloma
Ozanimod • Potential first-in-class BCMA CAR T for multiple myeloma
• Pivotal KarMMa™ trial enrollment completed
Fedratinib
• U.S. approval in highly refractory MM expected in H2:20
Luspatercept • Clinical program in earlier lines of MM advancing
− Ph III KarMMa™ 3 in 3rd line+
Liso-cel − Ph II KarMMa™ 2 in 2nd line
− Ph II NDMM trial planned in H2:19
bb2121
30
Program in collaboration with bluebird bioC HANGING THE C OURSE OF
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Q4 and Full Year 2018
Conference Call
January 31, 2019C HANGING THE C OURSE OF
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P URSUITS IN S CIENCE
Q&AC HANGING THE C OURSE OF
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P URSUITS IN S CIENCE
Use of Non-GAAP Financial
Measures and
Reconciliation TablesUse of Non-GAAP Financial Measures
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
34
severance costs which are not part of a formal restructuring program.Use of Non-GAAP Financial Measures
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.
35Use of Non-GAAP Financial Measures 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. 36
Reconciliation Tables
37Reconciliation Tables
38Reconciliation Tables
39C HANGING THE C OURSE OF
H UMAN H EALTH T HROUGH B OLD
P URSUITS IN S CIENCE
AppendixAdvancing a High Quality Pipeline with Significant Potential
REVLIMID® AG-270 ABRAXANE® AG-270
REVLIMID® JCARH125 Mat2A inhibitor
iMiD iMiD Mat2A inhibitor nab-paclitaxel
BCMA CAR T Solid tumors
NDMM, RRMM MCL NHL PanC, NSCLC, mBC
RRMM
ISTODAX® TRPH-222 MSC-1
CC-93269 HDAC inhibitor CD22 ADC
POMALYST® PTCL, CTCL Tislelizumab Anti-LIF
iMiD BCMA TCE NHL Anti-PD-1 Solid tumors
RRMM RRMM Solid Tumors
Multiple Solid
REVLIMID® NHL & CLL Liso-cel
Myeloma Tumors
THALOMID®
9 CC-92480
iMiD
R/R NHL 10 CD19 CAR T
R/R CLL
Marizomib
Proteasome inhibitor 9
CC-90010
iMiD CELMoD BET inhibitor
NDMM, RRMM RRMM GBM Solid Tumors
Liso-cel CC-90010
CD19 CAR T BET inhibitor
bb2121 bb21217 R/R NHL JTX-2011 Etigilimab
BCMA CAR T NHL ICOS agonist Anti-TIGIT
BCMA CAR T
RRMM CC-486 CC-90002 Solid Tumors Solid Tumors
RRMM CC-220 CC-90011
CELMoD DNMT inhibitor Anti-CD47 LSD1 inhibitor
RRMM NHL NHL Solid Tumors
REVLIMID® GEM333 OTEZLA® CC-99677
iMiD CD3xCD33 PDE4 inhibitor MK2 inhibitor
Del 5q MDS AML PSOR, PSA I&I
FT-1101 OTEZLA®
VIDAZA® PDE4 inhibitor CC-92252
DNMT inhibitor BET inhibitor IL-2 mutein
MDS, AML Behçet’s, Scalp PSOR
MDS, AML I&I
LEGEND Myeloid Ozanimod CC-90006
IDHIFA® S1P1/5 agonist I&I Anti-PD-1
IDH2 inhibitor Disease CC-90009
11
MS PSOR
Ph I Market IDH2 R/RAML
10 CELMoD
R/RAML
Ozanimod CC-90001
S1P1/5 agonist JNK inhibitor
Fedratinib Luspatercept UC
JAK2 kinase inhibitor IPF
TGFβ inhibitor
MF MF Ozanimod CC-220
CC-486 Luspatercept S1P1/5 agonist CELMoD
CD RPC-4046 SLE
DNMT inhibitor TGFβ inhibitor Anti-IL-13
AML MDS, Beta-thalassemia EoE
41
Celgene has an exclusive option to license and/or option to acquire: TRPH-222,JTX-2011, Etigilimab, AG-270, and MSC-12019 Milestones
Financial Performance Milestones Expected for Key Pivotal Assets
❑ Total revenue $17.0B-$17.2B Ozanimod
❑ REVLIMID® net sales ~$10.8B ❑ Submit NDA to FDA for ozanimod in RMS
❑ POMALYST® net sales ~$2.4B ❑ Submit MAA to EU for ozanimod in RRMS
❑ OTEZLA® net sales ~$1.9B ❑ Complete enrollment of Ph III TRUE NORTHTM in ulcerative colitis
❑ ABRAXANE® net sales ~$1.1B
Fedratinib
❑ Adj. operating margin ~57.5%
❑ FDA decision on NDA for fedratinib in myelofibrosis
❑ Adj. diluted EPS $10.60 to $10.80
❑ Submit EU MAA for Fedratinib in myelofibrosis
Maximize Commercial Assets ❑ Initiate Ph I/II combination trial with luspatercept
REVLIMID Luspatercept
❑ FDA decision on sNDA for REVLIMID® - AUGMENTTM in R/R iNHL ❑ Submit U.S. and EU regulatory applications for MEDALISTTM and BELIEVETM
❑ EMA CHMP decision on sNDA for RVd in NDMM ❑ Ph II myelofibrosis data in H2:2019
❑ Ph III ROBUST ® data – REVLIMID® in 1st line ABC-subtype DLBCL (event-driven) Liso-cel
POMALYST®/ IMNOVID® ❑ Pivotal TRANSCEND™ data in R/R DLBCL
❑ EMA CHMP decision on sNDA for PVd in RRMM ❑ Submit U.S. BLA in R/R DLBCL
❑ Japan PMDA decision on sNDA for PVd in RRMM ✓ Initiate the pivotal Ph II trial in R/R chronic lymphocytic leukemia (CLL)
bb2121
OTEZLA® ❑ Pivotal KarMMa™ data in RRMM
❑ FDA decision on sNDA for OTEZLA® in Behҫet’s disease ❑ Initiate Ph II trial in NDMM
❑ Japan PMDA decision on sNDA for OTEZLA® in Behҫet’s disease
❑ Submit sNDA for OTEZLA® in moderate to severe scalp psoriasis
ABRAXANE®
Research & Early Development Pipeline
❑ Ph III apact® data- ABRAXANE® in adjuvant PanC (event-driven) ❑ File at least 5 INDs or CTAs for novel assets 42Multiple Myeloma Late-Stage/Pivotal Programs
Patient
RRMM RRMM RRMM
Population
Molecule POMALYST®/IMNOVID® bb2121 bb2121
MM-007 BB2121-MM-001 BB2121-MM-003
Trial Name
OPTIMISMM® KarMMaTM KarMMa-3TM
Phase III II III
Target Enrollment 559 140 381
Arm A: POMALYST®/IMNOVID®
Arm A: bb2121 autologous CAR T
(4mg) + bortezomib (1.3 mg/m2 IV) +
bb2121 autologous CAR T cells cells (infused at a dose ranging from
low-dose dexamethasone to disease
(infused at a dose ranging from 15 - 15 - 45 x 107 CAR T cells after
Design progression
45 x 107 CAR T cells after receiving receiving lymphodepleting
Arm B: Bortezomib (1.3 mg/m2 IV) + lymphodepleting chemotherapy) chemotherapy)
low-dose dexamethasone to disease
Arm B: Physicians’ choice
progression
Primary Endpoint Progression Free Survival ORR PFS
Primary endpoint met
Status Data presented at ASCO 2018 Enrollment complete Not yet enrolling
Submitted in EU and Japan
43MDS/AML/MF Late-Stage/Pivotal Programs
Low risk/INT-1 transfusion- Post induction AML
Patient Population
dependent MDS Maintenance
CC-486 CC-486
Molecule
(Oral azacitidine) (Oral azacitidine)
Trial Name AZA-MDS-003 CC-486-AML-001
Phase III III
Target Enrollment 216 472
Arm A: CC-486 (300mg daily D1-21 of a Arm A: CC-486 (300mg D1-14 of 28-D
Design 28-D cycle) + best supportive care cycle)
Arm B: Placebo + best supportive care Arm B: Best supportive care
RBC-transfusion independence for
Primary Endpoint more than 8 weeks
Overall Survival
Enrollment complete Enrollment complete
Status
Data expected in 2Q2019 Data expected in 1H2019 (event driven)
44MDS/AML/MF Late-Stage/Pivotal Programs
Anemia in to Very Low-, Low-, or Red Blood Cell Transfusion Dependent
Patient Population
Intermediate-Risk MDS Beta-Thalassemia
Molecule Luspatercept Luspatercept
Trial Name MEDALISTTM BELIEVETM
Phase III III
Target Enrollment 229 335
Arm A: Luspatercept (starting dose of 1.0 mg/kg
Arm A: Luspatercept (1 mg/kg) + best supportive
subcutaneous injection every 3 weeks)
Design care
Arm B: Placebo (subcutaneous injection every 3
Arm B: Placebo + best supportive care
weeks)
Proportion of subjects with hematological
Red Blood Cell Transfusion Independence (RBC-TI)
Primary Endpoint ≥ 8 weeks
improvement from Week 13 to Week 24 compared to
12-week prior to randomization
Primary endpoint met Primary endpoint met
Status Data presented at ASH 2018 Data presented at ASH 2018
Regulatory submissions planned in H1:19 Regulatory submissions planned in H1:19
45MDS/AML/MF Late-Stage/Pivotal Programs
ESA Naïve Very Low, Low or Intermediate
Patient Population IDH2 Mutant RR AML
Risk MDS
Molecule Luspatercept IDHIFA®
Trial Name COMMANDSTM IDHENTIFYTM
Phase III III
Target Enrollment 350 316
Arm A: IDHIFA® (100 mg daily, 28-D cycle) + best
Arm A: Luspatercept (1.0 mg/kg SC every 3 weeks)
Design supportive care
Arm B: Epoetin alfa (450 IU/kg SC weekly)
Arm B: Best supportive care
Primary Endpoint Red blood cell transfusion independence at 24 weeks Overall Survival
Status Trial enrolling Trial enrolling
46Lymphoma Late-Stage/Pivotal Programs
Relapsed or Refractory Newly Diagnosed Follicular Untreated Activated B-Cell
Patient Population
Follicular Lymphoma Lymphoma DLBCL
Molecule REVLIMID® REVLIMID® REVLIMID®
AUGMENTTM ROBUST®
Trial Name RELEVANCE®
NHL-007 DLC-002
Phase III III III
Target Enrollment 358 1,031 570
Arm A: REVLIMID® (starting dose
Arm A: REVLIMID® (10-20mg, D1-21) +
20mg, D2-22 for up to 18 D cycles) +
rituximab (375 mg/m2 weekly for cycle 1 Arm A: REVLIMID® (15mg, D1-14) +
rituximab (starting dose 375 mg/m2
then D1 of cycles 2-5 for 5 28-D cycles) R-CHOP21 (6 21-D cycles)
Design weekly for up to 12 28-D cycles)
Arm B: Placebo (D1-21) + rituximab Arm B: Placebo + R-CHOP21
Arm B: Physician’s choice of
(375 mg/m2 weekly for cycle 1 then D1 (6 21-D cycles)
Rituximab-CHOP, Rituximab-CVP or
of cycles 2-5 for 5 28-D cycles)
Rituximab-bendamustine
Complete Response Rate and
Primary Endpoint Progression Free Survival
Progression Free Survival
Progression Free Survival
Primary endpoint met
Trial did not achieve superiority in co-
Submitted in US & EU primary endpoints Data expected in 2019 (event driven
Status trial
Submission in Japan planned for Data presented at ASCO 2018
1Q2019 47Lymphoma Late-Stage/Pivotal Programs
Patient Population Relapsed or Refractory Indolent Lymphoma Relapsed or Refractory B-cell NHL
Liso-cel
Molecule REVLIMID®
(lisocabtagene maraleucel; JCAR017)
MAGNIFYTM
Trial Name TRANSCEND-NHL-001
NHL-008
Phase III I
Target Enrollment 500 274
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
Arm A: JCAR017 single-dose schedule
Design progression, 28 D cycle)
Arm B: JCAR017 2-dose schedule
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)
Primary Endpoint Progression Free Survival Objective Response Rate; Safety
Trial enrolling Enrollment complete
Status
Data expected in 2020 Submission expected for 2H:2019
48Lymphoma Late-Stage/Pivotal Programs
Aggressive Relapsed or Refractory B-Cell Relapsed or Refractory Chronic Lymphocytic
Patient Population
Lymphoma Leukemia or Small Lymphocytic Lymphoma
Liso-cel Liso-cel
Molecule
(lisocabtagene maraleucel; JCAR017) (lisocabtagene maraleucel; JCAR017)
TRANSCEND-CLL-004
Trial Name TRANSCEND WORLD
(017004)
Phase II I/II
Target Enrollment 124 400
Phase I
JCAR017 +/- ibrutinib
Arm A: JCAR017 (1 x 108 positive transfected viable T cells
Design on D 1; 2 to 7 days after completion of lymphodepleting Phase II
chemotherapy).
Experimental: JCAR017 monotherapy at recommended dose
(RD)
Active comparator: standard of care
Phase I – Safety and RD of JCAR017 +\- ibrutinib
Primary Endpoint Overall Response Rate
Phase II – PFS with JCAR017 monotherapy
Status Trial enrolling Enrollment initiated in the Pivotal cohort
49Lymphoma Late-Stage/Pivotal Programs
Aggressive Relapsed or Refractory B-Cell non- Relapsed or Refractory Angioimmunoblastic T
Patient Population
Hodgkin Lymphomas Cell Lymphoma
Liso-cel CC-486
Molecule
(lisocabtagene maraleucel; JCAR017) (Oral Azacitidine)
TRANSFORM
Trial Name OA-CL-LYM-LYSARC-13134
(JCAR017-BCM-003)
Phase III III
Target Enrollment 182 86
Experimental Arm
Oral azacitidine 200 mg or 300 mg QD x 14 days of 28-day
cycle
Arm A: standard of care (SOC) per investigator’s decision
Control Arm
Design
Arm B: lymphodepleting chemotherapy followed by JCAR017 Investigator’s choice including the following:
infusion 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 enrolling
50Solid Tumor Late-Stage/Pivotal Programs
Adjuvant Therapy in Surgically Newly Diagnosed Stage III non-
Patient Population Newly Diagnosed Glioblastoma
Resected Pancreatic Cancer Small Cell Lung Cancer
Molecule ABRAXANE® Marizomib Tislelizumab (BGB-A317)
PANC-003 BGB-A317-NSCL-001
Trial Name EORTC-BTG-1709
apact® RATIONALE001
Phase III III III
Target Enrollment 866 750 840
Arm 1: tislelizumab + concurrent
chemoradiotherapy (cCRT) followed by
Arm A: Radiotherapy + temozolomide + tislelizumab monotherapy
Arm A: ABRAXANE® (125 mg/m2); marizomib followed by adjuvant
Gemcitabine (1000 mg/m2) D1,8,15 for 6 temozolomide + marizomib
Design 28-D cycles Arm 2: placebo + cCRT followed by
Arm B: Gemcitabine (1000 mg/m2) tislelizumab monotherapy
D1,8,15 for 6 28-D cycles Arm B: Radiotherapy + temozolomide
followed by adjuvant temozolomide
Arm 3: placebo + cCRT followed by
placebo monotherapy
Primary Endpoint Disease Free Survival Overall Survival Progression Free Survival
Enrollment complete
Status Trial enrolling Trial enrolling
Data expected in 2019 (event driven)
51I&I Late-Stage/Pivotal Programs
Mild to Moderate Plaque
Patient Population Active Behçet’s Disease Scalp Psoriasis
Psoriasis
Molecule OTEZLA® OTEZLA® OTEZLA®
BCT-002 SPSO-001 PSOR-022
Trial Name
RELIEF® STYLETM ADVANCETM
Phase III III III
Target Enrollment 208 300 574
Arm A: Placebo (for 12 weeks) followed
Arm A: Placebo (for 16 weeks) followed
by OTEZLA® (30mg twice daily for 52 Arm A: OTEZLA® 30 mg BID for 16
by OTEZLA® (30mg twice daily for 16
Design weeks) weeks
weeks)
Arm B: OTEZLA® (30mg twice daily for Arm B: Placebo BID for 16 weeks
Arm B: Placebo (for 32 weeks)
64 weeks)
Proportion of subjects with ScPGA Proportion of subjects with an sPGA
Area under the curve (AUC) for the
score of clear (0) or almost clear (1) score of clear (0) or almost clear (1) and
Primary Endpoint number of oral ulcers from baseline
with at least a 2-point reduction from with at least a 2- point reduction from
through week 12
baseline at Week 16 baseline at Week 16.
Met primary endpoint
Data presented at AAD 2018
Status Met primary endpoint Trial not yet enrolling
sNDA submitted; Additional regulatory
submissions planned
52I&I Late Stage Programs
Moderate to Severe Ulcerative Moderately to Severely Active Moderately to Severely Active
Patient Population
Colitis Crohn's Disease Crohn's Disease
Molecule Ozanimod Ozanimod Ozanimod
Trial Name TRUE NORTHTM RPC01-3201 RPC01-3202
Phase III III III
Target Enrollment 900 600 600
Arm A: Ozanimod (1mg daily) for
Arm A: Ozanimod (0.92 mg daily) with Arm A: Ozanimod (0.92 mg daily) with
induction and maintenance
Design a 7-D dose escalation a 7-D dose escalation
Arm B: Placebo induction and
Arm B: Placebo Arm B: Placebo
maintenance
Clinical remission assessed by Mayo
component sub-scores at week 10 Proportion of subjects with a CDAI Proportion of subjects with a CDAI
Primary Endpoint score < 150 at Week 12 score < 150 at Week 12
Clinical remission assessed by Mayo
component sub-scores at week 52
Enrollment expected to complete by
Status mid-2019
Trial enrolling Trial enrolling
53I&I Late Stage Programs
Maintenance for
Patient
Moderately to Severely Relapsing Multiple Sclerosis Relapsing Multiple Sclerosis
Population
Active Crohn's Disease
Molecule Ozanimod Ozanimod Ozanimod
Trial Name RPC01-3203 SUNBEAMTM RADIANCETM
Phase III III III
Target Enrollment 485 ~1,300 ~1,300
Arm A: Ozanimod (0.5mg daily) + Arm A: Ozanimod (0.5mg daily) + placebo IM
Arm A: Ozanimod (0.92 mg daily placebo IM weekly weekly
for 40 weeks) Arm B: Ozanimod (1mg daily) + placebo Arm B: Ozanimod (1mg daily) + placebo IM
Design IM weekly weekly
Arm B: Placebo (daily for 40
weeks) Arm C: Placebo (daily) + beta-interferon Arm C: Placebo (daily) + beta-interferon IM
IM weekly weekly
Proportion of subjects with a
CDAI score of < 150 at week 40
Primary Endpoint Proportion of subjects with a Annualized relapse rate at month 12 Annualized relapse rate at month 24
(SES-CD) score decrease from
baseline of ≥ 50% at week 40
Data presented at ECTRIMS 2017 and
Data presented at ECTRIMS 2017 and AAN
AAN 2018
2018
Status Trial not yet enrolling NDA expected to be resubmitted in
NDA expected to be resubmitted in Q1:19;
Q1:19; MAA expected to be submitted in
MAA expected to be submitted in Q1:19
Q1:19
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