Recent Advances In The Development
Of Innovative Therapies.
The Celgene Pipeline
Jean Caraux, MD PhD
BTG, Hong Kong - February 23th 2013
Targeting Unmet Medical Needs
Hematologic Malignancies
B-Cell
Malignancies
T-Cell
Malignancies
MDS and
Myeloproliferative
disorders
• Myeloma
• CTCL
• MDS
• CLL
• PTCL
• AML
• MF
• NHL
REVLIMID
POMALYST
ISTODAX
REVLIMID
VIDAZA
POMALYST
2
Targeting Unmet Medical Needs
Solid tumors
High Medical Need Malignancies
• Metastatic
• Pancreatic
Breast Cancer Cancer
• NSCLC
• Melanoma
Abraxane
3
Major Recent Accomplishments
Multiple Myeloma
• Lenalidomide (REVLIMID ®)
• Approved in China for RRMM,
• Reimbursed in Taiwan
• Pomalidomide (POMALYST®)
• Achieved an overall survival benefit in a Ph III trial
for RRMM after failure of prior options
• Approved in the US
• Access for Asian patients being set up
4
Pomalidomide-dex vs dex (phase III)
Improved Overall Survival
n= 455 HR=0.53
5
5
Pomalidomide : Myelofibrosis
• Global phase III trial fully accrued; Data
expected H1:13
• Combination of pomalidomide and
prednisone is active in myelofibrosis
• 62% of patients achieved transfusion
independence
6
Major Recent Accomplishments
Lymphomas
• MCL : Lenalidomide filed for approval in US
• FL : R2 highly active in first-line, phase III ongoing
• DLBCL : R2-CHOP21 is active and well tolerated.
MDS and AML
• CC-486 (oral aza) : Initiated Ph III program,
Chronic hypomethylation
7
Major Recent Accomplishments
Nab-paclitaxel (ABRAXANE®)
• NSCLC : approved (US)
• Pancreatic Cancer : Demonstrated an overall
survival benefit in Ph III trial
• Melanoma : Achieved the primary endpoint
of PFS with OS trend in Ph III trial
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Metastatic Melanoma : Abraxane
Improved PFS in phase III vs dacarbazine
• 529 pts
• nab-Paclitaxel superior to standard DTIC
chemotherapy
• PFS (primary endpoint) :
– significantly improved vs dacarbazine
– 4.8 vs 2.5 months (P = 0.044)
• OS :
– Interim OS analysis : trend in favor of the nabpaclitaxel arm
– Observed early and maintained throughout the
study
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Pancreatic : Abraxane + Gemcitabine
Improved Overall Survival in phase III
Variable
ABI007/Gemcitabine
(N=431)
Gemcitabine
(N=430)
Deaths
333 (77%)
359 (83%)
Censored
98 (23%)
71 (17%)
8.5
6.7
0.72
7.89 - 9.53
6.01 - 7.23
0.617 – 0.835
6 month
67%
55%
9 month
48%
36%
12 month
35%
22%
18 month
16%
9%
24 month
9%
4%
Median (months)
95% Confidence
Interval
Hazard Ratio
HR A+G/Ga
P-value
b
0.0000152
Survival Rate (%) at
a95%
CI from stratified Cox model
bStratified log-rank using IVRS strata of region, KPS, and presence of liver
mets
Confidential – for internal use only
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Apremilast
Adressing Frequent Unmet Needs
Apremilast Advantages
Large Underserved
Patient Populations
 Targets high unmet need
 Clinical activity in several
inflammatory diseases
PsA
 Safe and well tolerated
 Convenient – oral
Near-term Milestones
• Ph III ESTEEM data in psoriasis
expected in Q1
• Ph III PALACE-4 data in treatmentnaïve PsA expected in Q1
Psoriatic
Arthritis
~1M
Psoriasis
(moderate
to severe)
~2.5M
Ankylosing
Spondylitis
~2.5M
~ 6M patients in US / EU only
• Submit NDA for PsA in H1
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CC-292 (Avila)
Potential of BTK Inhibition
Monocytes
B Cells
Mast Cells
Osteoclasts
RA, IBD
RA, Multiple
Myeloma,
Osteolytic Bone
Disease
Leukemia,
Lymphoma
Allergy, MS
Asthma, RA
SLE, ITP
Vasculitides
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Focused Research and Early-Stage
Development
Exploit Our Unique Advantages
• IMiD technology platform
• Epigenetics
• Kinase inh. (TORKi, DNAPKi)
• Tumor progenitors
Highlights
• Avilomics – protein silencing
• Advanced 7 programs
into Ph I over last 2 yrs
Leverage Strategic Collaborations
& Disruptive Technologies
ACE-011
ACE-536
ARRY-111
ARRY-382
Genetic Lesion
DOT1L + HMTs
Cancer Stem
Cell Targets
Cancer
Metabolism
Antibody
Conjugates
Novel Antibody
Targets
Ca ImmunoRx
Target
• Expanded biologics
program
• Enhanced platforms to
accelerate discovery
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Thank you
February 3013