Recent Advances In The Development Of Innovative Therapies The Celgene Pipeline

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Recent Advances In The Development Of Innovative Therapies.

The Celgene Pipeline

Jean Caraux, MD PhD

BTG, Hong Kong - February 23th 2013

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Targeting Unmet Medical NeedsHematologic Malignancies

• Myeloma

• CLL

• NHL

• MDS

• AML

• MF

B-CellMalignancies

T-CellMalignancies

MDS and Myeloproliferative

disorders

REVLIMID

POMALYSTISTODAX

REVLIMIDVIDAZA

POMALYST

• CTCL

• PTCL

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Targeting Unmet Medical NeedsSolid tumors

• Metastatic Breast Cancer

• NSCLC

High Medical Need Malignancies

• Pancreatic Cancer

• Melanoma

Abraxane

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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

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Pomalidomide-dex vs dex (phase III) Improved Overall Survival

n= 455 HR=0.53

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• 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

Pomalidomide : Myelofibrosis

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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

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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 nab-

paclitaxel arm– Observed early and maintained throughout the

study

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Pancreatic : Abraxane + GemcitabineImproved Overall Survival in phase III

Variable ABI-007/

Gemcitabine(N=431)

Gemcitabine(N=430)

Hazard RatioHR A+G/Ga P-value b

Deaths 333 (77%) 359 (83%)

Censored 98 (23%) 71 (17%)

Median (months) 8.5 6.7 0.72 0.0000152

95% Confidence Interval 7.89 - 9.53 6.01 - 7.23 0.617 – 0.835

Survival Rate (%) at

6 month 67% 55%

9 month 48% 36%

12 month 35% 22%

18 month 16% 9%

24 month 9% 4%

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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Near-term Milestones

Apremilast Advantages

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Targets high unmet need

Clinical activity in several inflammatory diseases

Safe and well tolerated

Convenient – oralPsoriaticArthritis

Large Underserved Patient Populations

~ 6M patients in US / EU only

• Ph III ESTEEM data in psoriasis expected in Q1

• Ph III PALACE-4 data in treatment-naïve PsA expected in Q1

• Submit NDA for PsA in H1

~1M ~2.5M~2.5M

AnkylosingSpondylitis

Psoriasis (moderate to severe)

PsA

Apremilast Adressing Frequent Unmet Needs

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CC-292 (Avila)Potential of BTK Inhibition

Osteoclasts

RA, IBD Leukemia,Lymphoma

SLE, ITPVasculitides

Allergy, MSAsthma, RARA, Multiple

Myeloma,Osteolytic Bone

Disease

Monocytes B Cells Mast Cells

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Focused Research and Early-Stage Development

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Highlights

Leverage Strategic Collaborations & Disruptive Technologies

• Advanced 7 programs into Ph I over last 2 yrs

• Expanded biologics program

• Enhanced platforms to accelerate discovery

ARRY-111ARRY-382

ACE-011ACE-536

CancerMetabolism

Cancer StemCell Targets

Genetic LesionDOT1L + HMTs

Novel AntibodyTargets

Ca ImmunoRxTarget

Exploit Our Unique Advantages

• IMiD technology platform

• Epigenetics

• Kinase inh. (TORKi, DNAPKi)

• Tumor progenitors

• Avilomics – protein silencing

AntibodyConjugates

Thank you

February 3013