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Recent Advances In The Development Of Innovative Therapies. The Celgene Pipeline

Recent Advances In The Development Of Innovative Therapies. The Celgene Pipeline. Jean Caraux, MD PhD BTG, Hong Kong - February 23 th 2013. Myeloma CLL NHL. Targeting Unmet Medical Needs Hematologic Malignancies. B-Cell Malignancies. T-Cell Malignancies.

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Recent Advances In The Development Of Innovative Therapies. The Celgene Pipeline

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  1. Recent Advances In The Development Of Innovative Therapies. The Celgene Pipeline Jean Caraux, MD PhD BTG, Hong Kong - February 23th 2013

  2. Myeloma CLL NHL Targeting Unmet Medical NeedsHematologic Malignancies B-Cell Malignancies T-Cell Malignancies MDS andMyeloproliferativedisorders • CTCL • PTCL • MDS • AML • MF REVLIMID POMALYST ISTODAX REVLIMID VIDAZA POMALYST

  3. MetastaticBreast Cancer NSCLC Targeting Unmet Medical NeedsSolid tumors High Medical Need Malignancies • PancreaticCancer • Melanoma Abraxane

  4. 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

  5. Pomalidomide-dexvsdex (phase III) Improved Overall Survival n= 455 HR=0.53

  6. 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

  7. 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

  8. 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

  9. Metastatic Melanoma : AbraxaneImproved PFS in phase III vsdacarbazine • 529 pts • nab-Paclitaxel superior to standard DTIC chemotherapy • PFS (primary endpoint) : • significantly improved vsdacarbazine • 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

  10. Pancreatic : Abraxane + GemcitabineImproved Overall Survival in phase III 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 10

  11. ApremilastAdressing Frequent Unmet Needs • Apremilast Advantages Large Underserved Patient Populations • Targets high unmet need • Clinical activity in several inflammatory diseases • Safe and well tolerated • Convenient – oral PsA PsoriaticArthritis Psoriasis (moderate to severe) AnkylosingSpondylitis • Near-term Milestones • 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 ~ 6M patients in US / EU only

  12. CC-292 (Avila) Potential of BTK Inhibition Monocytes B Cells Mast Cells Osteoclasts Allergy, MS Asthma, RA Leukemia, Lymphoma RA, IBD RA, Multiple Myeloma, Osteolytic Bone Disease SLE, ITP Vasculitides

  13. Focused Research and Early-Stage Development Exploit Our Unique Advantages • IMiD technology platform • Epigenetics • Kinaseinh. (TORKi, DNAPKi) • Tumor progenitors • Avilomics – protein silencing Highlights • Advanced 7 programs into Ph I over last 2 yrs • Expanded biologics program • Enhanced platforms to accelerate discovery Leverage Strategic Collaborations & Disruptive Technologies ACE-011 ACE-536 ARRY-111 ARRY-382 Genetic Lesion DOT1L + HMTs Cancer StemCell Targets CancerMetabolism AntibodyConjugates Novel AntibodyTargets Ca ImmunoRxTarget

  14. Thank you February 3013

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