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Srdan (Serge) Verstovsek M.D., Ph.D. Professor of Medicine Department of Leukemia University of Texas MD Anderson Cancer Center Houston, Texas, USA JAK inhibitors and low blood cell count

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JAK inhibitorDiseases and studies CEP701 MF: phase II finished and I/II (new formulation) ongoing ET/PV: phase II completed AZD1480MF: phase I finished, development stopped XL019MF: phase I finished, development stopped NS-018MF: phase I ongoing BMS-911543MF: phase I/II ongoing LY2784544ET/PV/MF: phase I finished; MF: phase II ongoing CYT387 MF: phase I/II QD completed; phase I/II BID completed; phase III planned SB1518MF: phase I/IIx2 completed, phase III ongoing SAR302503/TG101348 MF: phase I/II completed; phase II completed, phase III completed; phase II second line ongoing ET/PV: phase II ongoing; PV: phase III planned INCB018424/Ruxolitinib MF: approved; phase II (low platelets) ongoing ET/PV: phase II completed; PV: phase III completed

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JAK2 Inhibitor Side Effects from Phase II Studies GI Anemia Platelets X X X X X X X Neuropathy X

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Ruxolitinib vs. Placebo: COMFORT-I Background Placebo-controlled, randomized, double-blind, phase III study Ruxolitinib starting doses: Baseline platelet count 100-20010 9 /L: 15 mg BID Baseline platelet count >20010 9 /L: 20 mg BID Doses individually titrated based on safety and efficacy Ruxolitinib treatment significantly reduced spleen size and improved myelofibrosis (MF_-related symptoms and QoL and was also associated with a survival advantage relative to placebo 1 Objective To describe long-term efficacy and safety of ruxolitinib with 1 year of additional follow-up beyond previously published data Data cutoff for current analysis: March 1, 2012. 1. Verstovsek S, et al. N Engl J Med. 2012;366(9):799-807. Verstovsek S, et al. Blood. 2012;120: Abstract 800.

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Patient Disposition at Current Analysis Patients, n (%) Ruxolitinib (n = 155) Placebo (n = 151) Placebo Ruxolitinib (n=111) Still on treatment100 (64.5)073 (65.8) Discontinued55 (35.5)40 (26.5)38 (34.2) Crossed over111 (73.5) Primary reasons for discontinuation Death13 (8.4)10 (6.6)11 (9.9) Adverse event11 (7.1)9 (6.0)7 (6.3) Consent withdrawn9 (5.8)6 (4.0)9 (8.1) Disease progression12 (7.7)12 (7.9)5 (4.5) Other10 (6.5)3 (2.0)5 (4.5) Noncompliance with study medication 1 (0.9) All patients receiving placebo at the primary analysis crossed over or discontinued within 3 months of the primary analysis Median time to crossover: 41.1 weeks Verstovsek S, et al. Blood. 2012;120: Abstract 800.

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Incidence of New Onset Grade 3 or 4 Anemia and Thrombocytopenia Over Time AnemiaThrombocytopenia All patients receiving placebo at the primary analysis crossed over or discontinued within 3 months of the primary analysis; therefore, data for patients receiving placebo is shown for 0