POM is a distinct oral immunomodulatory agent whose MOA consists of 3 primary effects 1 :

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1 MM-005: A Phase 1, Multicenter, Open-Label, Dose- Escalation Study to Determine the Maximum Tolerated Dose for the Combination of Pomalidomide, Bortezomib, and Low-Dose Dexamethasone in Patients With Relapsed or Relapsed/Refractory Multiple Myeloma Paul G. Richardson, 1 Craig C. Hofmeister, 2 David Siegel, 3 Sagar Lonial, 4 Mohamad Zaki, 5 Ye Hua, 5 Sheetal Shah, 5 Jianming Wang, 5 and Kenneth C. Anderson 1 1 Jerome Lipper Multiple Myeloma Center, Department of Medical Oncology, Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA; 2 Department of Internal Medicine, Division of Hematology, The Ohio State University, Columbus, OH; 3 Hackensack University Medical Center, Hackensack, NJ; 4 Division of BMT, Emory University, Winship Cancer Institute-Hematology and Medical Oncology, Atlanta, GA; 5 Celgene Corporation, Summit, NJ

2 Background POM is a distinct oral immunomodulatory agent whose MOA consists of 3 primary effects 1 : Potent direct antimyeloma activity Inhibition of stromal cell support Immune modulation When combined with LoDEX, POM has demonstrated encouraging clinical efficacy in relapsed and refractory patients (pts) treated with prior LEN and/or BORT 2 Combining an immune modulator (LEN), a proteasome inhibitor (BORT), and DEX has shown preclinical synergy as well as promising efficacy both as frontline and salvage treatment in myeloma pts 3-5 BORT, bortezomib; DEX, dexamethasone; LEN, lenalidomide; LoDEX, low-dose dexamethasone; MOA, mechanism of action; Pts, patients; POM, pomalidomide. 1. Quach H, et al. Leukemia. 2010;24: Vij R. J Clin Oncol. 2012;30(suppl):513s [oral presentation, abstract 8016]. 3. Mitsiades N, et al. Blood. 2002;99: Richardson PG, et al. J Clin Oncol. 2009;27: Richardson PG, et al. Blood. 2010;116:

3 MM-005 Study Design: POM + BORT + LoDEX Design (21-day cycles) Cohort POM (D1-14) BORT (D1, 4, 8, 11*) LoDEX (D1-2, 4-5, 8-9, ) 1 1 mg/day 1 mg/m 2 20 mg 2 2 mg/day 1 mg/m 2 20 mg 3 3 mg/day 1 mg/m 2 20 mg 4 4 mg/day 1 mg/m 2 20 mg 5 4 mg/day 1.3 mg/m 2 20 mg Expansion cohort (n = 6) at MTD/MPD *For cycles 1-8, then D1, 8 for cycles 9+; For cycles 1-8, then D1-2, 8-9 for cycles 9+; 10 mg for pts aged > 75 yrs Evaluation Every 21 Days (± 3 Days) Follow-Up for OS and SPM Until 5 Years Postenrollment Concomitant medications Required: thromboprophylaxis (aspirin or low-molecular-weight heparin) and antiviral prophylaxis (eg, acyclovir) Supportive care: RBC and platelet transfusions as needed, hematopoietic growth factors (after cycle 1), IV bisphosphonates BORT, bortezomib; IV, intravenous; LoDEX, low-dose dexamethasone; MPD, maximum planned dose; MTD, maximum tolerated dose; OS, overall survival; POM, pomalidomide; pts, patients; RBC, red blood cell; SPM, second primary malignancy.

4 MM-005: Key Eligibility Criteria Age 18 years Measureable disease by M-protein SPEP ( 0.5 g/dl) or UPEP ( 200 mg/dl) Relapsed or relapsed/refractory (RR) MM with 1-4 prior lines of therapy Prior therapy must have included 2 consecutive cycles of LEN Pts must be LEN-refractory Prior therapy must have included 2 consecutive cycles of a proteasome inhibitor Pts cannot be refractory to BORT (1.3 mg/m 2 twice weekly) No peripheral neuropathy grade 2 No significant hematologic or other abnormalities BORT, bortezomib; LEN, lenalidomide; pts, patients; RR MM, relapsed/refractory multiple myeloma; SPEP, serum protein electrophoresis; UPEP, urine protein electrophoresis.

5 MM-005: Endpoints Primary MTD Secondary Safety Response assessment per IMWG criteria 1 Overall survival Time to response Duration of response Exploratory Progression-free survival Time to progression Response assessment per modified EBMT criteria 2-4 POM plasma concentrations EBMT, European Group for Blood and Marrow Transplantation; IMWG, International Myeloma Working Group; MTD, maximum tolerated dose; POM, pomalidomide. 1. Durie BGM, et al. Leukemia. 2006;20: Bladé J, et al. Br J Haematol. 1998;102: Richardson PG, et al. N Engl J Med. 2003;348: Anderson K, et al. Leukemia. 2008;22:

6 MM-005: Patient Characteristics Characteristic Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Total (N = 15) Median age, yrs (range) 58 (57-61) 65 (36-75) 61 (47-73) 48 (41-57) 65 (60-66) 60 (36-75) Male, % ECOG status (0/1), % 67/33 33/67 33/67 33/67 67/33 47/53 ISS Stage (I/II/III/NA) 1/0/1/1 0/0/0/3 0/1/1/1 0/1/1/1 2/0/1/0 3/2/4/5 Median number of prior lines of therapy (range) 2 (2-4) 3 (1-4) 2 (1-3) 3 (2-3) 2 (2-4) 2 (1-4) Prior LEN and BORT, % Prior DEX, % Prior THAL, % Prior SCT, % Progression on or within 60 days of last LEN dose, % % of pts had progressed on LEN as their last prior regimen 53% of pts had relapsed and refractory disease 1,2 at study entry BORT, bortezomib; DEX, dexamethasone; ECOG, Eastern Cooperative Oncology Group; ISS, International Staging System; LEN, lenalidomide; NA, not available; SCT, stem cell transplant; TAL, thalidomide; pts, patients. 1. Richardson PG, et al. N Engl J Med. 2003;348: Anderson KC, et al. Leukemia. 2008;22:

7 MM-005: Patient Disposition Disposition, n Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Total (N = 15) On treatment Discontinued Disease progression Treatment completed Adverse event Death of 15 dose-escalation pts remain on study 5 pts have been added to the MTD/MPD expansion cohort, with 1 patient pending enrollment Total planned enrollment N = 21; 15 pts currently evaluable MPD, maximum planned dose; MTD, maximum tolerated dose; pts, patients.

8 MM-005: Dose-Limiting Toxicities (Within Cycle 1) Cohort Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Dose-Limiting Toxicity None None None None None No DLTs were observed at any dose level Confirmation of the MTD is ongoing; 5 of 5 pts enrolled in the expansion cohort without DLT at cycle 1: POM: 4 mg (D1-14) BORT: 1.3 mg/m 2 (D1, 4, 8, 11)* DEX: 20 mg (D1-2, 4-5, 8-9, 11-12) * For cycles 1-8, then D1, 8 for cycles 9+; 10 mg for pts aged > 75 yrs; For cycles 1-8, then D1-2, 8-9 for cycles 9+. BORT, bortezomib; DEX, dexamethasone; DLT, dose-limiting toxicity; MTD, maximum tolerated dose; POM, pomalidomide; pts, patients.

9 Summary of Adverse Events ( ( 20% of pts) No grade (G) 3/4 peripheral neuropathy (PN) observed G1 and 2 PN reported for 4 and 2 pts, respectively; none painful; 4 had prior PN /THAL No DVT was observed (any grade) No pts have discontinued treatment due to adverse events THAL, thalidomide. Patients (%)

10 MM-005: Dose Modifications Due to Adverse Events Patients, n Dose Interruption POM/BORT/DEX Dose Reduction POM/BORT/DEX Discontinuation POM/BORT/DEX Cohort 1 2 / 2 / 2 0 / 2 / 2 0 / 1 / 0 Cohort 2 1 / 1 / 1 0 / 0 / 2 0 / 0 / 0 Cohort 3 1 / 2 / 1 1 / 0 / 2 0 / 0 / 0 Cohort 4 2 / 1 / 1 0 / 1 / 2 0 / 0 / 0 Cohort 5 1 / 1 / 0 2 / 1 / 1 0 / 0 / 0 With appropriate dose adjustments, no patient discontinued treatment overall One patient discontinued BORT due to persistent grade 2 PN without discontinuing POM or DEX, as per protocol BORT, bortezomib; DEX, dexamethasone; POM, pomalidomide.

11 MM-005: Summary of Best Response IMWG Criteria Cohort Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Best Response 1 VGPR, 1 PR, 1 SD 1 PR, 2 SD 2 VGPR, 1 PR 1 VGPR, 2 PR 2 PR, 1 SD ORR ( PR): 73% VGPR: 27% SD: 27% Median time to response: 1 cycle (range 1-2) Most responses are ongoing IMWG, International Myeloma Working Group; N/A, not available; ORR, overall response rate; PR, partial response; SD, stable disease; VGPR, very good partial response.

12 MM-005: Duration of Response Cohort 1 Cohort 2 Discontinued PD Completed Treatment Discontinued PD Cohort 3 Cohort 4 Cohort 5 * * VGPR PR SD PD Ongoing Day 1 of Cycle Number Total number of completed cycles: 59 * Unconfirmed PR as of data cut. N/A, not available; PD, progressive disease; PR, partial response; SD, stable disease; VGPR, very good partial response. 11

13 MM-005: Cytogenetic Profiles Cohort Patient Cytogenetic Profile Best Response (IMWG) Cohort 1 Patient 1 Patient 2 Patient 3 1q21 +1, +11, +19 del(13q), del(17p) SD PR VGPR Cohort 2 Patient 1 Patient 2 Patient 3 +11q, +19p del(13q), t(4, 14) Pending SD SD PR Cohort 3 Patient 1 Patient 2 Patient 3 IgH-BA+, p53 deletion, RB1 deletion, D13S319+ Abnormal (details pending) del(17p) PR VGPR VGPR Cohort 4 Patient 1 Patient 2 Patient 3 del(13q), +1q, +19p, +5, ++11q Normal Not available PR PR VGPR Cohort 5 Patient 1 Patient 2 Patient 3 del(13q), del(17p), +3, +7, +15 del(13), del(14) Not available SD PR PR del, deletion; IMWG, International Myeloma Working Group; PR, partial response; SD, stable disease; VGPR, very good partial response.

14 Conclusions POM + BORT + LoDEX (PVD) well tolerated in pts with RR MM No DLTs observed at any of 5 dose levels tested No discontinuation of study treatment due to toxicity to date Confirmation of cohort 5 as the MTD/MPD is ongoing POM 4 mg; BORT 1.3 mg/m 2 ; DEX 10/20 mg PVD is active, with responses in RR MM across all cohorts ORR: 73% VGPR: 27% SD: 27% Responses were rapid; majority are ongoing Efficacy is encouraging with favorable tolerability observed in this population including those with adverse cytogenetics and relapsed/refractory disease BORT, bortezomib; DEX, dexamethasone; DLT, dose-limiting toxicity; LoDEX, low-dose dexamethasone; MPD, maximum planned dose; MTD, maximum tolerated dose; ORR, overall response rate; POM, pomalidomide; PR, partial response; RR MM, relapsed/refractory multiple myeloma; SD, stable disease; VGPR, very good partial response.

15 Future Directions MPD identified with this trial will serve as the recommended dose for the recently activated phase 3 trial comparing PVD vs BORT + LoDEX (VD) [MM-007] Estimated sample size: 782 RR MM pts Enrolment is anticipated to begin shortly Observed activity of PVD in RR MM provides a strong rationale for the use of POM in different combinations Phase 1/2 trials are ongoing in RR MM evaluating POM + steroids in combination with CFZ, CYC, clarithromycin, PLD BORT, bortezomib; CFZ, carfilzomib; CYC, cyclophosphamide; DEX, dexamethasone; LoDEX, low-dose dexamethasone; MTD, maximum tolerated dose; POM, pomalidomide; PLD, pegylated liposomal doxorubicin; PVD, pomalidomide-bortezomib-dexamethasone; RR MM, relapsed/refractory multiple myeloma.

16 Acknowledgments Our Patients and Families Investigators, including: David Vesole, Joshua Richter, Elizabeth Biolotti, Ann McNeill, Don Benson, Yvonne Efebera, Nancy Giallombardo, Katarina Luptakova, Dimitrios Tzachanis, Jacalyn Rosenblatt, Robert Schlossman, Patricia Renaudie, Jamie Mortellite, Mary McKenney, Kathleen Colson, Kimberly Noonan, Robin Joyce, James Levine, Nikhil Munshi, Tina Flaherty, Andrew Yee, Donna Fitzgerald, Jon Arnason, Noopor Raje, David Avigan, Deborah Doss, Jeffrey Zwicker, Katherine Conway, Irene Ghobrial, Anuj Mahindra, Virginia Dalton, Kate O'Brien, Jill Nelson Burke, Jacob Laubach, Leonard T. Heffner, Jonathan Kaufman, Christopher Flowers, Amelia Langston, Ajay Nooka Institutions/study sites Dana-Farber Cancer Institute, Harvard Medical School The Ohio State University Hackensack University Medical Center Emory University, Winship Cancer Institute The Multiple Myeloma Research Consortium Medical writing assistance: Richard Balzer, PhD (MediTech Media) Supported by Celgene Corporation