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1 Albumin-Bound Paclitaxel for the Treatment of Non-small Cell Lung Cancer Clinical data 2 Albumin-Bound Paclitaxel Trials in NSCLC: Monotherapy Trial Description Phase AB-paclitaxel dosed every-3-weeks as first-line therapy for patients with advanced NSCLC1 II AB-paclitaxel dosed weekly as initial chemotherapy for patients with stage IV NSCLC2 I/II AB, albumin-bound; NSCLC, non-small cell lung cancer. 1. Green MR, et al. Ann Oncol. 2006;17:1263-1268. 2. Rizvi NA, et al. J Clin Oncol. 2008;26:639-643. 3 Albumin-Bound Paclitaxel Trials in NSCLC: Combination Therapy Trial Description Phase Combination Therapy with Carboplatin Weekly AB-paclitaxel + carboplatin as first-line therapy for advanced NSCLC1 II Dose-finding study of weekly and q3w AB-paclitaxel + carboplatin as first-line therapy for advanced NSCLC2 II AB-paclitaxel + carboplatin vs CrEL paclitaxela + carboplatin as first-line therapy for advanced NSCLC3,4 III AB-Paclitaxel + carboplatin for patients at risk of bleeding from VEGF-directed therapies5 II AB-paclitaxel + carboplatin in 2 different schedules for patients with ES-SCLC6 II Combination Therapy with Carboplatin and Bevacizumab AB-paclitaxel + carboplatin + bevacizumab as first-line therapy for advanced nonsquamous NSCLC7 II In vivo assessment of the effects of bevacizumab in advanced NSCLC8 II Combination with Carboplatin and Radiotherapy AB-paclitaxel + carboplatin + thoracic radiation in locally advanced NSCLC9 ® Cremophor is a registered trademark of BASF. AB, albumin-bound; CrEL, Cremophor® EL; ESSCLC, extensive stage small cell lung cancer; NSCLC, non-small cell lung cancer; q3w, every 3 weeks; VEGF, vascular endothelial growth factor. I 1. Allerton JP, et al. ASCO. 2006. [Abstract 7127]. 2. Socinski MA, et al. J Thoracic Oncol. 2010;5(5):852-861. 3. Socinski MA, et al. ASCO. 2010 [Abstract LBA7511]. 4. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 5. Otterson GA et al. IASLC. 2011 [Abstract 2230]. 6. Grilley-Olson JE et al. IASLC. 2011 [Abstract 2221]. 7. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 8. Heist RS, et al. ASCO. 2010. [Abstract 7612]. 9. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 4 Albumin-Bound Paclitaxel as First-line Treatment for Non-Small Cell Lung Cancer Monotherapy Studies 5 Summary of Albumin-Bound Paclitaxel Monotherapy Studies in Non-Small Cell Lung Cancer Trial Description Phase Albumin-bound paclitaxel as first-line treatment in advanced NSCLC1 II Albumin-bound paclitaxel as first-line treatment in stage IV NSCLC2 I/II NSCLC, non-small cell lung cancer. 1. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 2. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 6 Summary of Albumin-Bound Paclitaxel Monotherapy Studies in Non-Small Cell Lung Cancer Trial Description Albumin-bound paclitaxel as first-line treatment in advanced NSCLC1 Albumin-bound paclitaxel as first-line treatment in stage IV NSCLC2 Phase Main Idea II q3w albumin-bound paclitaxel at 260 mg/m2 showed considerable activity and a favorable therapeutic index in advanced NSCLC I/II qw 3/4 albumin-bound paclitaxel at 125 mg/m2 demonstrated encouraging clinical efficacy in stage IV NSCLC NSCLC, non-small cell lung cancer; q3w, every 3 weeks; qw 3/4, first 3 of 4 weeks. 1. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 2. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 7 nab-paclitaxel®, a Novel, Cremophor® ELFree Albumin-Bound Particle Form of Paclitaxel for the Treatment of Advanced Non-Small Cell Lung Cancer M.R. Green, G.M. Manikhas, S. Orlov, B. Afanasyev, A.M. Makhson, P. Bhar, M.J. Hawkins Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 8 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Background • Cremophor® ELa paclitaxel plays a role in first-line therapy for advanced NSCLC1 – Cremophor EL, the solvent vehicle2, is associated with severe hypersensitivity reactions2,3 – To prevent severe hypersensitivity reactions, all patients should be premedicated prior to administration of Cremophor EL paclitaxel2 • Albumin-bound paclitaxel is a solvent-free formulation4 – Use of albumin as a vehicle eliminates solvent-related toxicities5 – No premedication to prevent hypersensitivity reactions is required prior to administration4 • Due to the limitations of treatments for NSCLC, Green et al. explored efficacy/safety of albumin-bound paclitaxel for NSCLC5 a Cremophor is a registered trademark of BASF; NSCLC, non-small cell lung cancer. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 3. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 4. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 5. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 9 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Objective • Objective – Evaluate the efficacy and safety of albumin-bound paclitaxel 260 mg/m2 q3w as first-line treatment for patients with advanced NSCLC • Primary efficacy endpoint – ORR • Secondary endpoints – TTP – OS – Toxicity • Other endpoints assessed – Overall disease control rate (confirmed responders + patients with SD ≥ 16 weeks) NSCLC, non-small cell lung cancer; ORR, overall response rate; OS, overall survival; q3w, every 3 weeks; SD, stable disease; TTP, time to tumor progression. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 10 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Key Eligibility Criteria • Key inclusion criteria – Men and non-pregnant women ≥ 18 years of age – Histologically or cytologically confirmed advanced NSCLC (at least 1 measurable stage IIIB or IV lesion) – Evidence of inoperable local recurrence or metastasis, but no other active malignancy – No prior therapy for metastatic disease – Expected survival of > 12 weeks – Adequate hematologic, hepatic, and renal function NSCLC, non-small cell lung cancer. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 11 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Key Eligibility Criteria (cont.) • Key exclusion criteria – Clinical evidence of brain metastasis – Serious concurrent illness – ECOG PS of ≥ 2 – Peripheral neuropathy ≥ grade 2 – Prior radiotherapy ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer; PS, performance status. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 12 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Phase II Study Design Albumin-bound paclitaxel 260 mg/m2 IV over 30 minutes Treatment repeated q3w until disease progression or unacceptable toxicity • Dose reduction (from 260 to 200 mg/m2) was permitted for: – Grade 4 hematologic toxicity – Neutropenic fever or sepsis – Grade 3 or 4 non-hematologic toxicity • Dose reduction (to 130 mg/m2) was recommended for all subsequent cycles if: – Adverse events listed above recurred after the initial resolution and reinitiation of albumin-bound paclitaxel dosing IV, intravenous; NSCLC, non-small cell lung cancer; q3w, every 3 weeks. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 13 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Baseline Patient Characteristics Baseline characteristic (N = 43) Value Sex, n (%) Men Women 33 (77) 10 (23) Age, years Median Range < 65, n (%) ≥ 65, n (%) 58 43 - 75 34 (79) 9 (21) ECOG PS, n (%) 0 1 9 (21) 34 (79) Dominant lesion site, n (%) Visceral Nonvisceral 36 (84) 7 (16) Total number of lesions, n (%) 1 2-3 >3 5 (12) 13 (30) 25 (58) Stage at primary diagnosis I II III IV Unknown 1 (2) 2 (5) 14 (33) 24 (56) 2 (5) ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer; PS, performance status. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 14 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Results: Overall Response and Response by Histology Subgroup/variable Overall responseb Response by histology Carcinoma/adenocarcinoma Squamous cell carcinoma Otherc Baseline, n Confirmed ORR, n (%) Disease control,a n (%) Death, n (%) 43 7 (16) 21 (49) 23 (53) 11 29 3 1 (9) 5 (17) 1 (33) 5 (45) 14 (48) 2 (67) 5 (45) 16 (55) 2 (67) ORR, overall response rate. a Includes responders + patients with stable disease ≥ 16 weeks. b Includes all partial responses. c Other categories of NSCLC include large-cell carcinoma, undifferentiated NSCLC, and mixed squamous and adenocarcinoma. NSCLC, non-small cell lung cancer. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 15 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Results: Time to Disease Progression (treated population) • Median TTP: 6 months (95% CI: 3.9 - 6.5) • Probability of not having progressed: 50% at 6 months,13% at 1 year CI, confidence interval; NSCLC, non-small cell lung cancer; q3w, every 3 weeks; TTP, time to tumor progression. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 16 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Results: Overall Survival (treated population) • Median survival was 11 months (95% CI: 9.5 - 16.2) • Probability of surviving 1 year was 45% CI, confidence interval; NSCLC, non-small cell lung cancer; q3w, every 3 weeks. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 17 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Results: Dose Adjustments and Discontinuations Due to AEs Dose adjustments and discontinuations due to treatment-related AEs (N = 43) n (%) Dose reductiona 2 (5) Dose interruption 0 Discontinuation of therapy 2 (5) AE, adverse event. aTwo dose reductions were permitted: from 260 to 200 mg/m 2 and from 200 to 130 mg/m 2. • Treatment was well-tolerated • 95% of patients received albumin-bound paclitaxel at the protocol-specified dose • 98% of all cycles were administered at the full dose • Patients received a median of 6 treatment cycles • 63% of patients received ≥ 6 treatment cycles AE, adverse event; NSCLC, non-small cell lung cancer. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 18 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Results: Treatment-Related Toxicities/Adverse Events Maximum grade, n (%) of patients Toxicity/AEa Patients with ≥ 1 treatment-related AE Hematologicb Anemia Neutropenia Leukopenia Thrombocytopenia Non-hematologic Alopecia Sensory neuropathy Arthralgia Fatigue Myalgia Fever All 1 2 3 4 40 (93) 7 (16) 24 (56) 9 (21) 0 32 (74) 21 (49) 10 (23) 6 (14) 24 (56) 9 (21) 7 (16) 6 (14) 8 (19) 8 (19) 3 (7) 0 0 4 (9) 0 0 0 0 0 0 33 (77) 28 (65) 15 (35) 14 (33) 8 (19) 7 (16) 4 (9) 21 (49) 10 (23) 3 (7) 5 (12) 2 (5) 29 (67) 5 (12) 4 (9) 8 (19) 3 (7) 5 (12) NA 2 (5) 1 (2) 3 (7) 0 0 NA 0 0 0 0 0 AE, adverse event; NA, not applicable. aReported in ≥ 10% of patients (N = 43). bBased on central laboratory values. NSCLC, non-small cell lung cancer. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 19 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Conclusions • Albumin-bound paclitaxel 260 mg/m2 administered IV over 30 minutes without premedication was well tolerated • Significant tumor responses and prolonged disease control were documented in these patients with NSCLC • Exploration of higher doses of albumin-bound paclitaxel alone and in combination with other drugs in NSCLC is warranted IV, intravenous; NSCLC, non-small cell lung cancer. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 20 Phase I/II Trial of Weekly Intravenous Albumin-Bound Paclitaxel as Initial Chemotherapy in Patients with Stage IV Non-Small Cell Lung Cancer N.A. Rizvi, G.J. Riely, C.G. Azzoli, V.A. Miller, K.K. Ng, J. Fiore, G. Chia, M. Brower, R. Heelan, M.J. Hawkins, M.G. Kris Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 21 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Advanced NSCLC Background • Cremophor® ELa paclitaxel plays a role in first-line therapy for advanced NSCLC1 – Cremophor EL, the solvent vehicle2, is associated with severe hypersensitivity reactions2,3 – To prevent severe hypersensitivity reactions, all patients should be premedicated prior to administration of Cremophor EL paclitaxel2 • Albumin-bound paclitaxel is a solvent-free formulation4 – Use of albumin as a vehicle eliminates solvent-related toxicities5 – No premedication to prevent hypersensitivity reactions is required prior to administration4 aCremophor EL is a registered trademark of BASF; NSCLC, non-small cell lung cancer. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 3. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 4. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 5. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 22 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Background (cont.) • In a phase II study of albumin-bound paclitaxel 260 mg/m2 IV q3w as first-line therapy for advanced NSCLC1: – ORR 16%; median TTP 6 months; median survival 11 months – No severe hypersensitivity reactions reported • In a phase I/PK dose-ranging study of weekly albumin-bound paclitaxel in patients with solid tumors2: – MTD in lightly pretreated patients was 150 mg/m2 – DLTs were grade 3 peripheral neuropathy and grade 4 neutropenia – PR in 1 patient with lung cancer previously treated with Cremophor® EL paclitaxel • Rizvi et al. explored the MTD of weekly albumin-bound paclitaxel in patients with untreated stage IV NSCLC3 DLT, dose-limiting toxicity; IV, intravenous; MTD, maximum-tolerated dose; NSCLC, non-small cell lung cancer; ORR, overall response rate; PK, pharmacokinetics; PR, partial response; q3w, every 3 weeks; TTP, time to tumor progression. 1. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 2. Nyman DW, et al. J Clin Oncol. 2005; 23(31):7785-7793. 3. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 23 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Objective • Objective – Determine MTD and single-agent activity of albumin-bound paclitaxel administered weekly as first-line therapy for patients with stage IV NSCLC • Primary endpoints – ORR* (CR and PR determined using RECIST) – Safety • Secondary efficacy endpoints – TTP – OS *Responses had to be confirmed at least 4 weeks after initial documentation; CR, complete response; MTD, maximum tolerated dose; NSCLC, non-small cell lung cancer; ORR, overall response rate; OS, overall survival; PR, partial response; RECIST, Response Evaluation Criteria in Solid Tumors; TTP, time to tumor progression. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 24 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Key Eligibility Criteria • Key inclusion criteria – Stage IV or recurrent NSCLC – Laboratory requirements • • • • • • AST and ALT ≤ 2.5X the upper limit of normal range Total bilirubin within the normal range Creatinine ≤ 1.5 mg/dL ANC ≥ 1.5 109 cells/L Platelets ≥ 100 109 cells/L Hemoglobin ≥ 9 g/dL • Key exclusion criteria – Prior chemotherapy for advanced NSCLC – Peripheral neuropathy > grade 1 – Radiotherapy received ≤ 3 weeks before study entry ALT, alanine aminotransferase; ANC, absolute neutrophil count; AST, aspartate aminotransferase; NSCLC, non-small cell lung cancer. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 25 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Study Design • Open-label, single-arm, phase I/II trial Phase I (N =15) Albumin-bound paclitaxel 100 → 125 → 150 mg/m2 IV qw 3/4 Phase II (N = 40) Albumin-bound paclitaxel 125 mg/m2 IV qw 3/4 IV, intravenous; NSCLC, non-small cell lung cancer; q8w, every 8 weeks; qw 3/4, first 3 of 4 weeks. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. Radiologic tumor assessment q8w 26 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Baseline Characteristics of Patients in Phase II Portion Baseline characteristic (N = 40) Value Sex, n (%) Men Women 19 (47) 21 (53) Age, years Median Range 70 43 - 84 Karnovsky PS, n (%) 90%-100% 70%-80% 10 (25) 30 (75) Prior chemotherapy, n (%) Neoadjuvant Adjuvant Prior gefitinib or erlotinib 3 (8) 6 (15) 5 (13) Histology, n (%) Adenocarcinoma Squamous cell carcinoma 32 (80) 8 (20) EGFR TKI—epidermal growth factor tyrosine kinase inhibitor NSCLC, non-small cell lung cancer; PS, performance status. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 27 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Results: Phase II Outcome (N = 40) Value ORR (PR + CR)* % 95% CI 30 16 - 44 SD ≥ 16 weeks n % 8 20 Median TTP months 95% CI 5 3-8 Median OS months 95% CI 11 7 - NR One-year OS % 41 *Responses had to be confirmed at least 4 weeks after initial documentation. CI, confidence interval; CR, complete response; NR, not reached; ORR, overall response rate; OS, overall survival; PR, partial response; SD, stable disease; TTP, time to tumor progression. NSCLC, non-small cell lung cancer. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 28 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Results: Treatment-Related Adverse Events Maximum grade, % AE* (N = 40) All 1 2 3 4 Hematologic Anemia Leukopenia Neutropenia 92 62 60 63 23 13 23 20 28 8 20 15 0 0 5 Non-hematologic Fatigue Neuropathy Alopecia Constipation Diarrhea Nausea Rash Edema Myalgia Anorexia Hypersensitivity 75 73 70 58 48 45 40 30 28 28 0 28 35 10 38 25 38 35 18 18 18 0 30 23 60 20 10 8 5 13 10 10 0 18 15 0 0 13 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 0 *Most frequent treatment-related AEs in patients treated with albumin-bound paclitaxel 125 mg/m2. AE, adverse event. NSCLC, non-small cell lung cancer. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 29 Albumin-Bound Paclitaxel Monotherapy as First-Line Treatment for Stage IV NSCLC Conclusions • Albumin-bound paclitaxel 125 mg/m2 administered qw 3/4 was well tolerated – No corticosteroid premedication was administered and no hypersensitivity reactions were seen • Albumin-bound paclitaxel 125 mg/m2 demonstrated encouraging single-agent activity • Additional studies of single-agent albumin-bound paclitaxel as well as platinum-based combinations are warranted NSCLC, non-small cell lung cancer; qw 3/4, first 3 of 4 weeks. Rizvi NA, et al. J Clin Oncol. 2008;26(4):639-643. 30 Albumin-Bound Paclitaxel for the Treatment of Non-Small Cell Lung Cancer Combination Therapy Studies 31 Summary of Albumin-Bound Paclitaxel Combination Therapy Studies in NSCLC Trial Description Phase Combination Therapy with Carboplatin Weekly AB-paclitaxel + carboplatin as first-line therapy for advanced NSCLC1 II Dose-finding study of weekly and q3w AB-paclitaxel + carboplatin as first-line therapy for advanced NSCLC2 II AB-paclitaxel + carboplatin vs CrEL paclitaxel + carboplatin as first-line therapy for advanced NSCLC3,4 III AB-Paclitaxel + carboplatin for patients at risk of bleeding from VEGF-directed therapies5 II Combination Therapy with Carboplatin and Bevacizumab AB-paclitaxel + carboplatin + bevacizumab as first-line therapy for advanced nonsquamous NSCLC6 II In vivo assessment of the effects of bevacizumab in advanced NSCLC7 II Combination with Carboplatin and Radiotherapy AB-paclitaxel + carboplatin + thoracic radiation in locally advanced NSCLC8 I Combination Therapy in SCLC AB-paclitaxel + carboplatin in 2 different schedules for patients with ES-SCLC9 II AB, albumin-bound; CrEL, Cremophor® EL; ES-SCLC, extensive stage small cell lung cancer; NSCLC, non-small cell lung cancer; q3w, every 3 weeks; VEGF, vascular endothelial growth factor. ® Cremophor is a registered trademark of BASF. References in slide notes. 32 Summary of Albumin-Bound Paclitaxel Combination Therapy Studies in NSCLC Trial Description Phase Main Idea Combination Therapy with Carboplatin Weekly AB-paclitaxel + carboplatin as first-line therapy for advanced NSCLC1 II AB-paclitaxel + carboplatin has a high degree of antitumor activity and an acceptable safety profile1 Dose-finding study of weekly and q3w ab-pac + carboplatin as first-line therapy for advanced NSCLC2 II AB-paclitaxel 100 mg/m2 weekly + carboplatin AUC = 6 q3w is an optimal dose and schedule for treating NSCLC2 AB-paclitaxel + carboplatin vs CrEL paclitaxel + carboplatin as first-line therapy for advanced NSCLC3,4 III AB-paclitaxel + carboplatin demonstrated a higher response rate than CrEL paclitaxel + carboplatin3,4 AB-paclitaxel + carboplatin for NSCLC patients at risk of bleeding from VEGF directed therapies5 II AB-paclitaxel + carboplatin showed a high ORR in NSCLC patients ineligible for VEGF directed therapies5 Combination Therapy with Carboplatin and Bevacizumab AB-paclitaxel + carboplatin + bevacizumab as first-line therapy for advanced nonsquamous NSCLC6 II PFS and OS were higher than previously reported in patients with advanced nonsquamous NSCLC and toxicity was generally acceptable5 In vivo assessment of the effects of bevacizumab in advanced NSCLC7 II PD and imaging biomarker studies in bevacizumabtreated NSCLC patients are feasible and may predict tumor response6 I Weekly AB-paclitaxel at 40 mg/m2 + carboplatin + thoracic radiation appears to be safe and well tolerated7 II qw and q3w schedules of AB-paclitaxel + carboplatin produced high ORRs in patients with ES-SCLC6 Combination with Carboplatin and Radiotherapy AB-paclitaxel + carboplatin + thoracic radiation in locally advanced NSCLC8 Combination Therapy in SCLC AB-paclitaxel + carboplatin in 2 different schedules for patients with ES-SCLC9 AB, albumin-bound; AUC, area under the curve; CrEL, Cremophor® EL; ES-SCLC, extensive stage small cell lung cancer; NSCLC, non-small cell lung cancer; OS, overall survival; PD, pharmacodynamic; PFS, progression-free survival; q3w, every 3 weeks; qw, weekly. References in slide notes. 33 Albumin-Bound Paclitaxel for the First-Line Treatment of Non-Small Cell Lung Cancer Combination Therapy with Carboplatin 34 A Phase II Evaluation of the Combination of Albumin-Bound Paclitaxel + Carboplatin in the First-Line Treatment of Advanced Non-Small Cell Lung Cancer J.P. Allerton, C.T. Hagenstad, T.R. Webb, G.B. Smith, R. Birch, T.F. Goggins, S.B. Katakkar, W. Khan, N.D. Mehta, F.A. Greco, Online Collaborative Oncology Group Allerton JP, et al. ASCO. 2006 [abstract 7127]. 35 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Background • Cremophor® EL paclitaxela + platinum agents are recommended as first-line chemotherapy for advanced NSCLC1 – In phase III trials, CrEL paclitaxel + carboplatin showed efficacy • Objective RR of 25%,2 overall RR of 32%,3 RR of 30%4 – CrEL use is associated with severe hypersensitivity reactions6 • All patients should be premedicated prior to CrEL paclitaxel administration to prevent severe hypersensitivity reactions5 • Albumin-bound paclitaxel is a solvent-free formulation of paclitaxel7 – Use of albumin as a vehicle eliminates solvent-related toxicities8 – No premedication to prevent hypersensitivity reactions is required prior to administration7 a Cremophor® EL is a registered trademark of BASF; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; RR, response rates. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Kelly K, et al. J Clin Oncol. 2001;19:3210-3218. 3. Scagliotti GV, et al. J Clin Oncol. 2002;20:4282-429. 4. Lilenbaum RC, et al. J Clin Oncol. 2005;23:190-196. 5. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 6. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 7. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 8. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 36 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Background (cont.) • Belani et al. studied weekly Cremophor® EL paclitaxel 100 mg/m2 qw 3/4 + carboplatin for advanced NSCLC1 – ORR 32% – Median TTP 30 weeks – Median survival time 49 weeks – 1-year survival rate 47% • Allerton et al. examined whether substituting albumin-bound paclitaxel for Cremophor® EL paclitaxel at an identical dose would improve ORR2 NSCLC, non-small cell lung cancer; ORR, objective response rate; qw3/4, weekly for 3 of 4 weeks; TTP, time to tumor progression. 1. Belani CP, et al. J Clin Oncol. 2003;21(15):2933-2939. 2. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 37 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Objective • Objective – Determine efficacy and safety of albumin-bound paclitaxel + carboplatin as first-line therapy for advanced NSCLC • Efficacy endpoints – Response rate – Response duration – TTP • Safety endpoints – Toxicities NSCLC, non-small cell lung cancer ; TTP, time to tumor progression. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 38 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Key Eligibility Criteria • Key inclusion criteria – Inoperable stage IIIB or IV NSCLC • ≥ 1 measurable indicator lesion • > 4 weeks from previous radiation therapy – ECOG PS of 0, 1, or 2 at screening and on first day of treatment – Life expectancy > 12 weeks • Key exclusion criteria – Previous treatment with chemotherapy – Peripheral neuropathy ≥ grade 2 ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer; PS, performance status. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 39 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Study Design • Open-label, phase II study Albumin-bound paclitaxel 100 mg/m2 IV on days 1, 8, and 15 q28 days Carboplatin AUC = 6 IV on day 1 AUC, area under the curve; IV, intravenous; NSCLC, non-small cell lung cancer; q28, every 28 days. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 40 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Baseline Patient Characteristics Baseline characteristic (N = 56) Value Age, years Median Range 66 37 - 83 Sex, n (%) Men Women 39 (70) 17 (30) Disease stage, n (%) Stage IIIB 14 (25) Stage IV with metastasis to, n Bone Liver Brain Lymph nodes 17 7 2 17 ECOG PS Median 0, n 1, n 2, n 1 18 33 2 ECOG, Eastern Cooperative Oncology Group; PS, performance status. NSCLC, non-small cell lung cancer. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 41 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Response Rate Response rate, n (%) Patients CR PR SD > 12 weeks All evaluable patients (N = 50) 1 (2) 24 (48) 18 (36) Stage IIIB (n = 12) 0 4 (33) 4 (33) Stage IV (n = 38) 1 (3) 20 (53) 14 (37) CR, complete response; PR, partial response; SD, stable disease. • Of 18 patients with SD – Stable at 12-29 weeks, n = 14 – Progressed at 20, 21, 23, and 28 weeks, n = 4 NSCLC, non-small cell lung cancer. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 42 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Time to Tumor Progression • Median TTP, 28 weeks; maximum follow-up, 39 weeks a Albumin-bound paclitaxel 100 mg/m2 was administered IV qw 3/4 and carboplatin AUC = 6 was administered on day 1 only of a 28-day cycle. AUC, area under the curve; NSCLC, non-small cell lung cancer; qw 3/4 weekly on 3 of 4 weeks; TTP, time to tumor progression. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 43 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Toxicities Grade 3 or 4 toxicitiesa n (%) Neutropenia 24 (44) Thrombocytopenia 14 (25) Anemia 5 (9) Neuropathy 0 Arthralgia 0 Myalgia 0 Nausea 1 (2) Vomiting 1 (2) Diarrhea 0 a Toxicities associated with albumin-bound paclitaxel followed by carboplatin. NSCLC, non-small cell lung cancer. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 44 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Conclusions • Efficacy conclusions – In first-line treatment of advanced NSCLC, albumin-bound paclitaxel + carboplatin had a high degree of objectively-defined antitumor activity • ORR 50% • Median TTP 28 weeks • Safety conclusions – Acceptable safety profile – Neutropenia was the most common severe toxicity – No patients had grade 3/4 peripheral neuropathy NSCLC, non-small cell lung cancer; ORR, overall response rate; TTP, time to tumor progression. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 45 A Dose Finding Study of Weekly and Every 3 Week Albumin-Bound Paclitaxel Followed by Carboplatin as First-Line Therapy in Patients with Advanced Non-Small Cell Lung Cancer M.A. Socinski, G.M. Manikhas, D.L. Stroyakovsky, A.N. Makhson, S.V. Cheporov, S.V. Orlov, P.K. Yablonsky, P.H. Bhar, and J. Iglesias Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 46 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Background • Cremophor® EL paclitaxela + platinum agents are recommended as first-line chemotherapy for advanced NSCLC1 – In phase III trials, CrEL paclitaxel + carboplatin showed efficacy: • Objective RR of 25%,2 overall RR of 32%,3 RR of 30%4 – CrEL use is associated with severe hypersensitivity reactions6 • All patients should be premedicated prior to CrEL paclitaxel administration to prevent severe hypersensitivity reactions5 • Albumin-bound paclitaxel is a solvent-free formulation of paclitaxel7 – Use of albumin as a vehicle eliminates solvent-related toxicities8 – No premedication to prevent hypersensitivity reactions is required prior to administration7 a Cremophor® EL is a registered trademark of BASF; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; RR, response rates. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Kelly K, et al. J Clin Oncol. 2001;19:3210-3218. 3. Scagliotti GV, et al. J Clin Oncol. 2002;20:4282-429. 4. Lilenbaum RC, et al. J Clin Oncol. 2005;23:190-196. 5. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 6. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 7. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 8. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 47 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Background Phase Patients, n ORR, % Median TTP, months CrEL paclitaxel monotherapy1 III 157 16 4 6.8 CrEL paclitaxel + carboplatin2 NA 290 17 3.1 8.1 q3w AB-paclitaxel monotherapy3 II 43 16 6 11 weekly AB-paclitaxel monotherapy4 II 40 30 5 11 weekly AB-paclitaxel + carboplatin5 II 50 50 7 NA NSCLC treatment studied Median OS, months AB, albumin-bound; CrEL, Cremophor EL; NA, not available; ORR overall response rate; OS, overall survival; q3w, every 3 weeks; TTP, time to tumor progression. • Based on the efficacy of ab-paclitaxel vs CrEL paclitaxel, Socinski et al. aimed to identify the optimal dose of ab-paclitaxel + carboplatin as first-line therapy in advanced NSCLC6 NSCLC, non-small cell lung cancer. 1. Ranson M, et al. J Natl Cancer Inst. 2000;92:1074-1080. 2. Schiller JH, et al. N Engl J Med. 2002;346:92-98. 3. Green MR, et al. Ann Oncol. 2006;17:1263-1268. 4. Rizvi NA, et al. J Clin Oncol. 2008;26:639-643. 5. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 6. Socinski MA, et al. J Thoracic Oncol. 2010;5(5):852-861. 48 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Objective • Objective – Identify optimal dose and schedule of albumin-bound paclitaxel + carboplatin as first-line therapy for advanced NSCLC • Primary efficacy endpoints – CR or PR based on RECIST • Secondary efficacy endpoints – SD ≥ 16 weeks – PFS – OS • Safety endpoints – – – – Serious AEs Treatment-related AEs Laboratory abnormalities Dose modifications, dose interruptions, premature discontinuation of study drug AE, adverse event; CR, complete response; NSCLC, non-small cell lung cancer; OS, overall survival; PFS, progression-free survival; PR, partial response; q3w, every 3 weeks; RECIST, Response Evaluation Criteria in Solid Tumors; SD, stable disease. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 49 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Key Eligibility Criteria • Key inclusion criteria – Men and nonpregnant, nonlactating women ≥ 18 years of age – Stage IIIB or IV NSCLC • Histologically or cytologically confirmed • Pleural effusion or evidence of inoperable local recurrence or metastasis • Measurable disease as defined by RECIST guidelines – No previous treatment for metastatic disease – Life expectancy > 12 weeks – ECOG PS of 0 or 1 – Adequate hematologic, hepatic and renal function ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer; PS, performance status; RECIST, Response Evaluation Criteria in Solid Tumors. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 50 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Key Eligibility Criteria • Key exclusion criteria – Evidence of active brain metastasis – Any other clinically serious concurrent illness – Radiotherapy or chemotherapy in the previous 4 weeks – Peripheral neuropathy > grade 1 – History of allergy or hypersensitivity to either of the study drugs NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 51 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Study Design • Open-label, multicenter, phase II study • AB-paclitaxel dosing schedule shown below • All patients also received q3w carboplatin AUC = 6 q3w AB-paclitaxel dosing weekly AB-paclitaxel dosing Cohort 1 Patients 1-25 225 mg/m2 on day 1 q3w Cohort 5 Patients 101-125 140 mg/m2 on day 1, 8 q3w Cohort 2 Patients 26-50 260 mg/m2 on day 1 q3w Cohort 6 Patients 126-150 100 mg/m2 on day 1, 8, 15 q3w Cohort 3 Patients 51-75 300 mg/m2 on day 1 q3w Cohort 7 Patients 151-175 125 mg/m2 on day 1, 8, 15 q3w Cohort 4 Patients 76-100 340 mg/m2 on day 1 q3w AB-paclitaxel, albumin-bound paclitaxel; AUC, area under the curve; NSCLC, non-small cell lung cancer; q3w, every 3 weeks. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 52 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Baseline Patient Characteristics Weekly (days 1, 8) q3w Baseline characteristic (N = 175) Weekly (days 1, 8, 15) Cohortsa 225 mg/m2 260 mg/m2 300 mg/m2 340 mg/m2 140 mg/m2 100 mg/m2 125 mg/m2 Age, years Mean 59.7 63.1 60.1 61.3 61.6 59.9 58.8 Sex, n (%) Men 23 (92) 18 (72) 17 (68) 20 (80) 22 (88) 21 (84) 20 (80) Stage, n (%) IIIB IV 10 (40) 15 (60) 8 (32) 17 (68) 4 (16) 21 (84) 3 (12) 22 (88) 4 (16) 21 (84) 4 (16) 21 (84) 7 (28) 18 (72) Histology, n (%) Nonsquamous Squamous Otherb 9 (36) 11 (44) 5 (20) 7 (28) 18 (72) 0 9 (36) 14 (56) 2 (8) 9 (36) 16 (64) 0 (0) 10 (40) 15 (60) 0 9 (36) 16 (64) 0 13 (52) 10 (40) 2 (8) ECOG PS, n (%) 0 1 1 (4) 24 (96) 0 25 (100) 3 (12) 22 (88) 7 (28) 18 (72) 5 (20) 20 (80) 4 (16) 21 (84) 3 (12) 22 (88) Preexisting peripheral neuropathy, n (%) Grade 0 Grade 1 23 (92) 2 (8) 23 (92) 2 (8) 24 (96) 1 (4) 21 (84) 4 (16) 25 (100) 0 24 (96) 1 (4) 25 (100) 0 AUC, area under the curve; ECOG PS, Eastern Cooperative Oncology Group performance status; q3w, every 3 weeks. a 25 patients per cohort; all patients received carboplatin AUC = 6. b Poorly differentiated or non-differentiated NSCLC. NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 53 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Response Rates and DCR for All Treated Patients Weekly (days 1, 8) q3w Clinical response (N = 175) Weekly (days 1, 8, 15) Cohortsa 225 mg/m2 260 mg/m2 300 mg/m2 340 mg/m2 140 mg/m2 100 mg/m2 125 mg/m2 10 (40) 20.8-59.2 6 (24) 7.3-40.7 6 (24) 7.3-40.7 8 (32) 13.7-50.3 14 (56) 36.5-75.5 12 (48) 28.4-67.6 9 (36) 17.2-54.8 CR, n (%) 0 1 (4) 0 0 0 1 (4) 1 (4) PR, n (%) 10 (40) 5 (20) 6 (24) 8 (32) 14 (56) 11 (44) 8 (32) SD ≥ 16 wks, n (%) 5 (20) 8 (32) 3 (12) 0 2 (8) 2 (8) 3 (12) 15 (60) 40.8-79.2 14 (56) 36.5-75.5 9 (36) 17.2-54.8 8 (32) 13.7-50.3 16 (64) 45.2-82.8 14 (56) 36.5-75.5 12 (48) 28.4-67.6 ORR, n (%) 95% CI DCRb n (%) 95% CI AUC, area under the curve; CI, confidence interval; CR, complete response; DCR, disease control rate; ORR, objective response rate; PR, partial response; q3w, every 3 weeks; SD, stable disease. a 25 patients per cohort, all patients received carboplatin at AUC = 6.. b DCR = CR + PR + SD ≥ 16 weeks. NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 54 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: PFS and OS for All Treated Patients Weekly (days 1, 8) q3w Weekly (days 1, 8, 15) Clinical response (N = 175) 225 mg/m2 260 mg/m2 300 mg/m2 340 mg/m2 140 mg/m2 100 mg/m2 125 mg/m2 Median PFS, months 95% CI 6.9 4.2-9.6 6.5 4.3-9.1 5.3 2.2-8.5 4.8 3.9-7.8 5.6 3.9-7.7 6.2 4.2-9.7 6.4 4.2-7.9 Median OS, months 95% CI 10.7 8.7-17.0 12.2 8.5-21.9 8.3 4.2-15.4 14.6 7.6-17.2 12.0 6.5-17.1 11.3 7.8- >20.1 15.0 10- >18.4 Cohortsa AUC, area under the curve; CI, confidence interval; OS, overall survival; PFS, progression-free survival; q3w, every 3 weeks. a 25 patients per cohort, all patiente received carboplatin AUC = 6. NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 55 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Progression-Free Survival AUC, area under the curve; NSCLC, non-small cell lung cancer; q3w, every 3 weeks. Note: all patients received carboplatin AUC = 6. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 56 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Overall Survival AUC, area under the curve; NSCLC, non-small cell lung cancer; q3w, every 3 weeks. Note: all patients received carboplatin AUC = 6. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 57 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Efficacy Results by Histologic Subtype, SCC Weekly (days 1, 8) q3w Weekly (days 1, 8, 15) Cohortsa 225 mg/m2 (n = 11 ) 260 mg/m2 (n = 18) 300 mg/m2 (n = 14) 340 mg/m2 (n = 16) 140 mg/m2 (n = 15) 100 mg/m2 (n= 16) 125 mg/m2 (n = 10) ORR, n (%) 95% CI 5 (45) 16.8-76.6 5 (28) 9.7-53.5 5 (36) 12.8-64.9 6 (38) 13.8-61.2 8 (53) 28.1-78.6 5 (31) 11.0-58.7 3 (30) 6.7-65.2 CR, n (%) 0 1 (6) 0 0 0 0 0 PR, n (%) 5 (45) 4 (22) 5 (36) 6 (38) 8 (53) 5 (31) 3 (30) SD ≥ 16 weeks, n (%) 2 (18) 6 (33) 1 (7) 0 1 (7) 1 (6) 1 (10) DCRb n (%) 95% CI 7 (64) 35.2-92.1 11 (61) 38.6-83.6 6 (43) 16.9-68.8 6 (37) 13.8-61.2 9 (60) 35.2-84.8 6 (38) 13.8-61.2 4 (40) 12.2-73.8 Median PFS, months 95% CI 8.1 4.2-10.4 8.4 5.7-21.7 5.3 1.9-15.5 6.0 4.4-7.8 5.0 3.9-6.1 4.5 2.1-9.7 4.2 4.1-7.9 Median OS, months 95% CI 13.2 5.4-18.5 12.2 8.5-23.9 8.0 3.1-17.8 15.1 10.5-18.5 9.4 7.8-14.0 12.6 5.3->18.8 10.9 9.2-16.3 Clinical response AUC, area under the curve; CI, confidence interval; CR, complete response; DCR, disease control rate; ORR, objective response rate; OS, overall survival; PFS, progression-free survival; PR, partial response; q3w, every 3 weeks; SCC, squamous cell carcinoma; SD, stable disease. a All patients received carboplatin AUC = 6. b DCR = CR + PR + SD ≥ 16 weeks. NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 58 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Efficacy Results by Histologic Subtype, Non-SCC Weekly (days 1, 8) q3w Weekly (days 1, 8, 15) Cohorts 225 mg/m2 (n = 9) 260 mg/m2 (n = 7) 300 mg/m2 (n = 9) 340 mg/m2 (n = 9) 140 mg/m2 (n = 10) 100 mg/m2 (n = 9) 125 mg/m2 (n = 13) 4 (44) 13.7-78.8 1 (14) 0.4-57.9 1 (11) 0.3-48.2 2 (22) 2.8-60.0 6 (60) 29.6-90.4 7 (78) 50.6-100 6 (46) 19.1-73.2 CR, n (%) 0 0 0 0 0 1 (11) 1 (8) PR, n (%) 4 (44) 1 (14) 1 (11) 2 (22) 6 (60) 6 (67) 5 (38) SD ≥ 16 weeks, n (%) 1 (11) 2 (29) 2 (22) 0 1 (10) 1 (11) 1 (8) 5 (56) 21.2-86.3 3 (43) 9.9-81.6 3 (33) 7.5-70.1 2 (22) 2.8-60.0 7 (70) 41.6-98.4 8 (89) 68.4-100 7 (54) 26.8-80.1 PFS in months, median 95% CI 5.8 4.0-9.6 5.5 2.5-10.2 5.3 3.5-7.0 4.4 3.7-8.7 7.7 3.5-15.9 6.6 5.7-17.0 18.3 4.6-18.3 OS in months, median 95% CI 12.4 10.3-21.0 10.7 7.3->22.0 10.5 7.3->25.1 11.9 4.4->22.3 13.1 4.8->18.4 9.8 7.8-11.3 >18.4 15.0->18.4 Clinical response ORR, n (%) 95% CI DCRa n (%) 95% CI AUC, area under the curve; CI, confidence interval; CR, complete response; DCR, disease control rate; ORR, objective response rate; OS, overall survival; PFS, progression-free survival; PR, partial response; q3w, every 3 weeks; SD, stable disease. a All patients received carboplatin AUC = 6. b DCR = CR + PR + SD ≥ 16 weeks. Non-SCC, nonsquamous cell carcinoma; NSCLC, non-small cell lung cancer; Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 59 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: ORR and DCR by Histologic Subtype DCR, disease control rate; Non-SCC, nonsquamous cell carcinoma; NS, not statistically significant; NSCLC, non-small cell lung cancer; ORR, overall response rate; q3w, every 3 weeks; SCC, squamous cell carcinoma. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 60 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: PFS and OS by Histologic Subtype Non-SCC, nonsquamous cell carcinoma; NS, not statistically significant; NSCLC, non-small cell lung cancer; OS, overall survival; PFS, progression-free survival; q3w, every 3 weeks; SCC, squamous cell carcinoma. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 61 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Treatment-Related Hematologic ≥ Grade 3 AEs in ≥ 5% of Patients Weekly (days 1, 8) q3w Weekly (days 1, 8, 15) Adverse event, n (%) (N = 175) 225 mg/m2 260 mg/m2 300 mg/m2 340 mg/m2 140 mg/m2 100 mg/m2 125 mg/m2 Neutropenia Grade 3 Grade 4 8 (32) 8 (32) 9 (36) 6 (24) 9 (36) 3 (12) 7 (28) 5 (20) 8 (32) 11 (44) 9 (36) 7 (28) 7 (28) 8 (32) Leukopenia Grade 3 Grade 4 8 (32) 1 (4) 6 (24) 0 (0) 7 (28) 0 (0) 9 (36) 1 (4) 12 (48) 0 (0) 6 (24) 0 (0) 5 (20) 1 (4) Thrombocytopenia Grade 3 Grade 4 7 (28) 3 (12) 5 (20) 1 (4) 5 (20) 2 (8) 5 (20) 1 (4) 5 (20) 3 (12) 4 (16) 1 (4) 5 (20) 4 (16) Anemia Grade 3 Grade 4 4 (16) 1 (4) 6 (24) 0 (0) 3 (12) 1 (4) 2 (8) 1 (4) 4 (16) 1 (4) 4 (16) 0 (0) 10 (40) 1 (4) Cohortsa AUC, area under the curve; q3w, every 3 weeks. a 25 patients per cohort, all patients received carboplatin AUC = 6. AE, adverse event; NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 62 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Treatment-Related Non-Hematologic ≥ Grade 3 AEs in ≥ 5% of Patients Weekly (days 1, 8) q3w Weekly (days 1, 8, 15) Adverse event, n (%) (N = 175) 225 mg/m2 260 mg/m2 300 mg/m2 340 mg/m2 140 mg/m2 100 mg/m2 125 mg/m2 Peripheral neuropathy Grade 3 Grade 4 3 (12) 0 (0) 4 (16) 0 (0) 6 (24) 0 (0) 12 (48) 0 (0) 2 (8) 0 (0) 2 (8) 0 (0) 4 (16) 0 (0) Fatigue Grade 3 Grade 4 3(12) 0 (0) 1 (4) 0 (0) 4 (16) 0 (0) 3 (12) 0 (0) 1 (4) 0 (0) 0 (0) 0 (0) 4 (16) 0 (0) Myalgia Grade 3 Grade 4 0 0 1 (4) 0 1 (4) 0 6 (24) 0 0 0 0 0 0 0 Arthralgia Grade 3 Grade 4 0 0 1 (4) 0 1 (4) 0 2 (8) 0 0 0 0 0 0 0 Cohortsa AUC, area under the curve; q3w, every 3 weeks. a 25 patients per cohort, all patients received carboplatin AUC = 6. AE, adverse event; NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 63 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Time to Improvement of Peripheral Neuropathy Weekly (days 1, 8) q3w Weekly (days 1, 8, 15) Cohortsa Improvement of peripheral neuropathy 225 mg/m2 (n = 3) 260 mg/m2 (n = 4) 300 mg/m2 (n = 6) 340 mg/m2 (n = 12) 140 mg/m2 (n = 2) 100 mg/m2 (n = 2) 125 mg/m2 (n = 4) Improved to grade ≤ 2, n (%) 2 (67) 4 (100) 2 (33) 7 (58) 1 (50) 2 (100) 1 (25) Time to improvement, Median daysa 95% CI 15.0 9.0-> 21.0 14.5 6.0-34.0 > 48.0 6.0-> 48.0 23.0 17.0-> 66.0 8.0 --- 15.5 13.0-18.0 > 24.0 8.0-> 24.0 AUC, area under the curve; CI, confidence interval; q3w, every 3 weeks. a All patients received carboplatin AUC = 6. b Time to improvement was defined as the time from first occurrence of grade 3 peripheral neuropathy to improvement of at least to grade 2. NSCLC, non-small cell lung cancer. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 64 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Results: Dose Modifications and Dose Interruptions • Dose modifications – Albumin-bound paclitaxel: • In the q3w cohorts, 40% of patients had ≥ 1 dose reduction • In the weekly cohorts, 51% of patients had ≥ 1 dose reduction • Hematologic toxicity was the most common reason for dose reductions – Carboplatin: • In the q3w cohorts, 6% of patients had a dose reduction • In the weekly cohorts, 16% of patients had a dose reduction • Dose interruptions – Albumin-bound paclitaxel: • None – Carboplatin: • In the q3w cohorts, 2 patients had a dose interruption because of a hypersensitivity reaction NSCLC, non-small cell lung cancer. q3w, every 3 weeks. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 65 First-Line Albumin-Bound Paclitaxel + Carboplatin in Advanced NSCLC Conclusions • Albumin-bound paclitaxel + carboplatin demonstrated antitumor activity in all cohorts and was well tolerated – 100 mg/m2 dose of albumin-bound paclitaxel, although not showing the highest DCR of all doses tested, provided the best clinical benefit-risk ratio – Weekly vs q3w albumin-bound paclitaxel was associated with improved clinical outcomes and less serious AEs • 100 mg/m2 weekly albumin-bound paclitaxel + carboplatin AUC = 6 q3w is an optimal dose and schedule for treating NSCLC – Based on the these results, a phase III study comparing 100 mg/m2 albumin-bound paclitaxel + carboplatin AUC = 6 q3w with standard dose Cremophor® EL paclitaxel + carboplatin for NSCLC was initiated AE, adverse event; AUC, area under the curve; DCR, disease control rate; NSCLC, non-small cell lung cancer; q3w, every 3 weeks. Socinski MA, et al. J Thoracic Oncol. 2010;5(6):852-861. 66 Survival Results of a Randomized, Phase III Trial of Albumin-Bound Paclitaxel and Carboplatin Compared With Cremophor-Based Paclitaxel and Carboplatin as First-Line Therapy in Advanced Non-Small Cell Lung Cancer M.A. Socinski, I. Bondarenko, N.A. Karaseva, A.M. Makhson, I.O. Vynnychenko, I. Okamoto, J. Hon, V. Hirsh, P. Bhar, G.I. Berks, J.L. Iglesias aCremophor BASF. is a registered trademark of Socinski MA et al. ASCO. 2011 [Abstract 7551]. 67 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Background • Cremophor® EL paclitaxela + platinum agents are recommended as first-line chemotherapy for advanced NSCLC1 – In phase III trials, CrEL paclitaxel + carboplatin showed efficacy: • Objective RR of 25%,2 overall RR of 32%,3 RR of 30%4 – CrEL use is associated with severe hypersensitivity reactions6 • All patients should be premedicated prior to CrEL paclitaxel administration to prevent severe hypersensitivity reactions5 • Albumin-bound paclitaxel is a solvent-free formulation of paclitaxel7 – Use of albumin as a vehicle eliminates solvent-related toxicities8 – No premedication to prevent hypersensitivity reactions is required prior to administration7 a Cremophor® EL is a registered trademark of BASF; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; RR, response rates. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Kelly K, et al. J Clin Oncol. 2001;19:3210-3218. 3. Scagliotti GV, et al. J Clin Oncol. 2002;20:4282-429. 4. Lilenbaum RC, et al. J Clin Oncol. 2005;23:190-196. 5. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 6. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 7. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 8. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 68 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Background (cont.) • Weekly CrEL paclitaxel 100 mg/m2 qw 3/4 + carboplatin AUC = 6 on day 1 achieved ORR of 32% in advanced NSCLC1 • Substituting AB-paclitaxel for CrEL paclitaxel at an identical dose achieved response rates of 50%2 • Based on the efficacy of AB-paclitaxel vs CrEL paclitaxel observed in these studies, a phase II trial was conducted3 – Showed that 100 mg/m2 weekly AB-paclitaxel + carboplatin AUC = 6 q3w is the optimal dose/schedule for treatment of advanced NSCLC3 • Based on these phase II results, a phase III regulatory pathway trial was designed4 – Investigated the efficacy and safety of AB-paclitaxel + carboplatin vs CrEL paclitaxel + carboplatin as first-line therapy in advanced NSCLC4 • Final survival outcomes are also presented here AB, albumin-bound: AUC, area under the curve; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; ORR, objective response rate; q3w, every 3 weeks; qw 3/4, weekly for 3 of 4 weeks. 1. Belani CP, et al. J Clin Oncol. 2003;21(15):2933-2939. 2. Allerton JP, et al. ASCO. 2006 [abstract 7127]. 3. Socinski MA, et al. J Thoracic Oncol. 2010;5(5):852-861. 4. Socinski MA, et al. ASCO. 2010 [abstract LBA7511]. 69 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Objective • Objective – Investigate efficacy and safety of AB-paclitaxel + carboplatin vs CrEL paclitaxel + carboplatin as first-line therapy for advanced NSCLC • Primary endpoint – ORR (CR + PR) by independent radiologic review based on RECIST • Secondary endpoints – PFS and OS – DCRa – Safety a DCR = CR + PR + SD ≥ 16 weeks. AB, albumin-bound ; CR, complete response; CrEL, Cremophor ® EL; DCR, disease control rate; NSCLC, non-small cell lung cancer; ORR, objective response rate; OS, overall survival; PFS, progression-free survival; PR, partial response; RECIST, Response Evaluation Criteria in Solid Tumors; SD, stable disease. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 70 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Key Eligibility Criteria • Key inclusion criteria – Adult patients with histologically/cytologically confirmed stage IIIB or IV NSCLC – Measurable disease by RECIST – No prior treatment for metastatic disease • Adjuvant therapy was allowed if it was > 1 year prior to study entry – ECOG PS of 0 or 1 – Adequate hematologic, hepatic, and renal function • Key exclusion criteria – Active brain metastases • Treated, controlled metastases were allowed – Baseline peripheral neuropathy ≥ grade 2 ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer; PS, performance status; RECIST, Response Evaluation Criteria in Solid Tumors. Socinski MA, et al. ASCO. 2010 [abstract LBA7511]. 71 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Study Design Chemotherapy-naïve ECOG PS 0-1 Stage IIIB/IV NSCLC (N = 1052) Randomized 1:1 mg/m2 AB-paclitaxel 100 days 1, 8, 15 Carboplatin AUC = 6 day 1 Cycles of 21 days No Premedication (n = 521) AB, albumin-bound; AUC, area under the curve; CrEL, Cremophor® EL; ECOG, Eastern Cooperative Oncology Group; NSCLC, nonsmall cell lung cancer; PS, performance status. CrEL paclitaxel 200 mg/m2 day 1 Carboplatin AUC = 6 day1 Cycles of 21 days Premedication with Dexamethasone + Antihistamines (n = 531) Socinski MA et al. ASCO. 2011 [Abstract 7551]. 72 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Baseline Patient Characteristics Baseline characteristic Age, years Median Range < 70 years, n (%) ≥ 70 years, n (%) Sex, n (%) Male Female Stage, n (%)a III IV Histology, n (%)a Adenocarcinoma Squamous cell carcinoma Large cell carcinoma Other ECOG PS, n (%) 0 1 Prior chemotherapy, n (%) Smoking status, n (%) Never smoked Smoked and quit Smoked and still smokes AB-paclitaxel + carboplatin (n = 521) CrEL paclitaxel + carboplatin (n = 531) 60 28 - 81 448 (86) 73 (14) 60 24 - 84 449 (85) 82 (15) 392 (75) 129 (25) 397 (75) 134 (25) 99 (19) 421 (81) 107 (20) 424 (80) 254 (49) 228 (44) 9 (2) 29 (6) 264 (50) 221 (42) 13 (2) 33 (6) 133 (26) 385 (74) 12 (2) 113 (21) 416 (78) 8 (2) 138 (27) 165 (32) 210 (41) 144 (28) 146 (28) 231 (44) AB, albumin-bound; CrEL, Cremophor EL ; ECOG PS, Eastern Cooperative Oncology Group performance status. a Data were missing for 1 patient at the time of this analysis. NSCLC, non-small cell lung cancer. Socinski MA, et al. ASCO. 2010 [abstract LBA7511]. 73 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Results: ORR, Stratified by Histologya a Not a pre-specified endpoint. AB, albumin-bound; CrEL Cremophor EL; NSCLC, non-small cell lung cancer; ORR, objective *response Not a pre-specified endpoint rate. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 74 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Primary Endpoint: Objective Responses -ITT AUC, area under the curve; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 75 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Results: Progression-Free Survival AB, albumin-bound; AUC, area under the curve; CI, confidence interval; CrEL, Cremophor EL; HR, hazard ratio; PFS, progression-free * Not a pre-specified endpoint survival. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 76 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Results: Progression-Free Survival AB, albumin-bound; Carbo, carboplatin; CI, confidence interval; CrEL, Cremophor EL; HR, *hazard Not a pre-specified endpoint ratio; PFS, progression-free survival. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 77 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Results: Overall Survival AB, albumin-bound; AUC, area under the curve; CI, confidence interval; CrEL, Cremophor EL; HR, ratio; OS, overall survival. *hazard Not a pre-specified endpoint Socinski MA et al. ASCO. 2011 [Abstract 7551]. 78 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Results: Overall Survival AB, albumin-bound; Carbo, carboplatin; CI, confidence interval; CrEL, Cremophor EL; HR, *hazard Not a pre-specified endpoint ratio; OS, overall survival. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 79 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Results: Safety Treatment-related grade 3/4 AEsa Hematologic, % Neutropenia Thrombocytopenia Anemia Febrile neutropenia Nonhematologic, % Fatigue Sensory neuropathy Anorexia Nausea Myalgia Time in days to improvement of sensory neuropathy to grade 1, median AB-paclitaxel + carboplatin (n = 514) CrEL paclitaxel + carboplatin (n = 524) P-value 42 18 24 1 48 7 6 1 .081 < .001 < .001 NS 5 3 2 <1 <1 6 12 <1 <1 2 NS < .001 NS NS .011 38 104 .238 AB, albumin-bound; AE, adverse event; CrEL, Cremophor EL; NS, not statistically significant;. a By National Cancer Institute Common Terminology Criteria for Adverse Events. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 80 Albumin-Bound Paclitaxel + Carboplatin vs Cremophor® EL Paclitaxel + Carboplatin in Advanced NSCLC Conclusions • The primary endpoint of a higher ORR albumin-bound paclitaxel plus carboplatin compared with Cremophor® EL palictaxel plus carboplatin was met - Overall: 33% vs. 25% (P = .005) • Within the squamous cell subset, the ORR of albumin-bound paclitaxel plus carboplatin was higher than Cremophor EL paclitaxel plus carboplatin: 41% vs. 24% (P < .001) • There was no significant difference in PFS or OS between the 2 arms - HRs trended in favor of the albumin-bound paclitaxel arm, particularly in the elderly and in patients with squamous cell histology • The albumin-bound paclitaxel arm exhibited higher rates of grade 3/4 anemia and thrombocytopenia and lower rates of grade 3/4 myalgia and sensory neuropathy HR, hazard ratio; NSCLC, non-small cell lung cancer; ORR, overall response rate; OS, overall survival; PFS, progression-free survival. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 81 Phase II Trial of Albumin-Bound Paclitaxel Plus Carboplatin in Patients With Advanced NSCLC at Risk of Bleeding From VEGF Directed Therapies G. A. Otterson, E. M. Bertino, N. A. Karim, K. Donthireddy, A. M. Ghany, R. Rupert, S. Cantrell, M. Rahmani, M. Lynn, S. P. Nana-Sinkam, G. S. Phillips, M. A. Villalona-Calero NSCLC, non-small cell lung cancer; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 82 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Background • A phase II trial of albumin-bound paclitaxel plus carboplatin and bevacizumab in patients with advanced NSCLC produced an ORR of 31% and a median OS of 16.8 months1 • Albumin-bound paclitaxel plus carboplatin produced a higher ORR vs Cremophor® EL paclitaxel plus carboplatin in a phase III trial of patients with advanced NSCLC2 – Difference particularly pronounced in patients with squamous histology (ORR = 41% vs 24%; P < .001) • Albumin-bound paclitaxel plus carboplatin may be a viable option for patients who can not receive bevacizumab due to characteristics such as squamous histology or risk of bleeding ® Cremophor is a registered trademark of BASF. NSCLC, non-small cell lung cancer; ORR, overall response rate; OS, overall survival; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 1. Reynolds C, et al. J Thorac Oncol. 2009;4(12):1537-1543. 2. Socinski MA et al. ASCO. 2011 [Abstract 7551]. 83 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Objective • Objective: to determine the efficacy of albumin-bound paclitaxel plus carboplatin in patients who are ineligible for treatment with bevacizumab • Primary endpoint: overall response rate by RECIST • Secondary endpoints – – – – Safety/toxicity OS PFS Exploratory • Tumor SPARC expression • Serum micro RNA expression profiles NSCLC, non-small cell lung cancer; OS, overall survival; PFS, progression-free survival; RECIST, Response Evaluation Criteria in Solid Tumors; SPARC, Secreted Protein Acidic and Rich in Cysteine; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 84 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Key Eligibility Criteria • Inclusion criteria – Advanced NSCLC • Stage IIIB with pleural effusion • Stage IV • Recurrent – Not eligible to receive bevacizumab because of • • • • Squamous histology Thrombotic or embolic events within 6 months History of controlled, non-life–threatening hemoptysis Cavitary lung lesions NSCLC, non-small cell lung cancer; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 85 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Key Eligibility Criteria (cont.) • Exclusion criteria – Prior treatment of advanced NSCLC – Pre-existing ≥ grade 2 neuropathy – Uncontrolled brain metastases – Major surgery within 4 weeks – Nonhealing wounds – Uncontrolled cardiac disease – HIV – Hepatitis B or C NSCLC, non-small cell lung cancer; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 86 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Study Design • Study design: phase II, single-arm, 2-stage Simon model - First stage: 27 patients Second stage: 36 patients Twenty-one day cycles Patients allowed to receive up to 6 cycles of therapy Albumin-bound paclitaxela 300 mg/m2 Day 1 a The dose of albumin-bound paclitaxel was reduced from 300 mg/m2 for the first 40 patients to 260 mg/m 2 for the subsequent patients due to excessive grade 3 neuropathy. AUC, area under the curve; NSCLC, non-small cell lung cancer; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. Carboplatin AUC = 6 Day 1 87 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Baseline Patient Characteristics Baseline characteristic N = 52 Age in years, mean 64 Race, n (%) White Black Other 42 (81) 9 (17) 1 (2) Tobacco pack years, mean Histology, n (%) Squamous Adenocarcinoma Adenosquamous Poorly differentiated NSCLC 49 37 (71) 8 (15) 2 (4) 5 (10) ECOG PS, n (%) 0 1 2 18 (35) 24 (46) 10 (19) ECOG PS, Eastern Cooperative Oncology Group performance status; NSCLC, non-small cell lung cancer. VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 88 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Results: Clinical Outcomes Clinical response, n (%) N = 52 Partial response 16 (31)a Stable disease 18 (35) Progressive disease 10 (19) Not evaluable 8 (15) a Thirteen (25%) of the 16 partial responses were confirmed responses. NSCLC, non-small cell lung cancer; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 89 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Results: Adverse Events Grade 3 or 4 adverse events in > 5% of patientsa (N = 52) Grade 3 Grade 4 Hematologic, n (%) Anemia Neutropenia Thrombocytopenia 2 (4) 4 (8) 5 (10) 1 (2) 8 (15) 1 (2) Nonhematologic, n (%) Sensory neuropathy Febrile neutropenia Infection in the absence of neutropenia Hyponatremia Hypoxia Dyspnea Dehydration Fatigue 13 (25) 7 (13) 5 (10) 7 (13) 1 (2) 7 (13) 6 (12) 10 (20) 0 2 (4) 5 (10) 0 2 (4) 2 (4) 3 (6) 0 a Adverse events per Common Terminology Criteria for Adverse Events, version 3.0. NSCLC, non-small cell lung cancer; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 90 Albumin-Bound Paclitaxel Plus Carboplatin in Patients With NSCLC at Risk of Bleeding From VEGF Directed Therapies Conclusions • This treatment regimen offers a reasonable first-line treatment option for patients with advanced squamous NSCLC who are not eligible to receive bevacizumab • Fifty-two patients have been enrolled in this ongoing study; 11 more patients are required to complete accrual • The clinical response data from the first stage of this trial (30% of the 27 patients exhibited partial responses) allowed for continuation to the second stage • Exploratory endpoints examining tumoral SPARC and serum micro RNA profiles are pending NSCLC, non-small cell lung cancer; SPARC, Secreted Protein Acidic and Rich in Cysteine; VEGF, vascular endothelial growth factor. Otterson GA, et al. IASLC 2011 [Abstract P3.183]. 91 Albumin-Bound Paclitaxel for the Treatment of Non-Small Cell Lung Cancer Combination Therapy with Carboplatin and Bevacizumab 92 Phase II Trial of Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in First-Line Patients With Advanced Nonsquamous Non-Small Cell Lung Cancer C. Reynolds, D. Barrera, R. Jotte, A. I. Spira, C. Weissman, K. A. Boehm, S. Pritchard, L. Asmar Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 93 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Background • Cremophor® EL paclitaxela + platinum agents are recommended as first-line chemotherapy for advanced NSCLC1 – In phase III trials, CrEL paclitaxel + carboplatin showed efficacy: • Objective RR of 25%,2 overall RR of 32%,3 RR of 30%4 – CrEL use is associated with severe hypersensitivity reactions6 • All patients should be premedicated prior to CrEL paclitaxel administration to prevent severe hypersensitivity reactions5 • Albumin-bound paclitaxel is a solvent-free formulation of paclitaxel7 – Use of albumin as a vehicle eliminates solvent-related toxicities8 – No premedication to prevent hypersensitivity reactions is required prior to administration7 a Cremophor® EL is a registered trademark of BASF; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; RR, response rates. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Kelly K, et al. J Clin Oncol. 2001;19:3210-3218. 3. Scagliotti GV, et al. J Clin Oncol. 2002;20:4282-429. 4. Lilenbaum RC, et al. J Clin Oncol. 2005;23:190-196. 5. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 6. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 7. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 8. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 94 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Background (cont.) • Bevacizumab is a VEGF-specific angiogenesis inhibitor1 – Indicated with CrEL paclitaxel + carboplatin as first-line treatment of patients with unresectable, locally advanced, recurrent, or metastatic nonsquamous NSCLC1 • In a phase III trial, AB-paclitaxel + carboplatin achieved a higher ORR than CrEL paclitaxel + carboplatin in advanced NSCLC2 – 33% vs 25%, P = .0052 • A phase II trial was designed to evaluate AB-paclitaxel + carboplatin + bevacizumab in advanced nonsquamous NSCLC3 AB-paclitaxel, albumin-bound paclitaxel; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; ORR, objective response rate; VEGF, vascular endothelial growth factor. 1. Avastin (bevacizumab) package insert. San Francisco, CA, Genentech, Inc., 2011. 2. Socinski MA, et al. ASCO. 2010 [abstract LBA7511]. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 95 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Objective • Objective – Evaluate the safety and antitumor activity of AB-paclitaxel + carboplatin + bevacizumab q21d in advanced stage IIIB/IV NSCLC • Primary endpoint – Response rate based upon RECIST criteria • Secondary endpoints – TTP – Duration of response – Safety – 1- and 2-year survival – Proportion of patients with SD ≥ 16 weeks – Changes in QOL using FACT-Taxane questionnaires AB, albumin-bound: FACT, Functional Assessment of Cancer Therapy; NSCLC, nonsmall cell lung cancer; q21d, every 21 days; QOL, quality of life; RECIST, Response Evaluation Criteria In Solid Tumors; SD, stable disease; TTP, time to disease progression. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 96 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Key Eligibility Criteria • Key inclusion criteria – Histologically or cytologically confirmed advanced stage IIIB (wet)/IV nonsquamous NSCLC – Evidence of inoperable local recurrence or metastasis – Measurable disease by RECIST – No prior chemotherapy – Prior RT permitted as long as the measurable disease • Was outside the field of radiation • Had progressed since completion of radiation – ECOG PS of 0-1 ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer; PS, performance status; RECIST, response evaluation criteria in solid tumors ; RT, radiation therapy. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 97 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Key Eligibility Criteria (cont.) • Key exclusion criteria – Another active malignancy – Pre-existing peripheral neuropathy of NCI grade > 1 • Exclusion criteria related to the administration of bevacizumab – Presence of CNS metastases – Gross hemoptysis – Unstable angina – Use of therapeutic anticoagulation CNS, central nervous system; NSCLC, non-small cell lung cancer; NCI, National Cancer Institute. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 98 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Study Design • Open-label, single arm, phase II study Albumin-bound paclitaxel 300 mg/m2 IV + Carboplatin AUC = 6 IV + Bevacizumab 15 mg/kg On day 1 of each 21 day cycle • Patients did not receive maintenance bevacizumab • Patients were to receive a minimum of 4 cycles of treatment AUC, area under the curve; IV, intravenous; NSCLC, non-small cell lung cancer . Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 99 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Baseline Patient Characteristics Baseline characteristic (N = 50) Age, years Median Range Sex, n (%) Male Female Histology, n (%) Adenocarcinoma Bronchioalveolar Large cell NOS ECOG PS, n (%) 0 1 Prior treatment, n (%) Surgery Radiation Number of metastatic sites per patient, n (%) 1 2 3 4 ECOG, Eastern Cooperative Oncology Group; NOS, not otherwise specified; PS, performance status. NSCLC, non-small cell lung cancer. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. Value 67 32 - 83 22 (48) 28 (56) 43 (86) 1 (2) 4 (8) 2 (4) 26 (52) 24 (48) 19 (30) 4 (8) 19 (38) 15 (30) 9 (18) 1 (2) 100 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Results: Response After Treatment Response (N = 48) Value ORR, % 31 Clinical benefit rate (CR + PR + [SD ≥ 6 months]), % 54 CR, n 0 PR, n (%) SD, n (%) ≥ 6 months, n < 6 months, n 15 (31) 26 (54) 11 15 PD, n (%) 2 (4) CR, complete response; ORR, overall response rate; PD, progressive disease; PR, partial response; SD, stable disease. • Median time to response: 1.3 months (range, 1.2 – 2.6) • Median duration of response: 8.9 months (range, 2.9 – 19.1) NSCLC, non-small cell lung cancer. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 101 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Results: Overall Survival NSCLC, non-small cell lung cancer; OS, overall survival. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 102 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Results: Progression-Free Survival NSCLC, non-small cell lung cancer; PFS, progression-free survival. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 103 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Results: Time to Progression NSCLC, non-small cell lung cancer; TTP, time to progression. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 104 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Results: Safety Treatment-related AEs occurring in > 1 patient (N = 48) Grade 3 Grade 4 Hematologic, n (%) Leukopenia Neutropenia Thrombocytopenia 2 (4) 8 (17) 4 (8) 0 18 (38) 1 (2) Nonhematologic, n (%) Anorexia Constipation Diarrhea Fatigue Febrile neutropenia Neuropathy Peripheral neuropathy 2 (4) 3 (6) 2 (4) 6 (13) 3 (6) 5 (10) 2 (4) 0 0 0 2 (4) 2 (4) 0 0 AE, adverse event. NSCLC, non-small cell lung cancer. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 105 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Results: Quality of Life • QOL was assessed using the FACT-Taxane scale1 • Over the course of the study, QOL decreased significantly with the exception of emotional well-being • Emotional well-being increased throughout cycles 1-5 and was significantly higher than baseline at cycle 3 (P = 0.05) FACT, Functional Assessment of Chronic Illness Therapy; NSCLC, non-small cell lung cancer; QOL, quality of life. 1. FACIT Functional Assessment of Chronic Illness Therapy, FACT-Taxane Questionnaire v4. Available at http://www.facit.org/FACITOrg/Questionnaires . Accessed May 20, 2011. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 106 First-Line Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced Nonsquamous NSCLC Conclusions • Efficacy conclusions – PFS and OS rates were higher than previously reported in patients with advanced NSCLC – PR occurred in 31% of patients – SD in 54% of patients • Safety conclusions – Toxicity was generally acceptable – Low incidence of grade 3 peripheral neuropathy – Absence of any obvious exacerbation of chemotherapy-induced myelosuppression by the addition of bevacizumab • Phase III evaluation of this combination would determine whether it is more efficacious than previous regimens NSCLC, non-small cell lung cancer; PFS, progression-free survival; PR, partial response; SD, stable disease; OS, overall survival. Reynolds C, et al. J Thoracic Oncol. 2009;4(12):1-7. 107 In Vivo Assessment of the Effects of Bevacizumab in Advanced Non-Small Cell Lung Cancer R. Suk Heist, D.G. Duda, D.V. Sahani, N.Pennell, J. Neal, M. Ancukiewicz, J. Engelman, T.J. Lynch, R.K. Jain Heist RS, et al. ASCO. 2010 [abstract 7612]. 108 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Background • Cremophor® EL paclitaxela + platinum agents are recommended as first-line chemotherapy for advanced NSCLC1 – In phase III trials, CrEL paclitaxel + carboplatin showed efficacy: • Objective RR of 25%,2 overall RR of 32%,3 RR of 30%4 – CrEL use is associated with severe hypersensitivity reactions6 • All patients should be premedicated prior to CrEL paclitaxel administration to prevent severe hypersensitivity reactions5 • Albumin-bound paclitaxel is a solvent-free formulation of paclitaxel7 – Use of albumin as a vehicle eliminates solvent-related toxicities8 – No premedication to prevent hypersensitivity reactions is required prior to administration7 a Cremophor® EL is a registered trademark of BASF; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; RR, response rates. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Kelly K, et al. J Clin Oncol. 2001;19:3210-3218. 3. Scagliotti GV, et al. J Clin Oncol. 2002;20:4282-429. 4. Lilenbaum RC, et al. J Clin Oncol. 2005;23:190-196. 5. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 6. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 7. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 8. Green MR, et al. Ann Oncol. 2006;17(8):1263-1268. 109 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Background (cont.) • Bevacizumab is a VEGF-specific angiogenesis inhibitor1 – Indicated with CrEL paclitaxel + carboplatin as first-line treatment of patients with locally advanced, recurrent, or metastatic nonsquamous NSCLC1 • Phase II trial evaluated the antitumor activity of AB-paclitaxel + carboplatin + bevacizumab2 – Demonstrated higher PFS and OS than previously reported in advanced nonsquamous NSCLC2 • Phase II trial was designed to identify biomarkers for bevacizumab in NSCLC patients treated with AB-paclitaxel + carboplatin + bevacizumab3 AB, albumin-bound; CrEL, Cremophor EL; NSCLC, non-small cell lung cancer; OS, overall survival; PFS, progression-free survival; VEGF, vascular endothelial growth factor. 1. Avastin (bevacizumab) package insert. San Francisco, CA, Genentech, Inc., 2011. 2. Reynolds C, et al. J Thorac Oncol. 2009;4:1-7. 3. Heist RS, et al. ASCO. 2010 [abstract 7612]. 110 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Objective • Objective – Identify biomarkers for bevacizumab in patients with NSCLC • Primary endpoint – 6-month PFS rate • Secondary endpoints – RR and OS – Safety • Exploratory correlative endpoints – Effect of bevacizumab on • Tumor perfusion • Serum levels of angiogenic cytokines and CECs – Relationship between tumor response and changes in • Tumor perfusion • Serum levels of angiogenic cytokines and CECs CECs, circulating endothelial cells; NSCLC, nonsmall cell lung cancer; OS, overall survival; PFS, progression-free survival; RR, response rate. Heist RS, et al. ASCO. 2010 [abstract 7612]. 111 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Study Design • Open-label, phase II study Day -14: Bevacizumab 15 mg/kg Day 1: Albumin-bound paclitaxel 100 mg/m2 + Carboplatin AUC = 6 + Bevacizumab 15 mg/kg Days 8 and 15: Albumin-bound paclitaxel 100 mg/m2 AUC, area under the curve; CECs, circulating endothelial cells; CT, computed tomography; FDG-PET, fluorodeoxyglucose positron emission tomography; NSCLC, non-small cell lung cancer. • Correlative studies included – Perfusion CT – FDG-PET – CECs – Angiogenic cytokines • Correlative studies performed – On day -14 – On day -2 (except FDG-PET) – After cycles 2 and 4 – At time of progression Heist RS, et al. ASCO. 2010 [abstract 7612]. 112 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Results: Preliminary Analysis of Correlative Studies Bev, bevacizumab; NSCLC, non-small cell lung cancer; RECIST, response evaluation criteria in solid tumors. Heist RS, et al. ASCO. 2010 [abstract 7612]. 113 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Results: Preliminary Analysis of Correlative Studies CT, computed tomography; FDG-PET, fluorodeoxyglucose positron emission tomography; NSCLC, non-small cell lung cancer; RECIST, response evaluation criteria in solid tumors; SUV, standardized uptake value. Heist RS, et al. ASCO. 2010 [abstract 7612]. 114 Albumin-Bound Paclitaxel + Carboplatin + Bevacizumab in Advanced NSCLC Conclusions • Pharmacodynamic blood and imaging biomarker studies in bevacizumab-treated NSCLC patients are feasible • Preliminary data suggest that such studies may predict tumor response • Enrollment and biomarker analyses are ongoing NSCLC, non-small cell lung cancer. Heist RS, et al. ASCO. 2010 [abstract 7612]. 115 Albumin-Bound Paclitaxel for the Treatment of Non-Small Cell Lung Cancer Combination Therapy with Carboplatin and Radiation 116 A Phase I Study of Albumin-Bound Paclitaxel With Carboplatin and Thoracic Radiation in Patients With Locally Advanced NSCLC V. L. Keedy, B. Lu, L. Horn, Y. Shyr, W. Conkright, D.P. Carbone, A. Sandler Keedy VL et al. ASCO. 2011 [Abstract 7046]. 117 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Background • In selected patients with unresectable stage III NSCLC, concurrent chemoradiation is superior to sequential therapy1,2 – NCCN-preferred1: cisplatin + etoposide3 and cisplatin + vinblastine4 – NCCN category 2B1,a: carboplatin + Cremophor® EL paclitaxel5,b • CrEL use is associated with severe hypersensitivity reactions7 – All patients should be premedicated prior to CrEL paclitaxel administration to prevent severe hypersensitivity reactions6 • Albumin-bound paclitaxel is a solvent-free formulation of paclitaxel8 – No premedication to prevent hypersensitivity reactions is required prior to administration8 • Phase I trial was designed to evaluate carboplatin + albumin-bound paclitaxel + thoracic radiation in unresectable stage III NSCLC9 a Recommendation is based on lower-level evidence and there is nonuniform NCCN consensus but no major disagreement. b Cremophor® is a registered trademark of BASF. CrEL, Cremophor EL; NCCN, National Comprehensive Cancer Network; NSCLC, nonsmall cell lung cancer. 1. NCCN Clinical Practice Guidelines in OncologyTM, Non-Small Cell Lung Cancer, v3.2011. 2. Auperin A, et al. J Clin Oncol. 2010;28:2181-2190. 3. Albain KS, et al. J Clin Oncol. 2002;20:3454-3460. 4. Curran WJ, et al. Proc Am Soc Clin Oncol. 2003;22:621[abstract 2499]. 5. Belani CP, et al. J Clin Oncol. 2005;23:5883-5891. 6. Taxol (paclitaxel) package insert. Princeton, NJ, Bristol-Myers Squibb Co, 2010. 7. Gelderblom H, et al. Eur J Cancer. 2001;37:1590-1598. 8. nab-paclitaxel (paclitaxel protein-bound particles) package insert. Bridgewater, NJ, Abraxis BioScience, LLC, a wholly owned subsidiary of Celgene Corporation, 2010. 9. Keedy VL, et al. J Clin Oncol. 2010;28(suppl):abstract e17504. 118 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Objective • Objective – Determine the MTD of weekly albumin-bound paclitaxel + carboplatin + concurrent thoracic radiation followed by consolidation therapy with albumin-bound paclitaxel plus carboplatin in patients with unresectable stage III NSCLC • Endpoints – Safety and tolerability NSCLC, non-small cell lung cancer. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 119 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Key Eligibility Criteria • Inclusion criteria – Nonmetastatic, inoperable Stage IIIA or IIIB NSCLC without pleural effusion – Measurable disease – Patients ≥ years of age – ECOG performance status = 0 or 1 – Adequate hepatic, renal, and bone marrow function – Force Expiratory volume in 1 second > 800 mL • Exclusion criteria – Known hypersensitivity to carboplatin or albumin-bound paclitaxel – Prior systemic chemotherapy, thoracic radiotherapy, or surgical resection – Peripheral neuropathy ≥ grade 2 – Concomitant malignancy – Pregnant or nursing women ECOG, Eastern Cooperative Oncology Group; NSCLC, non-small cell lung cancer. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 120 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Study Design • Dose-escalating 3 + 3 phase I trial of inoperable, stage III NSCLC patients receiving albumin-bound paclitaxel plus carboplatin plus XRT Consolidation period: 21-day cycle Χ 2 DLT period: 7 weeks Albumin-bound paclitaxel 40 – 60 mg/m2 qw Carboplatin AUC = 2 qw 21 days rest Albumin-bound paclitaxel 100 mg/m2 qw Restaging Carboplatin AUC = 6 Day 1 Concurrent radiation therapy 2 Gy qd Χ 33 days AUC, area under the curve; DLT, dose-limiting toxicity; MTD, maximum tolerated dose; NSCLC, non-small cell lung cancer; qd, daily; qw, weekly; XRT, radiation therapy. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 121 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Results: Baseline Patient Characteristics Baseline characteristic N = 11 Age in years, median (range) 67 (45 – 75) Smoker, n (%) 11 (100) Male, n (%) 9 (82) Race, n (%) White Black 10 (91) 1 (9) ECOG PS, n (%) 0 1 5 (45) 6 (55) Histology Adenocarcinoma Squamous Not otherwise specified 5 (45) 4 (36) 2 (18) Disease stage, n (%) IIIA IIIB 7 (64) 4 (36) ECOG PS, Eastern Cooperative Oncology Group performance status. NSCLC, non-small cell lung cancer. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 122 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Results: Antitumor Activity n = 9a Best response, n Partial response 9 Stable disease 0 a Of the 11 enrolled patients, 1 withdrew and 1 was not evaluable for response • Two dose-limiting toxicities occurred in the 60 mg/m2 arm - 40 mg/m2 determined to be the MTD of albumin-bound paclitaxel • Seven patients have progressed - At 3, 5, 6 (2 patients), 7, 8, and 20 months after enrollment Two patients remain progression-free at 10 and 34 months NSCLC, non-small cell lung cancer. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 123 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Results: Safety and Tolerability ≥ grade 2 AEs during concurrent chemoradiotherapy, n Albumin-bound paclitaxel dosea 40 mg/m2 (n = 6) 60 mg/m2 (n = 4) Grade 2 Grade 3 Grade 2 Grade 3 Esophagitis 2 0 1 1b Fatigue 1 0 3 0 Dehydration 2 0 1 0 Neutropenia 1 1 1 0 Hypoxia 1 1 0 0 Anemia 2 0 0 0 Febrile neutropenia 0 0 0 1 Thrombocytopenia 1 0 0 0 Chest pain 0 1 0 0 Nausea 1 0 0 0 Dermatitis 0 0 0 1b Dyspnea 1 0 0 0 AE, adverse event. a No grade 4 toxicities were observed. b Dose-limiting toxicity. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 124 Albumin-Bound Paclitaxel + Carboplatin + Thoracic Radiation in Unresectable Stage III NSCLC Conclusions • The recommended phase II dose of weekly albumin-bound paclitaxel + weekly carboplatin AUC = 6 + radiation therapy for patients with stage III NSCLC is 40 mg/m2 • A phase II study to evaluate this treatment is currently ongoing NSCLC, non-small cell lung cancer. Keedy VL et al. ASCO. 2011 [Abstract 7046]. 125 Albumin-Bound Paclitaxel for the Treatment of Small Cell Lung Cancer Combination Therapy with Carboplatin 126 A Randomized Phase II Study of Carboplatin and Albumin-Bound Paclitaxel With 2 Different Schedules in Patients With Extensive Stage Small Cell Lung Cancer J. E. Grilley-Olson, V. L. Keedy, A. Sandler, D. Moore, M. A. Socinski, T. E. Stinchcombe Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 127 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Background • The NCCN considers etoposide plus a platinum agent to be the standard treatment regimen for patients with SCLC1 • Clinicians often prescribe carboplatin over cisplatin in an effort to reduce the risk of emesis, neuropathy, and nephropathy1 • Etoposide treatment is associated with substantial toxicity, including myelosuppression, nausea, and vomiting2 • Albumin-bound paclitaxel given weekly or every 3 weeks in combination with carboplatin may lead to improved tolerability and acceptable efficacy in patients with ES-SCLC ES-SCLC, extensive stage small cell lung cancer; NCCN, National Comprehensive Cancer Network; SCLC, small cell lung cancer. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 1. NCCN Clinical Practice Guidelines in OncologyTM, Small Cell Lung Cancer, v2.2012. 2. Spira A, et al. N Engl J Med. 2004; 350(4):379-392. 128 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Objective • Objective: to evaluate the efficacy of 2 different dosing regimens of albumin-bound paclitaxel plus carboplatin in patients with ES-SCLC • Endpoints – Primary: ORR per RECIST – Secondary • • • • DoR PFS OS Toxicity profile per CTCAE version 3.0 CTCAE, Common Terminology Criteria for Adverse Events; DoR, duration of response; ESSCLC, extensive stage small cell lung cancer; ORR, overall response rate; OS, overall survival; PFS, progression-free survival; RECIST, Response Evaluation Criteria in Solid Tumors. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 129 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Key Eligibility Criteria • Inclusion criteria – Histologically or cytologically confirmed ES-SCLC – ECOG PS 0 - 2 – Adequate organ function – Patients ≥ 18 years of age – If brain metastases are present, they must be stable ECOG PS, Eastern Cooperative Oncology Group performance status; ES-SCLC, extensive stage small cell lung cancer. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 130 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Key Eligibility Criteria (cont.) • Exclusion criteria – Prior systemic chemotherapy, immunotherapy, or biologic therapy for ES-SCLC – Prior malignancies within 5 years – Serious concomitant illness – Grade ≥ 2 neuropathy – Previous anaphylactic reaction to carboplatin, paclitaxel, or docetaxel – Severe or uncontrolled cardiac disease ES-SCLC, extensive stage small cell lung cancer. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 131 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Study Design • Study design: randomized (1:1) phase II study of 2 different schedules of albumin-bound paclitaxel plus carboplatin in patients with ES-SCLC – Twenty-one day cycles – Response assessments were performed after cycles 2 and 4 Albumin-bound paclitaxela 240 - 300 mg/m2 IV Day 1 OR Albumin-bound paclitaxela 80 - 100 mg/m2 IV Days 1, 8, and 15 Carboplatin AUC = 6 IV Day 1 a Doses of albumin-bound paclitaxel were originally planned to be 300 mg/m 2 and 100 mg/m2 in the q3w and qw schedules, respectively, but excessive toxicity required reductions to 240 mg/m 2 and 80 mg/m2, respectively. AUC, area under the curve; ES-SCLC, extensive stage small cell lung cancer; IV, intravenous; q3w, every 3 weeks; qw, weekly. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 132 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Baseline Patient Characteristics Carbo + AB-Paclitaxel q3w (n = 14) Carbo + AB-Paclitaxel qw (n = 13) 60 (45 - 80) 67 (52 - 72) ECOG PS, n (%) 0 1 2 5 (36) 8 (57) 1 (7) 2 (15) 11 (85) 0 Male, n (%) 10 (71) 8 (62) White, n (%) 13 (93) 11 (85) Baseline Characteristic Age in years, median (range) AB, albumin-bound; carbo, carboplatin; ECOG PS, Eastern Cooperative Oncology Group performance status; q3w, every 3 weeks; qw, weekly. EGFR TKI—epidermal growth factor tyrosine kinase inhibitor ES-SCLC, extensive stage small cell lung cancer. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 133 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Results: Clinical Outcomes Carbo + AB-Paclitaxel q3w (n = 14) Carbo + AB-Paclitaxel qw (n = 13) 11 (79) 11 (85) PFS in months, median (95% CI) 5.8 (2.8 - 7.1) 5.2 (3.0 - 6.7) OS in months, median (95% CI) 8.6 (3.6 - 15.6) 11.6 (5.8 - 17.8) 36 (13 - 59) 42 (15 - 67) Clinical Outcome Partial response, n (%) 1-year survival, % (95% CI) AB, albumin-bound; carbo, carboplatin; CI, confidence interval; OS, overall survival; PFS, progression-free survival; q3w, every 3 weeks; qw, weekly. ES-SCLC, extensive stage small cell lung cancer. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 134 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Results: Adverse Events Carbo + AB-Paclitaxel q3w (n = 14) Carbo + AB-Paclitaxel qw (n = 13) Hematologic Neutropeniaa Anemia Thrombocytopenia 8 (57) 4 (29) 3 (21) 5 (38) 4 (31) 3 (23) Nonhematologic Sensory neuropathy Pain Fatigue Nausea Vomiting Diarrhea 3 (21) 4 (29) 0 0 0 1 (7) 0 0 1 (8) 3 (23) 2 (15) 1 (8) Grade 3 or 4 AEs, n (%) AB, albumin-bound; AE, adverse event; carbo, carboplatin; q3w, every 3 weeks; qw, weekly. a No cases of febrile neutropenia were reported in either arm. ES-SCLC, extensive stage small cell lung cancer. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 135 Carboplatin and Albumin-Bound Paclitaxel at 2 Different Schedules in Patients With ES-SCLC Conclusions • Response rates of 79% and 85% in the q3w and qw schedules of albumin-bound paclitaxel, respectively, demonstrate clinical activity of this combination therapy in patients with ES-SCLC • The majority of grade 3 or 4 adverse events were hematologic in both arms • Protocol dose reductions in both arms were necessary due to excessive toxicities at the originally specified doses ES-SCLC, extensive stage small cell lung cancer; q3w, every 3 weeks; qw, weekly. Grilley-Olson JE, et al. IASLC 2011 [Abstract P3.272]. 136 BACK UP NSCLC Clinical Data 137 nab-paclitaxel in Advanced NSCLC Trial CA031 Socinski1 Patients Advanced, 1st Line N=1052 Treatment ABX 100 mg/m2 qw + carboplatin paclitaxel + carboplatin ORR Median Survival Neuropathy Neutropenia Anemia Gr 3 Gr 4 Gr 3 Gr 4 Gr 3 Gr 4 33% OS: 12.1 mo 3% 0 33% 12% 22% 5% 25% OS: 11.2 mo 10% 0 33% 23% 6% 21% CA028 Socinski2 Advanced, 1st Line N=250 (dose-finding study) ABX 100 mg/m2 qw+ carboplatin 48% PFS: 6.2 OS: 11.3 8% 0 36% 28% 16% 0 CA015 Rizvi3 Advanced, 1st Line, N=40 ABX 125 mg/m2 qw 30% TTP: 5 mo OS: 11 mo 15% 0 15% 5% 8% 0 CA018 Green4 Advanced, metastatic 1st-line N=43 ABX 260 mg/m2 q3w 16% TTP: 6mo OS: 11 mo 5% 0 9% 0 0 0 ABX014 Allerton, Greco5 Advanced; 1st-line NSCLC N=50 ABX 100 mg/m2 qw + carboplatin 50% TTP: 28 wks 0 0 44% (Gr 3 or 4) 9% (Gr 3 or 4) ABX036 Reynolds6 Advanced nonsquamous; 1st-line N=48 ABX 300 mg/m2 q3w + carboplatin + BEV 31% PFS: 9.8 mo OS: 16.8 mo 10% 0 17% 37% NR NR ABX255 Otterson7 BEV-ineligible; advanced, 1st-line NSCLC N=52 (safety) N=27 (efficacy) ABX 260 mg/m2 q3w + carboplatin 30% NR 25% 0 8% 15% 4% 2% 1. Socinski IASCL 2011 2. Socinski JTO 2010. 3. Rizvi JCO 2008 4. Green Ann Onc 2006 5. Allerton ASCO 2006 6. Reynolds JTO 2009 7. Otterson IASLC 2011. 137 138 CA031 vs Historical Phase 3 Data with Taxol/Carboplatin Trial CA031 Socinski Patients Advanced, 1st Line N=1052 Treatment ABX 100 mg/m2 qw + carboplatin Taxol+ carboplatin Belani 2003 N=390 ORR Median Survival Anemia Gr 3 Gr 4 Gr 3 Gr 4 Gr 3 Gr 4 OS: 12.1 mo 3% 0 33% 12% 22% 5% 25% OS: 11.2 mo 10% 0 33% 23% 6% 21% TTP: 30 wks Taxol 100 mg/m2 qw + Carbo 32% Taxol 225 mg/m2 q3w + Carbo 17% 5% 22% OS: 49 wks 7% TTP: 3.1 mo N=1207 ECOG 4599 Sandler 2005 N=878 Taxol 200 mg/m2 q3w + Carbo 15% PFS: 4.5 mo OS: 10.3 mo N=1037 Taxol 225 mg/m2 q3w + Carbo 29% TTP: 5.0 mo OS: 9.9 mo N=926 Taxol 200 mg/m2 q3w + Carbo 24% PFS 5.4 mo OS: 10.6 mo ESCAPE Scagliotti 2010 Neutropenia 33% ECOG 1594 Schiller 2002 INTACT2 Herbst 2004 Neuropathy 10% 0 20% 43% 8% 2% 0 0S NR0 17% NR 1% OS: 8.1 mo 0.9% 0 5.9% 0 3% 0.6% 3% 1% 0% 138