AXICABTAGENE CILOLEUCEL (AXI‐CEL; KTE‐C19) IN PATIENTS WITH REFRACTORY AGGRESSIVE NON‐HODGKIN LYMPHOMAS (NHL): PRIMARY RESULTS OF THE PIVOTAL TRIAL ZUMA‐1 S.S. Neelapu* | F.L. Locke | N.L. Bartlett | L.J. Lekakis | D. Miklos | C.A. Jacobson | I. Braunschweig | O. Oluwole | T. Siddiqi | Y. Lin | J. Timmerman | P. Reagan | L. Navale | Y. Jiang | J. Aycock | M. Elias | J. Wiezorek | W.Y. Go Department of Lymphoma and Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, USA; Blood and Marrow Transplant and Cellular Immunotherapy, H. Lee Moffitt Cancer Center, Tampa, USA; Department of Medicine, Oncology Division, Medical Oncology Section, Washington University School of Medicine in St. Louis, St. Louis, USA; Hematology/Oncology, University of Miami Health System, Sylvester Comprehensive Cancer Center, Miami, USA; Stanford University School of Medicine, Blood and Marrow Transplantation, Stanford, USA; Hematologic Oncology, Dana‐Farber Cancer Institute, Boston, USA; Department of Oncology, Montefiore Medical Center, Bronx, USA; Department of Medicine, Vanderbilt University Medical Center, Nashville, USA; Department of Hematology & Hematopoietic Cell Transplantation, City of Hope, Duarte, USA; Department of Hematology, Mayo Clinic, Rochester, USA; Department of Medicine, University of California at Los Angeles, Santa Monica, USA; Department of Medicine, Hematology/Oncology School of Medicine and Dentistry, University of Rochester School of Medicine, Wilmot Cancer Center, Rochester, USA; N/A, Kite Pharma, Santa Monica, USA Introduction: Outcomes for pts with refractory aggressive NHL are poor with current therapies (Crump, ASCO 2016). Results from the interim analysis of (n = 62) of ZUMA‐1, the 1st multicenter trial of an anti‐CD19 chimeric antigen receptor (CAR) T cell, axi‐cel, in refractory aggressive NHL, showed an objective response rate (ORR) of 79% (complete response [CR] 52%; Blood2016;128:LBA‐6). Here we present results from the primary analysis of ZUMA‐1. Methods: Pts received a target dose of 2 × 10 anti‐CD19 CAR T cells/ kg after low‐ dose conditioning with cy/flu. Eligible pts (≥18 y) had diffuse large B cell lymphoma (DLBCL), primary mediastinal B cell lymphoma (PMBCL) or transformed follicular lymphoma (TFL); an ECOG performance status (PS) 0‐1; and refractory disease (progressive or stable disease as best response to last prior therapy, or relapsed ≤12 m of autologous stem cell transplant [ASCT]). The primary endpoint for this analysis was ORR in the combined DLBCL, PMBCL, and TFL population. Key secondary endpoints were duration of response (DOR), overall survival (OS), and frequency of adverse events (AEs). The primary analysis was triggered when 92 pts had at least 6 m of follow‐up. Results: As of January 27, 2017, 111 pts from 22 institutions were enrolled; 101 pts (91%) received axi‐cel. Median age was 58 y (range, 23‐76), 67% male, 85% stage III‐IV, 47% IPI 3‐4, 77% refractory to ≥2nd line of therapy, and 21% relapsed ≤12 m of ASCT. Axi‐cel was successfully manufactured in 110/111 (99%) pts with an average turnaround time from apheresis to the clinical site of 17 d. With an ORR of 82% (n = 92; P < .0001) the study met the primary endpoint. The ORR in the mITT analysis set of 101 pts was 82% (CR 54%, PR 28%), and was consistent across key covariates including disease subtype, refractory status, stage, and IPI score. At a median follow up of 8.7 m, 44% of pts were in response and 39% were in CR. The median DOR was 8.2 m overall and not reached for pts who achieved a CR. Median OS was not reached; 80% of pts remained alive at 6 m. The most common Gr ≥3 treatment‐emergent AEs were neutropenia (66%), leukopenia (44%), anemia (43%), febrile neutropenia (31%), and encephalopathy (21%). Gr ≥3 cytokine release syndrome (CRS) and neurologic events (NE) occurred in 13% and 28% of pts, respectively. All CRS and all NE resolved except 1 Gr 1 memory impairment. As previously reported, there were 3 Gr 5 AEs (3%). Peak and cumulative CAR T levels post–axi‐cel were associated with durable responses. Conclusions: Axi‐cel significantly improved ORR in pts with refractory aggressive NHL. The CR rate was 7‐fold higher compared to historical controls (Crump, ASCO 2016) and nearly half the patients have an ongoing response. Axi‐cel demonstrated significant clinical benefit with a manageable safety profile in pts lacking curative treatment options. *Drs Neelapu and Locke contributed equally to this study. Funding source: Kite Pharma and in part by funding from the Leukemia & Lymphoma Society (LLS) Therapy Acceleration Program®.