On November 3, 2025 Sumitomo Pharma America, Inc. (SMPA) reported three oral presentations and one poster presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition taking place in Orlando, Florida, from December 6-9, 2025. The presentations will include new clinical data supporting enzomenib, an investigational, oral small molecule menin inhibitor being researched as a monotherapy and in combination with venetoclax and azacitidine (VEN/AZA) for relapsed or refractory acute leukemia, and nuvisertib, an oral investigational highly selective small molecule PIM1 kinase inhibitor being evaluated as a monotherapy and in combination with momelotinib (MMB) for the treatment of relapsed or refractory myelofibrosis (MF).

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Updated clinical data from the ongoing Phase 1/2 study of enzomenib continue to show promising clinical activity across a wide range of potentially therapeutic doses in patients with relapsed or refractory acute leukemia with KMT2A-rearranged (KMT2Ar), NPM1-mutated (NPM1m), and other HOXA9/MEIS1-driven leukemia subtypes. Enzomenib is designed to target the menin and mixed-lineage leukemia (MLL) protein interaction, a key interaction for acute leukemia and cell growth in a variety of cancers. In this dose-escalation study of enzomenib monotherapy, enzomenib was escalated from 40 mg twice a day (BID) to 400 mg BID with no dose-limiting toxicities (DLTs) in 116 patients with sustained complete remission (CR) and complete remission with partial hematologic recovery (CRh) seen at doses of 200, 300 and 400 mg BID. Given the wide therapeutic window of enzomenib, these findings suggest that dosing may be tailored to the specific biology of different leukemia subtypes for potential optimal therapeutic effect.

Additionally, preliminary findings from a Phase 1 study of enzomenib in combination with VEN/AZA in patients with relapsed or refractory AML with KMT2Ar or NPM1m subtypes show enzomenib with VEN/AZA to be well-tolerated to up to 300 mg BID with no DLTs and no evidence of significant drug-drug interaction between enzomenib and VEN. Promising early clinical activity, particularly in patients without prior VEN or menin exposure, was also observed.

New clinical data from the Phase 1/2 study evaluating the safety and efficacy of nuvisertib in combination with MMB demonstrate early clinical activity and show that the treatment combination was well-tolerated supporting further development in patients with MF. Lastly, findings from the ongoing Phase 1/2 study of nuvisertib continue to support that nuvisertib monotherapy was well-tolerated with no DLTs and notable modulation of cytokine profiles demonstrating a strong correlation with clinical responses.

"Patients living with relapsed/refractory AML or MF desperately need effective therapies to overcome the poor prognoses typically associated with these cancers. The clinical data are highly compelling, especially for patients with particularly challenging forms of acute leukemia including those with KMT2A-rearranged and NPM1-mutated subtypes," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "Based on this progress, we look forward to sharing more comprehensive data further supporting the development of enzomenib and nuvisertib at the upcoming meeting in December and remain committed to advancing both of these programs."

Abstract Title

Detail

Lead Author

Nuvisertib, an oral investigational selective PIM1 kinase inhibitor, showed clinical responses strongly correlating with cytokine modulation in patients with relapsed/refractory myelofibrosis in the ongoing global phase I/II study

Poster Presentation

Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Poster I

Saturday, December 6.

5:30 – 7:30 p.m. EST

Presentation Time and Location: 5:30 p.m. EST

West Halls B3-B4 (Orange County Convention Center)

Lindsay A.M. Rein, M.D.

Preliminary data from the Phase I/II study of nuvisertib, an oral investigational selective PIM1 inhibitor, in combination with momelotinib showed clinical responses in patients with relapsed/refractory myelofibrosis

Oral Podium Presentation

Session: 634. Myeloproliferative Syndromes: Clinical and Epidemiological: Between a Rock and a Ropeg – Innovative Therapies for MPNs

Sunday, December 7.

9:30 – 11 a.m. EST

Presentation Time and Location: 9:45 a.m. EST

W414AB (Orange County Convention Center)

John Mascarenhas, M.D.

Monotherapy Update from Phase 1 Portion in Phase1/2 trial of the Menin-MLL Inhibitor Enzomenib (DSP-5336) in Patients with Relapsed or Refractory Acute Leukemia

Oral Podium Presentation

Session: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Menin inhibitors and FLT3 inhibitors in AML

Monday, December 8.

10:30 a.m. – noon EST

Presentation Time and Location: 10:30 a.m. EST

Chapin Theater (320) (Orange County Convention Center)

Naval G. Daver, M.D.

Preliminary data from the ongoing Phase 1 study of the menin-MLL inhibitor enzomenib

(DSP-5336) in combination with venetoclax and azacitidine in patients with relapsed or refractory Acute

Myeloid Leukemia

Oral Podium Presentation

Session: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: Menin inhibitors and FLT3 inhibitors in AML

Monday, December 8.

10:30 a.m. – noon EST

Presentation Time and Location: 11 a.m. EST

Chapin Theater (320) (Orange County Convention Center)

Justin M. Watts, M.D.

About Enzomenib
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.1,2 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations.1,3 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1, and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with MLL rearrangements and NPM1 mutation.4,5 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose escalation/dose expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in September 2024.

About Nuvisertib (TP-3654)
Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.6,7 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.7 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.6 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198). The FDA granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024. The FDA granted Fast Track Designation to nuvisertib for the indication of myelofibrosis in June 2025, and the European Medicines Agency granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in July 2025.

(Press release, Sumitomo Pharmaceuticals, NOV 3, 2025, View Source [SID1234659298])

On November 3, 2025 Pierre Fabre Pharmaceuticals Inc. (PFP) reported the transfer of the Biologics License Application (BLA) for tabelecleucel from Atara Biotherapeutics Inc. (Nasdaq: ATRA) with PFP now accountable for all aspects of the submission. Atara will continue to observe the regulatory process and provide support to PFP as needed. The tabelecleucel BLA has an FDA PDUFA target action date of January 10, 2026. If approved, the tabelecleucel will be indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with EBV+ PTLD who have received at least one prior therapy.

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Atara resubmitted the tabelecleucel BLA on July 11, 2025 of this year, in collaboration with PFP, and the FDA accepted the tabelecleucel BLA with Priority Review on July 23. With completion of the transfer of the BLA, Pierre Fabre Laboratories and its subsidiaries are now responsible for all clinical development regulatory, commercial and manufacturing activities for tabelecleucel worldwide. The innovative cell therapy is manufactured by PFP in the US for global clinical development and commercial access.

"Transfer of the BLA represents another critical milestone in our efforts to bring this innovative cell therapy to EBV+ PTLD patients in the US who have limited treatment options and lifespan measured in only a few weeks to months following failure of initial treatment," said Adriana Herrera, chief executive officer of PFP, the Pierre Fabre Laboratories Pharmaceutical subsidiary in the U.S. "We look forward to our continued engagement with the FDA in completing the review of tabelecleucel BLA as we seek to transform outcomes for this ultra-rare, acute, and potentially deadly blood malignancy that occurs after transplantation."

Tabelecleucel is an investigational, allogeneic, off the shelf, EBV-specific T-cell immunotherapy which targets and eliminates EBV-infected cells. The BLA includes data covering more than 430 patients treated with tabelecleucel including the ongoing pivotal ALLELE study investigating the therapy in adults and children two years of age and older with relapsed or refractory EBV+ PTLD following SOT or HCT.

About EBV+PTLD
EBV+ PTLD is an ultra-rare, acute, and potentially deadly hematologic malignancy that occurs after transplantation when patient T-cell immune responses are compromised by immunosuppression. It can impact patients who have undergone solid organ transplant (SOT) or allogeneic HCT. Poor median survival of 3 weeks and 4.1 months for HCT and SOT, respectively, is reported in EBV+ PTLD patients for whom standard of care failed, underscoring the significant need for new therapeutic options.

About Tabelecleucel
Tabelecleucel is an allogeneic, off-the-shelf, EBV-specific T-cell immunotherapy designed to selectively target and eliminate EBV-infected cells. Unlike autologous CAR-T therapies, allogeneic T-cells are derived from third-party donors and are not genetically modified. Immune cells are collected from the blood of healthy donors and exposed to Epstein-Barr virus antigens to enrich for T cells that recognize EBV. These EBV T cells are expanded, characterized, kept alive, and stored for future use in treating patients.

(Press release, Pierre Fabre, NOV 3, 2025, View Source;302601901.html [SID1234659297])

On November 3, 2025 Recordati Rare Diseases Inc. reported the presentation of new data related to its growing portfolio of treatments for rare hematologic disorders at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, which takes place December 6-9, 2025, in Orlando, Fla. In a series of nine poster presentations, Recordati researchers and independent investigators will present research detailing advances in Castleman disease (CD), cold agglutinin disease (CAD)/cold agglutinin syndrome (CAS), and immune-related complications of CAR T-cell therapy. Highlights include a report on the development of an artificial intelligence (AI) model that evaluates CD tissue samples; an analysis of the morbidity burden and healthcare costs among patients with idiopathic multicentric Castleman disease (iMCD); the first real-world evaluation of sutimlimab in the treatment of patients with CAD or CAS; and a prospective evaluation of siltuximab in the prevention or treatment of cytokine release syndrome (CRS) after CAR T-cell therapy.

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"We are particularly excited about the long-term safety data for sutimlimab in cold agglutinin disease and the growing body of research investigating the use of siltuximab in Castleman disease," said Milan Zdravkovic, Executive Vice President, Research & Development and Chief Medical Officer at Recordati. "Also notable is the poster describing an innovative, AI-based approach to lymph node analysis, which could represent a meaningful step toward earlier and more consistent diagnosis of Castleman disease, one of the most difficult hematologic conditions to diagnose accurately. Altogether, the datasets presented at ASH (Free ASH Whitepaper) 2025 reflect Recordati’s ongoing commitment to advancing care for people living with rare hematologic disorders."

"The ASH (Free ASH Whitepaper) Annual Meeting is an important opportunity to showcase research advances in hematologic diseases, and we are pleased that the meeting organizers have accepted nine abstracts related to our therapies," said Mohamed Ladha, President and General Manager at Recordati Rare Diseases North America. "Together with our independent research partners, Recordati is at the forefront of advancing potential therapeutic solutions for people living with devastating conditions such as Castleman disease. We look forward to these data sparking further dialogue and collaboration as we continue in our efforts to improve patients’ lives."

The ASH (Free ASH Whitepaper) 2025 Annual Meeting will feature the following poster presentations:

Castleman Disease

Poster number: 2606
Title: Retrospective real-world data analysis of morbidity burden and healthcare costs in idiopathic multicentric Castleman disease compared with matched controls (BURDEN-iMCD)
Presenting author: Sudipto Mukherjee, MD, PhD, MPH, Cleveland Clinic

Poster number: 3001
Title: Comprehensive analysis of subtype-specific outcomes and management in Castleman disease: a 20-year cohort study
Presenting author: Yoshito Nishimura, MD, PhD, MPH, Mayo Clinic

Poster number: 3002
Title: Automated grading of Castleman disease histopathology using an attention-based multiple-instance learning model
Presenting author: Muir Morrison, PhD, University of Utah

Poster number: 3009
Title: Pediatric idiopathic multicentric Castleman disease is often severe and responds to siltuximab
Presenting author: Bridget Austin, MS, Perelman School of Medicine, University of Pennsylvania, Center for Cytokine Storm Treatment & Laboratory

Poster number: 3006
Title: Epidemiology and clinical characteristics of idiopathic multicentric Castleman disease in Spain (ARCANA study): Prevalence cohort
Presenting author: José-Tomás Navarro, MD, PhD, Catalan Institute of Oncology, Josep Carreras Leukaemia Research Institute

Poster number: 4788
Title: Siltuximab-mediated suppression of CRP is associated with clinical response in idiopathic multicentric Castleman disease
Presenting author: Jean-Francois Rossi, MD, PhD, Université de Montpellier

Cold Agglutinin Disease (CAD)

Poster number: 6242
Title: Real-world safety of sutimlimab in patients with CAD/CAS: a multinational, multicenter, observational, prospective cohort study
Presenting author: Alexander Röth, MD, West German Cancer Center, University Hospital Essen, University of Duisburg-Essen

CAR T-Cell Therapy Toxicity (including in B-Cell Lymphoma)*

Poster number: 2385
Title: Safety and immunomodulatory effects of siltuximab prophylaxis prior to standard of care CD19 directed chimeric antigen receptor T-cell (CD19.CART) therapy for B-cell lymphomas: final Phase I trial results
Presenting author: Nathan Denlinger, DO, MS, The Ohio State University Wexner Medical Center, James Comprehensive Cancer Center

Poster number: 5919
Title: Siltuximab versus tocilizumab for the management of CAR T-cell associated cytokine release syndrome
Presenting author: Mayur Narkhede, MD, University of Alabama at Birmingham

*These studies were conducted as investigator-sponsored studies without research involvement by Recordati.

About Idiopathic Multicentric Castleman Disease (iMCD)
Idiopathic multicentric Castleman disease is a rare cytokine-driven disorder that may be life-threatening and can affect people of any age. Its symptoms often resemble those of malignant lymphoma, autoimmune, or infectious diseases, making it difficult to diagnose. iMCD is a subtype of Castleman disease (CD), which is a group of rare conditions that affect the immune system, characterized by swollen lymph nodes and a broad range of inflammatory signs and symptoms. The cause of iMCD is unknown, and there are no known risk factors. Some people with iMCD have elevated levels of interleukin 6 (IL-6), a cytokine that is produced in the body during inflammation and which plays a central pathological role in iMCD; IL-6 elevation may explain some of the symptoms patients experience, such as swollen lymph nodes, fever, unexplained weight loss, and night sweats.

About Cold Agglutinin Disease (CAD)
Cold agglutinin disease (CAD) is a rare type of autoimmune hemolytic anemia (AIHA), characterized as a low-grade lymphoproliferative disorder of the bone marrow. In CAD, autoantibodies bind to erythrocytes at temperatures ≤37°C, leading to complement-mediated hemolysis. CAD symptoms include severe, debilitating fatigue and other clinical manifestations (e.g., dyspnea, tachycardia) that can impact patients’ quality of life. While the median age of onset is approximately 60 years, CAD has been diagnosed in patients as young as 30.

(Press release, Recordati, NOV 3, 2025, View Source [SID1234659296])

On November 3, 2025 AAVivo, Inc., a pioneering biotechnology company developing novel, precision targeted biotherapeutics to enable in vivo generation of CAR-T cells, reported a presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2025, which is being held November 5-9, in National Harbor, MD.

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AAVivo will present a poster titled, "In Vivo Generation of CD19-Specific CAR-T Cells Using a Novel AAV Gene Therapy Platform to Treat B Cell Malignancies". The poster describes the Company’s lead candidate, AVO-100, a gene therapy that is designed using adeno-associated viruses (AAV) with capsids containing T cell-targeting (TCeT) moieties to produce CD19-specific CAR-T cells in patients. Preclinical data to date suggest that AVO-100 can be used to generate CD19-specific T cells from non-activated T cells in human PBMCs both in vitro and in vivo and the CAR-T cells can control the growth of Raji lymphoma in vitro and in NSG mice.

"We are pleased that a poster highlighting the potential of our iAAV platform to rapidly enable in vivo generation of CAR-T cells was accepted for presentation at SITC (Free SITC Whitepaper) 2025," said Haifeng Chen, Ph.D. Chief Technology Officer & Founder of AAVivo. "Having this research accepted for presentation at SITC (Free SITC Whitepaper), one of the preeminent oncology conferences, showcases the breadth of AAVivo’s patented technologies and the potential of AVO-100, our lead program targeting B-cell malignancies."

Details of the poster being presented at SITC (Free SITC Whitepaper) 2025 are as follows:

Abstract Number: 1001
Abstract Title: In Vivo Generation of CD19-Specific CAR-T Cells Using a Novel AAV Gene Therapy Platform to Treat B Cell Malignancies
Presenting Author: Haifeng Chen, Ph.D. Chief Technology Officer & Founder, AAVivo, Inc.
Session Date: Friday, Nov. 7

(Press release, AAVivo, NOV 3, 2025, View Source [SID1234659295])

On November 3, 2025 Gilead Sciences, Inc. (Nasdaq: GILD) and Kite, a Gilead Company, reported that it will present 21 abstracts, including 5 oral presentations, during the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition (December 6-9). These data showcase Kite’s continued progress in transforming blood cancer care and expanding the reach and impact of CAR T-cell therapy.

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"Kite is dedicated to advancing cell therapy as a path to cures, and our data at ASH (Free ASH Whitepaper) will reflect meaningful progress toward this goal," said Cindy Perettie, Executive Vice President of Kite. "Together with our partner Arcellx, we’ll unveil the updated results from the pivotal Phase 2 iMMagine-1 study. These findings will lay the foundation for our aspiration with anito-cel to deliver a differentiated treatment for relapsed/refractory multiple myeloma with strong potential for community oncology access and reduced burden on patients and caregivers."

Anito-cel Data Updates

Key presentations for anitocabtagene autoleucel (anito-cel) include updated results from the fully enrolled, ongoing iMMagine-1 Phase 2 pivotal study. No delayed neurotoxicities, including no Parkinsonism, no cranial nerve palsies, no Guillain-Barré syndrome, and no immune-mediated enterocolitis, have been observed to date.

Data on Next-Generation Pipeline

Kite will also share new data on its next-generation bicistronic autologous CAR T-cell therapies, KITE-363 and KITE-753. These therapies are designed to target two antigens (CD19 and CD20) found on cancer cells and use two co-stimulatory domains (CD28 and 4-1 BB) to help the immune system fight cancer more effectively. This dual-targeting approach may lower the chance of the cancer escaping treatment and could also improve safety, making it possible to treat patients outside of a hospital setting.

Survival Outcomes with Yescarta Based on ASCT Eligibility

A key presentation for Yescarta (axicabtagene ciloleucel) includes a joint analysis of 4-year follow-up data from ZUMA-7, which evaluated Yescarta as a second-line therapy in patients with relapsed/refractory (R/R) large B-cell lymphoma (LBCL) eligible for autologous stem-cell transplant (ASCT), alongside 2-year follow-up data from ALYCANTE that evaluated patients who were ASCT ineligible. Efficacy, safety, and health-related quality of life patterns were observed to be consistent across both ZUMA-7 and ALYCANTE populations, supporting the use of Yescarta regardless of transplant eligibility and effectively broadening eligibility to this potentially curative, one-time treatment.

Dates and times are listed in ET for the following accepted abstracts and presentations:

Oral Presentations

Abstract Details

Titles

Axicabtagene Ciloleucel Clinical Data

Abstract #671

Sunday, December 7, 2025

5:30 PM – 5:45 PM

OCCC – Tangerine Ballroom F3-4

Double-dose Axicabtagene Ciloleucel (Axi-Cel-2) for Second-Line High-Risk Large B-Cell Lymphoma (LBCL): Interim Results from a Phase 1b Study​

Brexucabtagene Autoleucel Clinical Data

Abstract #569

Sunday, December 7, 2025

1:00 PM – 1:15 PM

OCCC – Tangerine Ballroom F3-4

ZUMA-25 Preliminary Analysis: A Phase 2 Study of Brexucabtagene Autoleucel (Brexu-cel) in Patients (Pts) with Relapsed/Refractory (R/R) Burkitt Lymphoma (BL), Substudy C

Anitocabtagene Autoleucel Clinical Data

Abstract #256

Saturday, December 6, 2025

2:45 PM – 3:00 PM

OCCC – West Halls D1

Phase 2 Registrational Study of Anitocabtagene Autoleucel for the Treatment of Patients with Relapsed and/or Refractory Multiple Myeloma: Updated Results from iMMagine-1

Kite Next Generation CAR T Clinical Data

Abstract #265

Saturday, December 6, 2025

2:00 PM – 2:15 PM

OCCC – West Halls D2

A Phase 1 Study of KITE-753 or KITE-363 in Patients with Relapsed/Refractory B-Cell Lymphoma: Initial Safety and Preliminary Efficacy of KITE-753 and Updated Results of KITE-363

Translational Medicine

Abstract #805

Monday, December 8, 2025

10:30 AM – 10:45 AM

OCCC – W331

Blood and CSF Metabolomics Identifies Tryptophan Catabolism and Polyamine Synthesis as Drivers of CAR T-Cell-Associated Neurotoxicity

Poster Presentations

Axicabtagene Ciloleucel

Abstract #1799

Saturday, December 6, 2025

5:30 PM – 7:30 PM

OCCC – West Halls B3-B4

Patient Journey with Axicabtagene Ciloleucel for Relapsed or Refractory Large B Cell Lymphoma in Canada: Manufacturing experience and Impact of Patient Location to Treatment Centre​

Abstract #3714

Sunday, December 7, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Axi-Cel Delivers Similar Outcomes Regardless of ASCT-Eligibility in Second Line R/R LBCL: Combined Data from ZUMA-7 and ALYCANTE

Abstract #1894

Saturday, December 6, 2025

5:30 PM – 7:30 PM

OCCC – West Halls B3-B4

Diffuse Large B Cell Lymphoma in Brazil: Understanding the Patient Journey to Improve Healthcare Assistance​

Abstract #4510

Sunday, December 7, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Estimating the Survival Impact of Not Receiving CAR T-cell (CAR T) Therapy When Eligible in Patients with Relapsed or Refractory (R/R) Diffuse Large B-cell Lymphoma (DLBCL) in the United States (US)​

Abstract #6194

Monday, December 8, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

US Cost Consequence and Time Toxicity Model for Advanced Therapies in the Treatment for Relapsed/Refractory Third-line or Later Diffuse Large B-cell Lymphoma: A Comparison of Axicabtagene Ciloleucel with Bispecific Antibodies

Abstract #5356

Monday, December 8, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Real-World Effectiveness and Safety Outcomes by Age, Comorbidity, Frailty, and Treatments Prior to Infusion in Relapsed or Refractory (R/R) Follicular Lymphoma Patients Treated with Axicabtagene Ciloleucel​

Abstract #3717

Sunday, December 7, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Surrogate Endpoints as Prognostic Factors for Long-Term Outcomes Among Patients Receiving Axicabtagene Ciloleucel in Frontline High-risk Large B Cell Lymphoma​

Abstract #4503

Sunday, December 7, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Real-World Treatment Patterns and Survival Outcomes in Second and Third Line Settings in Large B-cell Lymphoma (LBCL)​

Brexucabtagene Autoleucel

Abstract #1588

Saturday, December 6, 2025

5:30 PM – 7:30 PM

OCCC – West Halls B3-B4

Endogenous Regulation of Inflammatory Response as a Determinant of Durable Remission Without Stem Cell Transplant Following Brexucabtagene Autoleucel (Brexu-Cel) Therapy in ALL

Abstract #1799

Saturday, December 6, 2025

5:30 PM – 7:30 PM

OCCC – West Halls B3-B4

Two-year Update of ZUMA-2 Cohort 3: Brexucabtagene Autoleucel (Brexu-cel) in Patients (Pts) with Relapsed/Refractory Mantle Cell Lymphoma (R/R MCL) Who Had Not Received Prior Bruton Tyrosine Kinase Inhibitor (BTKi) Therapy​

Abstract #3606

Sunday, December 7, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Real-world Effectiveness and Safety Outcomes by Age, Comorbidities, and Frailty Among Relapsed or Refractory (R/R) Mantle Cell Lymphoma (MCL) Patients Treated with Brexucabtagene Autoleucel (Brexu-Cel)​

Above Brand

Abstract #5882

Monday, December 8, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Pre-Treatment CD19 Antigen Density and Multi-Antigen Profiling by Calibrated Quantitative Flow Cytometry Correlates with CAR T Efficacy in LBCL​

Multiple Myeloma Unmet Clinical Need

Abstract #6344

Monday, December 8, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Visualizing Geographic Variation and Systemic Inequities of Disease Burden and CAR T-Cell Therapy Access in Multiple Myeloma in the US​

Abstract #6284

Monday, December 8, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Outcomes of Inpatient and Outpatient Chimeric Antigen Receptor T-cell Therapy (CAR T) in Newly Authorized Treatment Centers (ATCs) in the United States (US)​

Abstract #4411

Sunday, December 7, 2025

6:00 PM – 8:00 PM

OCCC – West Halls B3-B4

Real-world Healthcare Resource Utilization (HCRU) Following CAR T-Cell Therapy in US Patients Treated in Newly Authorized Treatment Centers​

Publication Only: Anitocabtagene Autoleucel Pre-Clinical Data

Abstract #7644

The Fast Off-Rate of Anito-cel’s D-Domain Binder Contributes to Its Distinctive Pharmacology Profile in Preclinical Models of Multiple Myeloma​

Investigator-Sponsored / Collaboration: Anitocabtagene Autoleucel Pre-Clinical Data*

Oral

Abstract #203

Sunday, December 7, 2025

10:30 AM – 10:45 AM

OCCC – Sunburst Room (W340)

Single-cell Transcriptomics Reveal Mechanisms of Efficacy and Toxicity in Anti-BCMA CAR-T Cell Therapies for Multiple Myeloma

For more information, including a complete list of abstract titles at the meeting, please visit: View Source

*Presentations independently led and sponsored feature Kite CAR T-cell therapies but are not included in total number of Kite accepted abstracts.

About Yescarta

INDICATIONS

YESCARTA is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy.
Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: YESCARTA is not indicated for the treatment of patients with primary central nervous system lymphoma.

Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
U.S. IMPORTANT SAFETY INFORMATION

WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, and SECONDARY HEMATOLOGICAL MALIGNANCIES

Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving YESCARTA. Do not administer YESCARTA to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab or tocilizumab and corticosteroids.
Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving YESCARTA, including concurrently with CRS or after CRS resolution. Monitor for neurologic toxicities after treatment with YESCARTA. Provide supportive care and/or corticosteroids as needed.
T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including YESCARTA.
CYTOKINE RELEASE SYNDROME (CRS)

CRS, including fatal or life-threatening reactions, occurred following treatment with YESCARTA. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including ≥ Grade 3 CRS in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including ≥ Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days).

CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including ≥ Grade 3 CRS in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS (≥ 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include, cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing ≥ Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Confirm that 2 doses of tocilizumab are available prior to infusion of YESCARTA. Monitor patients at least daily for 7 days following infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for 2 weeks after infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

NEUROLOGIC TOXICITIES

CRS, including fatal or life-threatening reactions, occurred following treatment with YESCARTA. CRS occurred in 90% (379/422) of patients with non-Hodgkin lymphoma (NHL), including ≥ Grade 3 CRS in 9%. CRS occurred in 93% (256/276) of patients with large B-cell lymphoma (LBCL), including ≥ Grade 3 in 9%. Among patients with LBCL who died after receiving YESCARTA, 4 had ongoing CRS events at the time of death. For patients with LBCL in ZUMA-1, the median time to onset of CRS was 2 days following infusion (range: 1-12 days) and the median duration was 7 days (range: 2-58 days). For patients with LBCL in ZUMA-7, the median time to onset of CRS was 3 days following infusion (range: 1-10 days) and the median duration was 7 days (range: 2-43 days).

CRS occurred in 84% (123/146) of patients with indolent non-Hodgkin lymphoma (iNHL) in ZUMA-5, including ≥ Grade 3 CRS in 8%. Among patients with iNHL who died after receiving YESCARTA, 1 patient had an ongoing CRS event at the time of death. The median time to onset of CRS was 4 days (range: 1-20 days) and median duration was 6 days (range: 1-27 days) for patients with iNHL.

Key manifestations of CRS (≥ 10%) in all patients combined included fever (85%), hypotension (40%), tachycardia (32%), chills (22%), hypoxia (20%), headache (15%), and fatigue (12%). Serious events that may be associated with CRS include, cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), renal insufficiency, cardiac failure, respiratory failure, cardiac arrest, capillary leak syndrome, multi-organ failure, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

The impact of tocilizumab and/or corticosteroids on the incidence and severity of CRS was assessed in 2 subsequent cohorts of LBCL patients in ZUMA-1. Among patients who received tocilizumab and/or corticosteroids for ongoing Grade 1 events, CRS occurred in 93% (38/41), including 2% (1/41) with Grade 3 CRS; no patients experienced a Grade 4 or 5 event. The median time to onset of CRS was 2 days (range: 1-8 days) and the median duration of CRS was 7 days (range: 2-16 days). Prophylactic treatment with corticosteroids was administered to a cohort of 39 patients for 3 days beginning on the day of infusion of YESCARTA. Thirty-one of the 39 patients (79%) developed CRS and were managed with tocilizumab and/or therapeutic doses of corticosteroids with no patients developing ≥ Grade 3 CRS. The median time to onset of CRS was 5 days (range: 1-15 days) and the median duration of CRS was 4 days (range: 1-10 days). Although there is no known mechanistic explanation, consider the risk and benefits of prophylactic corticosteroids in the context of pre-existing comorbidities for the individual patient and the potential for the risk of Grade 4 and prolonged neurologic toxicities.

Confirm that 2 doses of tocilizumab are available prior to infusion of YESCARTA. Monitor patients at least daily for 7 days following infusion for signs and symptoms of CRS. Monitor patients for signs or symptoms of CRS for 2 weeks after infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS occur at any time. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated.

HYPERSENSITIVITY REACTIONS

Allergic reactions may occur with the infusion of YESCARTA. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO) or residual gentamicin in YESCARTA.

SERIOUS INFECTIONS

Severe or life-threatening infections occurred after YESCARTA infusion. Infections (all grades) occurred in 45% of patients with NHL. Grade 3 or higher infections occurred in 17% of patients, including ≥ Grade 3 infections with an unspecified pathogen in 12%, bacterial infections in 5%, viral infections in 3%, and fungal infections in 1%. YESCARTA should not be administered to patients with clinically significant active systemic infections. Monitor patients for signs and symptoms of infection before and after infusion and treat appropriately. Administer prophylactic antimicrobials according to local guidelines.

Febrile neutropenia was observed in 36% of patients with NHL and may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

In immunosuppressed patients, including those who have received YESCARTA, life-threatening and fatal opportunistic infections including disseminated fungal infections (e.g., candida sepsis and aspergillus infections) and viral reactivation (e.g., human herpes virus-6 [HHV-6] encephalitis and JC virus progressive multifocal leukoencephalopathy [PML]) have been reported. The possibility of HHV-6 encephalitis and PML should be considered in immunosuppressed patients with neurologic events and appropriate diagnostic evaluations should be performed.

Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, has occurred in patients treated with drugs directed against B cells, including YESCARTA. Perform screening for HBV, HCV, and HIV and management in accordance with clinical guidelines before collection of cells for manufacturing.

PROLONGED CYTOPENIAS

Patients may exhibit cytopenias for several weeks following lymphodepleting chemotherapy and YESCARTA infusion. Grade 3 or higher cytopenias not resolved by Day 30 following YESCARTA infusion occurred in 39% of all patients with NHL and included neutropenia (33%), thrombocytopenia (13%), and anemia (8%). Monitor blood counts after infusion.

HYPOGAMMAGLOBULINEMIA

B-cell aplasia and hypogammaglobulinemia can occur in patients receiving YESCARTA. Hypogammaglobulinemia was reported as an adverse reaction in 14% of all patients with NHL. Monitor immunoglobulin levels after treatment and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement.

The safety of immunization with live viral vaccines during or following YESCARTA treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during YESCARTA treatment, and until immune recovery following treatment.

SECONDARY MALIGNANCIES

Patients treated with YESCARTA may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including YESCARTA. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes.

Monitor life-long for secondary malignancies. In the event that a secondary malignancy occurs, contact Kite at 1-844-454-KITE (5483) to obtain instructions on patient samples to collect for testing.

ADVERSE REACTIONS

The most common adverse reactions (incidence ≥ 20%) in:

patients with LBCL in ZUMA-7 included fever, CRS, fatigue, hypotension, encephalopathy, tachycardia, diarrhea, headache, musculoskeletal pain, nausea, febrile neutropenia, chills, cough, infection with unspecified pathogen, dizziness, tremor, decreased appetite, edema, hypoxia, abdominal pain, aphasia, constipation, and vomiting.
patients with LBCL in ZUMA-1 included CRS, fever, hypotension, encephalopathy, tachycardia, fatigue, headache, decreased appetite, chills, diarrhea, febrile neutropenia, infections with pathogen unspecified, nausea, hypoxia, tremor, cough, vomiting, dizziness, constipation, and cardiac arrhythmias.
patients with iNHL in ZUMA-5 included fever, CRS, hypotension, encephalopathy, fatigue, headache, infections with pathogen unspecified, tachycardia, febrile neutropenia, musculoskeletal pain, nausea, tremor, chills, diarrhea, constipation, decreased appetite, cough, vomiting, hypoxia, arrhythmia, and dizziness.
Please see full Prescribing Information, including BOXED WARNING and Medication Guide.

(Press release, Gilead Sciences, NOV 3, 2025, View Source [SID1234659293])