Abecma (idecabtagene vicleucel) / BMS - LARVOL DELTA

Successful BCMA-CAR T-cell salvage therapy in a patient with idiopathic inflammatory myositis relapsing after CD19-CAR T-cell therapy (EULAR 2025) - "The patient experienced a CMV-viremia under daratumumab, which was successfully treated with oral valganciclovir. Due to ongoing disease activity, plasma cell-targeting BCMA-CAR T-cell therapy was performed (Idecabtagene vicleucel, Bristol Myers Squibb). The patient experienced grade 1 cytokine release syndrome (CRS) on day 3, which resolved upon a single dose of the anti-interleukin-6 receptor monoclonal antibody tocilizumab. No neuro- or hematotoxicity were observed and, supported by a 100-day prophylaxis with letermovir, no CMV-reactivation occurred. BCMA-CAR T-cells expanded, cleared B cells in circulation and plasma cells in lymphoid tissue, reduced autoantibody levels, and re-induced stable drug-free remission with normalization of CK within three months. Learning points for clinical practice: These data demonstrate that (i) a switch of CAR T-cell target can restore drug-free remission after relapse of AID after the first CAR T-cell treatment, (ii) repeated..."

CAR T-Cell Therapy • Clinical • Immunology • Interstitial Lung Disease • Myositis • Pulmonary Disease • Rare Diseases • Respiratory Diseases • Rheumatology • Transplant Rejection • IL6R

Keeping an eye on immune effector cell-associated neurotoxicity syndrome (ICANS): Outcomes and predictors of mortality. (ASCO 2025) - " Among 3,910 CAR-T cell therapy admissions in 2022, Axicabtagene Ciloleucel was the most frequently used (52%), followed by Idecabtagene Vicleucel (17%) and Brexucabtagene Autoleucel (14%). ICANS remains a significant complication of CAR-T cell therapy affecting 1 in 5 hospitalized patients. Identifying risk factors like nutritional status, history of stroke, and seizures is important in hospitalized patients. Management plan based on type of CAR-T cell therapy can improve overall outcomes."

Cardiovascular • CNS Disorders • Epilepsy • Hematological Malignancies • Infectious Disease • Ischemic stroke • Oncology • Pneumonia • Respiratory Diseases

Incidence and risk factors for venous thromboembolism after CAR T-cell therapy: A systematic review and meta-analysis. (ASCO 2025) - "Following PRISMA guidelines, we searched Medline, Embase, and CENTRAL (Cochrane Central Register of Controlled Trials) through January 1, 2024, for studies enrolling >20 adults treated with any FDA-approved CAR T-cell product (axicabtagene ciloleucel, tisagenlecleucel, brexucabtagene autoleucel, lisocabtagene maraleucel, idecabtagene vicleucel, or ciltacabtagene autoleucel) that reported VTE events (pulmonary embolism, deep vein thrombosis, cerebral vein thrombosis, hepatic vein thrombosis, splenic vein thrombosis, or portal vein thrombosis) post infusion. Approximately 6–8% of patients develop VTE after CAR T-cell therapy, with higher risk among those who have grade >2 CRS, ICANS, or ECOG >1. Given CAR T's expanding role, these findings underscore the need for vigilant VTE assessment and management. Future investigations should evaluate prophylactic anticoagulation strategies to reduce VTE risk in this setting."

CAR T-Cell Therapy • Retrospective data • Review • Cardiovascular • Hematological Malignancies • Ischemic stroke • Oncology • Respiratory Diseases • Solid Tumor • Venous Thromboembolism

Outpatient administration of idecabtagene vicleucel in relapsed refractory multiple myeloma without planned prophylaxis or remote monitoring. (ASCO 2025) - "Fludarabine plus cyclophosphamide was the lymphodepletion regimen used for all patients...Most CRS events occurred within 1 day of infusion (83%) and Tocilizumab was used to manage all CRS events. Grade 1 ICANS was reported in one patient for whom steroids and Anakinra was used for management... Our findings indicate that outpatient administration of Idecabtagene Vicleucel is feasible with adequate institutional experience and proper toxicity monitoring and management. The majority of patients were still living, so longer follow up time is required to explore the full outpatient experience for patients on this treatment and to understand all pertinent patient outcomes."

Clinical • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • Oncology

Systematic review of BCMA-targeted therapies in relapsed/refractory (R/R) multiple myeloma (MM). (ASCO 2025) - "KarMMA-3 and CARTITUDE-4 use CAR-T cell therapies idecabtagene vicleucel (ide-cel) and ciltacabtagene autoleucel (cilta-cel)...DREAMM-3, DREAMM-7, and DREAMM-8 examine the ADC belantamab mafodotin (maf), as monotherapy or with dexamethasone (d) and bortezomib (V) or pomalidomide (P)... Innovative therapies such as CAR-T and ADCs outperform standard regimens, supporting the evolving landscape of R/R MM treatment. CAR-T cell therapies cilta-cel and ide-cell were approved for 2L+ and 3L+ MM, respectively, based on CARTITUDE-4 and KarMMa-3 trials. Meanwhile, the ADC belantamab maf conditional approval was withdrawn following DREAMM-3 failure to meet its primary endpoint, and it remains under evaluation."

Review • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology

Real-world outcomes of CAR-T in the outpatient setting. (ASCO 2025) - "The most commonly used products were axicabtagene ciloleucel (43.0%), idecabtagene vicleucel (15%), and brexucabtagene autoleucel (11.2%). Any-grade Cytokine release syndrome (CRS) was reported in 66.4% of patients for which Tocilizumab was administered in 92.7%. Any-grade neurotoxicity was reported in 27.7% of which steroids with/without Anakinra was administered in 95.8%... Our experience revealed that treatment with CAR-T in the outpatient setting is safe and feasible with adequate institutional experience. Proper toxicity monitoring and management could help optimize healthcare utilization, and CAR-T accessibility."

Clinical • Real-world • Real-world evidence • B Cell Lymphoma • Follicular Lymphoma • Hematological Malignancies • Infectious Disease • Large B Cell Lymphoma • Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Solid Tumor

Comparative assessment of colitis-related adverse events in multiple myeloma patients treated with teclistamab, ciltacabtagene, and idecabtagene: Incidence, outcomes, and risk assessment. (ASCO 2025) - "This analysis reveals notable differences in colitis-related AEs among patients treated with teclistamab, ciltacabtagene, and idecabtagene. Teclistamab showed the highest rates of severe colitis, including immune-mediated enterocolitis and Clostridium difficile infections. Ciltacabtagene had a broader spectrum of GI-related AEs and higher mortality rates, requiring careful monitoring."

Adverse events • Clinical • Crohn's disease • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Immunology • Infectious Disease • Inflammatory Bowel Disease • Multiple Myeloma • Oncology • Ulcerative Colitis

Comparative efficacy of idecabtagene vicleucel and ciltacabtagene autoleucel in relapsed/refractory multiple myeloma: Real-world analysis of overall survival and time to next treatment. (ASCO 2025) - "In this RW analysis, CC showed improved durability in delaying subsequent therapy compared to IC, as reflected by a significant TTNT benefit. However, this advantage did not translate into improved OS during the follow-up period. Differences in follow-up duration and RW data limitations, including potential missing data, may have influenced outcomes."

Clinical • Real-world • Real-world evidence • Hematological Malignancies • Multiple Myeloma • Oncology

Assessment of normal plasma cell biomarkers after arlocabtagene autoleucel (arlo-cel) treatment in patients with ≥3L relapsed refractory multiple myeloma (MM). (ASCO 2025) - P1, P2 | " Clinical endpoints included treatment-emergent adverse events for patients treated with arlo-cel (NCT04674813; n = 84) and idecabtagene vicleucel (ide-cel, NCT03361748; n = 137). Arlo-cel patients had higher levels of uiFLC from months 2–6, demonstrating greater anti-tumor specificity and preservation of humoral immunity. As a result, arlo-cel has the potential to achieve lower rates of hypogammaglobulinemia and infections compared to BCMA-targeting therapies, with fewer interventions."

Biomarker • Clinical • IO biomarker • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Oncology • GPRC5D

Impact of autoimmune disease on toxicity and outcomes after idecabtagene vicleucel in patients with multiple myeloma. (ASCO 2025) - "Two patients (with AIHA and ITP) were tapered off of prednisone prior to ide-cel. This retrospective study shows that pre-existing AD does not increase the risk of ICANS in patients with RRMM who were treated with ide-cel. Although LDH and ferritin levels were similar, levels of CRP were significantly higher in AD patients who experienced ICANS versus those that did not. Given notable differences in levels of inflammation and relapse rates among patients with AD, these patients may benefit from close monitoring."

Clinical • Endocrine Disorders • Hematological Malignancies • Immunology • Inflammatory Arthritis • Multiple Myeloma • Oncology

Investigating metabolic connectivity in patients with multiple myeloma receiving BCMA CAR T cell therapy. (ASCO 2025) - " Of the 108 consecutive patients (65 Cilta-cel, 43 Ide-cel), there were 61 men and 47 women (median age 65), with PET a median of 12 days prior to infusion 28 days post infusion. Patients with neurotoxicity after BCMA CAR T had an increased SUV in the putamen, but decreased in the frontal regions and basal ganglia at Day 28. Metabolic networks were globally less efficient and less dense and have changes that signify injury or attempts at compensation."

CAR T-Cell Therapy • Clinical • CNS Disorders • Hematological Malignancies • Movement Disorders • Multiple Myeloma • Oncology • Parkinson's Disease

Disease response at apheresis and association with long-term outcomes following CAR-T cells for relapsed/refractory multiple myeloma (RRMM). (ASCO 2025) - "Background: The two approved BCMA-targeted CAR-T products, cilta-cel and ide-cel, have significant efficacy in RRMM, but are not considered curative. Our data suggest that disease control (≥SD) at time of T-cell collection is associated with more durable responses, supporting use of CAR-T cells as a consolidation strategy in RRMM. We cannot conclude these associations are causal. Further analyses of apheresed T cell characteristics are planned."

CAR T-Cell Therapy • Hematological Malignancies • Multiple Myeloma • Oncology

Venous thromboembolism among cancer patients receiving chimeric antigen receptor-T cell therapy. (ASCO 2025) - "Eligibility included administration of any one of the six CAR-T therapies (Tisagenlecleucel, Axicabtagene (Axi-cel), Brexucabtagene, Lisocabtagene, Idecabtagene and Ciltacabtagene) at age ≥18y...The use of glucocorticoids (95% vs 77%, P<0.0001), alkylating agents (91% vs 71%, P<0.0001) or lenalidomide (18% vs 11%, P=0.0006) were significantly associated with VTE... There is 10% incidence of VTE within three months of CAR-T therapy. Particularly, VTE incidence is higher with Axi-cel, and DLBCL diagnosis. Patients with high –risk associations may benefit from thromboprophylaxis regimens, particularly during initial three months of CAR-T therapy."

CAR T-Cell Therapy • Clinical • B Cell Lymphoma • Cardiovascular • Diffuse Large B Cell Lymphoma • Dyslipidemia • Genetic Disorders • Hematological Malignancies • Hypertension • Ischemic stroke • Lymphoma • Mantle Cell Lymphoma • Nicotine Addiction • Non-Hodgkin’s Lymphoma • Obesity • Oncology • Respiratory Diseases • Venous Thromboembolism

Does the Occurrence of ICANS After CAR-T Therapy Increase the Risk of Late Neurological Complications: A Real-World Data Analysis (ASGCT 2025) - "A total of 4, 700 patients in this dataset received one of the US FDA-approved CAR-T therapies, including axicabtagene ciloleucel, tisagenlecleucel, lisocabtagene maraleucel, brexucabtagene autoleucel, and idecabtagene vicleucel. Future studies should consider potential confounding factors including age, prior cancer treatment, and underlying cancer type which could impact the risk of developing neurodegenerative disorders after CAR-T. Disease Focus of Abstract:Central Nervous System Disorders"

Clinical • Real-world • Real-world evidence • CNS Disorders • Movement Disorders • Oncology • Parkinson's Disease

Use of Novel Therapies for Multiple Myeloma in the United States: Important Differences in Patient Characteristics, Access to Care, and Real-World Treatment Challenges (ISPOR 2025) - "All pts had ≥1 claim for a CAR-T/BsAb tx including idecabtagene vicleucel, ciltacabtagene autoleucel, teclistamab, talquetamab, or elranatamab, and continuous enrollment ≥90 days before and after index date. Descriptively, these results suggest important differences in sociodemographic and clinical characteristics among pts who received novel tx for MM. Uptake of BsAbs was higher compared to CAR-Ts, indicating greater tx accessibility for BsAbs. This warrants further investigation into barriers to access and strategies to potentially ameliorate existing health disparities in U.S. MM pts."

Clinical • HEOR • Real-world • Real-world evidence • Hematological Malignancies • Multiple Myeloma • Oncology

Characteristics of RWE used in Regulatory Decision-Making for Marketing Authorization Applications (MAAs) (ISPOR 2025) - " Seven medicines were analyzed: idecabtagene vicleucel (ide-cel), omburtamab, sotorasib, alpelisib, palovarotene, tacrolimus, and omaveloxolone. MAAs containing RWE submitted to the FDA were predominantly for rare diseases medicines and for first-in-class indications. Acceptability of RWE varied based on entire body of evidence. Further investigation into factors influencing RWE acceptability and its integration into MAAs across other regulators such as EMA is warranted."

Hematological Disorders • Rare Diseases

Advancing Equity in CAR T-Cell Therapy: An Analysis of Health Technology Assessments by Canada's Drug Agency and the National Institute for Health and Care Excellence (ISPOR 2025) - " HTA reports from CDA and NICE for six CAR T-cell therapies (Abecma, Breyanzi, Carvykti, Kymriah, Tecartus, Yescarta) were reviewed by two researchers for equity considerations related to access disparities, capacity, socioeconomic determinants, and related clinical and economic evidence. This review identified that DEI considerations related to CAR T-cell therapy access like patient costs and geographical barriers were not routinely supported by evidence in CDA and NICE submissions. Evidence generation challenges in CAR-T therapy may inadvertently deprioritize equity concerns. Recent commitments to equity from HTA bodies offer opportunities to ensure fair access to novel, high-cost therapies."

CAR T-Cell Therapy • Reimbursement • US reimbursement • Hematological Disorders • Hematological Malignancies • Oncology

REAL-WORLD DATA ON BRIDGING STRATEGIES, TOXICITY AND OUTCOMES OF RELAPSED/REFRACTORY MULTIPLE MYELOMA PATIENTS TREATED WITH CAR-T-CELLS (EHA 2025) - "Our results demonstrate the importance of TB reduction by tailored BT, including polychemotherapy regimes and targeted therapies prior to CAR-T. This translates to very high ORR to both ide-cel and cilta-cel and low NRM. We observed significant reduction of CAR-T toxicities depending on the depth of response to BT."

CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • Hematological Malignancies • Multiple Myeloma • Oncology

INNOVATIVE SDAB-BASED CAR-T CELLS TARGETING BCMA OUTPERFORM CURRENT CAR-T THERAPIES FOR MULTIPLE MYELOMA (EHA 2025) - "Despite the high remission rates achieved with B-Cell Maturation Antigen (BCMA) CAR-T therapy, long-term responses remain limited, with median progression-free survival (PFS) of 13.8 months for ide-cel and 34.9 months for cilta-cel. In conclusion, we successfully identified and characterized multiple sdAb-based CAR-T cells with distinct affinities, demonstrating their potential to surpass the antitumor efficacy of currently approved MM CAR-T therapies. These findings highlight the promise of sdAb-based CAR-T cells as a novel and effective treatment for MM, paving the way for further clinical development and potential therapeutic application in relapsed or refractory patients."

CAR T-Cell Therapy • IO biomarker • Hematological Malignancies • Multiple Myeloma • Oncology • HAVCR2 • IFNG • IL2 • LAG3 • PD-1

MANAGEMENT OF IMMUNE EFFECTOR CELL-ASSOCIATED NEUROTOXICITY SYNDROME FOLLOWING CAR-T CELL THERAPY IN CLINICAL PRACTICE: A MULTICENTRIC, RETROSPECTIVE, REAL-WORLD ANALYSIS OF TREATMENT APPROACHES AND OUTCOMES (EHA 2025) - "The CAR T-cell products used were Axi-cel in 65 patients (62.5%), Brexu-cel in 15 (14.4%), Tisa-cel in 13 (12.5%), Liso-cel in 5 (4.8%), Ide-cel in 5 (4.8%), and Cilta-cel in 1 (1.0%)...Additionally, delayed initiation of corticosteroids and anakinra were strong determinants of extended ICANS duration (p5 days) was associated with inferior PFS. Early initiation of corticosteroids and IL-1 receptor antagonists was associated with shorter ICANS duration, suggesting that prompt intervention may mitigate prolonged neurotoxicity."

CAR T-Cell Therapy • Real-world • Real-world evidence • Retrospective data • Acute Lymphocytic Leukemia • B Cell Lymphoma • CNS Disorders • Epilepsy • Hematological Malignancies • Large B Cell Lymphoma • Leukemia • Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology

SEQUENTIAL BCMA CAR T-CELL THERAPY IN REFRACTORY MULTIPLE MYELOMA (EHA 2025) - "The first CAR-T was Ide-cel and the second was Cilta-cel in all patients. This study provides the first real-world evidence supporting the feasibility and efficacy of sequential BCMA-directed CAR-T therapy in refractory MM. Our findings indicate that retreatment with BCMA CAR-T cells can elicit meaningful responses, particularly in patients who initially experienced durable responses. These results highlight the potential for sequential CAR-T strategies and provide insights into patient selection for retreatment."

CAR T-Cell Therapy • IO biomarker • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology

ASSOCIATION BETWEEN BASELINE PATIENT-REPORTED OUTCOMES AND CLINICAL OUTCOMES OF CHIMERIC ANTIGEN RECEPTOR (CAR) T-CELL THERAPY (EHA 2025) - "The most frequently used CAR-T products were tisagenleccel (34%), followed by lisocabtagene maraleucel (16%), axicabtagene ciloleucel (13%), and idecabtagene vicleucel (12%). Baseline PROs are associated with OS post-CAR-T and risk of CRS and ICANS in CAR-T recipients. These findings underscore the potential utility of pre-CAR-T PROs as important prognostic factors for CAR-T outcomes."

Clinical • Clinical data • Patient reported outcomes • CNS Disorders • Depression • Hematological Disorders • Hematological Malignancies • Lymphoma • Mood Disorders • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Psychiatry

KarMMa-7: Safety and Efficacy of bb2121 (Ide-cel) Combinations in Multiple Myeloma (clinicaltrials.gov) - P1/2 | N=21 | Completed | Sponsor: Celgene | Active, not recruiting ➔ Completed | N=312 ➔ 21

Enrollment change • Trial completion • Hematological Malignancies • Multiple Myeloma • Oncology

Hematologic and lymphatic disorders associated with chimeric antigen receptor T-cell therapy: a pharmacovigilance analysis of the FDA adverse event reporting system (FAERS) database. (PubMed, BMC Cancer) - "Our study found that hematologic and lymphatic system AEs were more closely associated with anti-CD19 CAR-T and CAR-T containing CD28. Splenic hemorrhage, disseminated intravascular coagulation, and pancytopenia were identified as hematologic and lymphatic system AEs that, while less frequently reported clinically, were highly associated with mortality."

Adverse events • Journal • Aplastic Anemia • Hematological Disorders • Oncology

2seventy bio Reports First Quarter Financial Results and Provides Update on Proposed Acquisition by Bristol Myers Squibb (Businesswire) - "Acquisition by Bristol Myers Squibb on track to close in the second quarter of 2025: HSR waiting period expired on May 2, 2025; tender offer expected to expire on May 13, 2025. Abecma generated $59 million U.S. commercial revenue in the first quarter of 2025."

M&A • Sales • Multiple Myeloma