Yorwida (inaticabtagene autoleucel) / CASI, Juventas Cell Therapy - LARVOL DELTA

THE COST-EFFECTIVENESS ANALYSIS OF INATICABTAGENE AUTOLEUCEL FOR RELAPSED/REFRACTORY B-CELL ACUTE LYMPHOBLASTIC LEUKEMIA PATIENTS IN CHINA (ISPOR 2026) - "Given its high upfront cost and potential for survival benefits, this study aimed to evaluate the cost-effectiveness of inaticabtagene autoleucel compared with blinatumomab, inotuzumab ozogamicin, and allogeneic hematopoietic stem-cell transplantation (allo-HSCT) from the Chinese healthcare system perspective. An economic model was developed, combining a decision tree with a partitioned survival model to project lifetime costs and health outcomes. From the Chinese healthcare system perspective, inaticabtagene autoleucel is likely to be cost-effective for patients with R/R B-ALL when a willingness-to-pay threshold of twice the gross domestic product (GDP) per capita is applied. These findings informed the reimbursement and coverage decision-making for CAR-T cell therapies in China."

Clinical • Cost effectiveness • HEOR • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia

A Study of CNCT19 in the Treatment of Relapsed or Refractory Neurological Autoimmune Diseases (clinicaltrials.gov) - P1 | N=18 | Recruiting | Sponsor: Juventas Cell Therapy Ltd. | Not yet recruiting ➔ Recruiting | Initiation date: Dec 2025 ➔ Mar 2025

Enrollment open • Trial initiation date • CNS Disorders • Immunology • CD19

CASI Pharmaceuticals, Inc. reported that China’s National Medical Products Administration formally rejected its renewal application for the Import Drug Registration License for FOLOTYN in China. (StockTitan.net) - " The company had already stopped selling FOLOTYN in China after the prior license expired, in line with applicable regulations, so sales had ceased before this formal rejection....CASI highlights its focus on developing CID-103 and notes ongoing disputes and legal proceedings related to EVOMELA and CNCT-19, suggesting a portfolio in transition toward pipeline assets."

Pipeline update • Regulatory • Immunology • Oncology

Combinational therapy of CAR T-cell and HDT/ASCT demonstrates impressive clinical efficacy and improved CAR T-cell behavior in relapsed/refractory large B-cell lymphoma. (PubMed, J Immunother Cancer) - P1/2 | "The combinational therapy of HDT/ASCT and CNCT19 demonstrates impressive efficacy, improved CNCT19 behavior, and a favorable safety profile."

CAR T-Cell Therapy • Journal • Bone Marrow Transplantation • CNS Disorders • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Transplantation

Clinical Trial of Autologous CD19 CAR-T Cells (CNCT19) Therapy for Advanced Hepatocellular Carcinoma (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Zhejiang University | Not yet recruiting ➔ Recruiting

Enrollment open • Hepatocellular Cancer • Oncology • Solid Tumor

A Study of CNCT19 in the Treatment of Relapsed or Refractory Neurological Autoimmune Diseases (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: Juventas Cell Therapy Ltd.

New P1 trial • CNS Disorders • Immunology

Efficacy and safety profile of inaticabtagene autoleucel in Chinese patients with Philadelphia chromosome-positive b-ALL: Insights from real-world data (ASH 2025) - P | "Prior therapies included hematopoietic stem cell transplantation (HSCT) (24.1%), inotuzumab ozogamicin (14.8%), and blinatumomab (24.1%)...The median infusion dose was 0.60 (range: 0.44–1.00) × 10 8 viable CAR-T cells, with 88.9% of patients receiving bridge therapy...All patients recovered without sequelae: 9 received corticosteroids, and 11 received tocilizumab. Conclusion Real-world data demonstrate that Inati-cel exhibits excellent efficacy in patients with Ph+ B-ALL, yielding low toxicity, short treatment cycles, high complete molecular response rates, and favorable long-term survival. Extended follow-up could illuminate the long-term outcomes of Inati-cel use."

Clinical • Real-world • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Central Nervous System Leukemia • Hematological Malignancies • Leukemia • ABL1 • IKZF1

Efficacy and safety profile of inaticabtagene autoleucel in Chinese adult patients with B-cell acute lymphoblastic leukemia who relapsed after their transplant: Insights from real-world data (ASH 2025) - P | "The median infusion dose was 0.60 (range: 0.42–1.00) × 10 8 viable CAR-T cells...Patients with and without prior blinatumomab exposure had similar OS (p = 0.62) and RFS (p = 0.58); the same was true for prior inotuzumab ozogamicin exposure (p = 0.86 and p = 0.67, respectively)...Conclusion Real-world data demonstrate that Inati-cel exhibits excellent efficacy in patients with B-ALL who relapse after HSCT and in patients with active extramedullary disease; its safety was also established. Extended follow-up is warranted to fully characterize the long-term outcomes of Inati-cel use."

Clinical • IO biomarker • Real-world • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Central Nervous System Leukemia • Hematological Malignancies • Infectious Disease • Leukemia • Transplantation • IKZF1 • TP53

Efficacy and safety profile of inaticabtagene autoleucel in Chinese patients with B-cell acute lymphoblastic leukemia: Insights from real-world data (ASH 2025) - P | "Prior immunotherapies included: HSCT(21.4%), inotuzumab ozogamicin (18.6%), and blinatumomab (28.3%)...All patients recovered without sequelae, with 38 treatedwith corticosteroids, and 33 with tocilizumab or siltuximab.Real-world data on Inati-cel corroborates its robust response rate, favorable toxicity profile, and survivalbenefits. Real-world data on Inati-cel corroborates its robust response rate, favorable toxicity profile, and survivalbenefits. Inati-cel demonstrates efficacy in patients with active extramedullary diseases, particularlythose with CNSL. In vivo expansion was observed across different disease states."

Clinical • IO biomarker • Real-world • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Infectious Disease • Leukemia • CD4 • CD8 • IKZF1 • KMT2A • PAX5 • TP53

Deeper remission achievement and longer leukemia-free survival with inati-cel as consolidation therapy in allogeneic transplant-ineligible adolescent and adult patients with B-ALL (ASH 2025) - P1/2 | "All patients receivedlymphodepletion with fludarabine (30 mg/m2/d ×3 days) and cyclophosphamide (500 mg/m2/d×2 days) followed by Ina-cel infusion (0.6×108 live CAR-T cells)...Specially, only one patient had grade 3 CRS and grade 3 ICANS, characterized by high fever andhypotension, and was managed with tocilizumab in combination with dexamethasone (80 mg).Additionally, 7 patients (63.6%) suffered from cytopenia... Our preliminary results of the intervention study demonstrate inati-cel, a CD19 CAR-T celltherapy leads to high MRD-negativity conversion and survival rates with manageable toxicity intransplant-ineligible B-ALL patients. More patients are being enrolled and followed up. Long-termsurvival data are likely to support CD19 CAR-T cell therapy as a new paradigm for consolidation therapyfor B-ALL patients."

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Transplantation

A Real-world Study on the Treatment of Adult B-cell Acute Lymphoblastic Leukemia With CNCT-19 (clinicaltrials.gov) - P=N/A | N=270 | Active, not recruiting | Sponsor: Ruijin Hospital

New trial • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology

Real-world efficacy of inaticabtagene autoleucel in adult B-cell acute lymphoblastic leukemia patients with high-risk molecular alterations (ASH 2025) - P | "The CR1-MRD⁻ group exhibited a trend toward superior median RFS compared to the combined CR1-MRD⁺ and R/R group (not reached vs. 404 days [95% CI: 30-777 days], P=0.073).In terms of post-CAR-T management, 20 patients received combination therapy (including three patients received tyrosine kinase inhibitors, one patients received zanubrutinib, two patients received pomalidomide, three patients received venetoclax, one patients received blinatumomab, one patients received inotuzumab ozogamicin, and four patients received allo-HSCT), while 21 patients underwent observation only. Front-line consolidation with CAR-T during CR1-MRD⁻ in patients with high-risk molecular alterations may yield better RFS outcomes compared to treatment administered in R/R or MRD⁺ settings. No RFS benefit from post-CAR-T combination therapy was observed in this cohort, highlighting the need for validation with longer follow-up durations and larger patient cohorts."

Clinical • IO biomarker • Real-world • Real-world effectiveness • Real-world evidence • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • IKZF1 • KMT2A • TP53

Autologous hematopoietic stem cell transplantation combined with inaticabtagene autoleucel in adults with high-risk B-cell acute lymphoblastic leukemia: Interim analysis of a phase II study (ASH 2025) - P2 | "By July 20, 2025, twelve patients' results were included in the interim analysis. The median agewas 36 years (range: 19–52 years), and 66.7% were male. High-risk genetic abnormalities were detectedin 66.7% of the patients, including BCR-ABL1 (33.3%), MLL rearrangement (16.7%), IKZF1 mutation (8.3%),and MEF2D rearrangement (8.3%)."

Clinical • IO biomarker • P2 data • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Gastrointestinal Disorder • Hematological Malignancies • Infectious Disease • Leukemia • Pneumonia • Respiratory Diseases • Transplantation • ABL1 • BCR • CXCL8 • IKZF1 • IL10 • IL5 • IL6 • KMT2A • MEF2D • TNFA

Synergistic integration of CAR-T cell pharmacokinetics and NGS-MRD evolution enables precision risk stratification in relapsed/refractory B-cell acute lymphoblastic leukemia (ASH 2025) - P2 | " This study included 27 patients with relapsed/refractory B-cell acute lymphoblastic leukemia(r/r B-ALL) enrolled in a phase 2, multicenter study of inaticabtagene autoleucel (Inati-cel) for adult CD19+r/r B-ALL (ClincialTrials.gov Identifier: NCT04684147)... Both the 28-day cumulative CAR-T expansion area under the curve (AUC) and NGS-MRDtrajectory act as independent predictors of treatment outcomes in patients with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). When these two factors are integrated into acomprehensive predictive model, we can more accurately pinpoint high-risk patients and developtailored treatment strategies, which could ultimately improve long-term survival for this group ofpatients."

CAR T-Cell Therapy • IO biomarker • Next-generation sequencing • PK/PD data • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia

Inaticabtagene Autoleucel (YORWIDA) New Drug Application for Lymphoma Approved, China's innovative CAR-T Enters a New Era of Dual Indications (PRNewswire) - "The approval of this new indication is based on the positive results of a single-arm, open-label, multi-center pivotal Phase II clinical study (NCT04586478) for the treatment of r/r LBCL."

China approval • Large B Cell Lymphoma

Efficacy and Safety of Inaticabtagene Autoleucel in Children with Relapsed/Refractory (R/R) B-cell Acute Lymphoblastic Leukemia in China (ASH 2024) - P1/2 | "They received a single infusion of Inati-cel at a target dose of 1×106 CAR + viable T-cells/kg (±20%) after lymphodepletion with cyclophosphamide and fludarabine. Among the 6 pts in the phase Ib stage, one patient had a DLT event observed, related to high tumor burden and severe Grade 4 CRS, and was deemed manageable by the Steering Committee.CAR T-cell expansion occurred in all pts with a median time to maximum expansion (T max ) of 15 days (range : 9-23), and a peak copy number (Cmax) of 1.24×105 copies/μg gDNA.Conclusion : This trial demonstrates that Inati-cel is safe and effective in heavily treated children with R/R B-cell ALL. A pivotal trial evaluating Inati-cel at a target dose of 1×10^6 cells/kg (±20%) in children with R/R B-ALL is underway.*On behalf of Copenhagen Hospital Biobank and International Epidemiology Lymphoma Consortium"

Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Lymphoma • Oncology

Sustained Remission and Decreased Severity of CAR T-Cell Related Adverse Events: An Updated Report on the Pivotal Study of Inaticabtagene Autoleucel (Inati-cel; CNCT19) Treatment in Adult Patients with Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-Cell ALL) in China (ASH 2024) - P2 | "Inati-cel was infused 2 to 14 days after lymphodepletion with cyclophosphamide and fludarabine. The safety profile was favorable, with a low incidence of grade 3 or higher CRS and ICANS. With its distinct CAR structure containing a unique CD19 scFv (HI19a), Inati-cel provides effective treatment with potential long-term clinical benefits for adult patients with r/r B-cell ALL."

Adverse events • CAR T-Cell Therapy • Clinical • IO biomarker • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Oncology • IKZF1 • KMT2A • PBX1 • TP53

Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia After Three or More Lines of Therapy (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Juventas Cell Therapy Ltd. | Initiation date: Aug 2025 ➔ Dec 2025

Trial initiation date • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology

Long-term outcomes of CNCT19 chimeric antigen receptor T-cell therapy in relapsed or refractory aggressive B-cell lymphoma. (PubMed, Chin Med J (Engl)) - P1 | "CNCT19s exhibited favorable safety profiles and efficacy in patients with R/R DLBCL and FL3B. Long-term follow-up confirmed the curative potential of CNCT19s in R/R aggressive B-cell lymphoma."

Journal • B Cell Lymphoma • CNS Disorders • Diffuse Large B Cell Lymphoma • Follicular Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • Richter's Syndrome • TP53

A Multicenter, Open-label, Single-arm Phase I Clinical Trial on the Safety and Tolerability of Nachiotaxel Injection in the Treatment of Autoimmune Hemolytic Anemia That Has Failed at Least Three Lines of Treatment (clinicaltrials.gov) - P1 | N=6 | Not yet recruiting | Sponsor: Juventas Cell Therapy Ltd.

New P1 trial • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology

A Study of CNCT19 Treatment in Children and Adolescent r/r ALL Patients(Pediatric) (clinicaltrials.gov) - P1/2 | N=47 | Recruiting | Sponsor: Juventas Cell Therapy Ltd. | Trial completion date: Dec 2026 ➔ May 2027

Trial completion date • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • Hematological Malignancies • Leukemia • Oncology • Pediatrics • CD19

Inaticabtagene Autoleucel Injection in the Treatment of Refractory Systemic Lupus Erythematosus-related Immune Thrombocytopenia (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: Juventas Cell Therapy Ltd. | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Inflammatory Arthritis • Lupus • Systemic Lupus Erythematosus • Thrombocytopenia • Thrombocytopenic Purpura

Long-term Observational Study of Patients Treated With Inaticabtagene Autoleucel Injection (clinicaltrials.gov) - P=N/A | N=1000 | Recruiting | Sponsor: Juventas Cell Therapy Ltd. | Active, not recruiting ➔ Recruiting

Enrollment open

Inaticabtagene Autoleucel Injection in the Treatment of Autoimmune Hemolytic Anemia After Three or More Lines of Therapy (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: Juventas Cell Therapy Ltd. | Trial primary completion date: Sep 2025 ➔ Sep 2026

Trial primary completion date • Anemia • Autoimmune Hemolytic Anemia • Hematological Disorders • Immunology

EFFICACY AND SAFETY OF AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION COMBINED WITH CD19 CAR-T IN ADULTS WITH HIGH-RISK B-ALL: A PHASE II STUDY (EHA 2025) - P2 | "Inaticabtagene autoleucel has shown remarkable efficacy in relapsed/refractory B-ALL... Auto-CD19-CART combined with auto-HSCT did not affect the success of hematopoietic engraftment or induce additional adverse effects. Furthermore, CAR-T therapy may achieve deep remission in high-risk B-ALL patients. Larger sample sizes and longer-term follow-up are required to validate these findings."

Clinical • IO biomarker • P2 data • Acute Lymphocytic Leukemia • Agranulocytosis • Bone Marrow Transplantation • Granulocytopenia • Hematological Malignancies • Infectious Disease • Leukemia • Oncology • T Acute Lymphoblastic Leukemia • Transplantation