NXC-201 / Immix Biopharma - LARVOL DELTA

Phase 1 Study of HBI0101 CAR-T in Refractory B-Cell Autoimmune Diseases (clinicaltrials.gov) - P1 | N=60 | Recruiting | Sponsor: Polina Stepensky

New P1 trial • Immunology • Inflammatory Arthritis • Lupus • Myositis • Rheumatoid Arthritis • Rheumatology • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

Immix Biopharma Announces Class-Leading Safety Profile, Allowing Potential Future Indication Expansion (GlobeNewswire) - "Immix Biopharma...announced a class-leading safety profile for sterically-optimized CAR-T NXC-201, including absence of neurotoxicity of any grade in low-volume disease to-date, laying a foundation for potential future indication expansion."

Trial status • Amyloidosis

NEXICART-2: Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis (clinicaltrials.gov) - P1/2 | N=40 | Recruiting | Sponsor: Nexcella Inc. | Phase classification: P1 ➔ P1/2

Phase classification • Amyloidosis • Multiple Myeloma

Immix Biopharma Announces Accelerated NEXICART-2 Clinical Trial Progress in relapsed/refractory AL Amyloidosis (GlobeNewswire) - "Immix Biopharma, Inc...announced accelerated U.S. NEXICART-2 clinical trial progress in relapsed/refractory AL Amyloidosis. NEXICART-2 now includes 18 clinical trial sites in its national footprint....'These additional new sites further accelerate our path to BLA submission for FDA approval.'"

Trial status • Amyloidosis

Immix Biopharma Announces Primary Endpoint Met in positive NXC-201 Interim Results Presented at ASCO, Enabling Path to Best-in-Class Therapy for relapsed/refractory AL Amyloidosis (GlobeNewswire) - P1/2 | N=40 | NEXICART-2 (NCT06097832) | Sponsor: Nexcella Inc. | "After NXC-201 treatment, all patients normalized pathological disease markers. Complete responses (CRs) were observed in 70% (7 out of 10) of patients treated with NXC-201. The remaining 3 patients are bone marrow minimum residual disease (MRD) negative (10-6), predicting future CR (Immix believes remaining three MRD negative (10-6) patients could be confirmed as CRs in the coming weeks and months). Downstream clinical improvement, including cardiac and renal organ responses, were recorded after CRs. There have been no relapses recorded to-date and no safety signals identified. No neurotoxicity has been observed....Immix plans Biologics License Application (BLA) for FDA approval."

FDA filing • P1/2 data • Amyloidosis

Safety and efficacy data from Nexicart-2, the first US trial of CAR-T in R/R light chain (AL) amyloidosis, Nxc-201. (ASCO 2025) - P1 | "Pts must have been exposed to bortezomib and anti-CD-38 antibody with persistent or relapsed disease. Lymphodepletion was with fludarabine and cyclophosphamide...CRS was observed in 5 pts (grade 1 (N=4), grade 2 (N=1)); onset day 1 (N=3) or 3 (N=2), lasting <24 hours following 1 dose of tocilizumab in all pts... In this first reported U.S. CAR-T clinical trial experience in RR AL Amyloidosis, we demonstrate that NXC-201 can be given safely and resulted in rapid and deep hematologic responses in all pts treated. Our data suggests that the novel anti-BCMA CAR-T NXC-201 may become a valuable treatment option for RR AL pts."

Clinical • IO biomarker • Amyloidosis • Cardiovascular • Chronic Kidney Disease • Congestive Heart Failure • Febrile Neutropenia • Heart Failure • Hematological Disorders • Infectious Disease • Nephrology • Neutropenia • Oncology • Renal Disease

Immix Biopharma Announces Positive Results for NXC-201 at ASCO Oral Presentation, Enabling Pathway to Best-in-Class Therapy for relapsed/refractory AL Amyloidosis (GlobeNewswire) - P1b/2 | N=40 | NEXICART-2 (NCT06097832) | Sponsor: Nexcella Inc. | "The abstract summarizes positive results from the U.S. multi-site NEXICART-2 trial evaluating NXC-201 in relapsed/refractory AL Amyloidosis with a data cutoff of January 28, 2025....Complete responses (CRs) were observed in 71% (5 out of 7) of patients treated with NXC-201. The remaining 2 patients are bone marrow minimum residual disease (MRD) negative (10-6), predicting future CR (Immix believes remaining two MRD negative (10-6) patients could be confirmed as CRs in the coming weeks and months). Downstream clinical improvement, including cardiac and renal organ responses, were recorded after CRs. There have been no relapses recorded to-date and no safety signals identified."

P1/2 data • Amyloidosis

Immix Biopharma to Host KOL Event to Discuss NXC-201 Clinical Data Presented at ASCO 2025 in relapsed/refractory AL Amyloidosis (GlobeNewswire) - "Immix Biopharma...announced that it will host a virtual Key Opinion Leader (KOL) event to discuss interim clinical data from the NEXICART-2 Phase 1/2 clinical trial of cell therapy NXC-201 in patients with relapsed/refractory AL Amyloidosis following its 2025 American Society of Clinical Oncology (ASCO) Oral Presentation in Chicago, IL. The virtual KOL event will take place on Tuesday, June 3, 2025 at 3:00pm ET....The event will feature Heather Landau, MD (Memorial Sloan-Kettering Cancer Center), Shahzad Raza, MD (Cleveland Clinic), and Jeffrey Zonder, MD (Karmanos Cancer Institute) who will discuss their clinical experience with NXC-201 cell therapy and the evolving treatment landscape for relapsed/refractory AL Amyloidosis."

P1/2 data • Amyloidosis

Study of HBI0101 (NXC-201) CAR-T Therapy in Multiple Myeloma and Light-Chain Amyloidosis (clinicaltrials.gov) - P2 | N=180 | Recruiting | Sponsor: Polina Stepensky

New P2 trial • Amyloidosis • Hematological Malignancies • Multiple Myeloma • Oncology

CAR-T in high-risk plasma cell dyscrasias: A systematic review of AL amyloidosis and plasma cell leukemia [WITHDRAWN] (AACR 2025) - "Of 11 patients treated with HBI0101, 5 patients died of cardiac disease within 1 year of CAR-T (3 PD, 1 VGPR, 1 PR)... BCMA-directed CAR-T has been used in AL and PCL with encouraging responses, though the durability of these responses is unknown. Our review suggests that CAR-T may be safe in AL in patients without significant cardiac disease. Patients with PCL may be at an increased risk of serious adverse events, especially in the setting of active progressive disease."

Review • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • Plasma Cell Leukemia

Immix Biopharma’s NXC-201 NEXICART-2 Clinical Trial Data Selected for Oral Presentation at ASCO 2025 (GlobeNewswire) - "Immix Biopharma...announced that Phase 1/2 interim readout data from its U.S. NXC-201 NEXICART-2 trial in relapsed/refractory AL Amyloidosis has been selected for oral presentation at the upcoming 2025 American Society of Clinical Oncology Annual Meeting (ASCO 2025)..."

P1/2 data • Amyloidosis

Initial Safety and Efficacy Data from NEXICART-2, the First U.S. Trial of a CAR-T in R/R Light Chain (AL) Amyloidosis, NXC-201 (ASGCT 2025) - No abstract available

Clinical • Amyloidosis

VIRAL REACTIVATION IN MULTIPLE MYELOMA PATIENTS RECEIVING ANTI-BCMA CHIMERIC ANTIGEN RECEPTOR T-CELL THERAPY (EBMT 2025) - P1 | "This study evaluated the incidence and consequences of viral reactivation in MM patients treated with anti-BCMA CART using data from the phase 1a-b/2 clinical trial (HBI0101, NCT04720313) and compared it with anti-CD19 CART patients receiving treatment for B-cell lymphoma (BCL)...Both groups underwent lymphodepletion with fludarabine and cyclophosphamide on days -5 to -3, or bendamustine for patients with creatinine clearance less than 30 ml/min. Antiviral prophylaxis included acyclovir 800 mg twice daily... Viral reactivations are a common complication after CART therapy, particularly CMV occurring in 20% of MM patients receiving anti-BCMA CART. These reactivations generally lack clinical significance, showing no association with increased mortality or reduced PFS, which may suggest routine testing or preventive treatment are unnecessary. The protective effect of early post-infusion bacteremia should be tested in larger cohorts."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • B Cell Lymphoma • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology

VIRAL REACTIVATION IN MULTIPLE MYELOMA PATIENTS RECEIVING ANTI-BCMA CHIMERIC ANTIGEN RECEPTOR T-CELL THERAPY (EBMT 2025) - P1 | "This study evaluated the incidence and consequences of viral reactivation in MM patients treated with anti-BCMA CART using data from the phase 1a-b/2 clinical trial (HBI0101, NCT04720313) and compared it with anti-CD19 CART patients receiving treatment for B-cell lymphoma (BCL)...Both groups underwent lymphodepletion with fludarabine and cyclophosphamide on days -5 to -3, or bendamustine for patients with creatinine clearance less than 30 ml/min. Antiviral prophylaxis included acyclovir 800 mg twice daily... Viral reactivations are a common complication after CART therapy, particularly CMV occurring in 20% of MM patients receiving anti-BCMA CART. These reactivations generally lack clinical significance, showing no association with increased mortality or reduced PFS, which may suggest routine testing or preventive treatment are unnecessary. The protective effect of early post-infusion bacteremia should be tested in larger cohorts."

CAR T-Cell Therapy • Clinical • Acute Lymphocytic Leukemia • B Acute Lymphoblastic Leukemia • B Cell Lymphoma • Cytomegalovirus Infection • Epstein-Barr Virus Infections • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology

NEXICART-1: NXC-201 (formerly HBI0101) Multiple Myeloma (clinicaltrials.gov) - P1 | N=160 | Active, not recruiting | Sponsor: Hadassah Medical Organization | Recruiting ➔ Active, not recruiting | Trial completion date: Jan 2026 ➔ Jan 2027 | Trial primary completion date: Jan 2025 ➔ Jan 2026

Enrollment closed • Trial completion date • Trial primary completion date • Hematological Malignancies • Multiple Myeloma • Oncology

Immix Biopharma Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for NXC-201, sterically-optimized CAR-T for relapsed/refractory AL Amyloidosis (GlobeNewswire) - "Immix Biopharma, Inc...today announced that the U.S. Food and Drug Administration (FDA) has granted RMAT designation to sterically-optimized CAR-T NXC-201 for the treatment of relapsed/refractory AL amyloidosis....Receipt of FDA RMAT designation underscores the strength of our NXC-201 data and the potential for NXC-201 to provide a new treatment option for patients with relapsed/refractory AL amyloidosis....We look forward to sharing further information on our progress, including an update on NEXICART-2, in the first half of 2025."

FDA event • P1/2 data • Amyloidosis

Immix Biopharma Accelerates Enrollment in U.S. AL Amyloidosis Trial of NXC-201 CAR-T (GlobeNewswire) - "Immix Biopharma...announced successful completion of the six-patient Phase 1b safety run-in segment in the U.S. NEXICART-2 study of NXC-201, an investigational CAR-T therapy, in patients relapsed/refractory (R/R) AL Amyloidosis. Achievement of this milestone is expected to accelerate enrollment across U.S. study sites beginning in January 2025. NEXICART-2 is an open-label study designed to evaluate NXC-201 in patients with R/R AL Amyloidosis....We are on track for the next program update in Q1 2025."

Trial status • Amyloidosis

Efficacy and Safety of Chimeric Antigen Receptor T-Cell Therapy in Relapsed and Refractory Multiple Myeloma – a Meta-Analysis (ASH 2024) - "We included 7 trials each of Ciltacabtagene autoleucel (cilta-cel) and Idecabtagene vicleucel (ida-cel) while 21 trials used different CAR-T cell therapies including GPRC5D, HDS269B, C-CAR088, HBI0101, CART-ddBCMA, ALLO-715, ARI0002h. This analysis has limitations with significant heterogeneity among studies. There were different CAR T-cell therapies with diverse designs of studies which limit the generalization of results."

CAR T-Cell Therapy • Retrospective data • Anemia • Bone Marrow Transplantation • Cardiovascular • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukopenia • Multiple Myeloma • Neutropenia • Oncology • Thrombocytopenia

Immix Biopharma Announces Positive U.S. Clinical Data From First Four Patients in NEXICART-2 U.S. Trial of sterically-optimized CAR-T NXC-201 in relapsed/refractory Light Chain (AL) Amyloidosis (GlobeNewswire) - P1 | N=40 | NEXICART-2 (NCT06097832) | Sponsor: Nexcella Inc. | "Immix plans to host a conference call to review the results on Thursday, December 19, 2024 at 11:00 AM ET...Median follow-up was 85 days (range 29-141) as of the data cut-off date of November 14, 2024...All four patients treated with NXC-201 normalized their disease markers within 30 days of dosing...Bone marrow MRD negativity predicts future CR...No patient developed immune effector cell-associated neurotoxicity syndrome. No cytokine-release syndrome (CRS) was observed in two patients (patients NX2-001, NX2-002). Grade 1 (n=1), and grade 2 (n=1) CRS was observed; both onset day 3 and lasting less than 24 hours following one dose of tocilizumab (patients NX2-003 and NX2-004). Hematologic adverse events included neutropenia in all four patients...Company to provide next program update in H1 2025. Interim clinical data readout is expected Q2/Q3 2025. Final topline clinical data readout is expected Q2/Q3 2026."

P1 data • Pipeline update • Amyloidosis

Efficacy of HBI0101, an Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for Relapsed/Refractory Multiple Myeloma (ASH 2024) - P1 | "Most patients (73/84, 87%) were triple refractory, 32/84 (38%) were penta-refractory and 14/84 (17%) had received prior anti-BCMA therapy, mostly belantamab mafodotin. CONCLUSION : HBI0101 BCMA CART results demonstrate high efficacy and manageable safety in a frailer and higher risk population as compared with the registrational studies with commercial products. This data not only supports further utilization of HBI0101 CART therapy, but also of CART production at an academic setting in general, ensuring a sufficient CART supply in the light of the increasing demand."

CAR T-Cell Therapy • Clinical • Anemia • Hematological Disorders • Hematological Malignancies • Inflammation • Multiple Myeloma • Neutropenia • Oncology • Thrombocytopenia

Efficacy and Safety of Anti-BCMA Chimeric Antigen Receptor T-Cell (CART) for the Treatment of Relapsed and Refractory AL Amyloidosis (ASH 2024) - P1 | "In a phase Ia-b/2 study (NCT04720313), HBI0101 has demonstrated manageable safety with therapeutic efficacy in over 90 MM patient...Patients with creatinine clearance ≥30ml/min (n=13) received lymphodepletion with fludarabine 25mg/m2 and cyclophosphamide 250mg/m2 on days -5 to -3 before infusion, and patients with creatinine clearance <30ml/min (n=3) received lymphodepletion with bendamustine 90mg/m2 and on days -4 and -3 before infusion...RESULTS : Sixteen AL patients with relapsed/refractory disease were included, with a median of 4 prior lines of therapy (range : 3-10), all except one were refractory to their last line of therapy, 14/16 (88%) were triple refractory to PI, IMiD and anti-CD38 antibody and 6/16 (38%) were refractory to the anti-BCMA antibody drug conjugate belantamab mafodotin...Cytokine release syndrome (CRS) was observed in 14/16 (grade 1-2- 11 patients; grade 3- 3 patients; no grade 4 or 5 CRS), and was manageable with frequent use of..."

CAR T-Cell Therapy • Clinical • Acute Kidney Injury • Amyloidosis • Anemia • Cardiovascular • Congestive Heart Failure • Heart Failure • Hematological Disorders • Hematological Malignancies • Hepatology • Inflammation • Liver Failure • Multiple Myeloma • Myelodysplastic Syndrome • Nephrology • Neutropenia • Oncology • Renal Disease • Thrombocytopenia

Journal of Clinical Oncology Publishes NXC-201 Positive Clinical Results in relapsed/refractory AL Amyloidosis (GlobeNewswire) - P1 | N=160 | NEXICART-1 (NCT04720313) | "Immix Biopharma...announced today the Journal of Clinical Oncology (JCO) published NXC-201 clinical results in relapsed/refractory AL Amyloidosis. The data reported on 16 enrolled patients in NEXICART-1 who had received a median 4 prior lines of therapy prior to treatment with NXC-201. Results showed a complete response (CR) rate of 75% (12/16). Notably, findings demonstrated a favorable safety profile in frail relapsed/refractory AL Amyloidosis patients. Ex-U.S. study NEXICART-1 is the first NXC-201 clinical trial to treat relapsed/refractory AL Amyloidosis."

P1 data • Amyloidosis

Immix Biopharma Announces 75% Complete Response Rate (n=16); 31.5 months Best Response Duration (ongoing) for CAR-T NXC-201 in Relapsed/Refractory AL Amyloidosis Patients at ASH 2024 (GlobeNewswire) - P1b/2a | N=160 | NEXICART-1 (NCT04720313) | "At the NXC-201 ASH 2024 oral presentation, data were presented from 16 relapsed/refractory AL amyloidosis patients (including 3 new patients) in the ongoing Phase 1b/2 NEXICART-1 study, with median 4 lines of therapy prior to NXC-201...Safety and efficacy data: Overall response rate of 94% (15/16); Complete response rate of 75% (12/16) (9 out of 16 were MRD- 10-5); Organ response rate of 62% (8/13 evaluable); Best responder had a duration of response of 31.5 months as of December 9, 2024, with complete response ongoing; There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events; Median CRS duration was 2 days (range: 1-5): i) No grade 4 cytokine release syndrome (CRS) events; ii) 2 experienced no CRS; 3 experienced grade 1 CRS; 8 Experienced grade 2 CRS; 3 experienced grade 3 CRS."

P1/2 data • Amyloidosis

Efficacy and Safety of Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T-Cell for the Treatment of Relapsed and Refractory AL Amyloidosis. (PubMed, J Clin Oncol) - P1 | "This largest clinical trial of AL patients treated with anti-BCMA CART demonstrates acceptable and manageable toxicity in a highly frail and resistant population with remarkable efficacy, leading to fast organ responses. Among patients with baseline advanced cardiac disease, deaths in the first year were frequent, suggesting that this effective therapy should be considered earlier in the course of therapy. Anti-BCMA CART may become a powerful tool for improving organ function and survival in patients with AL."

CAR T-Cell Therapy • Journal • Amyloidosis • Heart Failure • Hematological Disorders • Inflammation

Immix Biopharma Presents Positive NXC-201 Clinical Data at 66th American Society of Hematology (ASH) Annual Meeting in 16 Relapsed/Refractory AL Amyloidosis Patients (GlobeNewswire) - "Immix Biopharma, Inc...today announced that additional NEXICART-1 NXC-201 clinical data in relapsed/refractory AL Amyloidosis has been selected for oral presentation at the upcoming 66th American Society of Hematology (ASH) Annual Meeting to be held in San Diego, California, December 7-10, 2024."

P1 data • Amyloidosis