NXC-201 / Immix Biopharma - LARVOL DELTA

BCMA-Targeted CART (HBI0101), a Safe and Efficacious Novel Modality of Treatment for LC Amyloidosis Patients (ASGCT 2023) - P1 | "In this largest cohort of AL patients reported to date, HBI0101 demonstrated acceptable toxicity in this frail patient population. Moreover, all patients responded to therapy, implying that CART therapy may become a powerful clinical tool to improve patient and organ survival even in advanced AL patients."

Clinical • IO biomarker • Late-breaking abstract • Amyloidosis • Anemia • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia

Clinical evaluation and determinants of response to HBI0101 (BCMA CART) therapy in relapsed/refractory multiple myeloma. (PubMed, Blood Adv) - P1 | "Multivariable analysis on patient/disease and CART cell-related characteristics revealed that high-risk cytogenetic, extramedullary disease, and increased number of effector-memory T-cells in CART products were independently associated with inferior PFS. In conclusion, comprehensive analyses of the parameters affecting the response to CART therapy are essential for improving patients' outcome."

Journal • Amyloidosis • Hematological Disorders • Hematological Malignancies • Inflammation • Multiple Myeloma • Oncology

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

NEXICART-2: Study of NXC-201 CAR-T in Patients With Light Chain (AL) Amyloidosis (clinicaltrials.gov) - P1/2 | N=45 | Active, not recruiting | Sponsor: Nexcella Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed • Amyloidosis

NEXICART-3: A Randomized Phase 3 Study to Evaluate the Efficacy and Safety of NXC-201 Compared With Daratumumab With Cyclophosphamide, Bortezomib and Dexamethasone (CyBorD) in Newly Diagnosed Systemic AL Amyloidosis (clinicaltrials.gov) - P3 | N=260 | Not yet recruiting | Sponsor: Nexcella Inc.

New P3 trial • Amyloidosis

Immix Biopharma Announces 95% Complete Response Rate in Interim Update From relapsed/refractory AL Amyloidosis Clinical Trial NEXICART-2 (The Manila Times) - "The NEXICART-2 CR rate is now 95% (19 out of 20 patients). All CRs were reached within 1 year of follow-up post-dosing. No relapses have been observed as of today for patients who have reached CR. All subsequently enrolled patients for whom MRD results are available are MRD-negative at one month...These results support the Company’s plan to initiate a multi-center, randomized Phase 3 trial in newly diagnosed AL Amyloidosis patients...'In September 2026 we plan to present updated data from our ongoing NEXICART-2 trial. By end of March 2027, we expect to present 1-year follow-up data of enrolled patients, which we expect will drive BLA submission and commercial launch.'"

New P3 trial • P2 data • Amyloidosis • Immunology

Immix Biopharma Announces Pricing of $150 Million Underwritten Offering of Common Stock (GlobeNewswire) - "Immix intends to use the net proceeds from this offering to fund NXC-201 development, working capital and general corporate purposes. Immix believes that the net proceeds from the offering, together with its existing cash and cash equivalents, will be sufficient to meet the Company’s operational needs into mid-2028."

Financing • Amyloidosis • Immunology

First 30-patient safety and efficacy data from NEXICART-2, the first U.S. trial of CAR-T in R/R light chain (AL) amyloidosis, NXC-201 (ASGCT 2026) - P1/2 | "It will enroll 40 patients who have been exposed to bortezomib and anti-CD-38 antibody with persistent or relapsed disease. Lymphodepletion is with fludarabine and cyclophosphamide...Dark blue: dFLC reduction percentage after treatment. Light blue: M-Spike reduction percentage after treatment."

CAR T-Cell Therapy • Clinical • IO biomarker • Amyloidosis • Atrial Fibrillation • Cardiovascular • Chronic Kidney Disease • Hematological Disorders • Infectious Disease • Inflammation • Nephrology • Neutropenia • Renal Disease

B-cell Maturation Antigen Targeted Chimeric Antigen Receptor T-cell Therapy for Refractory Systemic Lupus Erythematosus and Systemic Sclerosis. (PubMed, Arthritis Rheumatol) - "HBI0101 demonstrated an acceptable safety profile and early clinical activity in patients with severe refractory systemic lupus erythematosus or systemic sclerosis, supporting further prospective evaluation."

Journal • Hematological Disorders • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Neutropenia • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

Phase 1 Study of HBI0101 CAR-T in Refractory B-Cell Autoimmune Diseases (clinicaltrials.gov) - P1 | N=120 | Recruiting | Sponsor: Polina Stepensky | N=60 ➔ 120

Enrollment change • CNS Disorders • Immunology • Inflammatory Arthritis • Lupus • Multiple Sclerosis • Myasthenia Gravis • Myositis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Rheumatoid Arthritis • Rheumatology • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

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."

CAR T-Cell Therapy • Review • Hematological Malignancies • Leukemia • Multiple Myeloma • Oncology • Plasma Cell Leukemia

Immix Biopharma Announces Enrollment Completion of BLA-Enabling Relapsed/Refractory AL Amyloidosis Trial NEXICART-2, and Upcoming Milestones (GlobeNewswire) - "Topline NEXICART-2 Results Expected Q3 2026, followed by BLA submission and planned commercial launch."

Enrollment closed • FDA filing • Launch US • Amyloidosis • Immunology

PATTERNS AND PREDICTORS OF HEMATOLOGIC TOXICITY AFTER BCMA- AND CD19-TARGETED CAR-T THERAPY (EBMT 2026) - P1 | "The first cohort comprised patients with relapsed/refractory multiple myeloma (RRMM) (n=77) treated with a locally manufactured BCMA-directed CAR T-cell product (HBI0101)... Baseline neutropenia was the strongest predictor of post–CAR T-cell neutropenia, highlighting the importance of bone marrow reserve prior to infusion. Neutropenia was more frequent in the multiple myeloma patients treated with a BCMA-directed CAR T-cell construct compared with lymphoma patients, receiving CD19-directed products, likely reflecting differences in disease biology, prior treatment burden, and baseline cytopenias (as shown in supplementary figure baseline neutropenia was more severe in MM patients)."

CAR T-Cell Therapy • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Neutropenia • Non-Hodgkin’s Lymphoma • Plasma Cell Leukemia • Thrombocytopenia

BCMA CAR T-CELL THERAPY DEMONSTRATES EARLY SAFETY AND EFFICACY SIGNALS IN REFRACTORY SYSTEMIC SCLEROSIS AND ANTIPHOSPHOLIPID SYNDROME: PHASE I RESULTS IN AUTOIMMUNE DISEASES (EBMT 2026) - P1 | "We report updated safety and preliminary efficacy data from an ongoing Phase 1 trial of BCMA-directed CAR T cells (HBI0101) in patients with refractory autoimmune rheumatic diseases (ARDs) (NCT07085676)...Tocilizumab was administered to 6 patients (median dose 1.5, range 1–3), and high-dose corticosteroids were required in one patient... BCMA-targeted CAR T-cell therapy demonstrated a favorable safety profile with no dose-limiting toxicities or unexpected adverse events. All treated patients showed clinical or biological responses lasting up to 9 months. Notably, we report the first clinical responses to BCMA-directed CAR T-cell therapy in SSc and APS."

CAR T-Cell Therapy • Clinical • P1 data • Amyloidosis • Fibrosis • Genetic Disorders • Glomerulonephritis • Hematological Disorders • Hematological Malignancies • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Multiple Myeloma • Myositis • Nephrology • Neutropenia • Renal Disease • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

IMPACT OF CORTICOSTEROID THERAPY ON CLINICAL OUTCOMES, TOXICITY, AND CAR T-CELL KINETICS IN PATIENTS TREATED WITH ACADEMIC ANTI-BCMA CAR T CELLS (HBI0101) (EBMT 2026) - "Among patients receiving the anti-BCMA CAR T product HBI0101, corticosteroid administration for CRS/ICANS did not negatively impact CAR T-cell expansion or depth of response. Although an association with inferior OS was observed in univariate analysis, this effect was attenuated and did not reach statistical significance in multivariable modeling. These findings support the safety of clinically indicated corticosteroid use in managing CAR T-related toxicities, while underscoring the need for larger cohorts to confirm these results and refine toxicity management strategies for anti-BCMA CAR T therapies."

CAR T-Cell Therapy • Clinical • Clinical data • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • Neutropenia • GZMB • IFNG • TNFA

IMPACT OF CORTICOSTEROID THERAPY ON CLINICAL OUTCOMES, TOXICITY, AND CAR T-CELL KINETICS IN PATIENTS TREATED WITH ACADEMIC ANTI-BCMA CAR T CELLS (HBI0101) (EBMT 2026) - "Among patients receiving the anti-BCMA CAR T product HBI0101, corticosteroid administration for CRS/ICANS did not negatively impact CAR T-cell expansion or depth of response. Although an association with inferior OS was observed in univariate analysis, this effect was attenuated and did not reach statistical significance in multivariable modeling. These findings support the safety of clinically indicated corticosteroid use in managing CAR T-related toxicities, while underscoring the need for larger cohorts to confirm these results and refine toxicity management strategies for anti-BCMA CAR T therapies."

CAR T-Cell Therapy • Clinical • Clinical data • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Inflammation • Multiple Myeloma • Neutropenia • GZMB • IFNG • TNFA

PATTERNS AND PREDICTORS OF HEMATOLOGIC TOXICITY AFTER BCMA- AND CD19-TARGETED CAR-T THERAPY (EBMT 2026) - P1 | "The first cohort comprised patients with relapsed/refractory multiple myeloma (RRMM) (n=77) treated with a locally manufactured BCMA-directed CAR T-cell product (HBI0101)... Baseline neutropenia was the strongest predictor of post–CAR T-cell neutropenia, highlighting the importance of bone marrow reserve prior to infusion. Neutropenia was more frequent in the multiple myeloma patients treated with a BCMA-directed CAR T-cell construct compared with lymphoma patients, receiving CD19-directed products, likely reflecting differences in disease biology, prior treatment burden, and baseline cytopenias (as shown in supplementary figure baseline neutropenia was more severe in MM patients)."

CAR T-Cell Therapy • Hematological Disorders • Hematological Malignancies • Leukemia • Lymphoma • Multiple Myeloma • Neutropenia • Non-Hodgkin’s Lymphoma • Plasma Cell Leukemia • Thrombocytopenia

BCMA CAR T-CELL THERAPY DEMONSTRATES EARLY SAFETY AND EFFICACY SIGNALS IN REFRACTORY SYSTEMIC SCLEROSIS AND ANTIPHOSPHOLIPID SYNDROME: PHASE I RESULTS IN AUTOIMMUNE DISEASES (EBMT 2026) - P1 | "We report updated safety and preliminary efficacy data from an ongoing Phase 1 trial of BCMA-directed CAR T cells (HBI0101) in patients with refractory autoimmune rheumatic diseases (ARDs) (NCT07085676)...Tocilizumab was administered to 6 patients (median dose 1.5, range 1–3), and high-dose corticosteroids were required in one patient... BCMA-targeted CAR T-cell therapy demonstrated a favorable safety profile with no dose-limiting toxicities or unexpected adverse events. All treated patients showed clinical or biological responses lasting up to 9 months. Notably, we report the first clinical responses to BCMA-directed CAR T-cell therapy in SSc and APS."

CAR T-Cell Therapy • Clinical • P1 data • Amyloidosis • Fibrosis • Genetic Disorders • Glomerulonephritis • Hematological Disorders • Hematological Malignancies • Immunology • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Multiple Myeloma • Myositis • Nephrology • Neutropenia • Renal Disease • Rheumatology • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

First 23-Patient Safety and Efficacy Data from Nexicart-2, the First U.S. Trial of CAR-T in R/R Light Chain (AL) Amyloidosis, Nxc-201 (TCT-ASTCT-CIBMTR 2026) - P1/2 | "Pts have been exposed to bortezomib and anti- CD-38 antibody with persistent or relapsed disease with measurable disease dFLC >5mg/dl or dFLC >2mg/dl with abnormal k:l ratio, or M-spike >0.5g/dl . The novel anti-BCMA CAR-T NXC-201 may fill an unmet medical need for RR AL pts. CAR-T cells can be safely administered to patients with relapsed/refractory AL amyloidosis CAR-T cells result in deep hematologic responses in patients with relapsed/refractory AL amyloidosis CAR-T can fill an unmet medical need in patients with relapsed/refractory AL amyloidosis"

Clinical • IO biomarker • Amyloidosis • Atrial Fibrillation • Cardiovascular • Chronic Kidney Disease • Febrile Neutropenia • Heart Failure • Hematological Disorders • Infectious Disease • Inflammation • Nephrology • Neutropenia • Renal Disease

Immix Biopharma Receives U.S. FDA Breakthrough Therapy Designation for NXC-201 (The Manila Times) - "Breakthrough Therapy Designation granted to NXC-201 based on positive NEXICART-2 Phase 2 interim clinical results, presented at the American Society of Hematology (ASH) 2025 annual meeting; Final data expected this year followed by planned BLA submission....'We are looking forward to completing enrollment of NEXICART-2 with the goal of delivering this promising therapy to patients upon planned BLA submission this year.'"

Breakthrough therapy • FDA filing • P2 data • Amyloidosis • Immunology

Study of Naive HBI0101 CAR-T Therapy in Relapsed/Refractory Multiple Myeloma (clinicaltrials.gov) - P1 | N=60 | Recruiting | Sponsor: Hadassah Medical Organization

New P1 trial • Hematological Malignancies • Multiple Myeloma • Oncology

At ASH 2025 Oral Presentation, Immix Biopharma Reports Positive Phase 2 NXC-201 Results, Advancing Toward BLA Submission as a Potentially First- and Best-in-Class Therapy for relapsed/refractory AL Amyloidosis (GlobeNewswire) - "After NXC-201 treatment, complete responses (CRs) were observed in 75% (15 out of 20 patients) (at s/u IFE(-) level) by independent review committee. In four out of five pending patients, minimum residual disease (MRD) negativity in bone marrow predicts future complete response, potentially increasing the future CR rate to 95%. Downstream clinical improvement, including organ responses, were observed in 70% of evaluable patients (7/10)....NEXICART-2 final readout and BLA submission are planned for 2026."

FDA filing • P2 data • Amyloidosis • Immunology

2025 Update of Cellular Immunotherapy for Plasma Cell Disorders. (PubMed, Turk J Haematol) - "Ide-cel and Cilta-cel are CAR-T cells directed against BCMA, having received FDA approval for RRMM based on the Phase 2 KarMMa and CARTITUDE trials, respectively...Additional anti-BCMA targeted medicines, including LCAR-B38M, completely humanized CAR-T (FHVH-T), P-BCMA-ALLO-1, ALLO-715, and anti-BCMA CAR-NK, provide promising treatment options. Moreover, the anti-CD19 Fast-CAR, designed to shorten production time, and PHE885, which possesses in-vivo proliferation capability, are regarded as very efficacious...The development of academic CAR-Ts such as ARI0002h, HBI0101, eque-cel, zevor-cel, anito-cel, and Sleeping Beauty (utilizing a non-viral vector) have importance due to their accessibility and cost-effectiveness...To overcome these issues, strategies are being implemented, including combination therapy, the incorporation of gamma-secretase inhibitors etc. In conclusion, CAR-T treatments have evolved into an effective therapy modality being anticipated to be utilized..."

Journal • Amyloidosis • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Oncology • SLAMF7

High Rate of Cytokine Release Syndrome-Related Coagulopathy with Low Incidence of Bleeding and Thrombosis in Patients Treated with B-Cell Maturation Antigen (BCMA)-Targeted Chimeric Antigen Receptor T-Cells (CAR-T). (PubMed, Cancers (Basel)) - P1 | " We conducted a prospective analysis of 108 adult patients with multiple myeloma or light chain amyloidosis treated with the academic anti-BCMA CAR-T HBI0101 in a single-center trial (NCT04720313)...Laboratory markers, tocilizumab and blood product use, and thrombotic and bleeding complications were recorded. Patients received a short (3-day) or extended course of enoxaparin thromboprophylaxis as well as fresh frozen plasma in cases of severe coagulopathy... CRS-related coagulopathy is common following BCMA-targeted CAR-T treatment and correlates closely with CRS severity. Despite the high rate of laboratory coagulopathy, thrombosis and bleeding events were infrequent, suggesting the benefit of the prophylactic strategies used."

IO biomarker • Journal • Cardiovascular • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Neutropenia • Oncology • Thrombosis

Talquetamab induces deep responses in heavily pre-treated patients with systemic light-chain amyloidosis (ASH 2025) - "All were refractory to daratumumab, bortezomib, cyclophosphamide, and pomalidomide; four were refractory to lenalidomide and belantamab mafodotin; one patient with t(11; 14) was refractory to venetoclax. Three relapsed after anti-BCMA academic CAR-T (HBI0101). Additional therapies included ixazomib (2), carfilzomib (1), elotuzumab (1), and melphalan (1)... Talquetamab produced deep and durable hematologic responses in heavily pretreated patients with R/R AL amyloidosis. Assessment of organ response potential was limited by irreversible organ dysfunction at baseline and short follow-up. Treatment was feasible, including in end-stage renal disease, and the safety profile was manageable without unexpected toxicities."

Clinical • Amyloidosis • Cardiovascular • Congestive Heart Failure • Dental Disorders • Heart Failure • Hematological Disorders • Hematological Malignancies • Infectious Disease • Multiple Myeloma • Nephrology • Renal Disease • Septic Shock • Thrombocytopenia • Xerostomia