NXC-201 / Immix Biopharma - LARVOL DELTA

Prospective predictors of toxicity of BCMA‑directed CART therapy in multiple myeloma (ASH 2025) - P1 | "While risk factors forthese toxicities are established in lymphoma, often reflecting high tumor burden and pro-inflammatorystatus, their relevance in MM is unclear, and current evidence is largely limited to retrospective series.Identifying MM-specific predictors may inform patient selection, mitigate toxicity, and aid next-generationCART design.We included all patients with R/R MM enrolled in the NCT04720313 phase 1a/b study using the academicanti‑BCMA CART product HBI0101 who received the target dose of 800×10⁶ CART cells. ECOG >0, older age, anemia, and high involved FLC levels predicted severe inflammatory toxicities afteranti‑BCMA CART. Prior anti‑BCMA therapy (mostly belantamab mafodotin in our cohort) appearedprotective, an observation that may reflect differences in immune milieu or CART expansion kinetics andwarrants further investigation. These predictors outperformed the CAR‑HEMATOTOX score, and appearto have some similarities to what is known in..."

Clinical • Anemia • Hematological Disorders • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Lymphoma • Multiple Myeloma • Neutropenia • Rare Diseases • B2M • CD4 • CD8

First 20-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 (ASH 2025) - P1/2 | "Pts must have been exposed to bortezomib and anti-CD-38 antibodywith persistent or relapsed disease with measurable disease (dFLC >5mg/dl or dFLC >2mg/dl withabnormal k:l ratio, or serum M-spike >0.5g/dl). Lymphodepletion was with fludarabine andcyclophosphamide... In this first 20-patient reported U.S. CAR-T clinical trial experience in RR AL Amyloidosis, wedemonstrate that NXC-201 can be given safely and resulted in rapid and deep hematologic responses inall pts treated. Our data suggests that the novel anti-BCMA CAR-T NXC-201 may become a valuabletreatment option for RR AL pts."

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

BCMA-targeted CAR T-cell therapy in refractory autoimmune diseases: Initial Results from a Phase 1 clinical trial (ASH 2025) - "We conducted a phase 1, open-label, single-centertrial to evaluate the safety and preliminary efficacy of BCMA-targeted CAR T-cell therapy (HBI0101) inpatients with severe, treatment-refractory autoimmune diseases. HBI0101 is an autologous BCMA-targeted CAR T-cell product generated via retroviraltransduction of patient-derived T cells...Tocilizumab was administered to 4 patients, with high dose corticosteroidsneeded in one patient... BCMA-targeted CAR T-cell therapy demonstrated a favorable safety profile in this early-phase trial, with no dose-limiting toxicities or unexpected adverse events. No intervention from the DataSafety Monitoring Board was required. Among four patients with adequate follow-up, all showed signs ofsignificant clinical improvement across diverse autoimmune phenotypes."

CAR T-Cell Therapy • Clinical • P1 data • Amyloidosis • Fibrosis • 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

Summary analysis of the phase 1a/b study with HBI0101, an academic anti-BCMA chimeric antigen receptor T-cell (CART) for relapsed/refractory multiple myeloma (ASH 2025) - P1 | "HBI0101 anti-BCMA CART results demonstrate high efficacy and safety in a more difficult-to-treat population as compared with the registrational studies with commercial products. These data notonly support further utilization of HBI0101 CART therapy, but also demonstrate the feasibility of CARTproduction in an academic setting, ensuring a sufficient CART supply in light of the increasing demand.This study completed enrollment in February 2025, and was immediately followed by a phase 2 study,including R/R MM and AL amyloidosis patients at earlier stages."

CAR T-Cell Therapy • P1 data • Amyloidosis • Anemia • Hematological Disorders • Hematological Malignancies • Inflammation • Multiple Myeloma • Neutropenia • Thrombocytopenia

Immix Biopharma…announced that Phase 1/2 interim results from its U.S. NXC-201 NEXICART-2 trial in relapsed/refractory AL Amyloidosis has been selected for oral presentation at the upcoming American Society of Hematology 2025 Annual Meeting (ASH 2025)… (GlobeNewswire) - "Oral presentation Sunday, December 7, at ASH 2025."

P1/2 data • Amyloidosis

B Cell Maturation Antigen-Directed Chimeric Antigen Receptor T Cell Therapy in Refractory Autoimmune Diseases (KIDNEY WEEK 2025) - "Methods This Phase I, single-center, dose-escalation trial evaluates HBI0101, an autologous BCMA-directed CAR T-cell product developed at Hadassah Medical Center and validated in MM...Conclusion BCMA CAR T-cell therapy induced rapid, substantial improvement in three patients with refractory autoimmune diseases and was well tolerated. These findings underscore the potential of this strategy to redefine treatment in autoimmunity and justify its continued exploration as a targeted, durable, and disease-modifying intervention."

CAR T-Cell Therapy • IO biomarker • Glomerulonephritis • Hematological Malignancies • Immunology • Infectious Disease • Inflammation • Inflammatory Arthritis • Lupus • Lupus Nephritis • Multiple Myeloma • Myositis • Nephrology • Renal Disease • Rheumatoid Arthritis • Rheumatology • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

Immix Biopharma…announced that it will present a NXC-201 abstract at the upcoming American Society of Hematology (ASH) 67th Annual Meeting… (GlobeNewswire)

Clinical data • Amyloidosis

Immix Biopharma Announces 50% Enrollment Milestone Surpassed in its ongoing relapsed/refractory AL Amyloidosis Clinical Trial, NEXICART-2 (GlobeNewswire) - "Immix continues to enroll expediently toward BLA submission."

Enrollment status • Amyloidosis

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 Other Serious Diseases Strategy (GlobeNewswire) - "Immix Biopharma, Inc...announced important updates about its plan to address other serious diseases with its sterically-optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201 with a 'digital filter' that filters out non-specific activation. The Company plans to release forthcoming clinical data at upcoming scientific forums."

Clinical data • Amyloidosis

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