C-CAR168 / AbelZeta Pharma - LARVOL DELTA

AbelZeta Receives FDA IND Clearance for Phase 1b/2 Clinical Trial of C-CAR168 in Progressive Multiple Sclerosis (PRNewswire)

IND • New P1/2 trial • Multiple Sclerosis

A Study of C-CAR168 in Participants With Progressive Multiple Sclerosis (clinicaltrials.gov) - P1/2 | N=119 | Not yet recruiting | Sponsor: AbelZeta Inc.

New P1/2 trial • CNS Disorders • Multiple Sclerosis

C-CAR168 CAR T-Cell Therapy for the Treatment of Lupus Nephritis Refractory to Standard Therapy (clinicaltrials.gov) - P2 | N=50 | Not yet recruiting | Sponsor: AbelZeta Inc.

IO biomarker • New P2 trial • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology • Systemic Lupus Erythematosus

AbelZeta Receives FDA Clearance of Registrational Phase II Trial for C-CAR168 in Refractory Lupus Nephritis (PRNewswire) - "The Phase II trial will further evaluate the safety and efficacy of C-CAR168 in this patient population with significant unmet medical needs."

IND • New P2 trial • Lupus Nephritis

AbelZeta Pharma…announced that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has granted PRIority MEdicines (PRIME) designation for C-CAR168…for the treatment of refractory Systemic Lupus Erythematosus (SLE), with or without Lupus Nephritis (LN) (PRNewswire) - "Building on the clinical promise demonstrated by earlier CAR-T therapies, AbelZeta has applied extensive expertise in developing dual-target CAR-T technology to engineer its dual anti-CD20/BCMA-targeted CAR-T."

PRIME • Lupus Nephritis • Systemic Lupus Erythematosus

C-CAR168 CAR T Cell Therapy for Refractory Autoimmune Disease (clinicaltrials.gov) - P1/2 | N=0 | Withdrawn | Sponsor: AbelZeta Inc. | N=24 ➔ 0 | Not yet recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy (clinicaltrials.gov) - P1 | N=18 | Not yet recruiting | Sponsor: The Affiliated Hospital of Qingdao University

IO biomarker • New P1 trial • CNS Disorders • Immunology • Inflammatory Arthritis • Lupus • Multiple Sclerosis • Myasthenia Gravis • Myositis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Scleroderma • Systemic Lupus Erythematosus • Systemic Sclerosis

C-CAR168 CAR T Cell Therapy for Refractory Autoimmune Disease (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: AbelZeta Inc. | Trial completion date: Dec 2029 ➔ Jun 2030 | Initiation date: Jan 2026 ➔ Jul 2026 | Trial primary completion date: Nov 2028 ➔ May 2029

Trial completion date • Trial initiation date • Trial primary completion date • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

A Study of C-CAR168 in the Treatment of Central Nervous System Autoimmune Diseases Refractory to Standard Therapy (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Huashan Hospital

New P1 trial • CNS Disorders • Immunology • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases

Anti-CD20/BCMA Bispecific CAR-T Cell Therapy Promotes Immune Reset and Sustained Drug Free Remission in Refractory Autoimmune Diseases (ACR Convergence 2025) - P1 | "C-CAR168 therapy demonstrated durable treatment-free disease control in refractory autoimmune diseases. Its favorable safety profile, together with a renal response rate of 77.8% in refractory proliferative LN and rapid neurological improvement in SPMS, both achieved without background standard of care medications, coupled with mechanistic evidence of immune reset, provides a compelling rationale for further clinical evaluation of C-CAR168."

CAR T-Cell Therapy • Clinical • IO biomarker • Late-breaking abstract • CNS Disorders • Glomerulonephritis • Hematological Disorders • Immunology • Infectious Disease • Inflammatory Arthritis • Lupus • Lupus Nephritis • Multiple Sclerosis • Myositis • Nephrology • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Renal Disease • NEFL

AbelZeta Pharma…announced that clinical data from the first-in-human Phase I trial (NCT06249438) of C-CAR168 in patients with refractory autoimmune diseases will be presented as a late-breaking poster presentation at American College of Rheumatology (ACR) Convergence 2025 (PRNewswire)

Late-breaking abstract • P1 data • Multiple Sclerosis • Myasthenia Gravis • Neuromyelitis Optica Spectrum Disorder • Systemic Lupus Erythematosus • Systemic Sclerosis

C-CAR168 CAR T Cell Therapy for Refractory Autoimmune Disease (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: AbelZeta Inc. | Initiation date: Jul 2025 ➔ Jan 2026

Trial initiation date • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

AbelZeta Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for C-CAR168 for the Treatment of Refractory SLE, Including LN (PRNewswire) - "AbelZeta Pharma, Inc...announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational therapy, C-CAR168, for the treatment of refractory Systemic Lupus Erythematosus (SLE), including Lupus Nephritis (LN)...The RMAT designation, granted under Investigational New Drug (IND) 30283, is based on promising early clinical data demonstrating the potential of C-CAR168 to address serious unmet medical needs in patients with treatment-resistant forms of autoimmune disease....AbelZeta plans to request a multidisciplinary type B meeting with the FDA to discuss the next steps in the clinical and manufacturing development of C-CAR168."

FDA event • Lupus Nephritis • Systemic Lupus Erythematosus

AbelZeta Presents New Data on C-CAR168 for the Treatment of Autoimmune Diseases at LUPUS 2025 (PRNewswire) - P1 | N=30 | CAR-AID (NCT06249438) | "Overall, 10 patients have been treated with C-CAR168 including 7 LN patients, 1 Secondary Progressive Multiple Sclerosis (SPMS), 1 Neuromyelitis Optica Spectrum Disorder (NMOSD) and 1 Immune-Mediated Necrotizing Myopathy (IMNM) patient as of February 28, 2025; Early clinical data showed that C-CAR168 was well tolerated. Four LN patients and one SPMS patient experienced low-grade 1-2 CRS with median time to onset of 2 days post C-CAR168 treatment....Four LN patients treated with C-CAR168 reached the 6-month evaluation timepoint. All 4 achieved and remained in SRI (4), 2 patients achieved CR, and 1 PR based on Kidney Disease: Improving Global Outcomes (KDIGO) 2024 LN Response Criteria."

P1 data • Lupus Nephritis • Multiple Sclerosis • Neuromyelitis Optica Spectrum Disorder • Systemic Lupus Erythematosus

AbelZeta Announces Upcoming Oral Presentations at LUPUS 2025 (PRNewswire) - "AbelZeta Pharma, Inc...today announced the acceptance of an abstract related to C-CAR168 for presentation at the 16th International Congress on Systemic Lupus Erythematosus ('LUPUS 2025'), taking place in Toronto, Canada on May 21-24, 2025."

P1 data • Lupus Nephritis

C-CAR168 CAR T Cell Therapy for Refractory Autoimmune Disease (clinicaltrials.gov) - P1/2 | N=24 | Not yet recruiting | Sponsor: AbelZeta, Inc.

New P1/2 trial • Glomerulonephritis • Immunology • Inflammatory Arthritis • Lupus • Lupus Nephritis • Nephrology

AbelZeta Presents Preclinical Data from Study of C-CAR168 for the Treatment of Autoimmune Diseases at ACR Convergence 2024 (PRNewswire) - P1 | N=30 | NCT06249438 | "Potent In Vivo Cytotoxicity: In immunodeficient mouse models, a single dose of C-CAR168 demonstrated significant activity across multiple dosage levels, effectively inhibiting the growth of CD20 and BCMA single-positive or double-positive in various xenograft models....Clinical Development for C-CAR168: In an investigator-initiated trial in China targeting refractory/relapsing LN patients, 7 patients have been treated in a suboptimal dose and a potentially optimal dose. Depletion of both B cells and plasma cells in the blood in all patients were observed, accompanied by a favorable safety profile and early positive clinical signals at the suboptimal dose from patients that have had at least 3 months of follow up. Actively looking to expand into new indications such as Neuromyelitis Optica Spectrum Disorder (NMOSD), Immune-Mediated Necrotizing Myopathy (IMNM), and Systemic Sclerosis (SSc) to provide guidance for global trial to be initiated around mid 2025."

New trial • P1 data • Preclinical • Lupus Nephritis • Neuromyelitis Optica Spectrum Disorder • Systemic Sclerosis

C-CAR168 as a Novel Anti-CD20/BCMA Bispecific Autologous CAR-T Therapy for the Treatment of Autoimmune Diseases (ACR Convergence 2024) - "Based on the promising in vitro/in vivo efficacy results and safety data, C-CAR168 is being evaluated in clinical trials to treat patients with a variety of autoimmune diseases that are refractory to standard therapy."

B Cell Non-Hodgkin Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Immunology • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology • IL2

CAR-AID: A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy (clinicaltrials.gov) - P1 | N=30 | Recruiting | Sponsor: RenJi Hospital | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders • Immunology • Inflammatory Arthritis • Lupus • Multiple Sclerosis • Myositis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Systemic Lupus Erythematosus • IFNG • IL10 • IL2 • IL4 • IL6 • TNFA

CAR-AID: A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy (clinicaltrials.gov) - P1 | N=30 | Not yet recruiting | Sponsor: RenJi Hospital

New P1 trial • CNS Disorders • Immunology • Inflammatory Arthritis • Lupus • Multiple Sclerosis • Myositis • Neuromyelitis Optica Spectrum Disorder • Rare Diseases • Systemic Lupus Erythematosus • IFNG • IL10 • IL2 • IL4 • IL6 • TNFA