AbelZeta Pharma Presents Promising Early Clinical Data for C-CAR168 in Refractory Autoimmune Diseases at LUPUS 2025

ROCKVILLE, Md., May 22, 2025 – AbelZeta Pharma, Inc. (“AbelZeta”), a global clinical-stage biopharmaceutical company focused on innovative cell-based therapeutic products, today announced the presentation of encouraging Phase 1 first-in-human investigator-initiated trial (IIT) data for its novel therapy, C-CAR168 (NCT06249438). The early clinical results in patients with refractory autoimmune disease were shared by Principal Investigator Professor Nan Shen from Ren Ji Hospital, Shanghai Jiao Tong University School of Medicine, in a podium presentation at the 16th International Congress on Systemic Lupus Erythematosus (“LUPUS 2025”) in Toronto, Canada.

As of February 28, 2025, ten patients have been treated with C-CAR168, including seven with Lupus Nephritis (LN), one with Secondary Progressive Multiple Sclerosis (SPMS), one with Neuromyelitis Optica Spectrum Disorder (NMOSD), and one with Immune-Mediated Necrotizing Myopathy (IMNM).

Key Highlights from the Study:

• Favorable Safety Profile: C-CAR168 was generally well tolerated. Low-grade (1-2) Cytokine Release Syndrome (CRS) was observed in four LN patients and one SPMS patient, with a median onset of 2 days post-treatment. Notably, neither immune effector cell-associated neurotoxicity syndrome (ICANS) nor severe infections were reported.
• Robust Mechanism of Action: The therapy demonstrated robust C-CAR168 expansion and rapid, complete depletion of B cells, CD20dim T cells, and plasma cells in peripheral blood. Complete elimination of B cells and long-lived plasma cells was also observed in the bone marrow of the single patient examined. Evidence of C-CAR168-induced immune reset was observed through peripheral blood flow cytometry and RNA sequencing in LN patients.
• Promising Efficacy in LN: All four LN patients who reached the 6-month evaluation timepoint achieved and maintained a Systemic Lupus Erythematosus Responder Index (SRI(4)) response, with two achieving Complete Remission (CR) and one Partial Remission (PR) based on Kidney Disease: Improving Global Outcomes (KDIGO) 2024 LN Response Criteria. Most patients were able to discontinue immunosuppressive therapy (IS)/biologics after lymphodepletion and become steroid-free post C-CAR168 infusion. One LN patient experienced a flare before the 6-month mark.
• Encouraging SPMS Efficacy Signal: The treated SPMS patient showed a very promising early efficacy signal, including improvements in gait and orbital movement, significant reduction of brain inflammation and lesion size, and improvements in EDSS, 9-HPT, T25-FW, and biomarker levels.

“The early clinical results for C-CAR168 represent a significant stride forward in treating patients suffering from severe and refractory manifestations of Lupus Nephritis and Secondary Progressive Multiple Sclerosis, conditions with limited effective treatment options,” said Tony (Bizuo) Liu, AbelZeta Pharma’s Chairman and CEO. “These findings underscore the potential of our bi-specific CAR-T platform, expanding beyond anti-CD20/19 bispecific CAR-T for NHL to now include C-CAR168, an anti-CD20/BCMA therapy, for autoimmune diseases. Successful treatment with C-CAR168 holds the promise of enabling patients to discontinue long-term immunosuppressive therapy and substantially reduce disease activity. We look forward to collaborating closely with the FDA and the scientific community as we advance towards Phase 1b and Phase 2 studies.”

About the C-CAR168 Study (NCT06249438) This investigator-initiated, multicenter, open-label Phase 1 study is evaluating C-CAR168 for adult patients with autoimmune and neurological diseases, including refractory Lupus Nephritis (LN), Systemic Lupus Erythematosus (SLE), and progressive Multiple Sclerosis (MS) who are resistant to standard therapy. As of February 28, 2025, seven patients with refractory LN received C-CAR168 (four at 0.75×10^6 cells/kg and three at 1.5×10^6 cells/kg). The treated population had long-standing refractory disease (median SLE duration 9 years, LN 5 years) and had previously been exposed to a median of four immunosuppressive or biologic agents.

About C-CAR168 C-CAR168 is a novel autologous bi-specific CAR-T therapy engineered to target both CD20 and B-cell maturation antigens (BCMA). The U.S. Food and Drug Administration (FDA) has granted clearance for the Investigational New Drug (IND) application for C-CAR168. Preclinical data for C-CAR168 was previously presented at the American College of Rheumatology (ACR) Convergence 2024 in Washington, DC, in November 2024.