AbelZeta Unveils Promising Data on C-CAR168 for Autoimmune Diseases at LUPUS 2025 Event

AbelZeta Pharma, Inc., a renowned global biopharmaceutical innovator specializing in cell-based therapies, has recently shared exciting updates regarding its C-CAR168 treatment for autoimmune diseases. The data was presented during the prestigious LUPUS 2025 conference held in Toronto, Canada. This conference gathers experts in the field to discuss advances in research and treatment strategies for lupus and related illnesses.

Introduction to C-CAR168

C-CAR168 is an innovative autologous bispecific CAR-T therapy that targets both CD20 and B-cell maturation antigens (BCMA). Designed primarily for patients suffering from refractory autoimmune conditions such as lupus nephritis and multiple sclerosis, this groundbreaking therapy aims to provide a new avenue for those who have limited treatment options.

The Phase 1 first-in-human investigator-initiated trial (IIT), known as NCT06249438, has garnered significant attention due to its promising early results. The study, directed by Professor Nan Shen from the Department of Rheumatology at Ren Ji Hospital, Shanghai Jiao Tong University, has treated a mix of patients with various autoimmune diseases.

Key Findings from the Study

As of late February 2025, a total of ten patients participated in the trial, which included seven diagnosed with lupus nephritis (LN), one with secondary progressive multiple sclerosis (SPMS), one with neuromyelitis optica spectrum disorder (NMOSD), and another with immune-mediated necrotizing myopathy (IMNM).

The early clinical data from the study demonstrated that C-CAR168 was very well tolerated. Notably, four patients with lupus nephritis and one SPMS patient experienced only low-grade cytokine release syndrome (CRS) with mild symptoms, showcasing the treatment's safety profile. Importally, neither severe infections nor immune effector cell-associated neurotoxicity syndrome (ICANS) were reported, indicating a favorable safety outcome.

Mechanism of Action and Clinical Impact

The trial has provided evidence of C-CAR168's mechanism of action, showing robust expansion and effective depletion of harmful B cells that contribute to autoimmune diseases. Significant immune responses were observed in participants, with complete elimination of B cells and long-lived plasma cells in those whose bone marrow was examined. Such outcomes are critical, as they reveal the therapy's potential for changing autoimmunity pathways.

At the six-month evaluation point, remarkable results were shared. Four of the lupus nephritis patients achieved sustained remission, while two reached complete response and one partial remission according to the Kidney Disease Improving Global Outcomes (KDIGO) criteria.

Furthermore, a promising trend was noted in the SPMS patient whose condition showed marked improvement, including enhancements in gait and reduced brain inflammation, a vital aspect of recovery from multiple sclerosis symptoms.

Future Implications and Corporate Strategy

The preliminary outcomes from this study are significant not just for the patients involved but also for the future direction of AbelZeta. Tony Liu, the Chairman and CEO, highlighted the findings as pivotal in expanding their bi-specific CAR-T platform, which is now inclusive of C-CAR168 targeted at autoimmune diseases. The prospect of allowing patients to discontinue their dependence on immunosuppressive therapies stands to radically change their quality of life and improve clinical outcomes.

In light of these developments, AbelZeta commits to collaborating with regulatory bodies like the FDA to advance C-CAR168 into subsequent clinical phases, potentially leading to broader accessibility for patients across various autoimmune disorders. This direction aligns with AbelZeta's mission to innovate therapies that engage the body's immune system to combat complex diseases effectively.

Conclusion

The information unveiled at LUPUS 2025 not only sheds light on the potential of C-CAR168 but also emphasizes AbelZeta's role as a frontrunner in addressing unmet medical needs for autoimmune diseases. As research and development continues, many hope that C-CAR168 will bring about change and relief in the lives of patients battling the complexities of autoimmune conditions.