B cell Maturation Antigen Targeted Chimeric Antigen Receptor T Cell Therapy for Refractory Systemic Lupus...

Area of Science:

• Immunotherapy
• Rheumatology
• Cellular Therapy

Background:

• Systemic lupus erythematosus (SLE) and systemic sclerosis (SSc) are severe autoimmune diseases with limited treatment options.
• Refractory cases of SLE and SSc often require aggressive immunosuppression with significant side effects.
• Novel therapeutic strategies are needed to address the unmet medical needs in these conditions.

Purpose of the Study:

• To evaluate the safety and preliminary efficacy of HBI0101, a novel B-cell maturation antigen-targeted chimeric antigen receptor (CAR) T-cell therapy.
• To assess HBI0101 in patients with severe, refractory systemic lupus erythematosus (SLE) and systemic sclerosis (SSc).

Main Methods:

• An ongoing phase 1 clinical trial involving six patients (three with SLE, three with SSc).
• Patients had a median age of 41 years and a median disease duration of 2.7 years.
• Median follow-up was 6 months, with patients having received a median of 3.5 prior lines of immunomodulatory therapy.

Main Results:

• Grade 3-4 neutropenia occurred in two patients, managed with supportive care.
• Cytokine-release syndrome (CRS) was observed in five patients (grade 1 or 2); no immune effector cell-associated neurotoxicity syndrome (ICANS) was noted.
• Significant clinical improvements were seen in SLE patients (SLEDAI-2K decreased to 0 in two) and SSc patients (skin involvement improved in all).

Conclusions:

• HBI0101 demonstrated an acceptable safety profile in this early-phase trial.
• Preliminary clinical activity was observed in patients with severe refractory SLE and SSc.
• These findings support further prospective evaluation of HBI0101 for these autoimmune conditions.