A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects with Non-Oncology Plasma Cell-Related Diseases

Objective:

Primary Objective:

• The primary objective of this study is to assess the safety profile, including any DLT and identification of a MTD (if applicable), to support selection of the RP2D of anito-cel when administered to subjects with GMG.

Secondary Objectives:

• To quantify the clinical effect of anito-cel using standard clinical assessments, including Quantitative Myasthenia Gravis (QMG), Myasthenia Gravis Quality of Life (MG QoL 15R), Myasthenia Gravis Activities of Daily Living (MG ADL), MG Composite (MGC), Myasthenia Gravis Foundation of America (MGFA) Class, and MG Post Intervention Status (MG PIS)
• To determine the change from Baseline in the titer of myasthenia gravis-specific autoantibodies in all subjects following infusion of anito-cel
• To evaluate the PK in subjects treated with anito-cel in peripheral blood by VCN
• To characterize the treatment-free interval (period during which no non-steroidal therapy, including immunosuppressive therapy, is required) following cell infusion
• To characterize the corticosteroid dose reduction following cell infusion
• To determine the rate of disease related hospitalizations or treatment complications following treatment and descriptively compare to the rate of occurrence prior to treatment
• To assess for the presence of RCL in subjects