A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG). Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.
Subject must be 18 years of age or older
Must have MGFA clinical classification Grades 2-4A at time of screening
Subject must have clinically active disease and requiring ongoing therapy for GMG
MG-ADL score 6 and QMG score >10 at screening
Subject is pregnant or breastfeeding
Treatment with Anti-CD20 agents, calcineurin inhibitors, FcRN inhibitors, azathioprine, mycophenolate mofetil, methotrexate, or cyclophosphamide within the specified time frame prior to leukapheresis or prior to anito-cel infusion
Previous treatment with any gene therapy, chimeric antigen receptor therapy or T cell engager
Previous thymectomy within 6 months of screening
Single group
anito-cel
Biological/VaccineAnitocabtagene autoleucel BCMA directed CAR T-cell therapy using a novel, synthetic binding domain, called a D-Domain
Standard Lymphodepletion regimen
DrugStandard lymphodepletion regimen subject receive 5 days prior to CAR T infusion
Assess safety profile, including any DLT and MTD (if applicable)
Type, incidence, and severity of treatment-emergent adverse events (TEAEs), including DLT(s) and laboratory abnormalities
Selection of RP2D
Evaluate the MTD and establish the RP2D
Quantify Clinical Effect of Anito-cel in the Myasthenia Gravis Activities of Daily Living (MG ADL) score
The proportion of subjects achieving a ≥2-point change in the MG ADL score from Baseline at any timepoint after treatment. The MG-ADL is an 8-item patient-reported outcome measure assessing GMG symptoms and functional activities related to activities of daily living and producing a total score ranging from 0 to 24, where higher scores indicate greater severity of disease symptoms.
Quantify Clinical Effect of Anito-cel in the Quantitative Myasthenia Gravis (QMG) score
The proportion of subjects achieving a ≥3-point change in the QMG from Baseline at any timepoint after treatment. The QMG score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicate greater disease severity.
Quantify Clinical Effect of Anito-cel in the Myasthenia Gravis Composite (MGC) scale.
The proportion of subjects achieving a ≥3-point change in the MGC score from Baseline at any timepoint after treatment. The MGC is a 10-item assessment that measures signs and symptoms of GMG based on physical examination findings and patient history. The total score ranges from 0 to 50, where higher scores indicate more severe impairment.
Mean change in QMG score
The mean change in the QMG score from Baseline at any timepoint after treatment. The QMG score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicate greater disease severity.
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