Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Batoclimab weekly · 2 trials · 2 indications
MG-ADL is an 8-item, participant-reported questionnaire that assesses gMG symptoms and their effects on activities of daily living. Each item is assessed on a 4-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function. Total score ranges from 0 to 24, with higher scores indicating greater functional impairment and disability.
Relapse is defined as a worsening (increase) of \>=1 point on adjusted inflammatory neuropathy cause and treatment (AdjINCAT) score at any time point during Period 2 relative to Period 2 Baseline which is sustained at a Follow-Up visit 1 week later. The INCAT disability scale is a widely used and validated efficacy assessment of neurologic dysfunction in CIDP. Upper and lower limb dysfunction are each separately assessed on a scale of 0-5 and results are summed together for a total composite score of 0-10. Higher scores represent greater disability. The Adj INCAT disability score is identical to INCAT disability score with exception that changes in upper limb function from 0 (normal) to 1 (minor symptoms) and vice versa are excluded since minor symptoms in the fingers, which are implied by an upper limb score of 1, are not considered clinically significant in all participants. The Adj INCAT disability score will be used for participant selection and measurement of clinical response.
| Arm | Type | Description |
|---|---|---|
| Batoclimab Induction Dose 1 (Period 1) | EXPERIMENTAL | - |
| Batoclimab Induction Dose 2 (Period 1) | EXPERIMENTAL | - |
| Placebo Induction Dose (Period 1) | PLACEBO_COMPARATOR | - |
| Batoclimab Maintenance Dose 1 (Period 2) | EXPERIMENTAL | - |
| Batoclimab Maintenance Dose 2 (Period 2) | EXPERIMENTAL | - |
| Placebo Maintenance Dose (Period 2) | PLACEBO_COMPARATOR | - |
| Treatment Period 1: Cohort A, Dose 1 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort A, Dose 2 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort B, Dose 1 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort B, Dose 2 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort C, Dose 1 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort C, Dose 2 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort D, Dose 1 | EXPERIMENTAL | - |
| Treatment Period 1: Cohort D, Dose 2 | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort A, Dose 2 | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort A, Placebo | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort B, Dose 2 | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort B, Placebo | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort C, Dose 2 | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort C, Placebo | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort D, Dose 2 | EXPERIMENTAL | - |
| Withdrawal Period 2: Cohort D, Placebo | EXPERIMENTAL | - |
| LTE Period: With Relapse in Period 2: Dose 1 and Dose 2 | EXPERIMENTAL | Participants will receive Dose 1 for the initial 4 weeks only and Dose 2 for the remaining 48 weeks. |
| LTE Period: Without Relapse in Period 2: Dose 2 | EXPERIMENTAL | Participants will receive Dose 2 for all 52 weeks. |
| Name | Type | Description |
|---|---|---|
| Batoclimab 680 mg SC weekly | DRUG | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
| Batoclimab 340 mg SC weekly | DRUG | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
| Matching Placebo SC | DRUG | Placebo |
| Batoclimab 340 mg SC bi-weekly | DRUG | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
| Batoclimab 680 milligrams (mg) subcutaneous (SC) weekly | DRUG | Batoclimab is a fully human anti-neonatal fragment crystallizable receptor (FcRn) monoclonal antibody |
| Placebo | DRUG | Matching placebo SC |
Inclusion Criteria: 1. Are ≥ 18 years of age at the Screening Visit. 2. Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification Class II, III, or IVa at the Screening Visit. 3. Have a QMG score ≥ 11 at the Screening and Baseline Visits. 4. Have a MG-ADL score of ≥ 5...