Table 2.
Methodology of the phase 2 and phase 3 results.
| Therapeutic name | Phase | Treatment arms | Inclusion | Sample size | Route and dose of administration | Primary end-points | Secondary endpoints |
|---|---|---|---|---|---|---|---|
| Efgartigimod | 2 | Double blinded RCT (1:1). | AChR+gMG MG-ADL ≥ 5 MGFA II-IV |
24 | 10 mg/kg efgartigomod or placebo IV wkly 4 doses over 3 wks | Safety and efficacy | Change from baseline to 11 weeks: QMG MG-ADL MGC MGQoL15r |
| Efgartigimod | 3 | Double blinded RCT (1:1) | gMG MG-ADL ≥ 5 MGFA II-IV |
167 | 10 mg/kg efgartigomod or placebo IV wkly 4 doses and then repeat dosing if needed | MG-ADL responders. | QMG responders Early MG-ADL responders |
| Rozanolixizumab | 2 | Double blinded 2 Period RCT (1:1). | gMG QMG ≥11 | 43. | Period 1: 7 mg/kg rozanolixizumab. or placebo Qwkly for 3wks. Period 2: 7 mg/kg or 4 mg/kg Rozanolixizumab Qwkly for 3wks |
Change from baseline to day 29: QMG. | Change from baseline to day 29: MG-ADL MGC Safety |
| Rozanolixizumab | 3 | Double blinded RCT (1:1:1) | ACHR+ MG Musk+MG MGFA II-IVa MG-ADL ≥ 3 |
200 | 7 mg/kg Qwkly or 10 mg/kg Qwkly of rozanolixizumab for 6 wks. | Change from baseline to day 43: MG-ADL | Change from baseline to day 43: MGC QMG |
| Nipocalimab | 2 | Double blinded 5 arm RCT (1:1:1: 1:1) |
gMG | 68 | 5 mg/kg q4wkly 30 mg/kg q4wkly 60 mg/kg q2wkly 60 mg/kg q2wkly, or placebo q2wkly for 8 weeks |
Change from baseline to day 57 of MG-ADL Effect on total IgG and anti-ACHR ab level |
Change from baseline to day 57: QMG, MG-QoL15r |
| Nipocalimab | 3 | Double blinded RCT (1:1) | AChR+ gMG MGFA II-Iva MG-ADL ≥ 6 |
30 mg/kg at first infusion, 15 mg/kg thereafter Q2wks for 24 wks | Change from baseline to wks 22,23,24 of MG-ADL | ||
| Batoclimab | 2 | Double blinded 3arm RCT (1:1:1) with OLE | AChR+ gMG MGFA II-Iva QMG score ≥ 12 |
15 | Batoclimab 680 mg, Batoclimab 340 mg, Placebo Qwkly SC for 6wks | Safety and efficacy | Change from baseline to day 43: QMG MG-ADL MG-QoL15r |
| Batoclimab | 3 | Quadruple blinded 3 arm 2 period RCT (1:1:1) with OLE | gMG MGFA II-Iva QMG score ≥ 11 MG-ADL ≥ 5 |
Period 1: Batoclimab 680 mg, Batoclimab 340 mg, Placebo Qwkly SC for 6wks. Period 2: Batoclimab 340 mg QWkly, Batoclimab 340 mg Q2Wkly, Placebo Qwkly SC for 6wks |
Change from baseline to 12 wks of MG-ADL In ACHR+MG | Change from baseline to 12 wks of QMG in ACHR+MG Change from baseline to 24 wks of MG-ADL, QMG in ACHR+MG |