Abstract
Objective To assess efficacy and safety of cyclical rozanolixizumab treatment, based on study MG0003 with the open-label extensions (OLEs) MG0004 (NCT04124965) and MG0007 (NCT04650854).
Methods MG0004 was an OLE study of ≤52 weekly rozanolixizumab subcutaneous infusions. In MG0007, after an initial cycle, cycles were ‘symptom-driven’, administered on symptom worsening (at investigator’s discretion, e.g., MG-ADL increase ≥2/Quantitative Myasthenia Gravis [QMG] increase ≥3). Data were pooled for patients with ≥2 symptom-driven cycles across MycarinG, MG0004 (first 6 weeks) and MG0007 (interim data) (efficacy); ≥1 cycle across MycarinG (symptom-driven) and MG0007 (fixed/symptom-driven) (safety).
Results 127 (64.8%) patients received ≥2 symptom-driven rozanolixizumab cycles (7mg/kg, n=69; 10mg/kg, n=58). Treatment response was consistent across cycles 1–6; Day 43 mean change from baseline (CFB) in MG-ADL score for all rozanolixizumab-treated patients: Cycle 1 (n=127): −3.7; Cycle 2 (n=127): −3.9; Cycle 3 (n=98): −3.4; Cycle 4 (n=75): −3.8; Cycle 5 (n=51): −3.9; Cycle 6 (n=32): −4.5. Day 43 mean CFB in QMG score was: Cycle 1 (n=127): −5.4; Cycle 2 (n=125): −4.7; Cycle 3 (n=97): −4.7; Cycle 4 (n=74): −5.1; Cycle 5 (n=51): −4.5; Cycle 6 (n=32): −6.3. Myasthenia Gravis Composite scale reductions were consistent across cycles. Patients with >1 year participation had a median of 4 cycles in the first year. Treatment-emergent adverse events (most mild-to-moderate) occurred in 77.4% and 91.6% of patients receiving ≥1 cycle of rozanolixizumab 7mg/kg and 10mg/kg, respectively.
Conclusion Rozanolixizumab efficacy was maintained over up to 6 symptom-driven treatment cycles across multiple MG-specific endpoints with an acceptable safety profile.
Funding: UCB Pharma.
