[Correspondence] Unanswered questions from the NIMBLE trial

AI Summary

The correspondence questions the interpretation of the NIMBLE trial of cemdisiran alone and in combination with pozelimab for generalised myasthenia gravis. It notes that the reported clinical efficacy and complement blockade findings may be important but says three methodological issues remain unresolved.

Tuan Vu and colleagues reported that cemdisiran monotherapy (76·6% mean reduction from baseline in total complement haemolysis activity [CH50] inhibition) and cemdisiran–pozelimab combination therapy (99·6% CH50 inhibition) achieved similar clinical efficacy in patients with generalised myasthenia gravis, concluding that “substantial but incomplete complement blockade might be sufficient” for disease control.1 Although this finding is potentially important, three methodological issues remain unaddressed.