NIMBLE Trial Shows Efficacy of Cemdisiran in gMG: Tuan Vu, MD

Tuan Vu, MD, professor and division director of Neuromuscular Medicine & Electromyography at USF Health, spoke with The American Journal of Managed Care^® about the results of the NIMBLE trial, which will be presented at the 2026 American Academy of Neurology (AAN) Annual Meeting, held from April 18-22, 2026.

The NIMBLE trial (NCT05070858) is a randomized control trial that took place across the globe for 24 weeks. The goal was to evaluate the efficacy of cemdisiran monotherapy compared with placebo, pozelimab monotherapy, and the combination of cemdisiran and pozelimab in patients with generalized myasthenia gravis (gMG).

The primary end point was a change in score in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale.

The trial met its primary end point, as cemdisiran was considered more effective than placebo or pozelimab monotherapy. “Both the combo and the cemdisiran arm [were] statistically significant in terms of improvement vs placebo,” Vu explained.

These results were also corroborated when doing a responder analysis when the researchers used the Quantitative Myasthenia Gravis (QMG) score as the scale for the analysis. The responder analysis was defined as a 3-point improvement in the MG-ADL and a 5-point improvement in the QMG, which were found in both the cemdisiran group and the combination group compared with the placebo group.

“Overall, the result was fairly consistent across the board and both statistically and clinically significant,” said Vu.

These results were initially previewed in a press release in August 2025,¹ when Regeneron announced positive results from the NIMBLE trial. This announcement claimed that the phase 3 trial had met its primary end points as well as the key secondary end point of change in QMG score from baseline to the end of the study at 24 weeks. The official results were presented during the 2026 AAN Annual Meeting as part of the conference’s coverage of new results in neurology.

Reference

1. Regeneron announces positive results from phase 3 trial in generalized myasthenia gravis. News release. Regeneron. August 26, 2025. Accessed April 17, 2026. https://investor.regeneron.com/news-releases/news-release-details/regeneron-announces-positive-results-phase-3-trial-generalized