Pozelimab and cemdisiran combo holds potential in MG market

16 September 2025

US biotech Regeneron (Nasdaq: REGN) recently announced exciting results from a Phase III clinical trial called NIMBLE, evaluating the efficacy and safety of pozelimab + cemdisiran combination therapy and cemdisiran monotherapy for adult patients living with generalized myasthenia gravis (gMG).

The results showed that both cemdisiran monotherapy and pozelimab + cemdisiran combination therapy met their primary endpoints, displaying a significant improvement over 24 weeks, as measured by reductions in the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale score. This means that pozelimab and cemdisiran combination has the potential to challenge currently marketed disease-modifying therapies (DMTs), says pharma analytics company GlobalData.

Jos Opdenakker, neurology analyst at GlobalData, comments: “Regeneron’s pozelimab and Alnylam Pharmaceuticals’ cemdisiran work through mediating C5 complement inhibition. C5 is a protein that is thought to be involved in the damage of acetylcholine receptors and is a frequent target of DMTs within the MG treatment landscape. In the NIMBLE trial, the results were statistically significant, achieving the trial’s primary endpoint of a significant improvement in MG-ADL score.”

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