About MAGiNE

GNE myopathy is a slowly progressive muscle disease resulting in marked disability, including wheelchair use, and ultimately requiring dependent care and for which there is no treatment available. This trial will evaluate the efficacy of N-acetyl-D-mannosamine monohydrate (ManNAc) in a randomized-placebo- controlled, double-blind study of ManNAc in subjects with GNE myopathy.

ManNAc, the first committed precursor for the biosynthesis of Neu5Ac (sialic acid), and a substrate of the GNE enzyme defective in GNE myopathy, is being developed for this orphan indication. A first-in- human Phase 1 trial and an Open-Label Phase 2 trial have been completed. NN109 is a randomized, double-blinded, placebo-controlled trial to evaluate the efficacy and safety of ManNAc in subjects with GNE myopathy. We hope that this study will inform future trials and will eventually lead to FDA approval for ManNAc in GNE myopathy.