Switzerland-based MetrioPharm AG has received a rare paediatric disease designation from the US Food and Drug Administration for its candidate therapy for Duchenne muscular dystrophy (DMD). The drug, MP1032, is a small molecule immune modulator that targets dysregulated mitochondrial metabolism in macrophages. In a preclinical study it was able to increase muscle strength comparable to the corticosteroid prednisolone, but without the corticosteroid’s serious side effects.
