Roche Licenses Promising Huntington’s Therapy IONIS-HTTRx, Begins Extension Study

Roche will now be responsible for the development and marketing of Ionis Pharmaceuticals’ IONIS-HTTRx after exercising its option to license the investigational drug, designed to treat Huntington’s disease (HD). This move follows highly promising results from a Phase 1/2a clinical trial. The randomized, double-blind, placebo-controlled trial (NCT02519036) tested…

Investigational Drug, Olesoxime, Seen to Ease Huntingtin Fragmentation in Mouse Model

Beneficial effects of the investigational neuroprotective drug olesoxime have mainly been attributed to its effects on mitochondria, but new research suggests it also blocks calpain — a molecule directly involved in Huntington’s disease. The findings indicate that exploring the drug’s exact mechanism might bring scientists closer to effective treatments for Huntington’s.