Ionis' Partner Roche Set To Evaluate Tominersen For Huntington's Disease In New Phase 2 Trial

(RTTNews) - Ionis Pharmaceuticals, Inc. (IONS) said on Tuesday that its partner, Roche, is designing a new Phase 2 trial to evaluate tominersen, for Huntington's disease or HD, a rare genetic, progressive condition that causes the brain nerve cells to break down, leading to a disability.

The move follows the exploratory post-hoc analyses of Phase 3 GENERATION HD1 study, which suggested tominersen may benefit younger adult patients with lower disease burden. However, these results require further confirmation in a randomized, placebo-controlled study.

Roche is in the early stages of designing the Phase 2 clinical trial to explore different doses of tominersen in a younger adult patient population with less disease burden.

Roche has licensed the investigational drug tominersen, previously IONIS-HTTRx or RG6042, from Ionis in 2017. It is designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT.