Roche to examine tominersen in younger adult patients with Huntington’s disease

Roche is designing a new phase 2 trial to examine tominersen in Huntington’s disease, according to a press release from Roche’s partner, Ionis Pharmaceuticals Inc.

Tominersen is an investigational antisense medicine intended to reduce the production of all forms of the huntingtin protein. Following cessation of dosing in the phase 3 GENERATION HD1 trial, exploratory post-hoc analyses showed a possible benefit of tominersen among younger adult patients with lower disease burden; however, a randomized, placebo-controlled study is needed to confirm these results.

“These findings are promising and warrant a new study designed to test tominersen in this specific patient group,” C. Frank Bennett, PhD, Ionis' executive vice president, chief scientific officer and franchise leader for neurological programs, said in the release. “We are pleased that Roche has determined that there is a path to advance the tominersen development program. This is an encouraging development for the HD community.”

Roche will explore various tominersen doses in this patient population with less disease burden and then share additional details regarding the design of the new trial at future scientific meetings.