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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.


RTTNews | Jan 18, 2022 01:38AM EST

01:38 Tuesday, January 18, 2022 (RTTNews.com) - 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.

Read the original article on RTTNews ( https://www.rttnews.com/3255438/ionis-partner-roche-set-to-evaluate-tominersen-for-huntington-s-disease-in-new-phase-2-trial.aspx)

For comments and feedback: contact editorial@rttnews.com

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