Ionis’ partner to evaluate tominersen for Huntington’s disease in new Phase 2 trial

– Post-hoc analysis of GENERATION HD1 study data showed tominersen may have potential benefit in a subgroup of patients
– Ionis’ partner Roche is in early stages of designing trial for younger adult patient population with less disease burden
– Partner to participate in series of webinars for scientific and patient communities beginning Jan. 20, 2022

CARLSBAD, Calif., Jan. 18, 2022 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that its partner, Roche, is designing a new Phase 2 trial to evaluate tominersen in Huntington’s disease (HD). After halting dosing in the Phase 3 GENERATION HD1 study, exploratory post-hoc analyses suggest tominersen may benefit younger adult patients with lower disease burden. These results require confirmation in a randomized, placebo-controlled study.  

“These findings are promising and warrant a new study designed to test tominersen in this specific patient group. We are pleased that Roche has determined that there is a path to advance the tominersen development program,” said C. Frank Bennett, Ph.D., Ionis’ executive vice president, chief scientific officer and franchise leader for neurological programs. “This is an encouraging development for the HD community. We and Roche are grateful to the HD community’s continued partnership, which has led to these important insights and a new scientific hypothesis.”

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 will share further details about the design of the new Phase 2 trial at future scientific meetings. Beginning with the scientific community on Jan. 20, 2022, Roche will participate in a series of webinars to discuss the findings from their post-hoc analysis of the GENERATION HD1 study data and next steps for the tominersen program.

About tominersen and the clinical trials

Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense medicine designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. In December 2017, Roche licensed the investigational medicine from Ionis. The tominersen clinical development program included the following studies:

  • GENERATION HD1: a randomized, multicenter, double-blind, placebo-controlled Phase 3 clinical study evaluating the efficacy and safety of treatment with tominersen in people with manifest HD over 25 months. Study participants were randomized to either 120 mg every two months or 120 mg every four months with intrathecal injections of tominersen, or placebo. The study recruited 791 participants from 18 countries around the world. In March 2021, Roche announced that dosing would be stopped in the tominersen Phase 3 GENERATION HD1 study following a recommendation from the independent data monitoring committee (iDMC) based on an overall benefit/risk assessment. The study is ongoing without dosing to allow participants to be followed for safety and clinical outcomes. The study will complete at the last-patient last-visit, as planned in March/April 2022.
  • GEN-EXTEND: an open label extension study for participants coming from any Roche HD study. Participants received 120 mg tominersen every two months or every four months in the study. The study is ongoing without dosing to allow participants to be followed for safety and clinical outcomes. The study will complete in March/April 2022.
  • GEN-PEAK: a Phase I study aiming to better understand the pharmacokinetics of tominersen and how tominersen affects mHTT levels and other markers in the spinal fluid and blood, which studies different doses of tominersen over two administrations. The study will now be completed as all patients have completed Part 1 of the study and Part 2 will not be conducted.

Study participants should contact their local clinical trial site for questions or support about their situation.


Source: Ionis Pharmaceuticals


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