Roche to Give Huntington’s Candidate Another Shot in Phase II

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Roche to Give Huntington’s Candidate Another Shot in Phase II

Ionis Pharmaceuticals today announced that Roche, its partner firm, is designing a new Phase II trial that would assess the effectiveness of tominersen in younger adult patients diagnosed with Huntington’s disease.

The decision to carry on came after dosing for the Phase III GENERATION HD1 trial was halted following an overall risk/benefit assessment. The randomized, double-blind, placebo-controlled study tested tominersen in 791 patients with manifest HD across 18 countries. The participants were given either a placebo, 120 mg of tominersen every two months, or the same dose every four months via intrathecal injection. This trial is still active but no longer dosing to observe the participants for long-term clinical and safety outcomes.

Tominersen is an investigational antisense medicine designed to reduce the Huntington protein (HTT) production, including its mutated variant, mHTT. Ionis designed the drug, also called IONIS-HTTRx and RG6042. Roche licensed the drug from Ionis in December 2017.

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In March 2021, both companies announced they would discontinue dosing for the Phase III GENERATION HD1 trial after an unblinded Independent Data Monitoring Committee (iDMC) recommended it to be stopped. While the companies had been disappointed back then, they did declare that they would continue finding solutions in what could be a promising treatment. This latest announcement is a result of such efforts.

The new Phase II study that Roche will design will explore different doses of the drug in younger adult patients with a lower disease burden. Preparations are still in the early stages, and Roche said it will share details in the coming weeks. The company is expected to start appearing in a series of webinars beginning January 20 to talk about the results of its post-hoc analysis on the GENERATION HD1 data.

“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,” said C. Frank Bennett, Ph.D., the executive vice president, chief scientific officer, and franchise leader for Ionis’ neurological programs, in a statement.

“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,” added Dr. Bennett.

Huntington’s disease is a rare genetic and progressive disorder that causes brain nerve cells to break down. This causes problems in a person’s cognitive and motor functions, later leading to loss of independence and disability. Survival ranges from 10 to 20 years after motor symptoms emerge. There is no known cure for the disease as of this writing. There also are no approved treatments to address its underlying cause.

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