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Roche’s Huntington’s disease treatment disappoints in Phase III trial

pharmafile | March 23, 2021 | News story | Manufacturing and Production Huntington's, Phase III trial, Roche, huntington's disease, tominersen 

Roche has discontinued dosing in a Phase III trial of its tominersen treatment for Huntington’s disease, following discouraging initial data.

The decision was made following the results of a pre-planned review of the Phase III data, conducted by an Independent Data Monitoring Committee (iDMC). The iDMC made its recommendation based on the investigational therapy’s potential benefit/risk profile for study participants.

The randomised, double-blind GENERATION HD1 trial studied 791 participants from 18 countries, evaluating the efficacy and safety of treatment with tominersen in people with manifest Huntington’s over 25 months. Participants were randomised to 120mg of tominersen every two or four months, or given a placebo.

Roche will continue following participants for safety and clinical outcomes, without the dosing of the investigational medicine or placebo.

Tominersen is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT. There is no known cure for Huntington’s disease, and no approved therapies that treat the underlying cause.

A Phase I study, hoping to increase understanding of the pharmacokinetics of tominersen and how tominersen affects mHTT levels and other markers in the spinal fluid and blood, is ongoing, while dosing in Roche’s open label extension of the GENERATION HD1 trial will be paused while the data is analysed.

Dr Levi Garraway PhD, Roche’s Chief Medical Officer and Head of Global Product Development, said: “This is very unfortunate news to deliver on the tominersen Phase III study and we know it will be especially difficult for people with Huntington’s disease to hear. The HD community currently has no treatments to stop or slow the progression of this rare neurodegenerative disease that impacts families across generations.

“GENERATION HD1 is the largest clinical trial in Huntington’s disease to date and we do know that the data generated will significantly advance our understanding of huntingtin-lowering as a potential treatment approach. We would like to thank all of the individuals and families participating in the study for their contribution, as well as the broader HD community for their commitment and collaboration.”

Jack Goddard

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