Sunday, May 15, 2022

Roche ends tominersen study in manifest Huntington’s disease

Swiss pharma company Roche has announced that it will discontinue dosing in a Phase III study evaluating its investigational candidate tominersen in manifest Huntington’s disease.

This decision was based on the results of a pre-planned review of the data from the GENERATION HD1 study, conducted by an unblinded Independent Data Monitoring Committee (iDMC).

The iDMC made its recommendations on the potential risk/benefit profile of the investigational therapy for study participants.

In a statement, Roche said it will continue to follow participants for safety and clinical outcomes, without dosing of the investigational drug or placebo.

Roche will share its findings when full data from the Phase III study is available and analysed.

“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,” said Levi Garraway, chief medical officer and head of global product development, Roche.

“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,” he added.

Huntington’s disease is a rare genetic condition that causes the nerve cells in the brain to break down. This causes problems with a person’s ability to think, move and function, leading to increasing disability and loss of independence.

Tominersen is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT). Roche licensed the candidate from Ionis Pharmaceuticals in December 2017.

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