The largest clinical trial in Huntington’s disease, which has prompted global interest and excitement since its launch three years ago, has been discontinued.
The shock announcement was made by pharmaceutical company Roche, which announced the end of its Phase III GENERATION HD1 study of tominersen in manifest Huntington’s disease (HD).
“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,” says Dr Levi Garraway, Roche’s chief medical officer and head of global product development.
“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.”
The decision was made based on a review by the Independent Data Monitoring Committee (iDMC) based on the “investigational therapy’s potential benefit/risk profile for study participants”.
When the project was launched in 2017, hopes were high of a breakthrough in finding a treatment to stop or slow the progression of Huntington’s disease, a genetic, progressive condition that causes the nerve cells in the brain to break down, causing problems with a person’s ability to think, move and function, leading to increasing disability and loss of independence.
GENERATION HD1 comprised a randomised study, completed across multiple centres, which evaluated the efficacy and safety of treatment with tominersen in people with manifest HD over 25 months.
Study participants were randomised to either 120 mg every two months, or 120 mg every four months, of intrathecal injections of tominersen or placebo. Tominersen is an antisense oligonucleotide (ASO)—a snippet of DNA that is intended to block production of a mutant protein, huntingtin, that causes Huntington’s disease.
The study recruited 791 participants from 18 countries around the world.
“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,” adds Dr Garraway.
Roche added in a statement that it intends to continue following participants for safety and clinical outcomes, without the dosing of the investigational medicine or placebo, and will share its findings of the Phase III study once full data has been collected and analysed.
