Roche Halts 2 Huntington’s Drug Programs After Tominersen Misses Key Endpoint
Updated
Updated · en.hdbuzz.net · Jul 9
Roche Halts 2 Huntington’s Drug Programs After Tominersen Misses Key Endpoint
1 articles · Updated · en.hdbuzz.net · Jul 9
Summary
Roche ended the GENERATION HD2 and POINT-HD Huntington’s programs after tominersen failed to improve clinical outcomes and RG6496 was deemed unfit for long-term use.
In the Phase II tominersen trial, patients showed lower mutant huntingtin and neurofilament light levels, but the drug did not beat placebo on disease-progression measures including cUHDRS and TFC at 16 months.
Roche said neither shutdown was driven by participant safety: tominersen raised no new safety concerns, while only 3 people had received a single RG6496 dose before animal studies undermined repeated dosing plans.
The company said the decisions were unrelated and stressed the results do not settle whether huntingtin lowering can work, with fuller data still to come at future scientific meetings.
More than 1,500 families have participated in Roche’s Huntington’s programs over the past decade, and Roche said its RG6662 gene-therapy program will continue unchanged.