Roche discontinued two investigational Huntington's disease drugs, tominersen and RG6496, developed in collaboration with Ionis Pharmaceuticals.
The Phase 2 GENERATION HD2 study for tominersen failed to show clinical benefit for patients. While the drug successfully reduced the mutant huntingtin protein biomarker, it did not achieve therapeutic goals. This represents the second major setback for tominersen, which Roche previously halted during a Phase 3 trial in 2021.
Roche also terminated the early-stage POINT-HD trial for RG6496. Preclinical animal data indicated that repeated dosing would be unfeasible for long-term treatment.
These decisions remove two of the three clinical programs Roche was actively pursuing for Huntington's disease.