
Roche (ROG: SIX) has discontinued two Huntington’s disease programs, ending the long-running development of tominersen after a Phase II efficacy failure and stopping an early-stage study of RG6496 because animal findings indicated that the experimental therapy could not be administered repeatedly.
The 301-patient GENERATION HD2 study found that tominersen was well tolerated and significantly lowered mutant huntingtin protein and neurofilament light chain, a marker associated with damage to nerve cells. However, the treatment produced no meaningful clinical benefit compared with placebo after 16 months.
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