GENERATION HD2 Trial of Huntington Agent Tominersen Amended to Include Only Higher Dose
The phase 2 DIMENSION study results showed that SAGE-71 was well-tolerated but did not meet its primary end point, leading to Sage Therapeutics’ decision to discontinue its development.
Over a 12-month period, patients showed sustained improvements in disease progression through valbenazine, regardless of concomitant antipsychotic treatment.
VO659 was considered safe and well tolerated, with treated patients showing no sustained increase in neurofilament light protein through day 85 of treatment.
The application is based on results of Prilenia Therapeutics’ phase 3 study that assessed the safety and efficacy of pridopidine in patients with Huntington disease.
Overall, the treatment resulted in dose-dependent trends of improvement on key clinical measures including Total Functional Capacity and Composite Unified Hungtinton's Disease Rating Scale.
AMT-130, uniQure’s investigational gene therapy, is the first therapeutic candidate to receive regenerative medicine advanced therapy designation for Huntington disease.
The original approval for chorea associated with Huntington disease was based on the phase 3 KINECT-HD study, an 128-patient cohort trial in which valbenazine met its primary end point.
Teva’s VMAT2 inhibitor was previously approved in a twice-daily formulation. The once-daily extended-release treatment, marketed as Austedo XR, is expected to be available later in 2023 in doses of 6 mg, 12 mg, and 24 mg.
