A Phase II, Randomized, Double-Blind, Placebo-Controlled, Dose-Finding Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen in Individuals with Prodromal and Early Manifest Huntington’s Disease

Prodromal and Early Manifest Huntington’s Disease

1. Incidence and severity of adverse events, with severity determined according to the Adverse Event Severity Grading Scale, 2. Change from baseline in clinical laboratory results (CSF WBC and protein), 3. Safety MRI, 4. Percentage change from baseline in geometric means of CSF mHTT protein levels at Month 9, 5. Change from baseline in cUHDRS (non-U.S.) and TFC (U.S.) at 16 months

1. Change from baseline in vital signs, 2. Change from baseline in ECG parameters, 3. Change from baseline in plasma clinical laboratory results, 4. Change from baseline in MoCA, 5. Proportion of participants with suicidal ideation or behavior as assessed by C-SSRS score at each visit, including detailed focus on any individual cases identified as having severe ideation or behavior during the study conduct, 6. Change from baseline at 16 months for the assessments of TFC (non-U.S.)/cUHDRS (U.S.), SDMT, TMS, and SWR, 7. Change from baseline in CSF NfL levels at 16 months, 8. Incidence of anti-drug antibodies (ADAs) at specified timepoints relative to the prevalence of ADAs at baseline, 9. Titers will be determined if ADAs are identified

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