The purpose of this study is to evaluate the safety, tolerability, pharmacodynamics (PD) and pharmacokinetics (PK) of single or repeat doses of ALN-HTT02.
Phase 1 trials primarily test safety and dosing in a small number of participants (typically 20–100). Results do not yet speak to efficacy. FDA approval is typically many years away from this stage.
Currently recruiting: the trial is enrolling patients. A data readout is not expected until after enrollment closes and the follow-up period is complete.
Alnylam Pharmaceuticals (ALNY) is the sponsoring company for this trial. BiotechSign currently grades this company F (27/100) based on composite catalyst signals across its full pipeline. This trial is one data point in that overall catalyst picture.