Raptor Reports Results With RP103 In Huntington's Disease Phase 2/3 Trial

(RTTNews) - Raptor Pharmaceutical Corp. (RPTP) reported top line results from a planned 18-month analysis of an ongoing 3 year Phase 2/3 study of RP103 for the potential treatment of Huntington's disease or HD in collaboration with the Centre Hospitalier Universitaire d'Angers (CHU d'Angers). The company said that 96 patients with HD were randomized to treatment with RP103 or placebo, and 89 patients completed the initial 18-month phase.

Analysis of all 96 patients enrolled in the trial demonstrated a positive trend towards slower progression of Total Motor Score or TMS in patients treated with RP103 versus those patients on placebo, the primary endpoint of the trial. TMS progression was 32% slower in patients treated with RP103 versus those treated with placebo after 18 months treatment. In 66 patients not taking concurrent tetrabenazine, RP103 treatment led to a statistically significant slower progression in TMS versus the placebo group.

Due to the 36-month duration of the study, patients were allowed to continue their baseline medication regime, including antidepressants and tetrabenazine, the latter being an approved medication to treat chorea associated with HD. Patients were not randomized in the study based on concomitant medications.