uniQure jumps on new data for AMT-130 gene therapy for Huntington's disease

Shares of Dutch gene therapy company uniQure (Nasdaq: QURE) rose 26% to $19.15 on Thursday as it announced safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing Phase I/II clinical trial of AMT-130 for the treatment of Huntington’s disease (HD). Six of the 10 enrolled patients received AMT-130, and four patients received an imitation surgical procedure in this randomized, blinded clinical trial being conducted in the USA.

“We are encouraged by this 12-month update on the patients enrolled in the low-dose cohort,” stated Ricardo Dolmetsch, president of research and development at uniQure, adding: “Thus far in the clinical trial, AMT-130 has been well-tolerated, with no serious adverse events related to the gene therapy and NfL levels approaching baseline. We are also pleased to observe trends suggesting target engagement that are supported by the lowering of mHTT protein in evaluable patients receiving AMT-130. We look forward to presenting additional clinical data, including functional outcomes, on all patients from this important study next year.”

One-year observations on low-dose cohort of AMT-130