uniQure sheds 30% after data for Huntington’s disease candidate

Dutch biotech uniQure N.V. (NASDAQ:QURE) fell ~30% pre-market Wednesday after announcing interim data from its Phase 1/2 trial for AMT-130 in the treatment of neurogenerative disorder Huntington’s disease.

Citing up to 24 months of follow-up data from 26 patients, uniQure (QURE) said that in the cerebrospinal fluid (CSF) of those who received AMT-130, there was a temporary increase in Neurofilament Light Chain (NfL), a biomarker of neurodegeneration.

However, the company said that the NfL increases were not dose-dependent, and CSF NfL levels declined in all patients subsequently.

The U.S.-based trial was designed to evaluate low and high doses of the one-time gene therapy in patients with early-manifest Huntington’s disease.

Efficacy data from the trial indicate that clinical function was generally maintained for 24 months and 12 months in patients in the low-dose and high-dose cohorts, respectively.

The study drug was generally well tolerated with a manageable safety profile across low and high-dose cohorts, uniQure (QURE) said.

The company expects to disclose additional clinical data from its U.S. and EU Phase 1/2 studies for AMT-130 in Q4 2023 before meeting regulators in Q1 2024 to discuss the findings.

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