Artur Plawgo
uniQure (NASDAQ:QURE) shares traded higher premarket Tuesday after the Dutch biotech posted the latest data from its Phase 1/2 clinical trials for AMT-130, an investigational gene therapy for Huntington’s disease, a rare, inherited neurodegenerative disorder.
Based on a Mar. 31 data cut of 21 patients, the company said the one-time therapy at a higher dose led to a statistically significant and dose-dependent slowing of disease progression through two years.
During the same period, patients’ motor and cognitive function remained at near-baseline levels, uniQure (QURE) said, adding that the drug was also linked to a statistically significant reduction of a disease biomarker called NfL in cerebrospinal fluid (CSF).
No newly reported AMT-130-related serious adverse events occurred, and patients demonstrated a manageable safety profile for the experimental therapy at both dose levels.
The trials, conducted in the U.S. and Europe, evaluated patients who received either low or high doses of AMT-130 and those who underwent a so-called “imitation” surgery.
uniQure (QURE) said it would discuss the results with the FDA in H2 2024, seeking a faster development path for AMT-130 and potentially an accelerated approval.