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.
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