uniQure data suggest Huntington’s breakthrough
Dutch biotech uniQure (Nasdaq: QURE) has reported pivotal Phase I/II results showing its experimental gene therapy AMT-130 slowed Huntington’s disease progression by 75% at three years in patients given the higher dose. The milestone readout, unveiled on September 24, coincided with the launch of a $200 million share offering.
The trial enrolled 29 people in the USA and Europe, comparing outcomes with a matched dataset from the Enroll-HD registry. High-dose patients maintained motor and cognitive function longer than expected, with a key secondary measure of daily independence also showing a 60% slowing in decline. A supportive biomarker linked to disease severity fell, and the therapy was generally well tolerated.
UniQure said it intends to submit a biologics license application in early 2026, aiming for a US launch later that year if approved. Investor enthusiasm was immediate: shares surged from $13.66 to above $40 in Wednesday trading. The company’s offering was led by Leerink Partners, Stifel, Van Lanschot Kempen, and Guggenheim Securities.
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