UniQure plunges as Huntington's trial data disappoints investors

Sept 29 — UniQure said its gene therapy for Huntington's ​disease fell short of the goal of slowing disease progression significantly after four years of treatment, ‌sending its US-listed shares plummeting 48% in premarket trading on Tuesday.

The Dutch ⁠company earlier this month applied ​for US approval of ⁠the experimental treatment, AMT-130, after the FDA ended an ‌eight-month-long standoff by ‌reversing its position that there was insufficient data ⁠on the treatment.

AMT-130 slowed disease ⁠progression by 44% in 12 high-dose patients at 48 months on the cUHDRS scale, compared with an updated control group. But the result did not reach statistical significance, the company said.

The cUHDRS scale is ‌a composite measure of cognitive, motor ​and functional decline.

UniQure said control-group patients who discontinued follow-up appeared to experience faster disease progression than those who remained, meaning the control data may have understated AMT-130's potential benefit.

The therapy showed a 61% slowing at four years on TFC, a narrower functional measure ​that assesses a patient's ability to perform everyday activities, ‌such as working, ‌household ⁠tasks and self-care.

Guggenheim analyst Debjit Chattopadhyay said a steady measure of TFC counterbalances the softening in the cUHDRS scale.

Huntington's disease is a rare inherited brain disorder that causes ‌movement difficulties and triggers ​behavioral changes and cognitive decline. ‌No approved drugs ⁠exist that ​help slow progression.

(Reporting by Christy Santhosh in Bengaluru; Editing by ​Sahal Muhammed)

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