By Christy Santhosh
Sept 29 (Reuters) – 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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