UniQure plunges as Huntington's trial data disappoints investors

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Sept 29 — UniQure said its gene therapy for Huntington's disease fell short of the goal of slowing disease progression significantly after four years, sending its US-listed shares plummeting 40% on Tuesday.

The Dutch company ‌earlier this month applied for US approval of the treatment, AMT-130, after the FDA ended an ⁠eight-month-long standoff over the adequacy of data.

Its application was based on ⁠a 36-month analysis of the trial, in which the therapy slowed ‌progression of the brain ‌disorder by 75%.

Walid Abi-Saab, uniQure's chief medical officer, said it ⁠is difficult to speculate on whether the ⁠Food and Drug Administration will reconsider the application with the new data.

Tuesday's data showed that AMT-130 slowed disease progression by 44% in 12 high-dose patients at 48 months, compared with an updated control group. But the result did not reach statistical significance, the company ‌said.

UNIQURE CITES CHANGES TO CONTROL GROUP

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.

"We do think that, beyond three years, the data is really suffering from this survivor bias," Abi-Saab said.

The therapy's effect on disease progression was measured on the cUHDRS scale, a composite measure of cognitive, motor and functional decline.

It 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 on TFC counterbalances the lower reading on 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)