UniQure's update landed with a wrinkle: the high-dose group of 12 patients showed a 44% slower deterioration against an external comparator at the four-year mark on a widely used Huntington's disease rating scale, and the company said that difference did not reach statistical significance. A year earlier, the same metric looked markedly stronger at 75% and underpinned an approval filing submitted earlier this month. UniQure attributed the narrowing gap to "substantial missing data" in the historical control set, adding that a review suggested faster-declining patients dropped out of that group.
The picture was brighter on a second readout. On total functional capacity - which measures practical abilities such as maintaining employment and managing daily activities - high-dose patients declined 61% more slowly than controls over four years. Victor Sung - who treats several participants and is a neurologist at University of Alabama Medicine - said these functional measures have held up, noting, "Overall, my patients are not progressing very much at all, which is not the typical thing."
Chief Executive Officer Matt Kapusta said, "At 48 months, we continue to see evidence of meaningful disease slowing," while acknowledging the missing control data "makes these statistical comparisons and percent slowing more challenging." The three-year results marked the first time any treatment had slowed this fatal, inherited brain disorder.
Traders wasted no time. By 9:32 a.m. in New York on Tuesday, the stock had slid 38%, but as of Monday's close it remained 63% higher for the year.
It has been a whipsaw year. Shares ripped higher last September after initial data showed striking benefit, then tumbled in November after UniQure said the US Food and Drug Administration did not consider the small study adequate for approval anymore. In March, the company said the agency "strongly recommended" a new trial that would put a control group through a sham brain procedure, sparking debate over the ethics of lengthy anesthesia without a possible benefit.
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The therapy itself requires a roughly 10-hour operation, and doctors told Bloomberg a similar duration would be needed for controls to prevent unblinding. In June, UniQure said the FDA reversed course and allowed the company to file before running another study. A confirmatory trial will still be required, but the company said it would not include sham surgery.
The readout is nuanced, not neat. Leerink analyst Joseph P. Schwartz told clients the initial market reaction could skew negative, but he views the full four-year package as landing in neutral territory. "At the end of the day, we think these data remain approvable," he wrote, adding that any degree of slowing in Huntington's is unheard of. He argued it would be inappropriate to make patients wait for more data or alternatives when rare-disease communities often accept higher risk in exchange for time and options.
UniQure still plans a confirmatory study. Kapusta said it will enroll about 200 patients, and the company aims to start screening before year end.
Regulators appear a bit more accommodating after several FDA leaders exited this spring, among them former Commissioner Marty Makary and Vinay Prasad, who previously headed the center responsible for gene therapies like UniQure's.
Huntington's affects about 40,000 Americans, causing involuntary movements, trouble with balance and swallowing, mood changes, and eventually the loss of basic functions. Current drugs only ease symptoms, not the mutant Huntington protein that drives the disease.
UniQure's next catalysts now hinge on the FDA review of its filing and the setup for a roughly 200-patient follow-up study that it aims to kick off before the year wraps, which will shape the narrative from here.
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