The Biology’s Four-Year Test
In June, we wrote “The Biology Won” after the FDA agreed that uniQure (QURE 0.00%↑) could submit an accelerated-approval application for its Huntington’s disease treatment AMT-130 using three-year clinical data as the primary basis.
uniQure submitted that application on September 2. Tuesday’s four-year update sent QURE down 37%, making the durability of the treatment effect the immediate question.
Researchers measured disease progression with the composite Unified Huntington’s Disease Rating Scale (cUHDRS), which combines assessments of movement, thinking, and the ability to manage daily life.
Among 12 high-dose patients with four-year follow-up, the planned comparison using that combined score showed 44% slower progression than an updated external control, with p=0.144. Total Functional Capacity (TFC), the component focused on everyday functioning, showed 61% slower progression, with a nominal p=0.008. An expanded group of 15 high-dose patients at three years showed 80% slower progression on the combined score, with a nominal p=0.005. Those three-year data are the anchor for the submitted application; the new four-year results were released after the filing.
What the Control Data Leave Open
The external comparison needs close attention. At month 48, 53% of the matched ENROLL-HD control data were missing. uniQure says patients who stopped providing follow-up were progressing faster than those who remained, which could leave a healthier-looking control group and understate AMT-130’s effect. Huntington’s advocate Lauren Holder explains that potential bias clearly.
Patricia Huffman offered a concrete example: her husband contributed ENROLL-HD observations for a decade, but by 2025 could complete fewer than half the assessments.
Those observations illustrate the mechanism; the size of its effect on this comparison remains uncertain.
Several of our Market Watchers who follow uniQure shared their reactions to Tuesday’s update.
Peter Mantas called QURE a “massive buy” after the update.
Mantas bought shares when the stock fell into the teens last winter. Dr. Adam May emphasized that the application rests on the three-year data and that the four-year TFC comparison remained favorable.
The FDA will have to weigh the small treated cohort and the control-data questions. The low-dose group received active treatment and isn’t a placebo group.
Kevin Mak viewed the data as only mildly negative and the selloff as excessive. He says Bloomberg and STAT stories appeared around 7:05 a.m. ET, seven minutes before uniQure’s full release, with Bloomberg initially emphasizing the 44% result’s lack of statistical significance. His argument is that algorithms trading on incomplete headlines could have amplified the early selling and shaped investor sentiment.
Dr. Adam May highlighted Mak’s account.
We already have open QURE trades, and Tuesday’s 37% selloff gives us a reason to add: we think the price fell much further than AMT-130’s prospects weakened.
The FDA application rests on the stronger three-year data. At four years, patients still showed slower loss of everyday functioning, and missing observations from faster-progressing control patients may have made the headline comparison look weaker.
We’ll lead with today’s Top Names trade, then return to QURE for our new trade on it.
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Today’s Top Names Trade
Theme: Apparel retail and brand growth.
RSI: 51; Chartmill Technical Rating: 8; Setup Rating: 7; Fundamental Rating: 7.
