uniQure Plunges on Additional Data From Huntington's Disease Study
uniQure QURE announced mixed additional data from the ongoing phase I/II studies on investigational gene therapy, ifezuntirgene inilparvovec, for the treatment of Huntington’s disease.
The studies are evaluating the safety, tolerability and efficacy of ifezuntirgene inilparvovec for the treatment of Huntington’s disease.
However, the stock plunged 37% on Sept. 29, following the announcement of mixed data.
Year to date, the stock has gained 2.4% compared with the industry’s growth of 7.9%.
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More on QURE’s Huntington’s Disease Data
Across phase I/II clinical studies, 51 patients have been treated with ifezuntirgene inilparvovec. The U.S. randomized study enrolled 26 patients, with six receiving the low dose, 10 receiving the high dose and 10 undergoing a sham procedure. Four patients in the sham group subsequently crossed over to treatment after approximately 12 months.
The European open-label study enrolled 13 patients, including six on the low dose and seven on the high dose. A third cohort of 12 patients evaluated both doses in combination with immunosuppression, while a fourth U.S. cohort of six patients is evaluating the high dose in patients with lower striatal volumes than those enrolled in earlier cohorts.
Huntington’s disease is a rare, inherited neurodegenerative disorder characterized by progressive motor symptoms, including chorea, as well as behavioral changes and cognitive decline, ultimately leading to worsening physical and mental function.
The company announced results from the ongoing phase I/II studies at two timepoints, with a data cutoff as of June 30, 2026. Across the first two cohorts, 29 patients have been treated with ifezuntirgene inilparvovec, including 17 who received the high dose and 12 who received the low dose.
uniQure’s gene therapy, ifezuntirgene inilparvovec, appears to be slowing the progression of Huntington’s disease, with the effect becoming more evident in the updated 36-month data.
At 48 months, total Functional Capacity (TFC), the primary measure of the confirmatory study, continued to show a slowing of functional decline, with the absolute treatment benefit maintained through month 48.
The differences observed between the high- and low-dose groups on both the composite Unified Huntington’s Disease Rating Scale (cUHDRS) and TFC were also consistent with a dose-dependent treatment effect.
The updated 36-month analysis, which includes three additional high-dose patients, demonstrated a substantial treatment effect on both cUHDRS and TFC, further supporting the clinical data submitted as part of the company’s regulatory applications.
What Did the 36-Month Data Show?
At 36 months, cUHDRS showed 80% slowing of disease progression compared to the updated external control. TFC showed 67% slowing of disease progression compared to the updated external control. Mean cerebrospinal neurofilament light protein was 6% below baseline.
What Did the 48-Month Results Show?
The company also followed 12 high-dose patients for four years.
While cUHDRS showed a 44% slowing of disease progression compared to the updated external control, this result was not statistically significant. TFC showed 61% slowing of disease progression compared to the updated external control.
So, the 48-month results are somewhat mixed. While TFC continued to show a meaningful benefit, the cUHDRS result was weaker.
In a post-hoc sensitivity analysis using the prior ENROLL-HD external control, the 48-month analysis showed 53.5% slowing based on cUHDRS and 68.3% based on TFC.
The company compared treated patients with people from the ENROLL-HD natural-history database. Per the company, the treatment effect at 48 months was likely understated due to substantial missing data and survivor bias in the updated external control.
The observed differences between the high and low doses are consistent with a dose-dependent treatment effect.
Ifezuntirgene inilparvovec continues to show a generally manageable safety profile at both doses. Most adverse events were related to the administration procedure and have resolved. Five high-dose patients (17%) experienced treatment-related serious adverse events linked to central nervous system inflammation, all of which were resolved.
One suicide occurred in a low-dose patient about five years after treatment and was deemed unrelated to treatment by the investigator.
What Does This Mean for QURE Investors?
At a June 2026 Type B meeting, the FDA agreed that 36-month data from 12 high-dose patients would be sufficient to support the BLA submission under the accelerated approval pathway. The company subsequently submitted the BLA based on the data.
The submission was made before the latest results, so the data were not included in the BLA.
The key catalysts now are the FDA’s regulatory review of the BLA and additional clinical and regulatory validation of the durability of the treatment effect.
Ifezuntirgene inilparvovec is the first investigational Huntington’s disease therapy to receive both Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the FDA.
The investigational gene therapy has also received Fast Track designation from the regulatory body.
QURE's Zacks Rank & Stocks to Consider
uniQure currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the drug/biotech sector are AC Immune ACIU and Precigen PGEN, both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for AC Immune’s 2026 loss per share have narrowed from 84 cents to 60 cents. Over the same period, earnings estimates for 2027 have remained unchanged at 17 cents per share.
AC Immune’s earnings beat Zacks Consensus Estimate in each of the trailing four quarters, with the average surprise being 33.25%.
Over the past 60 days, estimates for Precigen’s 2026 bottom line have improved from a loss of 2 cents to earnings of 24 cents per share. Over the same period, earnings estimates for 2027 have risen from 25 cents to 73 cents. PGEN shares have gained 94% year to date.
Precigen’s earnings beat the Zacks Consensus Estimate in three of the trailing four quarters and missed in the remaining one, with the average surprise being 108.96%.
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This article originally published on Zacks Investment Research (zacks.com).
