uniQure Huntington’s gene therapy slowed decline on a measure of daily function by 61% at 48 months in a 12-patient high-dose group compared with an external control, the company said.
uniQure (NASDAQ: QURE) reported a nominal p-value of 0.008 on Total Functional Capacity, or TFC, for ifezuntirgene inilparvovec, formerly called AMT-130. The small, non-randomized comparison means the result requires cautious interpretation despite the reported treatment difference.
uniQure Huntington’s Gene Therapy Shows Mixed 48-Month Statistics
The high-dose group showed 44% slower progression on the composite Unified Huntington’s Disease Rating Scale, the prespecified primary endpoint, according to the company’s official clinical update. That comparison did not reach statistical significance, with a p-value of 0.144.
At 36 months, an updated analysis of 15 high-dose patients indicated 80% slower progression on the composite measure and 67% on TFC, with nominal p-values of 0.005 and 0.011. Those results were compared with matched patients from the observational ENROLL-HD dataset.
The company said missing data in 53% of the matched external controls at 48 months and the tendency for faster-progressing participants to leave follow-up may have understated disease progression in the comparison group.
Regulatory Review Relies on the Earlier Data Cut
The U.S. Food and Drug Administration previously told uniQure that 36-month data from 12 high-dose patients could serve as the primary basis for its biologics-license application under an accelerated-approval pathway. The newly reported results were not part of that submission.
Twenty-nine patients have received the therapy across the first two study cohorts. uniQure said it remained generally well tolerated, although five high-dose participants experienced treatment-related serious adverse events involving central-nervous-system inflammation; all resolved.
A suicide occurred in a low-dose patient approximately five years after treatment and was assessed by the investigator as unrelated. The company noted that suicide risk is elevated in Huntington’s disease.
The FDA’s review and the design of a confirmatory study are the next central milestones. The 48-month functional result adds follow-up, but approval will depend on the total evidence, including the limitations of comparing a small treated group with an external control rather than a concurrent placebo arm.
