UniQure AMT-130 Data Show Slower Effect at 48 Months
uniQure AMT-130 data showed a nonsignificant 48-month composite (44% slowing; p=0.144) while TFC was nominally positive, complicating regulatory outlook.
KEY TAKEAWAYS
- Primary composite cUHDRS at 48 months missed statistical significance (44.0% slowing; p = 0.144).
- Total functional capacity slowed 61.0% at 48 months with a nominal p = 0.008.
- Company had filed a BLA, and mixed 48-month results complicate the regulatory path for investors.
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uniQure N.V. (QURE) released data on Sept. 29, 2026, showing continued slowing of Huntington’s disease progression at four years for its investigational gene therapy AMT-130. The primary composite measure missed statistical significance, while a separate functional measure showed a nominal benefit, as the company said it had filed a biologics license application (BLA).
48-Month Efficacy Findings
uniQure reported 48-month outcomes for the first 12 high-dose patients in its Phase I/II trial, showing a 44% slowing of disease progression on the composite Unified Huntington’s Disease Rating Scale (cUHDRS), with a p-value of 0.144, which is not statistically significant. Total functional capacity (TFC), a separate measure of functional ability, slowed by 61% with a nominal p-value of 0.008. The term “nominal” indicates this p-value was not necessarily adjusted for multiple comparisons or other prespecified statistical controls.
The 48-month composite effect was smaller than the 75% slowing reported at 36 months. These comparisons were made against an external control group rather than a concurrent randomized placebo group. The cUHDRS and TFC assess different aspects of Huntington’s progression—cUHDRS is a composite performance metric, while TFC measures functional capacity—resulting in a mixed efficacy signal across endpoints.
Regulatory Filing and Implications
uniQure said it had filed a BLA for AMT-130, basing the submission primarily on the earlier 36-month dataset. The therapy remains investigational, and no approval has been granted. The company continues to pursue U.S. regulatory review, with the FDA reportedly willing to consider an accelerated-approval pathway.
Such a pathway would require FDA acceptance of the application and fulfillment of confirmatory trial requirements. The mixed 48-month results—a nonsignificant primary composite outcome alongside a nominally positive functional measure—complicate the regulatory assessment. This divergence and the small 48-month cohort increase uncertainty about how the FDA will weigh the evidence and the timing and conditions of any approval.
For investors, the conflicting endpoints and limited late-stage data make the overall efficacy picture harder to interpret than earlier reports suggested.
