UniQure’s Four-Year Huntington’s Gene-Therapy Result Misses Statistical Threshold

UniQure reported additional four-year data for its experimental Huntington’s disease gene therapy AMT-130 on September 29, 2026. Among 12 patients who received the high dose and had reached 48 months, a composite measure of disease progression favored the treatment by 44% against an external comparison group. That result did not meet the usual threshold for statistical significance (p=0.144), an important limit on what the analysis can establish.

What the four-year results show

The company reported a separate 61% slowing on Total Functional Capacity, a measure of daily functioning, at 48 months. It labeled the associated p value “nominal,” which means it should not be read as definitive proof across all analyses. The data cutoff was June 30, 2026, and the company compared treated patients with matched participants from a natural-history dataset rather than a concurrently randomized placebo group.

At 36 months, an updated analysis of 15 high-dose patients showed an 80% difference on the composite scale versus the updated external comparison group, according to uniQure. The 36-month information is the basis of the company’s previously submitted U.S. application under an accelerated-approval pathway. UniQure said the newly disclosed four-year results were not part of that submission.

Why interpretation is difficult

More than half of data were missing from the matched historical comparison group at 48 months, according to the company. UniQure argues that patients who stopped follow-up in that dataset had deteriorated faster, potentially making the treated group’s benefit look smaller. That is the sponsor’s explanation; the missing data also make the comparison less certain. Reuters reported that company officials could not say whether the FDA would reassess the filing in light of the new results.

AMT-130 remains investigational. The reported percentages describe differences within this small study analysis, not an individual patient’s expected response, and no U.S. approval follows from the announcement. For a separate rare-disease regulatory decision, see AskNovus’s report on the FDA’s Emcitate approval.

Sources and image credit

UniQure clinical update (September 29, 2026) and Reuters report (September 29, 2026). Featured image: illustrative photograph of clinicians reviewing medical imaging by Vitaly Gariev / Unsplash; it is unrelated to the trial.