uniQure releases mixed 48-month clinical data for AMT-130

uniQure has released 48-month long-term data from its Phase I/II clinical trials for the gene therapy AMT-130 (ifeunertirgene inilparvovec), leading to re-evaluations by financial institutions.

In a high-dose group of 12 patients, the data showed a 44 percent slowing of disease progression on the cUHDRS scale, though this result did not reach statistical significance (p-value of 0.144). However, the TFC scale showed a 61 percent slowing of progression. These 48-month results provide a more nuanced view compared to previous 36-month data, which had shown an 80 percent slowing on the cUHDRS scale.

Despite the mixed long-term data, Barclays has maintained a buy recommendation for uniQure with a price target of 60.00 USD. The company continues to use 36-month data for its FDA application. Financially, uniQure reported a revenue increase to 5.84 million USD in the second quarter of 2026, while its GAAP net loss expanded to 81.06 million USD.