uniQure (QURE) Q4 2025 earnings summary

Executive summary

• Reported full-year 2025 financial results, with revenue of $16.1 million, down from $27.1 million in 2024, mainly due to lower collaboration and contract manufacturing revenue, partially offset by higher license revenue.

• Focused on advancing AMT-130 for Huntington's disease, with 3-year Phase I/II data showing 75% slowing of disease progression and strong support from the HD community.

• Completed enrollment in key clinical studies and advanced regulatory discussions with the FDA, which recommended a Phase III sham-controlled trial for AMT-130; company is advocating for regulatory flexibility and leveraging natural history data.

• Pipeline progress includes AMT-191 for Fabry disease and AMT-260 for mesial temporal lobe epilepsy, with positive early clinical data and manageable safety profiles.

• Ended 2025 with a strong cash position of $622.5 million, expected to fund operations into the second half of 2029.

Financial highlights

• Revenue for 2025 was $16.1 million, a decrease from $27.1 million in 2024, mainly due to lower collaboration and contract manufacturing revenue, offset by higher license revenue.

• Research and development expenses were $140.7 million, down from $143.8 million in 2024, reflecting lower employee and facility costs after the Lexington facility divestiture, offset by higher direct R&D for AMT-130.

• Selling, general, and administrative expenses increased to $65.5 million, up from $52.7 million, driven by higher professional fees and commercialization preparation for AMT-130.

• Net loss for 2025 was $199.0 million ($3.46 per share), an improvement from $239.6 million ($4.92 per share) in 2024.

• Cash, cash equivalents, and investment securities totaled $622.5 million, up from $367.5 million, mainly due to $404.2 million raised from public offerings.

Outlook and guidance

• Engaged in ongoing discussions with the FDA and ex-US regulators to define the regulatory path for AMT-130, including a planned Type B meeting in Q2 2026.

• Plans to update the Phase I/II Statistical Analysis Plan for AMT-130 to include 4-year follow-up data, with analysis expected in Q3 2026.

• Additional clinical data from AMT-260 and AMT-191 expected in the first half of 2026.

• Cash runway expected to fund operations into the second half of 2029.