uniQure Announces 2025 Financial Results and Provides Recent Company Updates

Core Insights - uniQure N.V. reported updated Phase I/II data for AMT-191 in Fabry disease, showing durable, dose-dependent increases in α-Gal A enzyme activity [1] - The company has a strong financial position with approximately $622.5 million in cash and equivalents as of December 31, 2025, expected to fund operations into the second half of 2029 [1][13] - The CEO expressed confidence in AMT-130 as a potential first disease-modifying therapy for Huntington's disease, despite not yet aligning with the FDA on an approval pathway [2] Financial Performance - Revenue for the year ended December 31, 2025, was $16.1 million, a decrease from $27.1 million in 2024, primarily due to a $10.7 million drop in collaboration revenue [14] - Research and development expenses were $140.7 million for 2025, down from $143.8 million in 2024, mainly due to reduced costs following the divestment of the Lexington facility [16] - The net loss for the year ending December 31, 2025, was $199.0 million, or $3.46 per share, compared to a net loss of $239.6 million, or $4.92 per share, in 2024 [21][22] Clinical Developments - AMT-130 for Huntington's disease showed a statistically significant 75% slowing in disease progression at 36 months compared to an external control [5] - The company completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 for refractory mesial temporal lobe epilepsy, with additional data expected in the first half of 2026 [4][11] - AMT-191 demonstrated dose-dependent elevations in α-Gal A activity, with six of 11 patients withdrawing from enzyme replacement therapy [11] Regulatory Engagement - The company held a Type A meeting with the FDA regarding AMT-130 and plans to request a follow-up Type B meeting in the second quarter of 2026 [4][6] - The FDA indicated that the data from Phase I/II studies compared to an external control may not be sufficient for a marketing application, recommending a prospective, randomized, double-blind study [6] Upcoming Events - uniQure will host an earnings call on March 2, 2026, to discuss its financial results and business updates [1][23] - The company is scheduled to participate in several investor conferences in March and April 2026 [26]