uniQure N.V. Earnings Call: Pipeline Progress Amid Challenges

uniQure N.V. ((QURE)) has held its Q3 earnings call. Read on for the main highlights of the call.

During the recent earnings call, uniQure N.V. presented a mixed sentiment, highlighting significant advancements in its therapeutic pipeline, particularly for Huntington’s disease and epilepsy. Despite these advancements, the company faced regulatory hurdles and increased operational costs. Nevertheless, uniQure’s financial position remains robust, supporting its future operations. However, challenges arose with the FDA’s stance on AMT-130 and the pause in the AMT-162 program.

Positive Top-Line Data for AMT-130 in Huntington’s Disease

uniQure announced promising top-line data from their pivotal Phase 1/2 study of AMT-130, which demonstrated a statistically significant 75% slowing of disease progression over three years compared to a control group. Additionally, a 60% slowing was observed in a key secondary endpoint, marking a significant milestone in Huntington’s disease treatment.

Financial Growth

The company reported a notable increase in revenue for Q3 2025, reaching $3.7 million, up from $2.3 million in Q3 2024. This growth was primarily driven by $1.5 million in license revenues, showcasing a positive financial trajectory.

Strong Cash Position

uniQure’s financial stability is underscored by its cash, cash equivalents, and investment securities totaling $649.2 million as of September 30, 2025. This increase from $376.5 million at the end of 2024 is attributed to net proceeds from public offerings, providing a solid foundation for future endeavors.

Encouraging Initial Data for AMT-260 in Epilepsy

Initial data from the AMT-260 program for mesial temporal lobe epilepsy revealed a promising reduction in seizure frequency without serious adverse events. Following a favorable review, recruitment for the study has expanded, indicating potential progress in epilepsy treatment.

Fabry Disease Therapy Progress

The Phase 1/2a trial of AMT-191 for Fabry disease showed encouraging results, with one patient successfully withdrawing from enzyme replacement therapy while maintaining stable levels. This suggests the potential of AMT-191 as a one-time gene therapy.

FDA Meeting on AMT-130 BLA Submission

Challenges emerged as the FDA no longer agrees that the Phase 1/2 data of AMT-130 is sufficient for a Biologics License Application (BLA) submission. This introduces uncertainty in the approval timeline, posing a significant hurdle for uniQure.

AMT-162 Program Paused

The Phase 1/2 trial of AMT-162 for SOD1 ALS has been paused due to a dose-limiting toxicity observed in one patient, resulting in a serious adverse event. This pause reflects the cautious approach uniQure is taking in ensuring patient safety.

Increased Expenses

uniQure experienced increased research and development expenses by $3.8 million and a rise in selling, general, and administrative expenses by $7.8 million compared to the same period in 2024. These increases were driven by higher employee-related expenses and professional fees.

Forward-Looking Guidance

Looking ahead, uniQure provided updates on its gene therapy programs, including AMT-130 for Huntington’s disease, which showed promising results despite FDA concerns. The company also shared positive developments in its other pipeline programs, such as AMT-260 for epilepsy and AMT-191 for Fabry disease. Financially, uniQure reported a revenue increase and a strong cash position, although research and development expenses have risen.

In summary, uniQure’s earnings call reflected a blend of optimism and challenges. While the company made significant strides in its therapeutic pipeline, regulatory hurdles and increased expenses posed challenges. Nevertheless, uniQure’s strong financial position and promising data in key programs underscore its potential for future growth and innovation.