Debjit Chattopadhyay

Eliana Merle

Joseph Thome

Luca Issi

Patrick Trucchio

Paul Matteis

Salveen Richter

Yanan Zhu

Jenny Gonzalez-Armenta

Jenny Leigh Gonzalez-Armenta

Joseph Schwartz

Kristen Kluska

Lydia Erdman

Richard Miller

Samantha Corwin

Sami Corwin

Sushila Hernandez

Suzanne van Voorthuizen

Uy Ear

- High-dose AMT-130 achieved its primary endpoint, demonstrating a statistically significant **75% slowing of disease progression** as measured by the Composite Unified Huntington’s Disease Rating Scale (CUHDRS) at 36 months.  **[1590560_2155778_1]** **[1590560_2155778_4]** 
- The study also showed a statistically significant **60% slowing of disease progression** as measured by Total Functional Capacity (TFC) at 36 months, a key measure for regulatory agencies.  **[1590560_2155778_1]** **[1590560_2155778_4]** 
- uniQure plans to submit a Biologics License Application (BLA) for AMT-130 in the **first quarter of next year (Q1 2026)** with a request for priority review, following a pre-BLA meeting with the FDA in Q4 2025.  **[1590560_2155778_1]** **[1590560_2155778_4]** 
- AMT-130 continues to be generally well tolerated, with no new treatment-related serious adverse events observed since December 2022.  **[1590560_2155778_3]** **[1590560_2155778_4]**  The company estimates an initial addressable market of approximately **6,000 U.S. Huntington's disease patients** at launch.  **[1590560_2155778_6]**

uniQure Announces Positive Phase I/II Study Results for AMT-130 in Huntington's Disease

- **uniQure's high-dose AMT-130 met its primary and key secondary endpoints at 36 months** in the pivotal Phase I/II study for Huntington's Disease, demonstrating a **statistically-significant 75% slowing of disease progression based on cUHDRS (p=0.003)** and a statistically-significant slowing based on TFC (p=0.033).  **[1590560_2155828:5]** **[1590560_2155828:12]** 
- The study also showed **CSF NfL levels below baseline at 36 months** and favorable trends across other clinical subdomains of cUHDRS, with data as of June 30, 2025.  **[1590560_2155828:5]** **[1590560_2155828:10]** **[1590560_2155828:12]** 
- AMT-130 continued to be **generally well-tolerated with a manageable safety profile**, with no new drug-related serious adverse events observed since December 2022.  **[1590560_2155828:5]** **[1590560_2155828:10]** **[1590560_2155828:12]** 
- uniQure plans to hold a **pre-BLA meeting with the FDA in Q4 2025** and expects to submit a Biologics License Application (BLA) for AMT-130 with a request for priority review in **Q1 2026**.  **[1590560_2155828:11]**

uniQure's AMT-130 High-Dose Meets Primary and Secondary Endpoints in Huntington's Disease Study

- uniQure has secured a **$175 million senior secured term loan facility** from Hercules Capital to support the potential commercial launch of **AMT-130**, a gene therapy for Huntington’s disease.  **[1590560_dcff35f570eb4b2aaa7f0fe37324ef0d]** 
- The loan includes an immediate **$50 million** tranche, with an additional **$100 million contingent** upon uniQure raising over $150 million by March 15, 2027, and receiving Biologics License Application approval for AMT-130 by June 15, 2027.  **[1590560_dcff35f570eb4b2aaa7f0fe37324ef0d]** 
- This financing extends uniQure's debt term to **October 2030** and features a floating interest rate of approximately **9.70%**, an improvement from the previous debt's 11.95%.  **[1590560_dcff35f570eb4b2aaa7f0fe37324ef0d]** 
- The company anticipates a **2026 launch of AMT-130 in the U.S.**, with this loan providing enhanced financial flexibility.  **[1590560_dcff35f570eb4b2aaa7f0fe37324ef0d]**

uniQure Secures $175 Million Loan for AMT-130 Launch

- uniQure's experimental gene therapy **AMT-130** demonstrated significant efficacy in a trial for Huntington's disease, slowing clinical progression by approximately **75%** at **36 months** in the high-dose group and reducing functional decline by roughly **60%**.  **[1590560_54764ffa45f947e4b2a54aee1ec50cf9]** 
- The one-time therapy was generally well tolerated with **no new serious safety signals** since 2022, although the results are preliminary and require larger, longer randomized studies for confirmation.  **[1590560_54764ffa45f947e4b2a54aee1ec50cf9]** 
- uniQure is targeting a U.S. FDA filing for **AMT-130** in **early 2026**, with investigators highlighting its potential to preserve daily function and meaningfully slow disease progression.  **[1590560_54764ffa45f947e4b2a54aee1ec50cf9]**

uniQure Reports Positive AMT-130 Trial Results for Huntington's Disease

- uniQure N.V. entered into Amendment No. 3 to its loan agreement with Hercules Capital, Inc. on **September 22, 2025**, providing for **up to $175 million** in new term loans.  **[1590560_0001104659-25-092750_tm2526684d1_8k.htm:1]** 
- The company immediately drew **$50 million** from the 2025 Term Loan to refinance existing term loans and for general corporate purposes.  **[1590560_0001104659-25-092750_tm2526684d1_8k.htm:1]** 
- An additional **$100 million** is available upon achieving the Tranche 2 Milestone, which requires raising over $150 million by March 15, 2027, and obtaining Biologics License Application approval for **AMT-130** by June 15, 2027.  **[1590560_0001104659-25-092750_tm2526684d1_8k.htm:1]** 
- The 2025 Term Loan carries an interest rate equal to the greater of **9.45%** and the sum of the prime rate and 2.45%, with the principal and accrued interest due on **October 1, 2030**.  **[1590560_0001104659-25-092750_tm2526684d1_8k.htm:1]** 
- New financial covenants, effective **April 1, 2026**, require maintaining unrestricted cash of at least **65%** of the outstanding term loan advances, with potential reductions upon achieving specific milestones.  **[1590560_0001104659-25-092750_tm2526684d1_ex10-1.htm:45]**

uniQure N.V. Secures Up to **$175 Million** in New Term Loans from Hercules Capital

- uniQure N.V. announced **positive topline data** from the pivotal Phase I/II study of **AMT-130 for Huntington's disease**, with a data cutoff as of **June 30, 2025**.  **[1590560_0001104659-25-092740_qure-20250924xex99d1.htm:0]** **[1590560_0001104659-25-092740_qure-20250924x8k.htm:2]** 
- **High-dose AMT-130 met the primary endpoint**, demonstrating a **statistically significant 75% slowing of disease progression** as measured by cUHDRS at 36 months compared to an external control.  **[1590560_0001104659-25-092740_qure-20250924xex99d1.htm:0]** **[1590560_0001104659-25-092740_qure-20250924x8k.htm:2]** 
- A key secondary endpoint was also met, showing a **statistically significant 60% slowing of disease progression** as measured by TFC at 36 months compared to an external control.  **[1590560_0001104659-25-092740_qure-20250924xex99d1.htm:0]** **[1590560_0001104659-25-092740_qure-20250924x8k.htm:2]** 
- uniQure plans to **submit a Biologic License Application (BLA) in the first quarter of 2026**, with an anticipated U.S. launch later that year, pending approval.  **[1590560_0001104659-25-092740_qure-20250924xex99d1.htm:0]** **[1590560_0001104659-25-092740_qure-20250924xex99d1.htm:2]** **[1590560_0001104659-25-092740_qure-20250924x8k.htm:3]**

uniQure Announces Positive Topline Results for AMT-130 in Huntington's Disease Study

uniQure (QURE)