uniQure Advances Regulatory Path for AMT-130 in Huntington’s Disease
~ Continuous alignment with FDA supports accelerated approval journey ~
~ Biologics License Application submission is on track for early 2026 ~
~ Investor conference call scheduled for today at 8:30 a.m. ET ~
LEXINGTON, Mass. and AMSTERDAM — uniQure N.V. (NASDAQ: QURE), a pioneering gene therapy company focused on transformative treatments for serious medical conditions, has shared important updates regarding AMT-130, its innovative gene therapy aimed at addressing Huntington’s disease. Following productive discussions with the U.S. Food and Drug Administration (FDA), the company reports significant progress in preparation for a Biologics License Application (BLA) submission expected in the first quarter of 2026.
“We are thrilled with our ongoing constructive dialogue with the FDA and the advancements made toward our anticipated BLA for AMT-130 in early 2026,” stated Walid Abi-Saab, M.D., the chief medical officer of uniQure. “Our efforts are geared towards an accelerated approval pathway that is underpinned by extensive clinical data, reflecting the urgent need for effective treatments in Huntington’s disease. We appreciate the FDA's support and look forward to presenting three-year topline data later this calendar year.”
Understanding the Statistical Analysis Plan
In the mid-2025 frame, uniQure engaged in a Type B meeting with the FDA to discuss the application of external control data and a defined statistical analysis plan (SAP) to bolster the BLA submission for AMT-130. The FDA continues to accept the composite Unified Huntington’s Disease Rating Scale (cUHDRS) as a valid endpoint for accelerated approval. They confirmed that the primary efficacy measure will focus on the 3-year changes in the cUHDRS for patients treated with high-dose AMT-130 compared to a propensity score-adjusted external control arm. Data from the ENROLL-HD dataset is to be leveraged for this analysis.
The agreement also underscored the validity of ENROLL-HD as a large, prospective, longitudinal study monitoring Huntington’s disease patients. Approximately 33,000 individuals are currently involved in this study, offering a significant resource compared to smaller historical datasets like TRACK-HD and PREDICT-HD. This will potentially bolster the robustness and validity of uniQure's statistical approach, paving the way for effective regulatory advancements.
uniQure intends to submit an updated SAP in line with its discussions from the recent FDA meeting.
Chemistry, Manufacturing, and Controls (CMC) Insights
In the earlier part of 2025, another Type B discussion with the FDA addressed the essential CMC elements relevant to the upcoming BLA submission. The FDA acknowledged that leveraging expertise from the manufacturing process of etranacogene dezaparvovec-drlb (HEMGENIX) will facilitate the validation of AMT-130's manufacturing process, guided by additional large-scale Good Manufacturing Practice (GMP) batches.
Moreover, the FDA concurred with uniQure's proposed drug release testing strategy, including the potency assay, pending completion of the necessary validation activities.
Steps Ahead for BLA Submission
uniQure remains focused on preparing for the BLA submission, with the following critical timelines in mind:
- Q2 2025: Submission of an updated SAP to the FDA.
- Q3 2025: Initiation of the Process Performance Qualification (PPQ) run while presenting topline data from Phase I/II trials.
- Q4 2025: Pre-BLA meeting with the FDA.
- Q1 2026: Submission of the BLA requesting priority review status.
Investor Conference Call Insights
uniQure's management is set to host an investor conference call and webcast today at 8:30 a.m. ET. Interested parties can tune in through the Events & Presentations section of uniQure’s official website, where a replay will be available for the following 90 days. Participants seeking to join via phone must register through a dedicated online form to receive dial-in details.
Insights on AMT-130's Clinical Journey
uniQure is actively conducting multi-center, dose-escalating Phase I/II clinical trials assessing the safety and explore efficacy of AMT-130 for Huntington’s disease. One notable U.S. study included 26 patients with early-stage Huntington’s disease, dynamically assigned to receive either treatment or an imitation surgical procedure. There, participants received AMT-130 through a precisely guided neurosurgical approach. Tracking extends over a blinded 12-month core study period followed by a five-year unblinded follow-up for treated patients.
Across Europe, a Phase Ib/II open-label trial has enrolled 13 patients, while a third cohort across U.S. and EU sites aims to merge doses of AMT-130 with immunosuppression therapy.
Moreover, AMT-130 has achieved both FDA’s Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy designations — a groundbreaking advancement as it stands as the first therapy designed to combat Huntington’s disease to secure RMAT designation.
Understanding Huntington’s Disease
The landscape of Huntington's disease reveals a rare, inherited neurodegenerative condition that often culminates in various motor, cognitive, and behavioral challenges. The genetic basis of this disorder is well established, linked to a CAG repeat expansion in the huntingtin gene. Presently, research indicates that around 70,000 individuals in the United States and Europe are diagnosed with Huntington's disease, with many more at risk of inheritance. Despite a thorough understanding of Huntington's genetic causes, effective treatments to delay its progression remain elusive.
About uniQure
uniQure strives to fulfill the potential of gene therapy through innovative, potentially curative treatments. Their historical achievement in gaining approvals for gene therapy regarding hemophilia B signifies a noteworthy leap forward for genomic medicine. The company is expanding its pipeline of gene therapies targeted towards various severe conditions, including Huntington’s disease, refractory temporal lobe epilepsy, and Fabry disease. For more detailed information, please visit uniQure's official website.
Frequently Asked Questions
What is AMT-130?
AMT-130 is an investigational gene therapy developed by uniQure aimed at treating Huntington’s disease.
When is the BLA submission expected?
The submission of the Biologics License Application is planned for the first quarter of 2026.
What recent progress has uniQure made?
uniQure has aligned with the FDA on key components of their regulatory plan for AMT-130.
What designations has AMT-130 received from the FDA?
AMT-130 has received both RMAT and Breakthrough Therapy designations from the FDA.
How many patients are currently participating in clinical trials for AMT-130?
As of now, numerous patients are engaged in clinical trials assessing the efficacy and safety of AMT-130 across multiple centers.
