uniQure (NASDAQ:QURE) said it has aligned with the U.S. Food and Drug Administration on a potential accelerated-approval pathway for AMT-130, its investigational gene therapy for Huntington’s disease, and remains on track to submit a biologics license application in the third quarter of 2026.
Chief Executive Officer Matt Kapusta said the company held a Type B meeting with the FDA in June, during which the agency indicated that three-year Phase I/II data could serve as the primary basis for an accelerated-approval application. The company said it subsequently received final meeting minutes confirming that alignment.
AMT-130 Regulatory Plans and Four-Year Data
Abi-Saab said the company is working with the FDA to finalize the confirmatory trial design and has begun preparatory work for a global study. While uniQure did not disclose a proposed enrollment target or timeline, management said it believes it can initiate and complete the study within a reasonable period after a potential approval.
Patients randomized to standard care in the proposed confirmatory study would be eligible to receive AMT-130 after three years, provided treatment is safe for them, Abi-Saab said. The company also expects to use sites in countries where AMT-130 is not yet commercially available to support enrollment and trial completion.
AMT-130 has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA. Kapusta said uniQure expects to request priority review when it submits the BLA, though the decision remains at the FDA’s discretion. Management said it believes an FDA advisory committee meeting is likely and is preparing for that possibility.
The company also plans to submit a marketing authorization application to the U.K. Medicines and Healthcare products Regulatory Agency in the third quarter. uniQure expects to engage more fully with the European Medicines Agency in 2027.
UniQure plans to present four-year AMT-130 data in September. The analysis will include safety and tolerability data through four years, as well as top-line clinical data from 12 high-dose and 12 low-dose patients with four years of follow-up. An additional three high-dose patients will have reached three years of follow-up.
The analysis is expected to include cUHDRS, TFC and other clinical measures compared with a propensity-score-matched natural-history control from the Enroll-HD database. The updated Enroll-HD database includes about 26,000 Huntington’s disease participants, including roughly 6,000 added participants, the company said. UniQure also plans to report changes from baseline in cerebrospinal fluid neurofilament light chain at four years.
Kapusta said the company is in a quiet period regarding the four-year findings and will not provide further commentary before the September presentation. He added that the three-year statistical analysis plan has not changed since its submission to the FDA in 2025.
Early Updates in Epilepsy and Fabry Disease
UniQure also reported early clinical findings for AMT-260, its investigational therapy for refractory mesial temporal lobe epilepsy. As of a May 29 data cutoff, three of six patients in the first low-dose cohort experienced reductions in disabling seizures of 79% to 100% from baseline during months four through six.
The other three patients had outcomes ranging from a 33% reduction to a 36% increase in disabling seizures over the same period. There were no serious adverse events related to AMT-260 or the surgical procedure as of the presentation date, the company said. Reported adverse events in the low-dose cohort were mild or moderate, with headache reported in two patients. No immunosuppression was required.
Enrollment in a second, higher-dose cohort is expected to be completed imminently, and uniQure expects updated results from both cohorts in the first half of 2027.
For AMT-191 in Fabry disease, uniQure said all 11 dosed patients across three dose levels showed dose-dependent elevations in alpha-Gal A activity. Plasma lyso-Gb3 levels remained stable after dosing across cohorts, regardless of enzyme replacement therapy status, and all 11 patients remained off enzyme replacement therapy.
Additional dosing in the mid- and high-dose cohorts remains paused while the company works with the FDA on a monitoring and management plan following grade 3 liver enzyme elevations in two mid-dose patients. An independent data monitoring committee classified the events as dose-limiting toxicities. UniQure said the liver-function-test elevations had resolved following immunosuppression as of the end of May.
Commercial Preparation and Financial Position
Chief Customer and Strategy Officer Kylie O’Keefe said uniQure is preparing for a potential AMT-130 launch by working with Huntington’s disease centers of excellence in the United States and United Kingdom. The company is mapping institutional processes involving neurology, neurosurgery and other specialties, while also engaging with payers and patient communities.
O’Keefe said treatment-center capacity will vary by institution based on factors including the number of neurosurgeons, operating-room availability and competing priorities. She cited Zolgensma and Elevidys as potential commercial analogs for a hospital-administered therapy, while cautioning that no launch comparison is exact.
- Second-quarter revenue was $5.8 million, compared with $5.3 million a year earlier, driven by higher license revenue.
- Research and development expense declined to $34 million from $35.4 million in the prior-year period.
- Selling, general and administrative expense rose to $17.4 million from $13.5 million, reflecting hiring and other preparation for potential AMT-130 commercialization.
- Cash, cash equivalents and investment securities totaled $810.3 million as of June 30, up from $622.5 million at the end of 2025.
Chief Financial Officer Christian Klemt said uniQure expects its cash resources to fund operations into 2030. The estimate includes the confirmatory trial, potential commercial launches, ongoing clinical trials and possible investments to advance other pipeline candidates into late-stage development.
About uniQure (NASDAQ:QURE)
uniQure N.V. is a biotechnology company focused on the development and commercialization of gene therapies for patients with severe medical needs. Using its proprietary adeno‐associated viral (AAV) vector platform, the company designs single‐dose treatments aimed at addressing the underlying genetic causes of disease rather than solely managing symptoms. Its most advanced program, Hemgenix® (etranacogene dezaparvovec), received regulatory approval in the United States and Europe for adult patients with hemophilia B, marking one of the first gene therapies for a bleeding disorder to reach the market.
Beyond hemophilia B, uniQure’s pipeline includes preclinical and clinical-stage candidates targeting rare and debilitating conditions such as aromatic l-amino acid decarboxylase (AADC) deficiency, Huntington’s disease, and Parkinson’s disease.
