Uniqure Stock Price, News & Analysis

uniQure (NASDAQ: QURE), a gene therapy company, has announced a proposed $200 million public offering of ordinary shares and pre-funded warrants. The company will grant underwriters a 30-day option to purchase additional shares up to 15% of the total offering.

The offering will be managed by Leerink Partners, Stifel, Van Lanschot Kempen, and Guggenheim Securities as bookrunning managers. The securities will be offered through uniQure's automatically effective shelf registration statement, with a preliminary prospectus supplement and accompanying prospectus to be filed with the SEC.

uniQure (NASDAQ: QURE) has secured a $175 million non-dilutive senior secured term loan facility with Hercules Capital. The deal includes refinancing of existing $50 million debt with extended maturity from 2027 to 2030, plus access to $125 million in additional funding. The financing features three tranches: $50 million for current debt refinancing, $100 million tied to regulatory and financial milestones, and $25 million subject to Hercules' approval.

The new arrangement offers a more favorable floating interest rate of 9.70% (previously 11.95%) and aims to support the potential commercial launch of AMT-130, uniQure's gene therapy for Huntington's disease, anticipated in 2026.

uniQure (NASDAQ: QURE) announced breakthrough results from its pivotal Phase I/II study of AMT-130 for Huntington's disease treatment. The study achieved its primary endpoint, demonstrating a statistically significant 75% disease progression slowdown at 36 months using cUHDRS measurements compared to controls.

Key highlights include a 60% slowing in disease progression measured by Total Functional Capacity (TFC), favorable trends across cognitive and motor functions, and an 8.2% mean reduction in CSF neurofilament light protein from baseline. The high-dose AMT-130 showed a manageable safety profile with no new drug-related serious adverse events since December 2022.

The company plans to submit a Biologics License Application (BLA) in Q1 2026, with potential U.S. launch later that year. AMT-130 has received both Breakthrough Therapy and RMAT designations from the FDA.

uniQure (NASDAQ: QURE) has announced promising initial data from its Phase I/IIa trial of AMT-191, a gene therapy for Fabry disease. The first cohort of four patients demonstrated remarkable results, with α-Gal A enzyme activity increasing 27- to 208-fold above normal levels.

All patients in Cohort A (6x1013 gc/kg) successfully discontinued enzyme replacement therapy (ERT) while maintaining stable plasma lyso-Gb3 levels. The therapy showed a manageable safety profile, though some adverse events were reported. A second cohort with a lower dose (2x1013 gc/kg) has completed enrollment with no serious adverse events reported to date.

The company plans to present updated clinical results in the first half of 2026.

uniQure (NASDAQ: QURE) reported Q2 2025 financial results and significant progress across its gene therapy pipeline. The company achieved FDA alignment for AMT-130 in Huntington's disease, supporting a planned BLA submission in Q1 2026. Key highlights include a 92% seizure reduction in the first AMT-260 patient for epilepsy treatment and the appointment of Kylie O'Keefe as Chief Customer and Strategy Officer.

Financial results show $377.0 million in cash and investments as of June 30, 2025, expected to fund operations into H2 2027. Q2 revenues were $5.3 million, down from $11.1 million in Q2 2024. Net loss improved to $37.7 million ($0.69 per share) compared to $56.3 million ($1.16 per share) in the same period last year.

uniQure (NASDAQ: QURE), a leading gene therapy company, will release its second quarter 2025 financial results before market open on Tuesday, July 29, 2025. The company will host a conference call and webcast at 8:30 a.m. ET to discuss the results.

The earnings call will be accessible via webcast through uniQure's website under the Events & Presentations section, with a replay available for 90 days. Phone participants must pre-register online to receive dial-in details and are advised to join 15 minutes before the start time.

uniQure (NASDAQ: QURE) has appointed Kylie O'Keefe as Chief Customer and Strategy Officer, effective June 6, 2025. O'Keefe will lead the global commercialization strategy for AMT-130, the company's investigational gene therapy for Huntington's disease, with a potential U.S. launch in 2026. She brings extensive experience in rare diseases and gene therapy from her previous role as Chief Commercial Officer at PTC Therapeutics, where she managed global commercial operations across 50+ countries and led launches including Upstaza for AADC deficiency. At uniQure, O'Keefe will oversee commercial functions and medical affairs, leveraging her expertise in reimbursement strategies, payer engagement, and health economic assessments. The appointment strengthens uniQure's leadership team as they prepare to transition into a commercial-stage biotechnology company with AMT-130, which could become the first disease-modifying treatment for Huntington's disease.

uniQure (NASDAQ: QURE) has announced significant progress in its regulatory pathway for AMT-130, its gene therapy treatment for Huntington's disease. Following FDA meetings, the company has aligned on key components for a Biologics License Application (BLA) planned for Q1 2026 under an Accelerated Approval pathway. The FDA has agreed that the composite Unified Huntington's Disease Rating Scale (cUHDRS) can serve as a registrational endpoint, with primary efficacy analysis comparing 3-year changes in high-dose AMT-130 patients against external controls from ENROLL-HD database. The FDA also approved CMC requirements, allowing validation using experience from HEMGENIX process plus additional GMP batches. Key upcoming milestones include submitting updated Statistical Analysis Plan in Q2 2025, presenting Phase I/II topline data in Q3 2025, conducting Process Performance Qualification run, and holding pre-BLA meeting in Q4 2025.

uniQure has presented promising early results from the first participant in its Phase I/IIa GenTLE clinical trial of AMT-260, a gene therapy candidate for refractory Mesial Temporal Lobe Epilepsy (MTLE). The patient, who previously experienced an average of seven seizures monthly, showed a remarkable 92% reduction in seizure frequency over five months post-treatment, with no seizures reported in the last 60 days and no serious adverse events. AMT-260 is a one-time gene therapy designed to suppress the GRIK2 gene and GluK2 expression, which are believed to trigger seizures. The ongoing trial will evaluate two dose cohorts of six patients each across 12 U.S. sites, with additional sites planned by end of 2025.