Uniqure Stock Price, News & Analysis

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.

uniQure (NASDAQ: QURE) reported Q1 2025 financial results and key developments. The company's AMT-130 for Huntington's disease received FDA Breakthrough Therapy designation, with initial safety data showing good tolerability. The company held FDA meetings to advance BLA preparations, with a regulatory update expected in Q2 2025. Financial highlights include cash position of $409.0 million as of March 31, 2025, expected to fund operations into H2 2027. Q1 revenues were $1.6 million, with a net loss of $43.6 million ($0.82 per share). The company completed a public offering raising $80.5 million. Pipeline progress includes AMT-260 for epilepsy, AMT-191 for Fabry disease, and AMT-162 for ALS, with various data presentations expected throughout 2025.

uniQure (NASDAQ: QURE) has announced it will report its first quarter 2025 financial results on Friday, May 9, 2025, before market open. The company will host its inaugural quarterly earnings conference call at 8:30 a.m. ET on the same day. This initiative to begin regular earnings calls comes as uniQure advances its pipeline and AMT-130 toward a BLA submission and potential commercialization, aiming to enhance engagement with the investment community.

The event will be accessible via webcast through uniQure's website, with a 90-day replay available afterward. Phone participants must pre-register through an online form to receive dial-in details and are advised to join 15 minutes before the start time.

uniQure (NASDAQ: QURE) has received FDA Breakthrough Therapy designation for AMT-130, its gene therapy treatment for Huntington's disease, a rare inherited neurodegenerative disorder with no current disease-modifying therapies.

The designation is based on promising interim data from Phase I/II trials showing dose-dependent slowing of disease progression. The company presented 24-month data in July 2024, demonstrating meaningful disease progression slowdown based on cUHDRS scores compared to natural history.

AMT-130 has already received Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations. The treatment has been administered to 45 patients to date, with additional regulatory guidance on the Biologics License Application expected in Q2 2025.