QURE - Earnings announcements

~ uniQure to host earnings call on Tuesday, May 5, 2026 at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, April 28, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report first quarter 2026 financial results before market open on Tuesday, May 5, 2026. Management will then host a conference call at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Analysts wishing to participate in the question and

~ Held Type A meeting with FDA to discuss AMT-130 for Huntington's disease; Company evaluating Phase III development considerations and plans to request follow-up Type B meeting in the second quarter of 2026 ~ ~ Completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026 ~ ~ Presented updated Phase I/II data from AMT-191 in Fabry disease showing durable, dose-dependent increases in α-Gal A enzyme activity ~ ~ Cash, cash equivalents and current investment securities of approximately $622.5 million as of December 31, 2025 expected to fund operations into the second

LEXINGTON, Mass. and AMSTERDAM, Feb. 23, 2026 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report fourth quarter and full year of 2025 financial results before market open on Monday, March 2, 2026. Management will then host a conference call at 8:00 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Analysts wishing to participate in the question and answer session should access the live call by dialing (64

LEXINGTON, Mass. and AMSTERDAM, Nov. 06, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report third quarter 2025 financial results before market open on Monday, November 10, 2025. Management will then host a conference call at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Analysts wishing to participate in the question and answer session should access the live call by dialing (646) 307-1963 or

~ Pivotal study met primary endpoint; high-dose AMT-130 demonstrated statistically significant 75% disease slowing at 36 months as measured by cUHDRS compared to a propensity score-matched external control ~ ~ High-dose AMT-130 also demonstrated statistically significant slowing of disease progression as measured by TFC, a key secondary endpoint, and favorable trends across additional clinical measures ~ ~ Mean cerebrospinal fluid NfL levels were below baseline at 36 months ~ ~ AMT-130 continued to be generally well-tolerated with a manageable safety profile ~ ~ uniQure plans to submit a BLA in the first quarter of 2026, with anticipated U.S. launch later that year, pending approval ~

~ Achieved alignment with the FDA on the AMT-130 statistical analysis plan and CMC requirements to support a planned BLA submission in the first quarter of 2026; ~ Company expects to present AMT-130 topline three-year data in September 2025 ~ ~ Presented case study from first participant treated with AMT-260 for refractory mesial temporal lobe epilepsy showing 92% seizure reduction with no serious adverse events through first five months of follow up ~ ~ Appointment of Kylie O'Keefe as Chief Customer and Strategy Officer ~ ~ Cash, cash equivalents and current investment securities of approximately $

LEXINGTON, Mass. and AMSTERDAM, July 22, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report second quarter 2025 financial results before market open on Tuesday, July 29, 2025. Management will then host a conference call at 8:30 a.m. ET. The event will be webcast under the Events & Presentations section of uniQure's website at https://www.uniqure.com/investors-media/events-presentations, and following the event a replay will be archived for 90 days. Interested parties participating by phone will need to register using this online form. After registering for dial-in detai

~ Alignment with FDA continues to support Accelerated Approval pathway ~ ~ BLA submission planned for first quarter of 2026 ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 02, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today provided a regulatory update on AMT-130, its investigational gene therapy for the treatment of Huntington's disease. Following recent Type B meetings and further guidance from the U.S. Food and Drug Administration (FDA), the Company has reached alignment with the FDA on several key components of the statistical analysis

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, May 05, 2025 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs will report first quarter 2025 financial results before market open on Friday, May 9, 2025. Management will then host a conference call at 8:30 a.m. ET. As the Company advances its pipeline and AMT-130 toward a BLA submission and potential commercialization, uniQure is initiating quarterly earnings calls to provide regular updates on progress and to enhance engagement with the investment community. The event will be webcast under the Events & Presentations section of uniQure's

~ U.S. Food and Drug Administration (FDA) agrees that data from ongoing Phase I/II studies compared to a natural history external control may serve as the primary basis for a Biologics License Application (BLA) for Accelerated Approval ~ ~ FDA agrees that the composite Unified Huntington's Disease Rating Scale (cUHDRS) may serve as an intermediate clinical endpoint for Accelerated Approval ~         ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, Dec. 10, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company reached agreement

~ Achieved statistically significant, dose-dependent, and durable evidence of potential therapeutic benefit; Patients receiving high-dose AMT-130 showed 80% slowing of disease progression in the composite Unified Huntington's Disease Rating Scale (cUHDRS) at 24 months compared to a propensity score-weighted external control ~ ~ Achieved statistically significant lowering of CSF neurofilament light protein (NfL) compared to baseline at 24 months in patients treated with AMT-130; Mean CSF NfL levels for both doses were below baseline at 24 months ~ ~ Granted first-ever Regenerative Medicine Advanced Therapy (RMAT) designation in Huntington's disease; uniQure expects to meet with the FDA in t

~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline at 18 months ~ ~ AMT-130 continues to be generally well-tolerated across both doses ~ ~ Data support continuing clinical development of AMT-130 and pursuing regulatory interactions to discuss potential strategies for ongoing development ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, D

~ AMT-130 continues to be generally well-tolerated across both dose cohorts ~ ~ Patients treated with AMT-130 show preserved function compared to baseline and clinical benefits relative to natural history of the disease ~ ~ Neurofilament Light Chain (NfL) in cerebrospinal fluid (CSF) was below baseline at 24 months in patients treated with the low-dose of AMT-130 and declining towards baseline at 12 months in patients treated with the high-dose of AMT-130 ~ ~ Suppression of CSF mHTT in low-dose cohort supports AMT-130 target engagement; Greater variability observed in high-dose cohort ~ ~ Promising data support continuing clinical development of AMT-130 and pursuing regulatory interacti

~ Announced 12-month data on the lower-dose cohort of AMT-130 in Huntington's disease showed the investigative gene therapy was generally well tolerated at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF) ~ ~ Announced postponement of AMT-130 higher-dose procedures due to recent suspected unexpected severe adverse reactions at this dose; Lower-dose procedures are not affected and no impact is expected on anticipated data readouts in 2023 ~ ~ Advancing regulatory reviews of the U.S. and European marketing applications for etranacogene dezaparvovec in hemophilia B ~ ~ Investor conference call and webcast today at 8:30 a.m.

~ Treatment generally well-tolerated with no significant safety issues related to AMT-130 in treated patients through one year of follow-up ~ ~ A mean reduction of 53.8% of mutant HTT (mHTT) observed in cerebral spinal fluid (CSF) at 12 months in evaluable patients treated with AMT-130 ~ ~ Neurofilament Light Chain (NfL) in CSF near baseline at 12 months in patients treated with AMT-130 ~ ~ Biomarker and clinical data including 24-month follow-up in the low-dose U.S. cohort and 12-month follow up in the high-dose U.S. cohort expected in first half of 2023 ~ ~ Investor conference call and webcast today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, June 23, 2022 (GLOBE NEWSWIRE) --

~ Treatment was well tolerated with no significant safety issues related to AMT-130 in first two treated patients through one year of follow-up ~ ~ Neurofilament Light Chain (NfL) rose as expected immediately following surgery and returned to baseline in treated patients ~ ~ A total of 19 patient procedures have been performed in the U.S. Phase I/II clinical trial, with higher-dose cohort enrollment expected to be completed by mid-2022 ~ ~ Screening initiated in European open-label Phase I/II study ~ ~ Conference call today at 8:30 a.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, The Netherlands, Dec. 16, 2021 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company ad

~ Strong Presence at ASH Featuring Five Presentations, Including Late-BreakingOral Presentation on HOPE-B Pivotal Trial ~ ~ uniQure to Host Investor Webcast Tuesday, December 8, 2020 at 5:00 p.m. ET ~ LEXINGTON, Mass. and AMSTERDAM, Nov. 30, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that Steven Pipe, M.D., professor of pediatrics and pathology and pediatric medical director of the hemophilia and coagulation disorders program at the University of Michigan, will present clinical data from the HOPE-B pivotal trial of etranacogene dezaparvovec in hemophilia B