Welcome to our dedicated page for Uniqure news (Ticker: QURE), a resource for investors and traders seeking the latest updates and insights on Uniqure stock.
uniQure N.V. reports company news on its gene therapy pipeline, regulatory interactions, clinical data, operating results, and capital structure. The company develops genetic medicines for severe diseases, with disclosed programs including AMT-130 for Huntington’s disease, AMT-260 for refractory mesial temporal lobe epilepsy, AMT-191 for Fabry disease, ALS programs, and an approved gene therapy for hemophilia B.
Recurring updates cover FDA and MHRA discussions, clinical-trial observations, exploratory efficacy and safety data, quarterly and annual financial results, debt financing arrangements, corporate presentations, and governance or shareholder matters tied to its Nasdaq-listed public-company status.
uniQure (NASDAQ: QURE) reported Q1 2026 results and program updates on May 5, 2026. Key items: a Type B FDA meeting is scheduled to discuss AMT-130 trial design; a UK MAA for AMT-130 is expected in Q3 2026. AMT-260 Phase I/IIa enrollment continues; first-cohort data due June 18-19, 2026. AMT-191 showed durable increases in α-Gal A and all 11 dosed patients stopped enzyme replacement therapy. AMT-162 development discontinued. Cash totaled $586.6M, runway into H2 2029; Q1 revenue $3.6M; net loss $53.5M (EPS -$0.85).
uniQure (NASDAQ: QURE) held a Pre-Submission meeting with the UK MHRA and expects to file a Marketing Authorization Application (MAA) for AMT-130 in Huntington’s disease in Q3 2026. The planned MAA will rely on three-year Phase I/II data showing a 75% slowing of disease progression at the high dose (p=0.003) versus a propensity score-matched external control and a generally manageable safety profile. uniQure also secured a Type B meeting with the U.S. FDA in Q2 2026 to discuss potential Phase III design and the four-year analysis expected in Q3 2026, and is pursuing additional international regulatory pathways.
uniQure (NASDAQ: QURE) will report first quarter 2026 financial results before market open on Tuesday, May 5, 2026. Management will host a conference call at 8:30 a.m. ET the same day. The live webcast and a 90-day replay will be available in the Events & Presentations section of the company website.
Analysts may access the live call by dialing the provided numbers and entering conference ID 4607289. Participants are asked to join 15 minutes before start time.
uniQure (NASDAQ: QURE) reported 2025 results and program updates on March 2, 2026. Key highlights include $622.5 million in cash and investments as of Dec 31, 2025, expected to fund operations into H2 2029, and $16.1 million revenue for 2025 with a $199.0 million net loss.
Clinical updates: AMT-130 showed a 75% slowing on cUHDRS at 36 months but FDA recommended a prospective randomized, sham-controlled study; AMT-260 completed first cohort enrollment; AMT-191 showed durable, dose-dependent α-Gal A increases with dosing pauses for asymptomatic Grade 3 liver enzyme elevations; AMT-162 remains on voluntary pause.
uniQure (NASDAQ: QURE) received final FDA minutes from a Jan 30, 2026 Type A meeting about AMT-130 for Huntington’s disease. The FDA said Phase I/II data versus an external control are not sufficient to support a marketing application and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study.
uniQure plans further engagement and intends to request a Type B meeting in Q2 2026 to discuss Phase III design options.
uniQure (NASDAQ: QURE) will report fourth-quarter and full-year 2025 financial results before market open on Monday, March 2, 2026. Management will host a live conference call at 8:00 a.m. ET the same day.
The event will be webcast via the company’s Events & Presentations page and a replay will be archived for 90 days. Analysts may join the live Q&A by phone using conference ID 4607289; attendees are asked to dial in 15 minutes early.
uniQure (NASDAQ: QURE) reported updated preliminary Phase I/IIa AMT-191 data in 11 Fabry disease patients showing dose-dependent, sustained elevations in α-Gal A activity across three dose cohorts through a cutoff of Jan 8, 2026.
α-Gal A increases ranged from 0.34- to 312.52-fold versus mean normal by dose; six of 11 patients stopped enzyme replacement therapy, and plasma lyso-Gb3 remained stable. Safety showed manageable events, with protocol pause of additional mid/high dosing after two Grade 3 liver enzyme elevations at the mid dose.
uniQure (NASDAQ: QURE) announced a Type A meeting with the FDA to discuss the Biologics License Application (BLA) data package seeking accelerated approval for AMT-130, its investigational gene therapy for Huntington's disease. The company said it expects to provide a regulatory update after receipt of official meeting minutes and emphasized continued collaboration with the FDA and commitment to patient access.
This meeting represents a formal regulatory engagement on the pathway to potential accelerated approval but does not guarantee a regulatory decision.
uniQure (Nasdaq: QURE) five-year HOPE-B results published in NEJM on Dec 7, 2025 confirm long-term durability and safety of a single HEMGENIX infusion for adults with hemophilia B.
Key outcomes at year five: mean factor IX activity ~36.1%, adjusted annualized bleeding rate (ABR) for all bleeds reduced ~90% versus lead-in, joint bleeds reduced 93%, spontaneous bleeds reduced 94%, and 94% of patients remained off continuous prophylaxis. No serious adverse events were attributed to treatment; most treatment-related adverse events occurred in the first four months. Over 75 individuals across eight countries have received HEMGENIX in real-world use.
uniQure (NASDAQ: QURE) received final meeting minutes from the FDA following an Oct 29, 2025 pre-BLA meeting about AMT-130 for Huntington’s disease. The FDA conveyed that Phase I/II data are currently unlikely to provide the primary evidence to support a BLA. uniQure said it is evaluating the feedback and plans to urgently request a follow-up meeting in Q1 2026. Management reaffirmed commitment to working with regulators and noted community support from patients, families, clinicians and advocates.
The update signals additional regulatory engagement is needed before a BLA pathway can be confirmed.