Uniqure (QURE) Stock News

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.

uniQure (NASDAQ: QURE) reported Q3 2025 results and program updates on Nov 10, 2025. Key clinical highlights include AMT-130 Phase I/II topline 36-month data showing a 75% slowing in disease progression on the cUHDRS (p=0.003) and 60% slowing on TFC (p=0.033) versus an external control; safety remained manageable through 36 months. The company said preliminary FDA pre-BLA feedback introduced uncertainty about using Phase I/II versus external control as primary evidence and plans urgent follow-up.

Other updates: AMT-260 and AMT-191 have additional data expected H1 2026; AMT-162 enrollment paused after a dose-limiting toxicity. Financially, uniQure raised ~$323.7M net proceeds and held $694.2M in cash and investments as of Sept 30, 2025.

uniQure (NASDAQ: QURE) will report third quarter 2025 financial results before market open on Monday, November 10, 2025. Management will host a conference call at 8:30 a.m. ET the same day that will be webcast under the Events & Presentations section of uniQure’s investor website.

Analysts may join the live Q&A by dialing (646) 307-1963 or toll-free (800) 715-9871 with passcode 2196195. Attendees are asked to join 15 minutes before the start time. A replay of the webcast will be archived for 90 days.

uniQure (NASDAQ: QURE) on Nov. 3, 2025 announced FDA feedback from a pre-BLA meeting for AMT-130, its investigational gene therapy for Huntington’s disease.

uniQure said the FDA now appears to disagree that Phase I/II data versus an external control—as prespecified—may be adequate as primary evidence for a BLA, a material shift from prior Type B meeting guidance. As a result, the BLA timing is now unclear. uniQure expects final meeting minutes within 30 days and plans urgent interactions with the FDA while also engaging regulators in the EU and UK. AMT-130 holds Breakthrough Therapy (Apr 2025) and RMAT (May 2024) designations.

uniQure (NASDAQ:QURE) has successfully closed its upsized public offering, raising approximately $345 million in gross proceeds. The offering included 6,736,841 ordinary shares at $47.50 per share, including 947,368 additional shares from the underwriters' fully exercised option. Additionally, the company issued pre-funded warrants for 526,316 ordinary shares to certain investors.

The proceeds will fund commercialization readiness activities, potential commercial launch of AMT-130, development of clinical product candidates, business development initiatives, and general corporate purposes. The offering was managed by Leerink Partners, Stifel, Guggenheim Securities, and Van Lanschot Kempen as bookrunners, with H.C. Wainwright & Co. as lead manager.

uniQure (NASDAQ:QURE) has priced its upsized public offering of 5,789,473 ordinary shares at $47.50 per share, along with pre-funded warrants to purchase 526,316 ordinary shares. The offering is expected to generate approximately $300 million in gross proceeds before deducting underwriting costs and expenses.

The company has granted underwriters a 30-day option to purchase up to 947,368 additional ordinary shares. The offering is anticipated to close around September 29, 2025. Leerink Partners, Stifel, Guggenheim Securities, and Van Lanschot Kempen are serving as bookrunning managers, while H.C. Wainwright & Co. is acting as lead manager for the offering.