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. (NASDAQ: QURE) is a gene therapy company that regularly issues news on its clinical programs, regulatory interactions, and financing activities. The company describes itself as delivering single‑treatment gene therapies with potentially curative results and has reported that the approvals of its gene therapy for hemophilia B mark a major milestone in genomic medicine. News about uniQure often highlights progress in its pipeline for Huntington’s disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe conditions.
A major focus of QURE news coverage is AMT‑130, uniQure’s investigational gene therapy for Huntington’s disease. Press releases and related SEC filings detail pivotal Phase I/II trial results, including statistically significant slowing of disease progression at 36 months in high‑dose cohorts compared to a propensity score‑matched external control, as well as trends in motor and cognitive endpoints and biomarker changes. Investors also see updates on the company’s regulatory dialogue with the U.S. Food and Drug Administration, including pre‑BLA and Type A meetings and feedback on the adequacy of Phase I/II data for a potential Biologics License Application.
Beyond AMT‑130, uniQure news includes early‑stage data and enrollment updates for AMT‑260 in refractory mesial temporal lobe epilepsy, AMT‑191 in Fabry disease, and AMT‑162 in SOD1 ALS. Corporate news items cover public offerings of ordinary shares and pre‑funded warrants, amendments to loan facilities, and quarterly financial results. This QURE news page aggregates such disclosures so readers can follow clinical milestones, regulatory developments, and capital markets events that shape uniQure’s gene therapy business over time.
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.
uniQure (NASDAQ: QURE) reported its 2024 financial results and company progress. Key highlights include FDA alignment on Accelerated Approval pathway for AMT-130 in Huntington's disease and completion of patient enrollment in its third cohort Phase I/II study. The company initiated dosing in Phase I/II studies for multiple programs including AMT-260 for epilepsy, AMT-191 for Fabry disease, and AMT-162 for SOD1-ALS.
Financial position shows cash and equivalents of $367.5 million as of December 31, 2024, plus $80.7 million from recent financing, expected to fund operations into second half of 2027. Revenue increased to $27.1 million in 2024 from $15.8 million in 2023. The company reported a net loss of $239.6 million ($4.92 per share) compared to $308.5 million ($6.47 per share) in 2023.
CSL Behring announced four-year results from the pivotal HOPE-B study of HEMGENIX, their gene therapy for hemophilia B. The data demonstrates sustained efficacy and safety through four years post-treatment. Key findings include:
- 94% of patients remained free of continuous factor IX prophylaxis
- Mean factor IX activity levels maintained at 37% through four years
- Approximately 90% reduction in mean adjusted annualized bleeding rate from lead-in to year four
- Joint bleeds reduced from 2.34 to 0.09 mean ABR
The Phase III HOPE-B trial involved 54 adult male participants with severe or moderately severe hemophilia B. No serious adverse events related to HEMGENIX were reported, with 96% of treatment-related adverse events occurring in the first six months. HEMGENIX, approved by FDA in 2022, is the first gene therapy for hemophilia B that can treat patients with and without AAV5 neutralizing antibodies.
uniQure (NASDAQ: QURE) has announced the completion of enrollment in the first cohort of its Phase I/IIa trial for AMT-191, a gene therapy treatment for Fabry disease. The Independent Data Monitoring Committee (IDMC) reviewed safety data from the initial two patients and found no significant safety concerns, recommending progression to the second cohort.
The company plans to begin enrollment for the second dose cohort in Q1 2025. AMT-191 is an AAV5-based gene therapy utilizing a proprietary promoter to deliver a galactosidase alpha (GLA) transgene targeting the liver to produce GLA protein. This one-time intravenous treatment aims to address Fabry disease, a condition where GLA gene variants cause α-galactosidase A enzyme deficiency, leading to lipid accumulation in multiple cell types and resulting in a multi-system disorder.
uniQure (NASDAQ: QURE) announced a favorable recommendation from the Independent Data Monitoring Committee (IDMC) for its Phase I/II EPISOD1 clinical trial of AMT-162, a gene therapy for SOD1-ALS. The IDMC reviewed 28-day safety data from the first study cohort and found no significant safety concerns, recommending progression to the second cohort.
AMT-162 is an AAVrh10-based gene therapy designed to reduce mutated SOD1 protein expression through miRNA. The therapy targets SOD1-ALS, where patients produce misfolded SOD1 protein that damages motor neurons, leading to muscle weakness and eventual death. The treatment is administered intrathecally as a one-time dose, aiming to slow or stop SOD1-ALS progression. The company plans to begin enrollment for the second dose cohort in Q1 2025.
uniQure (Nasdaq: QURE) has announced the pricing of its public offering of 4,411,764 ordinary shares at $17.00 per share, expecting to raise approximately $75 million in gross proceeds before deducting underwriting costs and expenses. The company has also granted underwriters a 30-day option to purchase up to 661,764 additional shares at the same price.
The offering is expected to close around January 10, 2025. Leerink Partners, Stifel, and Guggenheim Securities are serving as bookrunning managers, while Chardan and H.C. Wainwright & Co. are acting as lead managers for the offering. The shares are being offered through an automatically effective shelf registration statement filed with the SEC on January 7, 2025.
uniQure (Nasdaq: QURE) has announced the launch of an underwritten public offering of ordinary shares and pre-funded warrants to purchase ordinary shares. The gene therapy company plans to grant underwriters a 30-day option to purchase up to an additional 15% of ordinary shares at the public offering price, less underwriting discounts and commissions.
Leerink Partners is serving as the bookrunning manager for the proposed offering. The securities will be offered through an automatically effective shelf registration statement on Form S-3 filed with the SEC on January 7, 2025. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC.