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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 N.V. (NASDAQ: QURE) announced that the U.S. FDA has granted Orphan Drug Designation to AMT-191, their investigational gene therapy for Fabry disease. This follows the dosing of the first patient in a U.S. multi-center, open-label Phase I/IIa trial in August 2024. AMT-191 is a one-time intravenously administered AAV5-based gene therapy targeting the liver to produce GLA protein.
The Phase I/IIa trial will include two cohorts of up to six adult male patients each, with low and high doses. Patients will be followed for 24 months to assess safety, tolerability, and early efficacy signs. The Orphan Drug Designation provides incentives including tax credits, grants, fee waivers, and seven years of market exclusivity upon approval.
uniQure N.V. (NASDAQ: QURE) has initiated dosing in a Phase I/IIa clinical trial of AMT-191 for Fabry disease treatment. The multi-center, open-label trial in the US will assess safety, tolerability, and early efficacy signs in two dose-escalating cohorts. AMT-191 is an AAV5-based gene therapy delivering a galactosidase alpha (GLA) transgene to the liver. It aims to address the α-galactosidase A enzyme deficiency in Fabry patients.
The trial includes two cohorts of up to six adult male patients each, with low (6x10^13 gc/kg) and high (3x10^14 gc/kg) doses administered intravenously. Patients will be followed for 24 months. This milestone aligns with uniQure's goal to advance three new gene therapies into clinical studies this year, alongside programs in Huntington's disease, temporal lobe epilepsy, and SOD1-ALS.
uniQure (NASDAQ: QURE) reported its Q2 2024 financial results and provided a company update. Key highlights include:
1. RMAT designation for AMT-130 in Huntington's disease and positive interim Phase I/II data showing slowing of disease progression.
2. Initiated patient screening for three Phase I/II studies in epilepsy, ALS, and Fabry disease.
3. Announced organizational restructuring, reducing headcount by 65% and lowering cash burn by $75 million annually.
4. Strong cash position of $524 million as of June 30, 2024, expected to fund operations through 2027.
5. Q2 2024 revenue of $11.1 million, up from $2.4 million in Q2 2023.
6. Net loss of $56.3 million ($1.16 per share) in Q2 2024, compared to $68.5 million ($1.44 per share) in Q2 2023.
uniQure has closed the sale of its global manufacturing facility in Lexington, Massachusetts to Genezen, a contract development and manufacturing organization. This strategic move is expected to reduce uniQure's annual cash burn by $40 million and streamline operations while maintaining preferential access to gene therapy manufacturing capabilities.
Key points:
- uniQure becomes a significant shareholder in Genezen
- CEO Matt Kapusta joins Genezen's Board of Directors
- $50 million in outstanding debt retired
- Amin Abujoub appointed as new Chief Technical Operations Officer
- Chief Operating Officer role eliminated
The company aims to focus future investments on projects with potential to increase shareholder value and plans to announce additional cost reduction measures later this quarter.
uniQure announced positive interim data from Phase I/II trials of AMT-130 for Huntington's disease, showing a statistically significant, dose-dependent slowing in disease progression. At 24 months, high-dose patients demonstrated an 80% slowing of disease progression compared to a propensity score-weighted external control. Additionally, a significant reduction in CSF neurofilament light protein (NfL) levels was observed. The trials also indicated that AMT-130 remains well-tolerated with no new serious adverse events. uniQure aims to meet with the FDA in the second half of 2024 to discuss expedited clinical development pathways for AMT-130.
uniQure announced the sale of its manufacturing facility in Lexington, Massachusetts, to Genezen for $25 million, consisting of $12.5 million in Series C Preferred Stock and a $12.5 million convertible note. This divestiture will allow uniQure to reduce its annual cash burn by $40 million and is part of a broader strategy to cut operating expenses. Genezen will now manufacture HEMGENIX® and support uniQure's pipeline programs, with the majority of current employees transitioning to Genezen. The deal is expected to close in early Q3 2024, and uniQure will use the proceeds to repay $50 million in debt, further saving on interest expenses.
Genezen announced its acquisition of uniQure's commercial gene therapy operations in Lexington, MA. The acquisition includes a state-of-the-art, commercially-licensed viral vector manufacturing facility, enhancing Genezen's ability to provide late-phase and commercial gene therapy development services. The Lexington site will become Genezen's global AAV center of excellence, complementing its existing operations in Indianapolis. Genezen will also enter into strategic supply agreements for uniQure's clinical portfolio and CSL's HEMGENIX® product. The transaction is supported by additional growth equity from Ampersand Capital Partners.
uniQure has received the FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for its investigational gene therapy, AMT-130, for Huntington’s disease. This designation is based on 24-month interim data from Phase I/II clinical trials released in December 2023 and represents a significant milestone.
AMT-130 is the first therapy for Huntington’s disease to receive RMAT designation, facilitating closer collaboration with the FDA and potentially expediting its development and approval process. This decision underscores AMT-130's potential to address the unmet medical needs of Huntington’s disease patients.
uniQure plans to present updated interim data from ongoing Phase I/II studies in mid-2024, including follow-up results on 29 treated patients, 21 of whom will have data spanning at least two years.
uniQure N.V. reported its first quarter 2024 financial results and recent progress, focusing on advancing AMT-130 for Huntington's disease and initiating new Phase I/II trials for Fabry disease, ALS, and MTLE. The company is on track to interact with the FDA in Q2 2024 and reduce expenses to increase shareholder value. Financially, uniQure's revenues increased to $8.5 million, with a cash position of $555.7 million as of March 31, 2024.
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