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uniQure N.V. - QURE STOCK NEWS

Welcome to our dedicated page for uniQure N.V. news (Ticker: QURE), a resource for investors and traders seeking the latest updates and insights on uniQure N.V. stock.

uniQure N.V. (NASDAQ: QURE) is a leading gene therapy company focused on developing transformative therapies for patients suffering from severe genetic and other devastating diseases. Based on its proprietary technology platform, uniQure aims to deliver single-treatment solutions with potentially curative results. The company is dedicated to addressing unmet medical needs in the areas of hemophilia, Huntington's disease, and cardiovascular diseases.

uniQure's core business revolves around advancing a robust pipeline of gene therapies, including its collaboration with Bristol Myers Squibb to develop treatments for cardiovascular diseases. The company’s most notable product, HEMGENIX®, is the first and only gene therapy approved for the treatment of adults with hemophilia B. Recent data from the HOPE-B study presented at the American Society of Hematology Annual Meeting confirmed the long-term efficacy and safety of HEMGENIX®, highlighting its ability to offer elevated and sustained factor IX activity levels for years post-treatment.

The company's financial highlights and strong partnerships underscore its commitment to innovation and leadership in the gene therapy field. uniQure continues to make significant strides with current projects and patient enrollment for new trials expected to begin in the first half of 2024.

With its validated modular technology platform and a dedicated focus on severe genetic diseases, uniQure’s work holds significant promise for transforming patient care and outcomes. The company's latest developments and financial performance are followed closely by investors and the biotechnology community, reflecting its pivotal role in advancing gene therapy solutions.

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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.

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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.

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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.

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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.

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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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uniQure N.V. (QURE) reported promising clinical updates for AMT-130 in Huntington’s disease, FDA clearance for IND applications, strategic reorganization for cost savings, and a strong cash position. Financially, the company holds approximately $618 million in cash as of December 31, 2023, with revenues of $15.8 million for 2023. However, there was a significant net loss of $308.5 million in 2023.
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uniQure N.V. (NASDAQ: QURE) announced updated interim data from Phase I/II clinical trials of AMT-130 for Huntington’s disease, showing preserved neurological function and favorable trends in neuronal degradation with generally well-tolerated gene therapy.
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CSL (ASX: CSL; USOTC: CSLLY) announced the three-year results from the pivotal HOPE-B study confirming continued long-term durability and safety of HEMGENIX® (etranacogene dezaparvovec-drlb) gene therapy for hemophilia B. The treatment offers elevated and sustained factor IX activity levels for years, reducing the rate of annual bleeds and the need for regular prophylactic infusions.
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uniQure N.V. (NASDAQ: QURE) granted 142,300 restricted share units and options to purchase 52,500 ordinary shares to 28 newly hired employees as inducement to commence their employment. Each option has an exercise price of $7.30 per share, with a ten-year term and vesting over four years. The restricted share units vest in three annual installments beginning on December 7, 2024.
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uniQure N.V. (NASDAQ: QURE) has received FDA clearance for its gene therapy candidate, AMT-191, for the treatment of Fabry disease. The company's chief medical officer, Walid Abi-Saab, M.D., highlighted the potential of AMT-191 as a one-time treatment incorporating proprietary technology. Patient enrollment for the Phase I/II trial is expected to begin in the first half of 2024.
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FAQ

What is uniQure N.V.?

uniQure N.V. is a gene therapy company focused on developing treatments for severe genetic and other devastating diseases, including hemophilia, Huntington's disease, and cardiovascular diseases.

What is HEMGENIX®?

HEMGENIX® is a gene therapy developed by uniQure for the treatment of adults with hemophilia B. It offers elevated and sustained factor IX activity levels following a one-time infusion.

What are the recent achievements of uniQure?

Recent achievements include the long-term efficacy and safety data from the pivotal HOPE-B study for HEMGENIX®, showing sustained factor IX activity levels and reduced annual bleeding rates for hemophilia B patients.

What are uniQure's current projects?

uniQure is currently advancing a pipeline of gene therapies, with new patient enrollment for trials expected to begin in the first half of 2024.

Who are uniQure's partners?

uniQure collaborates with Bristol Myers Squibb for the development of gene therapies for cardiovascular diseases.

Where is uniQure N.V. listed?

uniQure N.V. is listed on the NASDAQ stock exchange under the ticker symbol QURE.

What is the focus of uniQure's research and development?

uniQure focuses on developing gene therapies for severe genetic diseases, leveraging its modular and validated technology platform.

How does HEMGENIX® work?

HEMGENIX® uses a non-infectious viral vector to deliver genetic instructions to liver cells, enabling the body to produce stable levels of factor IX, which is deficient in hemophilia B patients.

What are the financial highlights of uniQure?

uniQure's financial highlights reflect its robust pipeline and ongoing advancements in gene therapy, supported by strategic partnerships and investor interest.

What is the significance of the HOPE-B study?

The HOPE-B study provides long-term follow-up data demonstrating the efficacy and safety of HEMGENIX®, reinforcing its potential as a groundbreaking treatment for hemophilia B.

uniQure N.V.

Nasdaq:QURE

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423.35M
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5.39%
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