Uniqure Stock Price, News & Analysis

uniQure N.V. (NASDAQ: QURE) reported Q3 2020 results with revenues of $1.8M, increasing from $1.0M in Q3 2019. The company enrolled four patients in the Phase I/II trial of AMT-130 for Huntington's disease and expects top-line data from the HOPE-B trial in hemophilia B by year-end. A licensing agreement with CSL Behring may yield $450M upfront and up to $1.6B in milestones. However, a net loss of $53.8M was recorded, up from $23.6M a year prior. Cash reserves stand at $279.5M, sufficient to fund operations into 2022, even without CSL Behring's payment.

uniQure (NASDAQ: QURE) announced the completion of two additional procedures in its Phase I/II clinical trial for AMT-130, aimed at treating Huntington’s disease. This follows an independent Data Safety Monitoring Board review, which reported no significant safety concerns. A total of four patients have now been enrolled, with two receiving AMT-130. The trial will assess safety and efficacy in a total of 26 patients. Enrollment is expected to continue after further DSMB reviews, with completion anticipated by mid-2021, aligning with the company's original clinical timeline.

uniQure (NASDAQ: QURE) announced that the independent Data Safety Monitoring Board (DSMB) reviewed 90-day safety data from the first two patients in its Phase I/II trial of AMT-130 for Huntington's disease. No significant safety concerns were noted, allowing the trial to proceed with enrollment of the next two patients. The double-blind study will assess safety and efficacy in 26 patients with early-stage Huntington's disease. The trial is expected to provide insights into the potential of AMT-130 as a treatment option.

uniQure N.V. (NASDAQ: QURE) announced participation in several upcoming virtual conferences in September 2020. Key events include:

• Citi’s 15th Annual BioPharma Virtual Conference (Sept. 9-10) with a live fireside chat on Sept. 9.
• Baird’s Virtual Global Healthcare Conference (Sept. 9-10) featuring one-on-one investor meetings.
• Wells Fargo Global Healthcare Conference (Sept. 9-10) with a fireside chat on Sept. 10.
• H.C. Wainwright 22nd Annual Global Investment Conference (Sept. 14-15) and Cantor Fitzgerald Virtual Global Healthcare Conference (Sept. 15-17).
• Barclays Boston Biotech Bus Trip (Sept. 23-25).

These events aim to provide insights into uniQure's gene therapy advancements.

uniQure N.V. (NASDAQ: QURE) appointed Ricardo Dolmetsch, Ph.D. as the new President of Research and Development, effective September 14, 2020, succeeding Sander van Deventer, M.D., Ph.D.. Dolmetsch, previously Global Head of Neuroscience at Novartis, brings extensive experience in gene therapy, notably in developing treatments for neurodegenerative diseases. CEO Matt Kapusta praised Dolmetsch's expertise and vision. Under Dolmetsch's leadership, uniQure aims to advance therapies for Huntington’s disease and expand its pipeline. The company plans an extraordinary shareholder meeting in Q4 2020 to re-elect Dr. Robert Gut as a non-executive director.

uniQure N.V. (NASDAQ: QURE) reported significant developments in its Q2 2020 results, including a global licensing agreement with CSL Behring for its hemophilia B gene therapy, potentially worth over $2 billion. The company completed initial dosing in a Phase I/II trial for AMT-130, targeting Huntington’s disease. Financially, uniQure held $314.3 million in cash, expected to fund operations into 2024, despite reporting a net loss of $42.6 million. The revenue declined to $1.5 million, a decrease from $2.5 million year-over-year.

CSL Behring has announced its acquisition of exclusive global rights to AMT-061, a novel gene therapy for hemophilia B from uniQure (QURE). The program, currently in Phase 3 trials, could offer long-term benefits to patients. An upfront payment of US$450 million will be made to uniQure, along with milestone payments and royalties. The therapy aims to restore Factor IX levels in patients, potentially eliminating the need for ongoing treatments. This acquisition supports CSL Behring's commitment to innovative therapies in hemophilia and expands its gene therapy portfolio.

uniQure has entered a licensing agreement with CSL Behring granting exclusive global rights to develop and commercialize etranacogene dezaparvovec, a gene therapy for hemophilia B. uniQure will receive over $2 billion in total, including $450 million upfront and $1.6 billion in milestone payments, along with royalties in the low-twenties percentage range of net sales. This collaboration aims to leverage CSL Behring's global reach to enhance patient access and enable uniQure to accelerate its pipeline, including advancing treatments for Huntington’s disease and Fabry disease.

uniQure (NASDAQ: QURE) announced the treatment of the first two patients in its Phase I/II clinical trial for AMT-130, aimed at Huntington’s disease. This double-blind study, involving 26 patients, will assess safety, tolerability, and efficacy signals. One patient received AMT-130 while the other underwent sham surgery. The trial features an 18-month core study followed by long-term follow-ups. The innovative delivery method involves MRI-guided surgery. This milestone reflects uniQure's commitment to developing effective therapies for Huntington’s disease, a condition with no currently approved treatments.