Uniqure Stock Price, News & Analysis

uniQure N.V. has announced that the waiting period for its licensing agreement with CSL Behring for etranacogene dezaparvovec has ended, making the deal effective from May 6, 2021. uniQure will receive a $450 million upfront payment due by May 13, 2021, with potential future payments up to $1.6 billion based on milestones. The agreement includes tiered royalties of up to low-twenties percentage on sales. uniQure remains responsible for the pivotal HOPE-B study and initial manufacturing, while CSL Behring will handle commercialization.

uniQure N.V. (NASDAQ: QURE) announced its participation in three upcoming virtual investor and scientific conferences. The Truist Securities Life Sciences Summit is scheduled for May 4-5, 2021, featuring one-on-one meetings. The American Society of Gene and Cell Therapy (ASGCT) virtual event will take place from May 11-14, 2021, showcasing five data presentations, including two oral presentations on May 12 and 13. Finally, at the RBC Capital Markets Global Healthcare Conference on May 18-19, 2021, there will be additional virtual meetings and a fireside chat with CEO Matt Kapusta.

uniQure N.V. (NASDAQ: QURE) announced five data presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual 2021 Annual Meeting from May 11-14. The presentations will focus on gene therapy advancements targeting hemophilia and Huntington’s disease, highlighting the AAV5 vector's efficacy amid pre-existing neutralizing antibodies. Notable sessions include two oral presentations on the Phase 3 HOPE-B trial and miQURE technology's proof-of-concept for liver targeting. These sessions aim to showcase uniQure's innovation in gene therapy for severe medical conditions.

uniQure N.V. (NASDAQ: QURE) has announced that the FDA has lifted the clinical hold on its hemophilia B gene therapy program, following a thorough review regarding a patient who developed hepatocellular carcinoma (HCC) during the HOPE-B trial. Investigations showed no evidence that the therapy contributed to the patient's condition. uniQure is set to release 52-week data from the HOPE-B trial soon. The company has conducted extensive monitoring of patients, with no further HCC cases reported across over 100 participants.

uniQure N.V. (NASDAQ: QURE) announced that three preclinical studies on its gene therapy candidate AMT-130 for Huntington's disease were published in notable journals. The studies demonstrate the safety and efficacy of AMT-130, which showed significant long-term reductions in mutant huntingtin protein levels in minipig models. At 12 months post-administration, reductions were 85% in the putamen and 80% in the caudate. The results support ongoing Phase I/II clinical trials for AMT-130, showcasing a promising biomarker for monitoring therapy.

uniQure N.V. announced the successful completion of patient enrollment in the first dose cohort of a Phase I/II clinical trial for AMT-130, aimed at treating early-stage Huntington's disease. The trial involves a total of 26 patients, with 10 in the first cohort and 16 planned for the second dose cohort, slated to begin in 3Q 2021. Additionally, uniQure intends to initiate an open-label clinical study in Europe later this year, potentially impacting an estimated 75,000 Europeans affected by Huntington's disease.

uniQure N.V. (NASDAQ: QURE) announced its participation in multiple upcoming virtual investor and scientific conferences. Key events include the Guggenheim Healthcare Talks on April 1, where management will engage in discussions on gene therapy challenges. Following this, the Wells Fargo Corporate Access Days will take place from April 6-8, and the Needham Virtual Healthcare Conference from April 12-15. Additional panels will cover topics related to Huntington's disease and patient advocacy. The company's pipeline focuses on therapies for various severe conditions including hemophilia and Huntington's disease.

uniQure N.V. (NASDAQ: QURE) announced that an independent investigation suggests etranacogene dezaparvovec is highly unlikely to have caused hepatocellular carcinoma (HCC) in a patient from the HOPE-B pivotal trial. The analysis of over 220,000 cells indicated rare AAV vector integration events, constituting just 0.027% of the sample, with no evidence of clonal expansion. The tumor exhibited genetic mutations indicative of HCC, independent of the vector. The company is preparing to discuss these findings with the FDA, emphasizing the safety of their gene therapy program.

uniQure N.V. (NASDAQ: QURE) reported significant developments in its gene therapy pipeline in a recent press release. Key highlights include the completion of patient procedures in the Phase I/II study for Huntington's disease and the HOPE-B trial for hemophilia B, which showed promising data. Financial results for 2020 showed revenues surged to $37.5 million from $7.3 million in 2019, largely due to licensing agreements. However, the company faced a net loss of $125 million. A strong cash position of $244.9 million positions them well for upcoming trials and regulatory filings.