uniQure N.V. - QURE STOCK NEWS

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.

uniQure N.V. (NASDAQ: QURE) has announced its participation in several upcoming virtual investor and scientific conferences. Key events include the Cowen 41st Annual Health Care Conference from March 1-4, featuring a panel on neurological therapies, and the H.C. Wainwright Global Life Science Conference on March 9-10 with a fireside chat by CEO Matt Kapusta. Additionally, the company will participate in the Gene Therapy for Blood Disorders workshop on March 9-11 and Gene Therapy Patient Engagement on March 23-25. The Stifel CNS Day will occur from March 31 to April 1.

uniQure (NASDAQ: QURE) announced positive progress in its Phase I/II clinical trial of AMT-130 for Huntington's disease. An independent Data Safety Monitoring Board confirmed no significant safety concerns after reviewing six-month safety data from the first two patients and 90-day data from two others. All remaining patients in the first cohort are cleared for enrollment, targeting full enrollment by mid-2021. The trial aims to assess safety, tolerability, and efficacy in 26 patients, with initial biomarker and imaging data expected by year-end 2021.

uniQure N.V. (NASDAQ: QURE) has announced its participation in several key virtual investor and scientific conferences in February 2021. Highlighted events include the 14th Annual EAHAD Congress, where uniQure will present data on their gene therapy candidate, etranacogene dezaparvovec, for hemophilia B. Other presentations will focus on medical affairs and healthcare economics. The company will also engage in a fireside chat during the SVB Leerink Global Healthcare Conference. Additional details on their presentations will be disclosed closer to the event dates.

uniQure N.V. (NASDAQ: QURE) has announced that its hemophilia B gene therapy program, including the pivotal Phase III HOPE-B study, is on clinical hold by the FDA after a serious adverse event linked to liver cancer was reported in a patient. Although patient dosing is complete, no new patients will be treated. A full surgical resection of the liver lesion is scheduled, with no other cases of liver cancer reported in over 100 patients. The company is investigating the event and plans to monitor patients without impacting regulatory timelines.