Shares Of Uniqure NV Surge Following Release Of Clinical Trial Data

Shares of Uniqure NV QURE surged higher by more than 13 percent on Thursday after the company detailed positive topline results from a clinical study. Uniqure, a pharmaceutical company engaged in the field of gene therapy for orphan diseases, announced preliminary topline results from the low-dose cohort of an ongoing Phase I/II clinical trial being conducted in adult hemophilia B patients treated with uniQure's novel AAV5/FIX gene therapy, AMT-060. The company said that all 5 patients in the low-dose cohort had Factor IX (FIX) phenotypic features of severe or moderately-severe emophilia including documented Factor IX (FIX) levels less than 1-2% and required chronic treatment with prophylactic recombinant FIX (rFIX) therapy at the time of enrollment. Uniqure stated it will present a more complete analysis at a scientific conference in the second quarter. The company also anticipates initiating enrollment of the hgih-dose cohort during the current quarter. "Thus far, the overall tolerability and FIX expression profile in the low-dose cohort is encouraging for patients with hemophilia B and support the continuation of the study," commented Professor Frank W.G. Leebeek, M.D. Ph.D. of the Erasmus Medical Center in Rotterdam, an investigator in the study. "Previous studies have demonstrated that maintaining durable FIX expression around 3% to 5% of normal may have a significant clinical benefit as measured by significant reduction in consumption of units of FIX concentrate and lower risk of spontaneous bleeding episodes."