uniQure gains on preliminary hemophilia B data

uniQure N.V. (NASDAQ:QURE) gained $2.37 (16%) to $17.28 on Thursday after reporting preliminary data from the low-dose cohort of an ongoing Phase I/II trial of AMT-060 to treat hemophilia B. The five-year trial is enrolling patients with Factor IX (FIX) phenotypic features of severe or moderately severe hemophilia, including documented FI levels less than 1-2% and required chronic prophylactic recombinant FIX (rFIX) treatment at enrollment.

uniQure said that as of Dec. 16, the first enrolled patient had FIX expression levels of 5.5% of normal after more than 20 weeks of follow-up and the second patient had levels 4.5% of normal with at least 12 weeks of follow up. As of Jan. 6, four of the five patients had discontinued prophylactic therapy. According to uniQure, maintaining durable FIX expression around 3%-5% of normal may have a significant clinical benefit. ...