UniQure's shares rise on early study data for haemophilia B gene therapy

Shares in uniQure rose as much as 21 percent Thursday after the company released study data suggesting that its AAV5/FIX gene therapy AMT-060 can increase levels of Factor IX in patients with haemophilia B. Top-line results from the Phase I/II trial demonstrated that the first two patients given a low dose of the therapy had Factor IX expression levels of 5.5 percent and 4.5 percent of normal. CEO Dan Soland remarked "these preliminary...results support our hypothesis that AAV5/FIX can deliver clinically meaningful expression levels of FIX for patients with haemophilia B."

In the study, five adults with haemophilia B were treated with a low dose of AMT-060. All patients exhibited Factor IX phenotypic features of severe or moderately severe haemophilia including documented Factor IX levels of less than 1 percent to 2 percent and required chronic treatment with prophylactic recombinant Factor IX therapy at the time of study enrolment.

uniQure noted that the remaining three patients have not completed 12 weeks of follow-up at the cut-off date. The company said that four of the five patients, including the first two patients, have achieved the secondary endpoint of full discontinuation of prophylactic recombinant Factor IX therapy.

uniQure stated that it will present a more comprehensive analysis of data from this low-dose cohort at a scientific conference in the next quarter. The drugmaker also intends to initiate patient enrolment in the high-dose cohort in the current quarter, pending data monitoring committee approval.

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