UniQure Up On Positive Data From Mid-Stage Hemophilia Study

uniQure N.V. (NASDAQ:QURE) announced encouraging initial top-line data from a phase IIb dose-confirmation study evaluating its AAV5-based gene therapy candidate, AMT-061, in patients with severe and moderately severe hemophilia B. The candidate has achieved and sustained the required (therapeutic) levels of Factor IX activity in all patients over the duration of treatment. Factor IX promotes blood clotting and lack of it causes hemophilia B, a genetic disorder causing uncontrolled bleeding.

Shares of the company rallied 35.7% on Nov 15, following the announcement. uniQure’s shares have increased 57.9% so far this year against the industry's decline of 20.6%.

The phase IIb study evaluated single infusion of a dose 2x10^13 vc/kg of AMT-061 in three patients with severe hemophilia B for tolerability of the dose and improvement in Factor IX activity over a period of six weeks. However, the company will continue to observe the patients for a year.

Data from the study showed that the candidate achieved a mean Factor IX activity of 31% of normal level, which is considered enough to significantly reduce the risk of bleeding events. A study, Den Uijl IE et al Haemophilia 2011, showed that 12% of normal Factor IX activity is sufficient for substantial reduction or elimination of spontaneous bleeding in hemophilia B patients. None of the patients required a Factor IX infusion and Immunosuppression and there have been no reports of bleeding events so far.

uniQure expects Factor IX activity to increase over the long term based on data from a phase I/II on its another pipeline candidate, AMT-060. In patients treated with AMT-060 in the study, Factor IX level has continued to increase beyond the levels achieved during six to 10 weeks.

Currently approved treatments for hemophilia B typically require patients to receive periodic infusion of Factor IX. However, successful development of AMT-061 will provide the company a significant opportunity as it requires single infusion and Factor IX activity levels continue to increase over the long term.

AMT-061 enjoys Breakthrough Therapy and Priority Medicine ("PRIME") designation in the United States and Europe, respectively.

The company expects to submit final data from the study to the FDA by the end of the year. Following the submission, uniQure intends to initiate a phase III study to evaluate AMT-061 in the first quarter of 2019.

Please note that uniQure has a collaboration with Bristol-Myers (NYSE:BMY) for developing its gene therapies. AMT-061 is also being developed as part of this agreement.

We remind investors that there are several pharma companies developing gene therapies for the treatment of hemophilia B. Pfizer’s (NYSE:PFE) fidanacogene elaparvovec is currently being evaluated in a phase III study as a one-time treatment option. Pfizer gained the development rights for the candidate from Spark Therapeutics (NASDAQ:ONCE) , which also has a long gene therapy pipeline.

Zacks Rank

uniQure currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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