UniQure (QURE) Shares Rally on Positive Results in Gene Therapy Trials
Shares of uniQure N.V. (QURE) were trading up +2.50 or +16.77 percent to $17.41 per share in Thursday’s premarket after the company announced positive preliminary results in a Phase I/ II clinical trial that assessed the company’s gene therapy in patients with hemophilia B. uniQure stock closed at $14.91, down -1.26 or -7.79 percent in Wednesday’s regular trading session.
Early this morning, uniQure announced it had obtained preliminary topline results from the low dose cohort of an ongoing Phase I/II clinical trial being conducted on adult patients with hemophilia B. The patients in the trial were being treated with uniQure’s AAV5/FIX gene therapy, AMT-060.
The five subjects involved in the trials had documented Factor IX (FIX) levels of less than 1-2 percent, requiring chronic prophylactic treatment with recombinant FIX (rFIX) when enrolling in the program. A previous study performed at St. Jude Children’s Hospital demonstrated that a durable FIX expression of -5 percent of normal produced long term clinical benefits.
Two of the participants that completed at least 12 weeks of follow up had FIX expression levels of 5.5 percent and 4.5 percent respectively, while three subjects received dosages but did not achieve the full twelve week follow up. Nevertheless, two of the three that did not achieve the twelve week follow up met a secondary endpoint of discontinuing prophylactic rFIX based on FIX expression levels.
Hemophilia B, the disease being treated by uniQure’s AMT-060 is a rare inherited disease which has insufficient blood clotting as its main symptom. The disease can lead to episodes of internal bleeding following trauma through accidents or medical procedures. Episodes can cause long term damage to joints or be fatal if they occur in the brain.
Blood clotting deficiency in hemophilia B is the result of the lack of functional human Factor IX or hFIX. Hemophilia B occurs in approximately one in every 30,000 births. The condition is currently being treated by prophylactic or on demand protein replacement therapy with frequent intravenous administrations of plasma-derived or recombinant hFIX needed to arrest or prevent bleeding.
Dan Soland, uniQure’s newly appointed Chief Executive Officer said in the company’s press release that, “These preliminary topline results support our hypothesis that AAV5/FIX can deliver clinically meaningful expression levels of FIX for patients with hemophilia B. So far, our AAV5-based gene therapies have been systemically administered to 13 adult patients across two clinical studies in two different disease states, and via direct central nervous system administration in four children in a third study, providing us with a strong safety dataset on the AAV5 vector and our proprietary insect cell based manufacturing technology.”
Soland concluded saying, “Today, we are the only AAV gene therapy company in the world with both proprietary, commercial-scale manufacturing capabilities and encouraging clinical data across multiple diseases. These preliminary results further support our modular platform approach and leadership in gene therapy.”
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