Uniqure NV (QURE) Announces Positive Top-Line Data For Hemophilia B Phase I/II Trials


After yesterday’s selloff, shares of Uniqure NV (NASDAQ:QURE) are soaring in pre-market trading early this morning. The stock is currently trading higher by $1.69 or 11.33% after the company announced preliminary top-line data related to their Hemophilia B Phase I/II trials for adults who were treated with the company’s AAV5/FIX gene therapy, AMT-060. According to Uniqure NV (NASDAQ:QURE) results are very promising.

“These preliminary topline results support our hypothesis that AAV5/FIX can deliver clinically meaningful expression levels of FIX for patients with hemophilia B,” commented Dan Soland, Chief Executive Officer of uniQure. “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.”

While this is great news for shareholders, it may be even better news for the 1 in 30,000 people born with Hemophilia B who suffer from a lack of blood clotting. The stock decreased 7.79% or $1.26 during the last trading session, hitting $14.91. Approximately 64,174 shares traded hands. QURE shares have declined 49.87% since June 3, 2015 and are currently downtrending. It has underperformed the S&P500 by 45.17%.

From a total of 8 analysts covering uniQure N.V. (NASDAQ:QURE) stock, 8 rate it a “Buy”, 0 a “Sell”, and 0 a “Hold”. This means that 100% of the ratings are positive. The highest target price is $60 while the lowest target price is $33. The mean of all analyst targets is $44 which is 195.10% above today’s ($14.91) stock price. uniQure N.V. was the topic of 15 analyst reports since August 7, 2015 according to the firm StockzIntelligence Inc. Janney Capital initiated shares on December 1 with a “Buy” rating. Piper Jaffray maintained shares with a”Overweight” rating and a $40 target share price in their report from an August 28. Roth Capital reinitiated QURE stock in a recent report from November 30 with a “Buy” rating. Finally, Chardan Capital Markets maintained the stock with a “Buy” rating in a report they issued on an October 1.

Uniqure NV is a pharmaceutical company based in the Netherlands. The company has a market cap of $343.87 million. It is primarily engaged in the field of gene therapy for orphan diseases. It currently has negative earnings. The Firm is focused on patients suffering from genetic or acquired diseases by directing the expression of a therapeutic protein or restoring the expression of a missing protein through a single administration.