uniQure Announces Oral Presentation Of Clinical Data From Ongoing Phase I/II Study Of AMT-060 In Hemophilia B At The 21st Congress Of The European Hematology Association

Presentation to Include Most Recent Clinical Data from the Five Patient, Low-dose Cohort

Company to Host Conference Call on Monday, June 13, 2016 at 2:00 pm CET/ 8:00 am EDT

Amsterdam, the Netherlands, May 19, 2016 - uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced that additional data from its Phase I/II trial of AMT-060 in hemophilia B will be presented on June 11, 2016 at the 21st Congress of the European Hematology Association ("EHA") in Copenhagen, Denmark. The oral presentation by the clinical investigator will include new and updated data on five patients enrolled in the study's low-dose cohort.

European Hematolgy Association Data Presentation Details

Abstract #S467: First Results from a Dose-Escalating Study with AAV5 Vector Containing Wild Type Human Factor IX Gene Therapy in Patients with Severe or Moederately-Severe Hemophilia B

Session: Bleeding Disorders

Lead Author: Dr. Frank Leebeek, Professor at the Erasmus University Medical Center in Rotterdam

Date & Time: Saturday, June 11, 2016, 11:30 am CET

Location: Room H4 at the Bella Center in Copenhagen, Denmark

uniQure Conference Call and Webcast

uniQure will host a webcast conference call at 2:00 pm CET/ 8:00 am EDT on Monday, June 13, 2016, to discuss the data from the study. The conference call can be accessed by dialling one of the numbers listed below five minutes prior to the start of the call and providing the confirmation code: 5314178.

Local - New York, United States of America: +1 646 254 3367

Local - Amsterdam, Netherlands: +31 (0) 20 716 8256

Local - Toronto, Canada: +1 416 216 4141

Local - Brussels, Belgium: +32 (0) 2 620 0138

Local - London, United Kingdom: +44 (0) 20 3427 1912

Local - Paris, France: +33 (0) 1 76 77 22 29

Local - Frankfurt, Germany: +49 (0) 69 2222 10624

Local - Milan, Italy: +39 02 3600 9869

Investors may also listen to the webcast of the conference call live through a link provided on both the "Investor" and the "Events" sections of uniQure's website, www.uniQure.com. To ensure a timely connection to the webcast, it is recommended that users register at least 15 minutes prior to the scheduled start time. The webcast replay will be available for at least 72 hours following the call.

About Hemophilia B

Hemophilia B is a serious and rare inherited disease in males characterized by insufficient blood clotting. The condition can lead to repeated and sometimes life-threatening episodes of external and internal bleeding following accidental trauma or medical interventions. The episodes can cause long-term damage, for example to the joints, and can be fatal if they occur in the brain. The deficient blood clotting results from the lack of functional human Factor IX, or hFIX. Treatment of hemophilia B today consists of prophylactic or on-demand protein replacement therapy, in which frequent intravenous administrations of plasma-derived or recombinant hFIX are required to stop or prevent bleeding. Hemophilia B occurs in approximately 1 out of 30,000 live births.

About uniQure

uniQure is delivering on the promise of gene therapy - single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with CNS, liver/metabolic and cardiovascular diseases. www.uniQure.com

Back to news