11/3/2016 9:07:33 AM
-- Poster presentation on Saturday, December 3, 2016
-- Investor breakfast event on Monday, December 5 at 7:00 am PST/10:00 am EST -
Lexington, MA and Amsterdam, the Netherlands, November 3, 2016 - uniQure N.V. (NASDAQ: QURE), a leader in human gene therapy, today announced that clinical data from a Phase I/II dose-escalating study of AMT-060, its investigational gene therapy in adult patients with severe hemophilia B, will be presented at the 58th American Society of Hematology (ASH) Annual Meeting, held in San Diego, CA from December 3 to 6, 2016.
Updated data on AMT-060 will be presented by study investigator Professor Frank W.G. Leebeek, M.D. Ph.D. of the Erasmus Medical Center in Rotterdam, the Netherlands. AMT-060 is an AAV5-FIX gene therapy developed by uniQure using its proprietary insect-cell based technology platform. The updated data will include up to 52 weeks of follow-up on the first patient cohort receiving 5x1012 gc/kg and up to 26 weeks of follow-up on the second patient cohort receiving a higher dose of 2x1013 gc/kg.
The conference abstract is available today and can be accessed at http://www.hematology.org/Annual-Meeting. The abstract includes previously reported data of up to nine months from the first patient cohort in the study.
"We will present updated clinical data at ASH on long-term durability of FIX activity now out to one year," stated Christian Meyer, M.D., Ph.D., chief medical officer of uniQure. "We also will present the additional shift in disease phenotype through data from the second, higher-dose cohort, and an update on previously reported results that demonstrate that four out of five patients no longer require chronic infusions of prophylaxis. We look forward to Professor Leebeek's presentation and to hosting an investor event at the meeting."
Title: Interim results from a dose escalating study of AMT-060 (AAV5-hFIX) gene transfer in adult patients with severe hemophilia B
Presenter: Professor Frank W.G. Leebeek, M.D. Ph.D.
Poster Session: 801. Gene Therapy and Transfer: Poster I
Date and Time: Saturday, December 3, 2016, 5:30 pm PST/8:30 pm EST
Location: Hall GH, San Diego Convention Center
Investor and Analyst Breakfast and Webcast
uniQure management will host an investor breakfast meeting featuring Professor Leebeek to review the updated data on AMT-060 and outline progress taking place with its ongoing clinical development. The meeting will be webcast live along with slides and can be accessed by visiting the investor relations section of the Company's website at www.uniQure.com
Date and Time: Monday, December 5, 2016 at 7:00 am PST/ 10:00 am EST
Location: Marriott Gaslamp Hotel, Room Presidio A, 660 K Street, San Diego, CA 92101
To request attendance at the meeting, please RSVP to Investors@uniQure.com by November 30, 2016, as space is limited.
A replay of the investor event will be available on the uniQure website, approximately three hours after the presentation and will be archived on the site for at least 30 days.
AMT-060 Phase I/II Trial Overview
The Phase I/II, open-label, multi-center study includes 10 patients each receiving a one-time, 30-minute, intravenous administration of AMT-060, without the use of immunosuppression therapy. The study includes two dose cohorts of five patients each, with the first cohort receiving 5x1012 gc/kg and the second cohort receiving 2x1013 gc/kg. All patients in the trial had documented severe or moderately-severe hemophilia, including documented FIX levels less than 1-2%, and required chronic infusions of prophylactic or on-demand Factor IX therapy at the time of enrolment. uniQure will follow all 10 patients in the study for a period of five years to further assess long-term safety and durability.
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.
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
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