uniQure to Present Clinical Data from Study of AMT-060 in Hemophilia B at Upcoming ASH Meeting

uniQure to Present Clinical Data from Study of AMT-060 in Hemophilia B at Upcoming ASH Meeting

uniQure, a company specializing in human gene therapy, will present clinical data from a Phase 1/2 dose-escalating study of AMT-060 in adults with hemophilia B at the upcoming 58th American Society of Hematology Annual Meeting (ASH 2016), to be held in San Diego, Calif., in Dec.3-6. The study is currently recruiting participants.

Frank W. G. Leebeek, MD, PhD, professor at the Erasmus Medical Center in Rotterdam, the Netherlands, will be presenting the data on AMT-060.

The presentation is titled “Interim results from a dose escalating study of AMT-060 (AAV5-hFIX) gene transfer in adult patients with severe hemophilia B,” and will be available during the poster session #801 “Gene Therapy and Transfer: Poster I,” taking place Dec. 3 between 5:30 and 8:30 p.m. at Hall GH, San Diego Convention Center.

“We will present updated clinical data at ASH on long-term durability of FIX activity now out to one year,” Christian Meyer, MD, PhD, uniQure’s chief medical officer, said in a press release.

AMT-060 is an AAV5-FIX gene therapy being developed for hemophilia B, an orphan blood-clotting disorder.

The Phase 1/2 open-label, multi-center clinical trial (NCT02396342) comprises 10 patients, each receiving a one-time, 30-minute, intravenous (IV) administration of AMT-060 without immunosuppressant therapy. Patients are split into two cohorts, one receiving a lower dose of AMT-060 (5×1012 gc/kg) and one receiving a higher dose of AMT-060 (2×1013 gc/kg).

All participants had documented severe or moderate-to-severe hemophilia. All patients will be followed for a period of five years after the end of the trial, to further assess long-term safety and durability.

The updated data presented will include the results of up to 52 weeks of follow-up on the first patient group receiving the lower dose, and of up 26 weeks of follow-up on the second patient group receiving the higher dose.

“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,” Meyer said.

uniQure management also will host an investor and analyst breakfast and webcast event, featuring Leebeek, to review the data and outline progress. The meeting can be accessed by visiting the investor relations section of the uniQure website.

The investor’s event will take place on Dec. 5 at 7 a.m. To request attendance at the meeting. A replay will be available online for up to 30 days after the meeting.

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