Data supports nonclinical comparability plan agreed upon with FDA
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Dec. 09, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe unmet medical needs, today announced new nonclinical data on AMT-061, its investigational gene therapy for patients with hemophilia B. AMT-061 combines an AAV5 vector with the FIX-Padua variant. The data will be presented today in a poster session at the 59th American Society of Hematology (ASH) Annual Meeting taking place in Atlanta, GA.
The data presented from the study in non-human primates demonstrated dose-dependent expression of human Factor IX protein (hFIX) and FIX clotting activity. Animals receiving the same dose of AMT-060 and AMT-061 showed comparable levels of hFIX protein, but the animals receiving AMT-061 demonstrated approximately 6.5-fold higher FIX activity compared to those receiving AMT-060.
"These data establish that AMT-061 is well-tolerated and provides a substantial increase in FIX activity compared to AMT-060, consistent with the gain-of-function reported in previous preclinical studies of FIX-Padua," stated Sander van Deventer, M.D., Ph.D., chief scientific officer of uniQure. "At a dose of 2x1013 gc/kg, the same dose used in the second patient cohort of our ongoing Phase I/II clinical trial of AMT-060, AMT-061 has the potential to achieve mean FIX activity in humans of approximately 30 to 50 percent of normal."
Key Data Findings Presented at ASH
The goal of this non-human primate study was to evaluate the efficacy and safety of introducing the FIX-Padua variant into the AMT-060 cassette. AMT-061 leverages the same AAV5 capsid and insect cell-based manufacturing platform as AMT-060. The study also compared AMT-061 to AMT-060 with respect to liver transduction, circulating FIX protein levels, circulating FIX activity levels and toxicity.
"The data confirms that the biological activity of AMT-060 and AMT-061 are highly comparable, but the FIX clotting activity of AMT-061 is substantially higher than AMT-060," added van Deventer. "We believe the enhanced potency of AMT-061 has the potential to provide substantial benefit to patients, and we look forward to advancing this promising gene therapy candidate into a pivotal study in 2018."
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 hemophilia, Huntington's disease and cardiovascular diseases. www.uniQure.com
uniQure Forward-Looking Statements
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Maria E. Cantor Eva M. Mulder Tom Malone
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