uniQure plots to initiate a three-pronged clinical trial program next year as key assets from its in-house pipeline and Bristol-Myers Squibb collaboration advance. uniQure aims to move its hemophilia B gene therapy, AMT-060, into a pivotal trial while working to advance a Huntington's disease asset and Bristol-Myers-partnered heart failure candidate into the clinic. <\/p>\n
Matthew Kapusta, who took over as CEO of uniQure in September, detailed the strategy in a talk(registration required)at Leerinks healthcare conference. The plan will follow the strategy uniQure set out in November when it laid off workers and stepped down its interest in Sanfilippo B and Parkinsons disease to prioritize investment in the aforementioned three programs. <\/p>\n
The closely watched hemophilia B remains the centerpiece of the company. While investors weredisappointed by the Factor IX activity presented in a succession of readoutsespecially when compared to data from Spark Therapeutics rival gene therapyuniQure maintainedAMT-060 has a compelling efficacy and safety profile. <\/p>\n
Kapusta claimed patients, physicians and the FDA share uniQures focus on reductions in FIX transfusions and rates of spontaneous bleeding rather than FIX activity. <\/p>\n
In the interaction that we had with the FDA, it was very clear that they were looking at defined clinical benefits. And I think they were defining clinical benefit not in terms of Factor IX activity but in terms of impact on annualized bleeding rate, he said. <\/p>\n
uniQure will have another chance to hear the FDAs views at its end of phase 2 meeting set to take place before the end of the quarter. The meeting will mark the start of a process intended to lead uniQure into a pivotal trial next year. <\/p>\n
The start of phase 3 should contribute to a busy year in the clinic for uniQure. Following a year ofcollaboration with Bristol-Myers working on manufacturing matters, uniQure is now in the middle of animal studies that could set it up to file an INDand possibly start a trialnext year. uniQure isrunning a dose expression study of the heart failure gene therapy in healthy and diseased pigs. <\/p>\n
In parallel, uniQure is working to get its Huntington's disease asset into the clinic. The candidate,the result of a five-year project, uses the AAV5 vector to deliver a microRNA intended to silence the Huntington gene. In mice, administration of the gene therapy into the striatum led to a 50% knockdown in the cortex. <\/p>\n
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uniQure details three-pronged gene therapy trial program - FierceBiotech<\/a><\/p>\n","protected":false},"excerpt":{"rendered":" uniQure plots to initiate a three-pronged clinical trial program next year as key assets from its in-house pipeline and Bristol-Myers Squibb collaboration advance. uniQure aims to move its hemophilia B gene therapy, AMT-060, into a pivotal trial while working to advance a Huntington's disease asset and Bristol-Myers-partnered heart failure candidate into the clinic. Continue reading