http://www.fiercebiotech.com/r-d/uniqure-details-three-pronged-gene-therapy-trial-program The closely watched [UniQure] hemophilia B remains the centerpiece of the company. While investors were disappointed by the Factor IX activity presented in a succession of readouts—especially when compared to data from Spark Therapeutics’ rival gene therapy—uniQure maintained AMT-060 has a compelling efficacy and safety profile.
Kapusta claimed patients, physicians and the FDA share uniQure’s focus on reductions in FIX transfusions and rates of spontaneous bleeding rather than FIX activity.
“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.