UniQure N.V. shares (NASDAQ:QURE) were on the uptick in premarket trade after an early-stage trial of a treatment for severe hemophelia B recorded positive results.
In a statement, the company said the 18-month follow-up data from a Phase I/II trial of AMT-060 in the second-dose cohort found "substantial improvement" in disease state in all five patients, and that all those who previous required routine prophylactic FIX infusions were able to stop them.
Pivotal study in 2018
UniQure plans to start a pivotal study in 2018 with AMT-061, which combines an AAV5 vector with the FIX-Padua mutant.
AMT-061 and AMT-060 are identical in structure apart from two nucleotide substitutions in the coding sequence for FIX.
Hemophelia is an inherited disease that affects males. It stops their blood from clotting after a bleed.
The condition can cause life-threatening episodes of external and internal bleeding and requires regular injections of plasma-derived human Factor IX.
The data were presented to the 58th American Society of Hematology (ASH) annual meeting in Atlanta, Georgia on Monday morning.
"We continue to observe a therapeutic benefit from AMT-060 that is clearly superior to patients' previous prophylactic FIX replacement therapy regimen, with stable elevations in Factor IX levels and a cessation of spontaneous bleeds," said Professor Frank W.G. Leebeek, of the Erasmus University Medical Center in Rotterdam.
In premarket, uniQure shares were up 3.25% at US$18.40.