(Reuters) – Drug developer UniQure NV said on Friday its gene therapy to treat hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a small study.
The company’s shares rose 5.5 percent in light premarket trading.
The updated data from a mid-stage study showed that all three patients treated with a single dose of the therapy, AMT-061, showed increased and sustained levels of factor IX (FIX), the company said.
Hemophilia B is a genetic disorder where the lack of enough FIX prevents blood from clotting, resulting in severe bleeding.
FIX activity at 12 weeks increased to 38 percent of normal, which exceeded the threshold levels generally considered enough to eliminate or cut down the risk of bleeding events, UniQure said.
“Increases in FIX activity levels across patients would be a ‘nice-to-have’, considering that QURE has already achieved activity levels expected to prevent bleeding,” Evercore ISI analyst Josh Schimmer said in a client note ahead of the data release.
UniQure in November reported initial data from the same trial, showing FIX activity of 31 percent of normal after six weeks of administration.
The positive data follows a bleak day for companies developing gene therapies and gene editing technology, after drugs developed by two small firms failed to show promise in separate clinical trials.
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