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UniQure’s Gene Therapy Performing well in Hemophilia B Trial

June 14, 2016
UniQure’s Gene Therapy Performing well in Hemophilia B Trial

UniQure recently announced data from a phase I/II clinical trial of AMT-060, one of its proprietary gene therapy technologies, to treat patients with hemophilia B (factor IX deficiency). UniQure is an Amsterdam-based company specializing in developing gene therapies to treat rare conditions, such as central nervous system disorders, and liver/metabolic and cardiovascular diseases.

UniQure’s hemophilia B technology employs adeno-associated viruses (AAV). These small viruses, which do not cause disease and produce mild immune responses, are used as vectors (delivery vehicles) to introduce a functioning factor IX (FIX) gene into the liver cells of patient’s with hemophilia B. The goal of the trial is to trigger long-term FIX protein production through a single administration of the therapy. This could dramatically reduce the frequency of bleeding episodes in people with hemophilia B.

The data were reported via a press release and an oral presentation given by lead investigator Frank Leebeek, MD, PhD, Professor of Hematology at Erasmus University Medical Center in Rotterdam, at the 21st Congress of the European Hematology Association (EHA) in Copenhagen, Denmark, June 9-12, 2016. The trial of 10 patients divided them into two groups: five received a low-dose administration of AMT-060 and five received a considerably higher dose. The data presented at the EHA Congress were from the low-dose group.

Before the trial, the five patients in the low-dose group all had FIX activity levels consistent with moderate-to-severe hemophilia B. Their median recorded activity was less than 1.5%, requiring prophylactic infusions of FIX concentrate to control their bleeding. Six months after receiving a single low-dose administration of AMT-060, all five patients showed an increase in FIX activity, with a median level of 5.4%. This seemingly modest increase is significant. It represents a change in severity from primarily severe to mild/moderate, indicating both a notable decrease in spontaneous bleeding and improvement in quality of life.

According to uniQure, AMT-060 has so far been well tolerated by patients in the first group. One patient had a temporary increase in the level of the liver enzyme alanine aminotransferase 10 weeks after receiving the therapy. This was quickly corrected with a short regimen of the steroid prednisone. In addition, none of the patients developed inhibitor antibodies.

“After six months of follow-up, I can say as a clinician who regularly treats hemophilia patients that the impact on the quality of life for these patients treated with AMT-060 is very positive,” said Leebeek. “The increases in FIX activity and the overall stability of the activity observed over a 6-month period are cause for optimism, as they are associated with meaningful clinical benefits as well as reduced need for ongoing infusions of recombinant FIX therapy.”

uniQure has also announced that all five patients in the second, high-dose group have received a one-time administration of AMT-060. That dose is four times higher than the one administered to the low-dose group. According to the company’s news release, these patients are currently in the early stages of follow-up. Initial data are expected to be presented before the end of 2016.

Source: UniQure press release dated June 11, 2016