How AMT-061 works
Hemophilia B is a genetic condition caused by a mutation in the F9 gene, which carries the instructions to make clotting factor IX, a protein required for normal blood clotting. Patients with hemophilia B either have reduced levels of factor IX or an abnormal version of the protein, both of which cause problems in the clotting process, resulting in a bleeding disorder.
Gene therapy is an emerging therapeutic strategy to treat genetic conditions like hemophilia B. The goal is to replace a faulty gene with a healthy copy in the patient’s body.
AMT-061 is designed to provide a highly functional copy of the F9 gene called FIX Padua to help the body make more of the factor IX protein. FIX Padua is different from the normal healthy copy of the F9 gene; it is a variant with a change that renders the produced factor IX protein eight times more active than normal. AMT-061 uses a harmless virus called adeno-associated virus 5 (AAV5) to deliver the gene to the body. The treatment is administered intravenously (into the vein) into the bloodstream.
AMT-061 in clinical trials
A Phase 2b dose-confirmation trial (NCT03489291) is underway to assess the safety and efficacy of AMT-061 in three patients with severe or moderately severe hemophilia B. Each patient receives a single dose of AMT-061, and is kept under observation for 24 hours for any adverse events. The patients will be monitored for a total of five years. Six-month interim data from the trial were presented at the 2019 Scientific Symposium of the Hemostasis & Thrombosis Research Society. In all three patients, there was a significant increase in factor IX levels, and no serious adverse effects of thrombotic events were reported. Factor IX activity was markedly higher than normal in all three patients lowering their bleeding risk, the team noted. The study will continue monitoring the patients throughout the rest of the study period.
A Phase 3 study (NCT03569891) called HOPE-B is recruiting participants with severe to moderate hemophilia B to evaluate the safety, efficacy, and tolerability of AMT-061. The trial aims to recruit 56 male patients, ages 18 and older in the U.S., U.K., and Europe. The participants will receive a single intravenous injection of AMT-061 and will be monitored for any signs of adverse events. Factor IX activity levels will be assessed as the primary outcome while annualized bleeding rate (number of bleeding episodes per year) will be monitored as the secondary outcome. The treatment has already been administered to the first patient in the study.
AMT-061 has been granted breakthrough designation by the U.S. Food and Drug Administration and PRIME eligibility by the European Medicines Agency. Both these designations support the rapid development of AMT-061.
The technology used to create AMT-061 is patented by uniQure in both the U.S. and Canada.
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