How does AMT-061 work?
Hemophilia B is a genetic condition resulting from 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.
Researchers designed AMT-061 to provide a highly functional copy of the F9 gene, called FIX Padua, to help the body make more 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 is an intravenous (into a vein) treatment that uses a harmless virus called adeno-associated virus 5 (AAV5) to deliver the gene to the body.
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. Researchers presented six-month interim data from the trial at the 2019 Scientific Symposium of the Hemostasis & Thrombosis Research Society. There was a significant increase in factor IX levels in all three patients, and none reported any serious adverse effects of thrombotic events. Factor IX activity was markedly higher than normal in all three patients lowering their bleeding risk. Researchers will continue monitoring the patients throughout the rest of the study period.
A Phase 3 study (NCT03569891) called HOPE-B is also evaluating the safety, efficacy, and tolerability of AMT-061 in people with severe to moderate hemophilia B. The trial recruited 54 male patients, ages 18 and older in the U.S., U.K., and Europe. Participants received a single intravenous injection of AMT-061 and researchers will monitor them for any signs of adverse events. They will assess factor IX activity levels as the primary outcome and will monitor the annualized bleeding rate (number of bleeding episodes per year) as the secondary outcome.
The U.S. Food and Drug Administration (FDA) granted AMT-061 breakthrough designation and the European Medicines Agency (EMA) granted it PRIME eligibility. Both these designations support the rapid development of AMT-061.
uniQure holds patents on the technology used to create AMT-061 in the U.S. and in Canada.
Last updated: June 26, 2020
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