AMT-060 is a new potential therapy developed by the biotechnology company UniQure for the treatment of hemophilia B, a serious and rare inherited disease characterized by insufficient blood clotting.

How AMT-060 Works

People with hemophilia B, predominantly males, have a mutation in the gene encoding for the blood clotting factor IX, which leads to no factor IX being synthesized by the body. AMT-060 works by restoring the production of factor IX in hemophilia patients by delivering a functional copy of the factor IX gene into the liver cells of patients diagnosed with hemophilia B. The functional copy of the gene is delivered via a harmless virus called adeno-associated viruses (AAV). These viruses trigger only a mild immune response and are used as vectors, or vehicles, to deliver genes to the body.

AMT-060 and clinical trials

Early results from an ongoing two-cohort Phase 1/2, non-randomized, open-label, multi-center study (NCT02396342), which included 10 patients with severe or moderately severe hemophilia B, demonstrated a clinically significant and sustained increase in factor IX activity, a substantial reduction in factor IX replacement usage, and a complete cessation of spontaneous bleeding episodes. Of the 10 patients, five received a lower dose of AMT-060; the other five received a higher dose of AMT-060 intravenously for 30 minutes.

The results were reported after 52 weeks of follow-up from the low-dose cohort and up to 31 weeks from the high-dose cohort. All five patients in the low-dose cohort continued to maintain constant and clinically meaningful levels of factor IX activity for up to 52 weeks post-treatment, resulting in a complete cessation of spontaneous bleedings during the last 14 weeks of observation. The bleeding in these patients was previously uncontrollable despite being managed with prophylactic therapy.

In addition, at both doses that were evaluated, AMT-060 appeared to be safe and well-tolerated with no loss of factor IX activity, no activation of T-cell response (a type of immune response), and no development of inhibition for any of the 10 patients.

UniQure has announced that it aims to continue the follow-up with all 10 patients in the study for a period of five years to further assess the long-term safety and durability of AMT-060 in treating hemophilia B.

Based on the results of the study, AMT-060 has been granted a breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) and also has received European Medicines Agency (EMA) Priority Medicines (PRIME) designation for hemophilia B.

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