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Updated trial data show uniQure's gene therapy AMT-060 induces sustained Factor IX activity in patients with hemophilia B
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“The majority of patients in this low-dose cohort of AMT-060 are showing FIX activity in the range of 5% of normal, and clinical experience has shown that patients in this range generally do not require prophylactic factor replacement and have a very low frequency of spontaneous joint bleeding episodes,” Professor Miesbach added. “Furthermore, at up to 9 months of follow up, AMT-060 continues to be well-tolerated and show durable, clinically relevant levels of FIX activity, which is the key goal of an effective gene therapy approach.”
- Through up to 9 months of follow-up, the mean steady-state FIX activity for the four patients that discontinued prophylactic (precautionary) FIX therapy was 5.4% of normal, with a range from 3.1% to 6.7% of normal. This level of FIX activity is consistent with the highest achieved FIX activity observed in a previous study conducted by St. Jude Children’s Research Hospital (St. Jude), which utilized the same wild-type FIX gene cassette incorporated in AMT-060. The results of the St. Jude study were published in the New England Journal of Medicine in 2011 and 2014 and demonstrated therapeutically relevant and durable clinical benefits for up to four years as of the time of its last publication. The FIX gene cassette used in the St. Jude study is exclusively licensed by uniQure.
- Total usage of FIX concentrate declined substantially among the four patients that discontinued prophylactic FIX therapy. These patients demonstrated a mean reduction in annualized total FIX usage of 82% after treatment with AMT-060. For all five patients in the low-dose cohort, the mean annualized total FIX usage declined 75% after treatment with AMT-060. The one patient who remained on prophylactic therapy has sustained an improved disease phenotype and also required materially less FIX concentrate after treatment with AMT-060.
- AMT-060 continues to be well-tolerated. As noted previously, one patient in the low-dose cohort experienced a mild, transient and asymptomatic elevation of alanine aminotransferase (ALT) 10 weeks after treatment. No cellular immune response to AAV5 was evident. Per the study protocol, the patient received a short course of prednisolone and rapidly returned to baseline ALT levels with no evidence of loss of FIX expression. No other patients have experienced elevated ALT levels and none of the five patients developed inhibitory antibodies against FIX.
- AMT-060 continues to demonstrate a very low screening failure rate, with all patients screened in the study testing negative for pre-existing anti-AAV5 NABs. To date, 25 patients have been screened for pre-existing anti-AAV5 NABs with a fully validated assay across several clinical studies with only one patient excluded due to a borderline positive result. This collective data set suggests that a large proportion of the hemophilia patient population may be eligible for treatment with AMT-060.
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Jul 27, 2016
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Related Keywords Hemophilia B, Gene Therapy, Amt-060, Factor Ix, Prophylactic Fix Infusions, Advanced Joint Disease, Frequent Bleeding Episodes