uniQure N.V.

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Uniqure Announces Positive Top-Line Data From The Hope-B Pivotal Trial Of Etranacogene Dezaparvovec Gene Therapy In Patients With Hemophilia B

Published: 11/19/2020 14:18 GMT
Uniqure NV (QURE) - Uniqure Announces Positive Top-line Data From the Hope-b Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients With Hemophilia B.
Uniqure NV - Phase Iii Study in 54 Patients Met Primary Endpoint With Mean Factor Ix Activity of 37% of Normal at 26 Weeks.
Uniqure NV - Increases in Factor Ix Activity Were Sustained for Up to 18 Months With Near Elimination of Bleeding in Study.
Uniqure NV - Patients Achieved Significant Increases in Factor Ix Activity Irrespective of Pre-existing Neutralizing Antibodies in Hope-b Trial.
Uniqure NV - Etranacogene Dezaparvovec Was Well-tolerated With No Treatment-related Serious Adverse Events.
Uniqure NV - Plan to Incorporate Fix Activity and Bleeding Rates at 52 Weeks As Additional Co-primary Endpoints in Study.
Uniqure NV - in Study Mean Annualized USAge of Fix Replacement Therapy Declined by 96% After Dosing Versus Observational Lead-in Period.
Uniqure NV - Look Forward to Holding Pre-bla Meeting With FDA and Completing Last Patient's 52-week Follow-up Visit in Q1 of 2021.