Actionable news
All posts from Actionable news
Actionable news in QURE: UNIQURE N.V.,

uniQure Announces Updated, Long-Term Clinical Data from Ongoing Phase I/II Trial of AMT-060 In Patients with Severe Hemophilia B

Clinical Benefit Maintained in All Patients, with FIX Activity Persisting At Up To 18 Months of Follow-Up

Second-dose Cohort Demonstrates Dose Response Up to One Year, with 84% Reduction in
Spontaneous Bleeds and All Patients Free of Prophylactic FIX Replacement Therapy

No Activation of T-Cell Responses or Loss of FIX Activity in Any Patient Up To 18 Months

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, July 10, 2017 (GLOBE NEWSWIRE) -- uniQure N.V. (QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, will today announce updated results from its ongoing, dose-ranging Phase I/II trial of AMT-060, its proprietary, investigational gene therapy in patients with severe hemophilia B. The data includes up to 18 months of follow-up from the low-dose cohort and up to one year of follow-up from the second dose cohort.

The AAV5-based AMT-060 remains safe and well-tolerated with up to a year and a half of follow-up, with no serious adverse events and no development of inhibitors. All patients are now past one year of follow up with no loss of Factor IX (FIX) activity and no capsid-specific T-cell activation.

One-year follow-up data from the second-dose cohort continue to show a dose response with substantial improvement in disease state in all five patients, including the discontinuation of routine prophylactic FIX infusions in all patients that previously required chronic replacement therapy. The annualized spontaneous bleeding rate for the second dose cohort declined 84% to a mean of 0.5 annual bleeds after gene transfer. During more than 1,700 cumulative patient days of observation, only one patient in the second cohort reported two unconfirmed spontaneous bleeds, and no such bleeds were reported by any patient during the last six months of observation.

These clinical data will be presented today in an oral presentation at the 26th Biennial Congress of the International Society on Thrombosis and Haemostasis (ISTH), taking place this week in Berlin, Germany.

“We continue to observe a therapeutic benefit from AMT-060 that is clearly superior to their previous prophylactic FIX replacement therapy regimen, even in patients with advanced joint disease who experienced a high rate of bleeds prior to gene transfer,” stated Professor Wolfgang Miesbach, M.D., of the University Hospital Frankfurt, Germany.

“Importantly, AMT-060 appears to be safe and well-tolerated, and quite differentiated, with no loss of FIX activity, no activation of T-cell response and no development of inhibitors for any of the ten patients in the study. The safety profile observed in this study suggests that the AAV5 vector offers long-term safety, efficacy and the potential for broad application in hemophilia B patients,” he added.

Phase 1/2 Trial Overview

The AMT-060 gene therapy consists of a codon-optimized wild type FIX gene cassette, the LP1 liver promoter and an AAV5 viral vector manufactured by uniQure using its proprietary insect cell-based technology platform. It is the only hemophilia gene therapy that combines a gene cassette with clinically proven...