uniQure Announces New Gene Therapy Trial Data

uniQure recently announced updated data related to a phase IIb clinical study of AMT-061, the company’s investigational gene therapy candidate for patients with severe and moderately severe hemophilia B. The update came in the form of an oral presentation at the XXVII Congress of the International Society on Thrombosis and Haemostasis (ISTH), July 6-10, 2019 in Melbourne, Australia.

AMT-061 consists of adeno-associated virus serotype 5 (AAV5), which has been demonstrated to be safe and well-tolerated, acting as a delivery vector carrying a gene cassette with the Padua variant of factor IX (FIX). AAV5 is a variant of the type of the adeno-associated virus (AAVs) vectors that are being investigated in multiple gene therapy trials. The update at ISTH, included data for three severe hemophilia B patients who have received a one-time intravenous infusion of AMT-061.

According to a uniQure press release, data at 36 weeks-post administration show that all three patients have sustained overall increases in FIX levels. Mean FIX activity for the three patients was 45% of normal. The first patient achieved FIX activity of 54% of normal, the second patient achieved FIX activity of 30% of normal and the third patient achieved FIX activity of 51% of normal.

In addition, there have been no reports of bleeding episodes that necessitated infusions of FIX replacement therapy. One patient underwent hip surgery due to a pre-existing condition for which he was treated perioperatively with short-acting factor replacement. This was reported by the investigator as a serious adverse event unrelated to AMT-061.

“These updated data continue to show that a single administration of AMT-061 is well-tolerated and has the potential to increase FIX activity into the normal range for people living with hemophilia B,” stated Matt Kapusta, chief executive officer of uniQure. “We continue to be very pleased with these results, which as of this data report show durable increases in FIX activity with no bleeds and no requirement for infusions of FIX replacement therapy or immunosuppression.”

Source: uniQure press release data July 6, 2019

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