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uniQure Confirms First Patient Dosed in Hemophilia B Gene Therapy Study with AMT-061

August 23, 2018

uniQure recently announced that it has treated the first patient in its Phase IIb dose-confirmation of AMT-061, the company’s investigational gene therapy treatment for patients with severe and moderately severe hemophilia B. 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. This variant of Factor IX has been reported to provide an approximate 8 to 9-fold increase in factor activity compared to the wild-type FIX protein used in AMT-060, according to uniQure’s press release. 

This phase IIb study of AMT-061 is an open-label, single-arm, single-dose trial of approximately three patients receiving a single intravenous infusion and evaluated over a period of six to eight weeks to assess their FIX activity. AMT-061 was granted Breakthrough Therapy Designation by the US Food and Drug Administration.

Enrollment for the global Phase III HOPE-B clinical trial is underway and dosing of patients is expected to start in the first quarter of 2019. After a six-month observation period, approximately 50 adult severe to moderately-severe hemophilia B patients will receive a single intravenous administration of AMT-061.

“As a one-time administered therapy, AMT-061 has the potential to transform the treatment paradigm for hemophilia B patients,” said Annette von Drygalski, M.D., associate clinical professor at the University of California San Diego and director of its hemophilia and thrombosis treatment center. “By incorporating both AAV5 and the FIX-Padua variant, AMT-061 has the potential to deliver clinically relevant increases in FIX activity with low risk of cellular immune responses, which could expand patient eligibility for treatment with gene therapy.”

Source: uniQure press release dated August 23, 2018