CSL Acquires Hemophilia B Gene Therapy Candidate from uniQure

CSL Behring recently announced that it has agreed to acquire exclusive global license rights from uniQure, to commercialize etranacogene dezaparvovec (AMT-061), an investigational gene therapy candidate for patients with severe and moderately severe hemophilia B.

Etranacogene dezaparvovec 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 CSL press release announcing the acquisition references positive data, first reported in late 2019, from a phase 2b dose-confirmation study of etranacogene dezaparvovec. Those results showed that all trial participants had demonstrated stabilized and sustained FIX activity at functionally high levels for one year following a single administration of the therapy. By increasing FIX activity up to 50% (mean of 41%) of normal, patients had achieved levels “considered sufficient to eliminate or significantly reduce” bleeding risk.

“Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” said CSL’s CEO and Managing Director Paul Perreault. “With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy.”

“We are thrilled to enter into this commercialization and license agreement with CSL Behring, an ideal commercial partner with global reach and decades of expertise in hemophilia,” stated Matt Kapusta, chief executive officer of uniQure. “We believe that through this arrangement, we are ideally positioned to deliver our innovative gene therapy to the largest number of hemophilia B patients as quickly as possible. The transaction represents a major milestone in the development of etranacogene dezaparvovec and, when closed, we expect that it will provide uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates, anchored by AMT-130 in Huntington’s disease, as well as to invest further in our leading manufacturing and technology platform.”

As part of the agreement uniQure will complete an ongoing phase 3 clinical study currently investigating etranacogene dezaparvovec in a total of 54 patients, with new trial data expected by the end of 2020. The therapy has already been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration.

Source: CSL Behring press release dated June 24, 2020

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