Hemophilia

uniQure has built a potential first- and best-in-class hemophilia B gene therapy program.

uniQure is advancing a promising clinical program focused on hemophilia B, a severe orphan blood clotting disorder. Our gene therapy product candidate etranacogene dezaparvovec (AMT-061) consists of an AAV5 viral vector carrying a gene cassette with the Padua variant of Factor IX (FIX-Padua). 

uniQure is now conducting the Phase III HOPE-B pivotal study of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B. 

In June 2021, we announced positive 52-week data on all patients from the trial, the first clinical data from a Phase III gene therapy study in hemophilia B and, with 54 patients, the largest set of hemophilia B patients receiving a gene therapy investigational product to date. These data show that participants continued to demonstrate durable, sustained increases in Factor IX (FIX) activity at one year post-infusion with a mean FIX activity of 41.5 percent of normal, compared to a mean FIX activity of 39.0 percent of normal at 26-weeks of follow-up. 

AAV5-based gene therapies have been demonstrated to be safe and well-tolerated in a multitude of clinical trials, including five uniQure trials conducted in nearly 80 patients in hemophilia B and other indications. No patient treated in clinical trials with the uniQure’s AAV5 gene therapies has experienced any confirmed cytotoxic T-cell-mediated immune response to the capsid. Additionally, pre-clinical and clinical data show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). uniQure holds multiple issued patents in the United States and Canada broadly, with patent pending in the EU, covering methods of treating bleeding disorders, including hemophilia B, using AAV gene therapy with the FIX-Padua variant. 

Etranacogene dezaparvovec has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration and access to the Priority Medicines (PRIME) regulatory initiative by the European Medicines Agency. 

We believe that etranacogene dezaparvovec has the potential to be a first- and best-in-class gene therapy in hemophilia B, providing durable, functionally curative benefits to nearly all patients with hemophilia B.  These factors, along with our commercial-scale manufacturing capabilities, differentiate etranacogene dezaparvovec from other hemophilia gene therapies in development.

 

Licensing Agreement with CSL Behring

uniQure and CSL Behring have entered into a licensing agreement providing CSL Behring exclusive global rights to etranacogene dezaparvovec.  Under the terms of the agreement, which was completed in May 2021, uniQure receives a $450 million cash payment and will be eligible to receive up to $1.6 billion in payments based on regulatory and commercial milestones. uniQure will also be eligible to receive tiered double-digit royalties in a range of up to a low-twenties percent of on net sales of products arising from the collaboration based on sales thresholds.

uniQure is responsible for the completion of the HOPE-B pivotal study, manufacturing process validation, and the manufacturing supply of etranacogene dezaparvovec until such time that these capabilities are transferred to CSL Behring. Clinical development and regulatory activities performed by uniQure under the agreement will be reimbursed by CSL Behring. CSL Behring will be responsible for regulatory submissions and commercialization of etranacogene dezaparvovec. 

The collaboration combines uniQure’s differentiated gene therapy candidate in hemophilia B and CSL Behring’s strong global reach and commercial infrastructure in hematology to accelerate access of etranacogene dezaparvovec to hemophilia B patients around the world.

 

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