uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 for the global licensing agreement with CSL Behring for etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia B, expired on May 5, 2021, and the agreement became fully effective on May 6, 2021. The agreement was contingent on completion of review under antitrust laws in the United States, Australia and the United Kingdom. The antitrust review process is now complete in all three countries.
“As a global leader in hematology and thrombosis, CSL Behring is an ideal commercial partner, and we are excited to embark on our relationship together with a shared goal of delivering this potentially transformative therapy to patients around the world living with hemophilia B,” stated Matt Kapusta, chief executive officer of uniQure. “This transaction positions etranacogene dezaparvovec to be made available to the largest number of hemophilia B patients as quickly as possible and provides uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates.”
As previously announced in June 2020, uniQure and CSL Behring entered into a licensing agreement providing CSL Behring with exclusive global rights to etranacogene dezaparvovec. Under the terms of the agreement, uniQure will receive a $450 million cash payment from CSL Behring by May 13, 2021 and is 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 percentage of net product sales arising from the collaboration.
Additionally, uniQure will be 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.