CSL To Acquire Rights to Hemophilia B Gene Therapy in $2-Bn Deal

By Miranda Greenberg -

July 1, 2020

CSL Behring has agreed to acquire exclusive global license rights to commercialize an adeno-associated virus (AAV) gene therapy for treating hemophilia B from uniQure, an Amsterdam-based gene-therapy company, in a deal worth up to $2 billion ($450 million upfront and up to $1.6 billion in milestone payments).

Under the agreement, CSL Behring will have the exclusive global right to commercialize AMT-061 (etranacogene dezaparvovec), which is currently in Phase III clinical trials. Under the agreement, uniQure will complete the Phase III trial and scale up manufacture for early commercial supply under an agreed plan with 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.

Under the agreement, uniQure will receive a $450-million upfront cash payment and 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 percentage of net product sales arising from the collaboration.

This acquisition adds to CSL Behring’s gene-therapy portfolio. The company is currently developing a stem-cell gene therapy, CSL200, for treating sickle-cell disease and has recently established an alliance with Seattle Children’s Research Institute to develop a stem-cell gene therapy for primary immunodeficiency diseases.

The transaction is subject to customary regulatory clearances before closing.

Source: CSL Behring and uniQure