uniQure NV (NASDAQ:QURE) has announced that it has entered a licensing agreement with CSL Behring. The agreement offers CSL Behring exclusive rights globally to uniQure’s hemophilia B gene therapy etranacogene dezaparvovec.
uniQure to receive $450 million upfront
Etranacogene dezaparvovec comprises of AAV5 viral vector that carries a gene cassette with Padua variant of Factor IX. According to the terms of the transaction, uniQure gets an upfront payment of $450 million and will also be eligible for up to $1.6 billion in commercial and regulatory milestone payments. Similarly, the company will also receive tiered double-digit royalties that range up to percentage in low twenties for net product sales accruing from the partnership.
The partnership will leverage the strong commercial infrastructure and global reach if CSL Behring in hematology. This will help expedite the availability of etranacogene dezaparvovec to individuals suffering from hemophilia B across the globe. uniQure CEO Matt Kapusta indicated that they were delighted with the license and commercialization deal with CSL Behring. The CEO added that CSL was an ideal partner because of its global reach and hemophilia expertise. uniQure believes that deliver the innovative Haemophilia B gene therapy to patients as soon as possible.
uniQure announce treatment of first patients in AMT-130 study
Recently the company announced that two patients have been treated in the Phase 1/2 AMT-130 clinical study for the treatment of Huntington’s disease patients. The study is a randomized, double-blind clinical study conducted in the US with one patient having been treated using AMT-130 while another receiving imitation surgery.
uniQure CEO stated that the company has been committed to advancing the first human Huntington’s disease AAV gene therapy into clinical trials. Kapusta stated that the treatment of the first patients is a huge milestone for the company considering there are two AAV gene therapy candidates in clinical testing. Following the treatment of the first patients in the study, the company has taken steps to advance AMT-130. This moves uniQure closer to its goal of developing a therapy inhibiting the production of the modified Huntington protein.