Biotechnology giant CSL could potentially pay more than $US2 billion ($A2.9 billion) for a gene therapy for haemophilia B, which may help people with the blood clotting disorder to stop bleeding.
UniQure N.V. on Wednesday said it entered a licensing deal which will provide CSL Behring exclusive global rights to etranacogene dezaparvovec, an investigational gene therapy for patients with hemophilia.
Last year, uniQure reported phase 2b results with AMT-061 - which aims to replace missing Factor IX in patients with haemophilia B - showing that the therapy raised levels of the clotting factor well above the level needed to reduce bleeding episodes.
Patients would only need one treatment, which would remove the need for ongoing therapies.
UniQure said it "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".
Under the agreement terms, uniQure will complete the phase three trial and scale up the manufacture for early commercial supply.
Clinical development and regulatory activities performed by uniQure under the agreement will be reimbursed by CSL Behring.
Matt Kapusta, uniQure's CEO, said the transaction will provide the company with "significant financial resources" to advance its pipeline of other gene therapy candidates, including AMT-130 in Huntington's disease.
For CSL Behring, the acquisition will enhance its gene therapy portfolio. The company is now developing a stem cell gene therapy (CSL200) for the treatment of 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 - another rare disease area where CSL Behring has leading capabilities.
"We hope that it provides patients with an alternate best-in-class treatment option, building on our legacy of delivering lifesaving innovations in hematology", Paul stated.
CSL Behring has put patients first by addressing the world's most serious, complicated and rare diseases for over 100 years.
AMT-061 contains an AAV5 viral vector which has shown to be safe and well-tolerated in many clinical trials.
uniQure plans to continue to leverage its leading gene therapy platform, including the Company's deep expertise with AAV5, to develop potentially best-in-class gene therapies. No patient treated in clinical trials with uniQure's AAV5 gene therapies has experienced any cytotoxic T-cell-mediated immune response to the capsid.