uniQure announces achievement of $100 million milestone related to hemophilia B gene therapy

Milestone payment triggered by first commercial sale of HEMGENIX in U.S. by CSL Behring

LEXINGTON, Mass. and AMSTERDAM, June 20, 2023 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, announced the achievement of a $100 million milestone associated with the first commercial sale of HEMGENIX in the United States by its partner, global biotechnology leader CSL (ASX: CSL). HEMGENIX (etranacogene dezaparvovec) is a one-time administered gene therapy for the treatment of adults with hemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage or have repeated, serious spontaneous bleeding episodes.

“The first commercial sale in the U.S. is a major milestone for uniQure as it marks the fulfillment of our promise to deliver genetic medicines that have the potential to transform people’s lives,” said Matt Kapusta, chief executive officer of uniQure. “uniQure’s successful development of HEMGENIX further validates our AAV platform built on the back of 25 years of scientific leadership and innovation in the field of gene therapy. We look forward to our continued collaboration with CSL Behring as they work to bring this important treatment to those living with hemophilia B.”

uniQure conducted the multi-year research and clinical development program for HEMGENIX, which included three clinical trials across 34 global sites and involved 67 adults with hemophilia B. In May 2021, uniQure and CSL completed a licensing transaction providing CSL Behring with exclusive rights to commercialize and continue clinical development of HEMGENIX globally. uniQure is responsible for the global manufacturing of the product at its licensed facility in Lexington, Massachusetts. Under the agreement with CSL Behring, the milestone payment is due within 30 days after achievement of the milestone.

HEMGENIX is the first approved gene therapy for hemophilia B in the United States, European Union (EU) and European Economic Area (EEA), and the UK.

Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene defect, resulting in insufficient production of factor IX, a protein primarily produced by the liver that helps blood clots form. Treatments for moderate to severe hemophilia B include prophylactic infusions of factor IX replacement therapy to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules. They may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease. For appropriate patients, HEMGENIX has been shown in clinical trials to allow people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding.