uniQure N.V. is to sell a portion of its royalty rights from the net sales o its hemophilia B gene therapy etranacogene dezaparvovec-drbl to HealthCare Royalty and Sagard Healthcare for a gross purchase price of up to $400m in cash. “This  financing provides uniQure with immediate, non-equity-dilutive capital to continue to invest in our AAV gene therapy pipeline and platform and to advance potentially breakthrough gene therapies for patients, including AMT-130 in Huntington’s disease, AMT-260 for refractory temporal lobe epilepsy, AMT-162 for SOD-1 ALS and other product candidates,” statedMatt Kapusta, CEO of uniQure.
Etranacogene dezaparvovec-drbl is a gene therapy that reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.
Under the terms of the agreement, uniQure will receive an upfront cash payment of $375m in exchange for the lowest royalty tier on CSL Behring’s worldwide net sales of etranacogene dezaparvovec-drbl up to 1.85 times the purchase price until June 30, 2032 or, if such cap is not met by June 30, 2032, up to 2.25x the purchase price through December 31, 2038.