RARE Daily

UniQure Sells Royalty Interest in Hemgenix for Up to $400 Million

May 15, 2023

Rare Daily Staff

UniQure said it entered into a definitive agreement to sell a portion of the royalty rights due to UniQure from CSL Behring from the net sales of Hemgenix to HealthCare Royalty and Sagard Healthcare for a gross purchase price of up to $400 million in cash.

“This attractive 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,” said Matt Kapusta, CEO of UniQure. “The partial monetization of this royalty stream validates the significant potential of Hemgenix global sales while substantially reducing commercialization risk for UniQure.”

Hemgenix is the first gene therapy approved for adults with hemophilia B. It reduces the rate of abnormal bleeding by enabling the body to continuously produce factor IX, the deficient protein in hemophilia B.

UniQure led the multi-year development of Hemgenix and completed an exclusive global license and commercialization agreement with CSL Behring in May 2021. Hemgenix is approved for the treatment of hemophilia B in the United States, Europe, and United Kingdom. CSL Behring is also pursuing registration in additional countries.

Under the terms of the agreement with HealthCare Royalty and Sagard, UniQure will receive an upfront cash payment of $375 million in exchange for the lowest royalty tier on CSL Behring’s worldwide net sales of Hemgenix 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.

UniQure also is eligible to receive an additional $25 million milestone payment if 2024 net sales of Hemgenix exceed a pre-specified threshold. UniQure will retain the rights to all other royalties under its existing agreement with CSL Behring, as well as contractual milestones totaling up to $1.5 billion, including the $100 million milestone for the first U.S. product sale and the $75 million milestone for the first product sale in one of five major European countries if achieved prior to July 2, 2023. The transaction is expected to close no later than 15 business days from signing.

UniQure expects the transaction will extend its cash runway into the second quarter of 2026, assuming receipt of the contractual milestone payment for the first sale of Hemgenix in the United States.

Photo: Matt Kapusta, CEO of UniQure

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