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UniQure Selling Portion of Royalties From Hemgenix to Fund Other Gene Therapy Programs

NEW YORK – UniQure on Monday said it is selling a portion of its royalty rights from the net sales of hemophilia treatment Hemgenix (etranacogene dezaparvovec-drbl) to HealthCare Royalty and Sagard Healthcare for up to $400 million in cash.

UniQure, headquartered in the Netherlands, plans to invest the capital in its gene therapy pipeline, which includes AMT-130, an investigational treatment for Huntington's disease; AMT-260, a potential treatment for refractory temporal lobe epilepsy; and AMT-162 for SOD-1 amyotrophic lateral sclerosis.

UniQure led development of Hemgenix, an adeno-associated virus-based gene therapy that delivers the Padua gene variant of factor IX to target cells in the liver, improving the activity of factor IX proteins and reducing abnormal bleeding in hemophilia B patients. It inked a global license and commercialization agreement with CSL Behring in May 2021 for Hemgenix, which was subsequently approved as the first gene therapy for hemophilia B in the US, Europe, and other countries.

Under the terms of the agreement, UniQure will receive an upfront cash payment of $375 million for selling its rights to the lowest royalty tier on CSL Behring's worldwide net sales of Hemgenix. UniQure is eligible to receive up to 1.85 times the purchase price until June 30, 2032, or if such cap is not met by then, up to 2.25 times the purchase price through Dec. 31, 2038.

UniQure also is eligible for an additional $25 million milestone payment if 2024 net sales of Hemgenix surpass a pre-specified threshold. In the deal, UniQure retains the rights to other royalties it is eligible for under its agreement with CSL Behring, as well as to contractual milestones totaling up to $1.5 billion, which include milestone payments tied to the first Hemgenix sales in the US and in one of five European countries prior to July 2 of this year.

UniQure expects the transaction, slated to close 15 days from signing, to extend its cash runway into Q2 2026, assuming it receives the $100 million contractual milestone payment from the first sale of Hemgenix in the US. Moelis & Company was UniQure's financial adviser in this transaction, and Ropes & Gray was its legal adviser. HealthCare Royalty was represented by Sidley Austin, and Sagard was represented by Mintz, Levin, Cohn, Ferris, Glovsky, and Popeo.