After Hemgenix approvals, uniQure cashes out some of its royalty rights for $375M-plus

Months after the U.S. and European approvals of its CSL-partnered hemophilia A gene therapy Hemgenix, uniQure is selling some royalty rights for up to $400 million.

In a deal with HealthCare Royalty (HCRx) and Sagard Healthcare, uniQure agreed to part ways with a portion of its Hemgenix royalty rights. The company previously licensed the drug to Australia's CSL Behring, and it's entitled to milestone and royalty payments as the launch progresses.

Now, though, it's trading some of those royalty rights for a quick payday. Under the new deal with HCRx and Sagard Healthcare, uniQure will get $375 million upfront and another $25 million if an undisclosed sales threshold is achieved.

The cash lets uniQure “continue to invest” in its adeno-associated virus vector gene therapy pipeline, CEO Matt Kapusta said a statement. The pipeline includes a Huntington’s disease prospect, a refractory temporal lobe epilepsy candidate and an SOD-1 amyotrophic lateral sclerosis therapy.

“The partial monetization of this royalty stream validates the significant potential of Hemgenix global sales while substantially reducing commercialization risk for uniQure,” Kapusta said in the statement.

UniQure expects the deal to extend its cash runway through the second quarter of 2026.

As for HCRx, it is “pleased to be able to provide uniQure with the opportunity to immediately monetize their royalty assets,” CEO Clarke B. Futch said in a statement.

Under uniQure's deal with CSL, the Australian biopharma giant shelled out $450 million upfront for rights to the drug. CSL also agreed to pay up to $1.5 billion in milestones based on regulatory and commercial advances.

Hemgenix scored FDA approval in November and was cleared in Europe in February. The drug costs $3.5 million per treatment in the U.S. and is the most expensive drug available in the country.

Elsewhere in the hemophilia treatment field, BioMarin’s hemophilia A gene therapy Roctavian in March had its FDA decision date pushed back by three months. Sanofi’s Altuviiio bagged an FDA approval for the same hemophilia type in February.