- UniQure said Monday it has sold a portion of the royalties it expects to receive on sales of a newly approved gene therapy for hemophilia B.
- HealthCare Royalty and Sagard Healthcare will pay $375 million upfront and up to $25 million in milestone payments in exchange for the “lowest royalty tier” for sales of Hemgenix, which won U.S. approval in November for the less common form of the bleeding disorder.
- Three years ago, UniQure licensed the therapy to Australian drugmaker CSL Behring for $450 million in cash. The royalty sale “is a smart move,” Stifel analyst Paul Matteis wrote in a Monday note, because it extends UniQure’s cash runway into 2026, beyond trial readouts for its experimental Huntington’s disease gene therapy.
When Hemgenix gained U.S. approval, it became the first marketed gene therapy for hemophilia B and, at a list price of $3.5 million, the country’s most expensive medicine on a single-use basis.
While Hemgenix was long one of UniQure’s top assets, the company has broader ambitions it has been working toward following the 2020 deal with CSL. Monday’s royalty agreement gives it more cash to fund those plans.
“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,” the company’s CEO Matt Kapusta said in a statement.
UniQure’s pipeline includes a therapy for Huntington’s disease, called AMT-130, experimental treatments for amyotrophic lateral sclerosis it acquired from Apic Bio in January, and a therapy called AMT-260 for a type of epilepsy, he said.
Under its 2020 deal with CSL, UniQure is eligible to receive tiered double-digit royalties up to a low-twenties percentage. A portion of those royalties will now go to HealthCare Royalty and Sagard Healthcare, up to 1.85 times the purchase price through 2032 or, if that cap is not reached, up to 2.25 times the purchase price by 2038.
UniQure said the royalty deal will extend its cash runway into the second quarter of 2026, assuming it receives a $100 million milestone payment from CSL for the first commercial sale of Hemgenix in the U.S.
Hemgenix received EU approval in February, but there is a chance its first commercial sale on the continent won’t be recorded by a July 2 deadline for UniQure to get another $75 million from CSL, Matteis wrote. UniQure is working with CSL to see if they can extend the deadline, he added.
On the AMT-130 therapy for Huntington’s disease, Matteis said there is a “real chance” that initial readouts in second or third quarter 2023 could be inconclusive because of the limited number of patients being followed out to two years and the slow clinical progression of the disease.