A hemophilia gene therapy initially developed in the labs of uniQure is set to start generating revenue following its regulatory approvals in the U.S. and Europe, but the company is cashing in on that treatment’s financial promise now through a royalty deal that could pay up to $400 million.
Amsterdam-based uniQure said Monday that it has agreed to sell some of the royalties it is due for the therapy, Hemgenix. The buyers are HealthCare Royalty and Sagard Healthcare. UniQure plans to deploy the new cash toward clinical development of other gene therapies in its pipeline.
FDA approval of Hemgenix last fall made it the first gene therapy for hemophilia B. The one-time treatment delivers a gene intended to restore production of the blood-clotting protein deficient in hemophilia B patients. Three years ago, when Hemgenix was still in late-stage clinical development, CSL Behring acquired rights to the uniQure gene therapy, committing $450 million up front and another $1.6 billion tied to milestones. That deal also put uniQure in line for tiered double-digit royalties from CSL Behring’s sales of the therapy, whose $3.5 million list price makes it the most expensive therapy of any type.
According to the terms of the agreement announced Monday, uniQure will receive a $375 million up front payment in exchange for the lowest royalty tier on CSL Behring’s global sales of Hemgenix. That royalty is capped at 1.85 times the purchase price until June 30, 2032. If the cap is not met by that date, it rises to 2.25 times the purchase price through Dec. 31, 2038. The agreement also makes uniQure eligible for an additional $25 million milestone payment if 2024 net sales of Hemgenix exceed a pre-specified threshold that was not disclosed.
UniQure retains all other royalties that were established by its licensing agreement with CSL Behring, along with the milestone payments that were part of that deal. For the first U.S. product sale, uniQure will receive $100 million. Another $75 million milestone is due for the first product sale in one of the five major European countries—if that sale happens before July 2.
With the royalty deal, uniQure is extending its financial runway by another year. In its report of first quarter 2023 financial results, the company said it expects to have enough cash to last into the first half of 2025. UniQure said Monday that the royalty deal provides the company with enough cash to last into the second quarter of 2026. That timeframe assumes the receipt of the $100 million milestone payment for the first Hemgenix sale in the U.S.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In a statement, uniQure CEO Matt Kapusta said the royalty agreement provides immediate, non-dilutive capital that allows for continued investment in other gene therapies. The next most advanced program in the uniQure pipeline is Huntington’s disease gene therapy AMT-130, which has reached Phase 1/2 testing. AMT-162, a potential gene therapy for amyotrophic lateral sclerosis driven by the SOD1 genetic mutation, is on track to begin a Phase 1/2 study in the second half of this year, the company said in its annual report. The biotech also has gene therapies in preclinical development for temporal lobe epilepsy, Fabry disease, Parkinson’s disease, and Alzheimer’s disease.
“The partial monetization of this royalty stream validates the significant potential of Hemgenix global sales while substantially reducing commercialization risk for uniQure,” Kapusta said.
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