UniQure sells hemophilia gene therapy royalty rights

UniQure made an announcement that it has sold a portion of the royalties worth $400 million it expects to get from the sale of a newly authorized gene treatment for hemophilia B. This took place a few months after the company received its final nod from the US and European regulatory bodies.

In a recent agreement with HealthCare Royalty and Sagard Healthcare, UniQure has consented to selling a portion of its royalty rights related to Hemgenix. The medicine was originally licensed to CSL Behring, where the company will receive payments in the form of royalties once the medicine is fully launched.

But it’s now swapping some of those royalties for a faster way of earning. UniQure will get $375 million upfront and an additional $25 million if an unknown sales benchmark is met under the new HCRx and Sagard Healthcare agreement.

According to CEO Matt Kapusta, the funds would allow UniQure to continue to further invest in its adeno-associated viral vector gene therapy pipeline. A Huntington’s disease candidate, a refractory temporal lobe epilepsy candidate, and a SOD-1 amyotrophic lateral sclerosis treatment are all in development. This additional stream of royalties indicates the potentially rising sales of the medication with very little risk for the company.

UniQure is entitled to receive royalty payments within the bracket of 20% under its 2020 agreement with CSL. HealthCare Royalty and Sagard Healthcare will receive a share of the payments, which will be 1.85 times the purchase price until 2032. However, if the ceiling is not reached, the payments can go up to 2.25 times the purchase price by 2038.

Healthcare Royalty CEO Clarke believes that the company is honored to support UniQure in increasing its royalty assets.

UniQure licensed the medication to Australian pharmaceutical CSL Behring three years ago for $450 million in cash. In addition, CSL committed to pay up to $1.5 billion in milestone payments dependent on regulatory and commercial advancements. According to Stifel analyst Paul Matteis, the royalty sale will support UniQure’s funding for its experimental Huntington’s disease gene treatment until 2026.

Hemgenix received FDA approval in November and European clearance in February. The medicine costs $3.5 million per treatment in the United States and is the most expensive medication on the market.

Matteis stated that there is a real chance that early readouts in the second or third quarter of 2023 would be inconclusive due to the small number of patients being tracked for two years and the delayed clinical course of the disease.

In other hemophilia treatment news, the FDA delayed BioMarin’s hemophilia gene therapy Roctavian by three months in March. Sanofi’s Altuviiio received FDA clearance for the same hemophilia type.

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