- UniQure has sold off the rights to its experimental gene therapy for hemophilia B, announcing Wednesday evening that it is licensing the treatment to CSL Behring for $450 million in cash.
- The Dutch gene therapy developer could receive as much as $1.6 billion more in conditional payments if the gene therapy, currently in late-stage testing, progresses as expected. Per deal terms, CSL will also reimburse UniQure's remaining development costs, cover expenses related to regulatory submissions and give UniQure royalties on net sales.
- The deal, which sent shares in UniQure down sharply, gives the biotech enough cash to fund itself for another four years and develop its other experimental gene therapies. But licensing its lead drug candidate makes a buyout less likely — UniQure was seen as a potential acquisition target after sales of other gene therapy makers like AveXis, Spark Therapeutics and Audentes Therapeutics.
UniQure has had one of the more dramatic turnarounds of any gene therapy developer.
The Dutch firm, formed as Amsterdam Molecular Therapies in 1998, is a gene therapy pioneer, responsible for developing the first approved gene therapy in Europe. But that treatment, called Glybera, fizzled commercially, and UniQure withdrew it from the market in April 2017, five years after its historic approval.
UniQure's shares were worth just $5 apiece at the time. But the company restructured and focused on hemophilia B, slowly turning around its fortunes. At the same time, the gene therapy field vaulted forward with the approvals of Luxturna, for an inherited form of blindness, and Zolgensma, for spinal muscular atrophy.
The developers of both of those gene therapies — Spark and AveXis — were both acquired in multi-billion-dollar buyouts. That made UniQure and its advancing hemophilia B treatment, which could deliver late-stage results later this year, a potentially attractive target. Shares climbed to more than $82 apiece in 2019.
So the news that UniQure is selling the therapy, dubbed EntranaDez, is a "surprising transaction," wrote Stifel analyst Paul Matteis. Other gene therapy buyouts had made UniQure a "plausible M&A name," he said, and the partnership with CSL Behring "will be viewed as for now, taking that off the table." Shares fell 15% in postmarket trading Wednesday after UniQure announced the licensing.
Yet UniQure has other considerations. Within a few years, the company could have had one of two marketed gene therapies for hemophilia B — a Pfizer program, too, is in its final tests — dividing up an already small market with other treatment options.
It's also unclear how long the benefits of EntranaDez will last, or whether payers will cover the likely high price tag of a one-time treatment. Hemophilia affects some 400,000 people worldwide, but hemophilia B is four times less common than hemophilia A, according to the National Hemophilia Foundation.
The hemophilia B market "is small, global, competitive, and hard for [UniQure] to build a business around," Matteis wrote.
"This decision was based specifically on analyzing the competitive dynamics associated with the hemophilia market," CEO Matt Kapusta said on a conference call Wednesday. "Partnering this globally was the best way to get EntranaDez to as many patients globally as efficiently as possible and as fast as possible within this specific market."
Solving the commercial challenges will now be up to CSL, which already sells hemophilia treatments globally.
UniQure will use the cash it receives through the deal to place more gene therapy bets. One of its other programs, called AMT-130, recently became the first gene therapy to enter human testing in Huntington's disease.The company is also developing gene treatments for rare diseases like Fabry and it may add more through deals around neurological and liver-directed diseases.
Kapusta added that the company does aim to eventually sell other gene therapies on its own. "We still aspire to be a fully vertically integrated commercial entity, and we do plan to take Huntington's disease and other gene therapies to the market," he said.
UniQure also noted that it will stop developing a gene therapy for hemophilia A, which is well behind rival programs. BioMarin Pharmaceutical's hemophilia A gene therapy Roctavian could win approval in the U.S. this summer.