CSL Behring has struck a deal to buy uniQure’s late-phase hemophilia B gene therapy AMT-061 for $450 million upfront. The agreement will catapult CSL to the front of the race to bring a hemophilia B gene therapy to market, pitting it against companies including Pfizer. 

UniQure has turned perceptions of its hemophilia B gene therapy around in recent years. After clinical data suggested it would struggle to match the efficacy of Pfizer and Spark Therapeutics’ SPK-9001, uniQure switched from wild-type factor IX (FIX) to gene variant FIX Padua and began posting stronger results. The change in fortunes dialed up uniQure’s value and talk of a takeover.

Now, CSL is set to pick up the hemophilia B gene therapy without acquiring the rest of uniQure. The deal features a $450 million upfront payment and up to $1.6 billion in milestones, $300 million of which are tied to regulatory events and first commercial sales. CSL is also on the hook for tiered royalties that top out a little above 20%.

In return for the outlay, CSL is set to gain the global rights to a gene therapy that, based on cross-trial comparisons involving small numbers of patients, could be best in class. AMT-061 also looks likely to come to market before SPK-9001, its closest competitor. If AMT-061 lives up to that promise, it could enable CSL to grow its $1 billion a year hemophilia business significantly.

Investors have called the wisdom of taking the other side of the deal into question, though. Shares in uniQure fell 12% following news of the CSL agreement, which dashed near-term hopes of the biotech being bought out.  

Spark also courted a company that offered $450 million upfront for hemophilia gene therapy assets but went on to accept a $4.3 billion takeover offer from Roche. With other gene therapy companies such as Audentes Therapeutics and AveXis also landing multibillion dollar buyouts, investors viewed uniQure as next in line.

The fact that uniQure ended up offloading one asset, not the whole company, may say something about the lack of potential buyers with a broad enough focus to place value on all its assets. UniQure CEO Matt Kapusta outlined the dynamics on a conference call with investors. 

“M&A for pharma companies is about the lead program, it’s about the pipeline and it’s about the platform. For an acquisition to make sense, you’ve got to be able to value all of that. The reality is when you look at hemophilia B, and you look at Huntington’s or CNS assets, in order to value all of that requires a specific therapeutic focus area of the acquirer,” Kapusta said. 

UniQure will emerge from the deal with a narrower focus. As well as selling AMT-061, uniQure is deprioritizing its hemophilia A asset AMT-180, resulting in a pipeline centered on CNS and, to a lesser extent, liver-directed diseases. UniQure plans to focus on those areas after exiting hemophilia. 

Kapusta said the CSL deal “could make [uniQure] potentially a more attractive target for an acquirer.” UniQure still has manufacturing assets along with aspirations to become a vertically integrated commercial entity, but it lacks validated clinical-phase assets.

A Huntington’s disease program that began dosing patients in a first-in-human study this month will be uniQure’s most advanced prospect when the CSL deal closes. UnIQure also has preclinical gene therapies against Fabry disease and spinocerebellar ataxia type 3 and plans to use some of the $720 million it will have after the CSL deal to in-license or acquire additional candidates.  

The new uniQure will lack a late-phase asset but also be free from the need to build commercial infrastructure to support its entry into the hemophilia B market. In accepting that trade-off, uniQure is set to revert to being an earlier-stage biotech, albeit one funded through to the second half of 2024 and with a five-year plan to have up to five commercial or pivotal-stage programs.

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