- UniQure said Thursday it closed a deal with CSL Behring, selling an experimental gene therapy for hemophilia B after regulators in three countries signed off on the transaction.
- By May 13, UniQure will receive $450 million from CSL, and could take home as much as $1.6 billion in additional payments contingent on reaching certain regulatory and commercial milestones. The Dutch biotech will also be reimbursed for the costs of development and regulatory activities undertaken during the transfer of responsibilities to CSL.
- The deal's clearance is the latest indication that biopharma dealmaking may not yet be slowed by the Federal Trade Commission's plans to look more closely at acquisitions. Concern that the FTC may hold up the alliance weighed down UniQure's shares, according to some Wall Street analysts. The company's stock climbed 7% in trading Thursday.
According to Paul Matteis, an analyst at Stifel, the closing of the deal removes a question mark hanging over UniQure's stock. Regulators, especially those in the U.S., are taking harder looks at pharmaceutical acquisitions, suggesting that approval of the UniQure-CSL asset sale wasn’t assured.
The CSL deal, originally announced in June 2020, surprised some investors who saw UniQure as a takeover target. By selling its most closely watched experimental medicine, the company likely removed itself from the running to be bought anytime soon.
Meanwhile, the transaction offered UniQure an immediate infusion of cash needed to develop other products, including an experimental therapy for Huntington’s disease. Analysts expect initial data from the treatment by the end of the year.
CSL's experience in the hemophilia market should translate to higher sales of the hemophilia treatment than UniQure could have managed alone, wrote Cantor Fitzgerald analyst Kristen Kluska in a note to clients. Kluska added that she believes the deal is favorable for UniQure, which will receive royalties on sales.
UniQure shares rose about 7% to just over $32 apiece in Thursday morning trading. Still, shares are well below the $67 they traded at right before the agreement with CSL was announced.
The gene therapy at the center of UniQure and CSL's deal is known as AMT-061. UniQure has been testing it in patients with moderate to severe hemophilia B, the less common form of the rare bleeding disorder. Clinical trial results made public last year showed that, after receiving AMT-061, most patients were producing enough blood clotting protein to no longer need the protein-replacement therapy that's commonplace in hemophilia treatment.
While AMT-061 has been proven effective, it still faces obstacles. A rival gene therapy from partners Pfizer and Spark Therapeutics, a biotech Roche acquired in 2019, is not far behind in development. There are also multiple, effective drugs — including some sold by CSL — already available to hemophilia B patients, meaning there could be a considerable amount of pricing pressure on AMT-061 should it come to market.
UniQure also just had to deal with a testing issue related to AMT-061. Late last month, the Food and Drug Administration lifted a clinical hold on the therapy, which was put in place after a patient treated with it unexpectedly developed liver cancer. The FDA ended up agreeing with UniQure's assessment that there were no signs its treatment caused the cancer.