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Uniqure share prices drop shows csl got the tech A lot cheaper than taking over uniqure.


In an unexpected twist, CSL grabs worldwide rights to uniQure's PhIII hemophilia B gene therapy in $2B-plus dealJohn CarrollEditor & FounderIn a surprise move, CSL Behring reported after the market closed on Wednesday that it is grabbing rights to uniQure’s Phase III hemophilia B gene therapy with a $2 billion-plus deal fronted with $450 million in cash.The money — which includes $1.6 billion in milestones — gives the Australian pharma giant global rights to the pivotal stage AMT-061 (etranacogene dezaparvovec), an AAV5 viral vector carrying a gene cassette with the Padua variant of Factor IX, which hemophilia B patients lack. Top-line Phase III data are expected before the end of this year from the HOPE-B study.“Our vision with hemophilia B patients is to offer transformational treatment paradigms that help free them from the lifelong burden of this disease,” said CSL’s CEO Paul Perreault. “With more than three decades of providing lifesaving innovations for the global bleeding disorders community, we are well positioned to maximize the potential benefit of this therapy. Upon approval, we believe this next-generation therapy will be highly complementary to our existing best-in-class hemophilia B product portfolio with an alternate best-in-class treatment option.”advertisementadvertisementGenerally a low-key pharma, CSL has been on a deal spree recently. The pharma company bought out the IL-6 player Vitaeris a few days ago. But gene therapy is clearly the big new focus. Two weeks ago it partnered with Seattle Children’s Research Institute to develop stem cell gene therapies for rare primary immunodeficiency diseases like Wiskott-Aldrich syndrome and X-linked Agammaglobulinemia. This latest deal marks a dramatic turn into the spotlight, taking on Pfizer’s rival gene therapy in a closely-watched late-stage matchup.In a recent note, SVB Leerink’s Joseph Schwartz commented:Recall that in a Ph.2b dose-confirmation study, QURE reported ~41% Factor IX (FIX) activity out to 1 year (n=3; LINK). 26-week FIX data from all 54 pts. enrolled in the Ph.3 HOPE-B trial remains on schedule, and mgmt. continues to anticipate topline data from the pivotal trial by YE20. QURE also reiterated plans to complete a BLA submission in 2021.A major supplier of plasma, CSL also sells treatments for hemophilia A and B that could soon be eclipsed by a wave of gene therapies headed to regulators.It’s a major turning point for uniQure, which has had its ups and downs over the years. The sudden injection of cash gives uniQure enough cash to push ahead with AMT-130 in Huntington’s disease, where it just dosed patients, launch IND-enabling studies of AMT-150 in spinocerebellar ataxia type 3, select a lead candidate in Fabry disease and move on other current and new candidates for central nervous system disorders and rare liver-directed diseases.But the news left some uniQure investors feeling a little flat, especially after they were buzzing for months over a potential buyout that would have been far more rewarding for the shareholders. The stock slid 8% after the news hit.AUTHORJohn CarrollEditor & [email protected]@JohnCendptsJohn Carroll on LinkedIn