Amsterdam-based Uniqure NV’s regulatory hitch with its highly regarded hemophilia B program had analysts questioning during a conference call what the development might mean for the company’s deal with CSL Ltd.
Positive top-line data from the pivotal phase III study of AAV5-based etranacogene dezaparvovec by Uniqure NV, shows 54 patients met the primary endpoint in treating severe to moderate hemophilia B.
CSL Behring has licensed global rights to Uniqure NV's late-stage hemophilia B gene therapy candidate, AMT-061, for $450 million up front, plus up to $1.6 billion in potential milestone payments and royalties. Uniqure positioned the deal as a boon for its broader gene therapies pipeline, anchored by a Huntington's disease (HD) program. But the move appeared to sour investors hoping for a rumored near-term M&A takeout, sending company shares (NASDAQ:QURE) falling 21.8% to $49.22 on June 25.
CSL Behring has licensed global rights to Uniqure NV's late-stage hemophilia B gene therapy candidate, AMT-061, for $450 million up front, plus up to $1.6 billion in potential milestone payments and royalties. Uniqure positioned the deal as a boon for its broader gene therapies pipeline, anchored by a Huntington's disease (HD) program. But the move appeared to sour investors hoping for a rumored near-term M&A takeout, sending company shares (NASDAQ:QURE) falling 21.8% to $49.22 on June 25. Despite the market reception, analyst reaction was largely supportive of the move, embracing CSL as a natural fit for the program and, in the words of SVB Leerink analyst Joseph Schwartz, appreciating the transfer of "any potential remaining risk in hemophilia B to CSL Behring as QURE rings the cash register."