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.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising.
A first half of the year progress report from the international advocacy group Alliance for Regenerative Medicine (ARM), finds that the regenerative medicine and advanced therapy sector is in very good shape and has performed well in terms of both clinical development and fundraising despite the challenges posed by the ongoing COVID-19 pandemic.
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."
