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Posted by James Tannahill

On 05/17/19, RumorHound platform alerted a potential takeover of UniQure Biopharma N.V. (NasdaqGSM:QURE; $59.74) where no potential acquirer was mentioned. Our analyst identified Bristol-Myers Squibb (NYSE:BMY; $46.85) as a potential acquirer, although several other large pharma are likely following QURE closely. See below:


QURE rumor is the latest in a series of 10 picked up by RumorHound dating back to February 2018. The Company is advancing their lead product- a gene therapy for Hemophilia B (AMT-061) currently in PhIIb- alongside a variety of established CNS and liver-directed candidates. Updated ‘061 data presented at the Hemostasis & Thrombosis Research Society (HTRS) 2019 Scientific Symposium earlier this month suggested a durable clinical response lasting up to 6-months following administration, where Factor IX (FIX) activity was sustained at 51% and 57% of normal in two patients in the dosing cohort after a one-time infusion, respectively. Notably, two patients in this cohort had been excluded from a different gene therapy trial at a competing biotech due to presence of neutralizing antibodies against the implemented AAV vector. No SAEs have been reported-while the durable and sustained clinical response of ‘061 suggests an on time HOPE-B PhIII kickoff by YE19 (https://clinicaltrials.gov/ct2/show/NCT03569891?cond=AMT-061&rank=1).


This rumor may be substantiated by heightened interest in the gene therapy space- e.g. large takeover events such as Roche (RHHBY) acquiring Spark Therapeutics (ONCE) for $4.8B (120% premium)- included three bidders who placed serious money on the table for ONCE’s Hemophilia programs and inherited retinal disease product Luxturna, according to their Schedule 14D-9 filed prior to the February 2019 merger (https://www.sec.gov/Archives/edgar/data/1609351/000119312519066393/d720810dsc14d9.htm). Other YTD gene therapy deals include Biogen (BIIB) takeout of Nightstar Therapeutics (http://investors.biogen.com/news-releases/news-release-details/biogen-announces-agreement-acquire-nightstar-therapeutics) for $800M, representing a 68% premium and Novartis (NVS) acquiring AveXis for $8.7B, who secured a 88% premium.


QURE has a robust patent estate (http://www.uniqure.com/gene-therapy/gene-therapy-intellectual-property.php) and has developed one of the largest gene therapy cGMP manufacturing facilities in the world, which would be able to produce commercial-scale product in-house while incubating ongoing pipeline development activities. These Of note, BMY has partnered with QURE and holds more than 6% of QURE shares outstanding- signaling that their foot and pocketbook are already in the door. Although the commercial prospects for gene therapy are still unclear, QURE represents a tasty next addition to the gene therapy MA list for 2019.


About uniQure 
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntington’s disease and other severe genetic diseases. www.uniQure.com


About Bristol-Myers Squibb

Bristol-Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol-Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube and Facebook.

Topics: Rumor Hound