UniQure weighs sale after hemophilia gene therapy turnaround: report

uniQure
UniQure has a market cap of $2.7 billion. (uniQure)

Gene therapy specialist uniQure is considering selling up amid interest from large pharma buyers, according to Bloomberg. The report follows the revitalization of uniQure’s hemophilia B prospect and accompanying triple-digit stock price surge.

According to unnamed sources, uniQure is working with advisers to assess options including a sale or partnership but is yet to decide which path to take. 

The report is credible given interest in the sort of assets and infrastructure uniQure possesses. But, as the postmortems of deals involving rivals such as Nightstar Therapeutics shows, uniQure may be in talks about a range of partnership structures and buyout offers while also evaluating the possibility of raising money and going it alone. There is no guarantee uniQure will opt to accept a buyout.

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Analysts have touted Novo Nordisk, Pfizer and Sanofi as among the companies that may be interested in acquiring uniQure. Three companies were in the running to partner with or buy uniQure’s chief rival for the hemophilia A gene therapy market, Spark Therapeutics. Roche won that race with a $4.8 billion offer, leaving two companies with an unsated desire to pick up a hemophilia A gene therapy.

Whether the companies want to buy uniQure or license its lead program will depend in part on their infrastructure and gene therapy strategies. Following a move from wild-type factor IX (FIX) to gene variant FIX Padua, uniQure’s lead program, renamed AMT-061, has begun generating data that are in line with results posted by Spark’s SPK-9001. And uniQure looks to have stolen a lead in the clinic, with its phase 3 underway and Pfizer’s pivotal assessment of SPK-9001 yet to start enrollment.

AMT-061 also looks to be effective in patients with neutralizing antibodies to the AAV vector used in SPK-9001, giving the asset a mix of positive attributes that could be attractive to multiple companies.

A partnership deal would allow a company to gain access to AMT-061 without taking on uniQure’s other assets and infrastructure. But for some companies, the resources uniQure possesses beyond AMT-061 could be attractive. Notably, uniQure runs a 55,000-square-foot manufacturing facility in Massachusetts. 

The progress of AMT-061 and demonstrations of big biopharma interest in gene therapy players have driven uniQure’s stock price up more than 160% this year, giving it a market cap of $2.7 billion. 

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