Seeking cost savings, uniQure cuts R&D staff and trims pipeline projects


Embattled gene therapy specialist uniQure is planning to cut projects and R&D staff to reduce costs as it strives to bring its next product to market.

The Dutch firm said it intends to cut 20%-25% of its total headcount--some 50 to 60 jobs--by the end of 2017 as it tries to slice €5 million to €6 million off its cost base next year and extend cash resources "into 2019". 

Interim chief executive Matt Kapusta said the company intends to focus its efforts on its hemophilia B candidate AMT-060--currently in a Phase I/II trial--as well as a Huntington's disease therapy and a congestive heart failure candidate partnered with Bristol-Myers Squibb which remain in preclinical development.

That means two other programs, AMT-110 for the treatment of Sanfilippo B and a project in Parkinson's disease, are no longer a priority and may be discontinued or licensed out, added Kapusta.

uniQure has seen its share price go on the slide in the last 12 months after abandoning plans to file for U.S. approval of Glybera (alipogene tiparvovec), a gene therapy for lipoprotein lipase deficiency that was launched onto the market in Europe in 2012.

That decision was followed by the departure of then CEO Joern Aldag—as well as replacement Daniel Soland after just nine months—along with an array of other senior executives. Adding to the pressure, the company has also faced questions about the competitive position of AMT-060 versus a rival gene therapy in development at Spark Therapeutics ($ONCE). 

The recent news of an unwanted immune response to Spark's SPK-9001 drug in a seven-patient trial could shift the balance once again, and all eyes will be on the two programs when updated results are presented at the American Society of Hematology (ASH) congress in December. Meanwhile, another potential rival fell by the wayside when Shire opted to discontinue its BAX 335 program in hemophilia B a few weeks ago.

"We are enthusiastic about the interim data from our ongoing Phase I/II study of AMT-060 in hemophilia B and will allocate necessary resources to expedite bringing AMT-060 to market," said Kapusta on a conference call with investors today, in which he also detailed plans to consolidate the firm's manufacturing  at its facility in Lexington, MA.

The company also announced that head of neurological Charles Richard and metabolic research chief Deya Corzo will leave the company by the end of the year, and that it now hopes savings could reach €11 million to €15 million over the next two years.

News of the cost clampdown and re-focused R&D priorities seemed to be well received by investors, with the stock edging up a couple of points in early trading this morning to $8.30. It's still languishing well below its year-long high of almost $23 however.