Struggling UniQure Cuts Gene Therapies, Jobs, in Big Restructuring

Xconomy Boston — 

UniQure may have been the first company to win approval of a gene therapy, but that hasn’t translated to commercial success or a booming share price. Now it’s cutting jobs and restructuring to try to gain some momentum.

Amsterdam- and Lexington, MA-based UniQure (NASDAQ: QURE) said this morning that it’s completed a strategic review, and as a result it will cut some experimental gene therapy programs and focus its resources around others. Specifically, UniQure will rally around programs for hemophilia B, Huntington’s disease, and heart disease—the last of which being the focus of UniQure’s alliance with Bristol-Myers Squibb (NYSE: BMY). The company will try to find partners to advance experimental gene therapies it’s been developing for a form of Sanfilippo syndrome and Parkinson’s disease, meanwhile, and develop or acquire new treatments for rare diseases.

UniQure will also cut between 50 and 60 jobs—about 20 to 25 percent of its global workforce—by the end of 2017 to save about €5 to €6 million per year. It’s restructuring its R&D operations in the Netherlands, where it will maintain a smaller research team in a new Amsterdam facility, and focus all of its manufacturing in Lexington. Additionally, the company’s heads of neurological (Charles Richard) and metabolic research (Deya Corzo) will leave the company by the end of the year.

The restructuring moves could save UniQure another €11 to €15 million over the next two years, which should give the company enough cash to get to 2019.

“We are confident that these efforts will significantly streamline operations and position us well for the future,” said interim CEO Matthew Kapusta.

UniQure made history in 2012 when it became the first company to win approval of a gene therapy—a type of treatment designed to provide a long-lasting, if not permanent fix for a genetic disease. European regulators approved UniQure’s Glybera for a rare metabolic disease called lipoprotein lipase deficiency, an important milestone in gene therapy’s recent renaissance. But the product—which hasn’t been approved in the U.S.—has struggled commercially, and UniQure has changed CEOs twice since 2012 and rallied around other products. In the meantime, shares have suffered. The company went public in 2014 at $17 per share, and now trades at $8.30.

Additionally, UniQure’s most advanced prospect, for the blood disease hemophilia B, is part of an ultra-competitive race between several companies. Like UniQure, Spark Therapeutics (NASDAQ: ONCE) has a hemophilia B gene therapy in early stage testing. Others, such as BioMarin Pharmaceutical (NASDAQ: BMRN), are looming with experimental treatments of their own. Kapusta said in a statement that UniQure plans to start a pivotal trial of its hemophilia B treatment pending talks with regulators.

Here’s more on UniQure, Glybera, and gene therapy.