uniQure said today it will eliminate 50 to 60 positions—20% to 25% of its workforce—by the end of 2017 under a restructuring the gene therapy developer said was intended to refocus its pipeline while consolidating its manufacturing and overhauling its R&D operations.

The company identified three R&D priorities going forward—its clinical programs to develop treatments for hemophilia B, Huntington’s disease, and cardiovascular disease, the subject of an up-to-$1 billion collaboration with Bristol-Myers Squib (BMS) launched last year.

The BMS collaboration will advance, uniQure said, with a specific focus on developing S100A1 in congestive heart failure. uniQure also said it will launch a pivotal trial in hemophilia B pending discussions with regulators, as well as file an IND application for Huntington’s disease following completion of ongoing preclinical studies.

“Further details on how we expect to position our hemophilia program will be provided after we have met with regulatory authorities early next year to discuss our plans for a pivotal study,” Philip Astley-Sparke, chairman of uniQure’s Board of Directors, said in a statement.

uniQure said it has begun talks with an unnamed potential partner to end licensing discussions for AMT-110 for the treatment of Sanfilippo B. The company said two of four patients in an ongoing Phase I/II trial for its Sanfilippo B treatment have completed their 30-month evaluations, with 30-month data on all four patients expected to be available in the first quarter of 2017.

The company added that it will pursue partnering opportunities for an academic-sponsored program in Parkinson’s disease, with the goals of accelerating completion of an ongoing study and further developing the program.

Another priority, uniQure said, will be developing new products, with an emphasis on liver-directed diseases. That will include internal development or licensing of new product candidates in rare and orphan diseases where the company aims to apply its next-generation vector and promoter platform and manufacturing capabilities.

uniQure said it would consolidate all GMP manufacturing at its Lexington, MA, facility, which has the capability of scaling up to 2000-L capacity—a capability the company said positions it for late-stage development and commercialization of its gene therapies.

The company is also eliminating its R&D structure based on therapeutic areas and is changing its R&D leadership. Christian Meyer, M.D., Ph.D., CMO, will assume full responsibility for the company’s hemophilia B gene therapy program, while Harald Petry, Ph.D., CSO, will continue to lead uniQure’s preclinical research, including IND-enabling studies in Huntington’s disease.

Two other executives will be leaving uniQure by year’s end: Deya Corzo, M.D., svp of the liver and metabolic therapeutic area, and Charles Richard, M.D., Ph.D., svp of the central nervous system therapeutic area.

uniQure added that it will restructure its R&D organization in The Netherlands while consolidating manufacturing in the U.S.

uniQure said its job cuts would generate €5 million to €6 million ($5.4 million to $6.4 million) in annual cost savings, while its pipeline refocusing would reduce expenses an additional €11 million to €15 million ($11.8 million to $16 million) over the next 2 years.

“We have simplified the structure of the Company to drive focus and provide for a strong foundation upon which to execute our key priorities,” Astley-Sparke stated.

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