NEW YORK – UniQure on Thursday said it was cutting 114 positions, or 20 percent of its total workforce, and discontinuing more than half of its research and technology projects as part of a strategic reorganization aimed at saving $180 million.
The changes are expected to extend UniQure's cash runway into Q2 2027. The Amsterdam-based company clarified that the workforce reduction will not impact its ability to manufacture the hemophilia gene therapy Hemgenix (etranacogene dezaparvovec-drbl) as stipulated under a global license and commercialization agreement with CSL Behring.
"We are taking important actions today to cut operating expenses while ensuring that we have the necessary resources to advance our prioritized clinical-stage programs as rapidly as possible to proof of concept," UniQure CEO Matt Kapusta said in a statement. "Following an extensive review, we plan to discontinue more than half our research and technology projects and focus our R&D efforts on programs that leverage our CNS and liver-targeted gene therapy expertise, have the potential for expedited clinical proof of concept, and have attractive risk-value profiles."
UniQure is discontinuing its Parkinson's disease gene therapy candidate AMT-210 and other undisclosed programs, but it is still prioritizing several clinical-stage gene therapies. AMT-260 and AMT-162 will enter human trials in the fourth quarter in refractory mesial temporal lobe epilepsy and SOD-1 ALS, respectively. The company plans to meet with the US Food and Drug Administration to discuss future development of its investigational Huntington's disease gene therapy, AMT-130, in Q1 2024, and present longer-term follow-up data from Phase I/II studies next year.
The firm will also submit an investigational new drug application for AMT-191 seeking permission to start human trials in Fabry disease in 2024. UniQure is also still committed to developing AMT-161 in c9orf72 amyotrophic lateral sclerosis, AMT-240 in autosomal-dominant Alzheimer's disease, and next-generation AAV capsids.
UniQure operates a research lab and manufacturing facility in Lexington, Massachusetts. The firm will close and sublease the research lab and consolidate all good manufacturing practices activities into the manufacturing facility and consolidate process and analytical development into its Amsterdam facility.
The firm will incur a one-time restructuring cost of $2.3 million largely in Q4 2023. As of June 30, the company had $628.6 million in cash, cash equivalents, and investment securities, excluding a $100 million milestone payment from CSL Behring.
As part of reprioritization, Ricardo Dolmetsch, UniQure's current CSO, will leave the company, and Rich Porter, the firm's chief business officer, will lead research as well as nonclinical and vector development efforts.