RARE Daily

UniQure Cuts Staff in Reorganization to Reduce Operating Expenses and Support Clinical-Stage Programs

October 5, 2023

Rare Daily Staff

Gene therapy biotech UniQure said it has begun a reorganization, including cutting 20 percent of its workforce, to significantly reduce operating expenses while supporting focused execution to rapidly advance multiple clinical-stage programs to proof-of-concept.

“To accomplish our mission and generate near-term value for our stakeholders, we will implement a strategic restructuring of our business,” said Matt Kapusta, CEO of UniQure. “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.”

Following an extensive review, the company plans to discontinue more than half of its research and technology projects and focus R&D efforts on programs that leverage its CNS and liver-targeted gene therapy expertise, have the potential for expedited clinical proof of concept, and have attractive risk-value profiles.

Projects to be discontinued include AMT-210 for the treatment of Parkinson’s disease and multiple undisclosed programs. The company will focus its research efforts on a limited number of projects believed to have optimal risk, value, and speed attributes, including AMT-161 for c9orf72 amyotrophic lateral sclerosis, AMT-240 for autosomal dominant Alzheimer’s disease, and next-generation AAV capsid development. As a result of the reprioritization, the company will be closing a research lab in Lexington, Massachusetts with plans to sublease this space, and will consolidate all GMP manufacturing into its Lexington, Massachusetts manufacturing facility and consolidate process and analytical development into its Amsterdam, Netherlands facility. Commercial manufacturing of Hemgenix for CSL Behring will be unaffected by these actions.

As a result of the restructuring plan, UniQure will eliminate 114 positions, representing 28 percent of the workforce not committed to Hemgenix manufacturing obligations and approximately 20 percent of the total workforce. The company expects total cost savings of approximately $180 million over the next three years, which will extend its cash runway into the second quarter of 2027.

UniQure will prioritize four clinical programs: AMT-130 in Huntington’s disease, which is in a phase 1/2 study; near-term initiation of clinical trials for AMT-260 in refractory mesial temporal lobe epilepsy; AMT-162 in SOD1-ALS for which the company plans to initiate patient screening in  the 4th quarter; and AMT-191 in Fabry disease for which the company expects to submit an application to begin human clinical trials before the end of the year and begin patient dosing in 2024.

As a result of reprioritization, Chief Scientific Officer Ricardo Dolmetsch will leave UniQure and Chief Business Officer Rich Porter will assume responsibilities for research as well as nonclinical and vector development.

Photo: Matt Kapusta, CEO of UniQure

Stay Connected

Sign up for updates straight to your inbox.