uniQure cuts staff and R&D projects to extend gene therapy cash runway

uniQure cuts staff and R&D projects to extend gene therapy cash runway

uniQure, a biotech company focused on gene therapy, is implementing a significant workforce reduction as part of a strategic restructuring effort aimed at ensuring the continuation of its gene therapy trials until 2027. This restructuring will result in the elimination of 20% of the company’s workforce.

uniQure plans to discontinue multiple undisclosed programs, including the potential Parkinson’s disease therapy AMT-210, which aimed to reduce misfolded alpha-synuclein protein in the brain but did not advance to clinical trials. The restructuring will not affect staff involved in manufacturing the hemophilia B drug Hemgenix, developed in partnership with CSL Behring.

As part of the restructuring, UniQure will close a research lab in Lexington, Massachusetts, while consolidating its Good Manufacturing Practice (GMP) manufacturing into a facility in the same area. The company will shift its focus to process and analytical development at its Amsterdam facility in the Netherlands.

Also Read: UniQure Craters On The Gene Therapy Data From Huntington Disease

“At uniQure, our highest priority is to deliver innovative, life-changing therapies to patients with significant unmet needs. To accomplish our mission and generate near-term value for our stakeholders, we will implement a strategic restructuring of our business. 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, 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. We remain fully committed to carefully managing costs, prudently allocating capital, rigorously assessing our clinical development priorities as new data emerges, and thoughtfully evaluating strategies that can enhance value for shareholders.”

– Matt Kapusta, chief executive officer of uniQure

uniQure will prioritize gene therapy programs that are already in clinical trials or in the pipeline. The lead candidate, AMT-130, is undergoing phase 1/2 studies in both the U.S. and Europe for Huntington’s disease, although it experienced a temporary dosing pause due to safety signals. Early-stage trials for AMT-260 in refractory mesial temporal lobe epilepsy and AMT-162 in amyotrophic lateral sclerosis are set to begin in early 2024. Additionally, UniQure plans to initiate a trial for AMT-191 in Fabry disease, pending FDA permission later this year.

The restructuring is expected to save approximately $180 million over the next three years, providing funding for operations through the second quarter of 2027. As of the end of June, UniQure had $628.6 million in cash, with an additional $100 million received from CSL Behring as a milestone payment related to their approved gene therapy for hemophilia B, Hemgenix, which is the most expensive drug in the US with a price tag of $3.5 million per dose.

Also Read: Despite CSL’s Hemophilia B Gene Therapy, NICE Is Not Persuaded

“These were difficult but necessary decisions. “I’m truly grateful for the commitment and contributions of our many colleagues, and we remain fully committed to delivering on our promise to patients in need. We also look forward to sharing longer-term, interim data on our Huntington’s disease program in late fourth quarter of this year and initiating clinical trials for our other programs as rapidly as possible.”

– Matt Kapusta, chief executive officer of uniQure

To oversee the changes, Richard Porter, Ph.D., who joined UniQure through its acquisition of Corlieve Therapeutics in 2021, will take on the combined role of Chief Business and Scientific Officer, while Ricardo Dolmetsch, Ph.D., will remain as a scientific consultant until the end of the year.

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