uniQure offloads 5th of staff, half of R&D projects to keep gene therapies in clinic into 2027

The “strategic restructuring” will see “multiple undisclosed programs” shut down, along with potential Parkinson’s disease therapy AMT-210. The one-time AAV gene therapy was designed to reduce the amount of misfolded alpha-synuclein protein in the brain, but the drug never made it into the clinic and has been notably absent from the company’s recent earnings reports.

A total of 114 positions—accounting for 20% of the company’s total workforce—will be laid off, although the biotech was keen to emphasize that this won’t impact staff involved in the manufacturing of the hemophilia B dug Hemgenix for its partner CSL Behring.

Instead, uniQure will close a research lab in its U.S. hometown of Lexington, Mass., as well as consolidate its GMP manufacturing into a facility in the same area while focusing on process and analytical development at its Amsterdam facility in the Netherlands.

The biotech is now focusing on gene therapy programs that are already in the clinic or are en route. Chief among them is AMT-130, which is in phase 1/2 studies in both the U.S. and Europe for Huntington’s disease. The therapy hasn’t had a smooth ride so far, with safety signals prompting a pause in dosing for two groups in the European trial, while investors appeared to take fright at an interim readout from the U.S. study in June despite the company itself lauding the results as showing that AMT-130 was generally well tolerated.

Early-stage trials of two other assets are due to start dosing patients in early 2024—namely AMT-260 in refractory mesial temporal lobe epilepsy, and AMT-162 in amyotrophic lateral sclerosis. The company will also persevere with AMT-191, for which it plans to secure FDA permission later this year to start a trial in Fabry disease.

“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,” CEO Matt Kapusta explained in the Oct. 5 release.

“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,” Kapusta added.

The “significant reduction in research activities” means that Chief Scientific Officer Ricardo Dolmetsch, Ph.D., is now surplus, although he will be retained as a scientific consultant until the end of the year. In his place will step Richard Porter, Ph.D., who will take on the combined role of chief business and scientific officer. Porter joined uniQure through its acquisition in 2021 of Corlieve Therapeutics, where he was CEO.

The combined changes are expected to save around $180 million over the next three years, funding operations to the second quarter of 2027. At the end of June, uniQure had $628.6 million in the bank, with an extra $100 million since handed over by CSL Behring as a milestone payment.

CSL Behring and uniQure secured approval of Hemgenix in November. The treatment is not only the first gene therapy approved for hemophilia B but also the most expensive drug in the U.S., sporting an eye-popping price tag of $3.5 million per dose.

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