- Gene therapy developer UniQure is laying off 114 employees and halting more than half of its research and technology projects, the company said Thursday.
- The Dutch biotechnology company said the cost-cutting measures would save the company $180 million over the next three years, extending its cash runway into 2027.
- UniQure’s stock price rose by as much as 14% on news of restructuring, but remains about two-thirds lower than where it traded earlier this summer before the company revealed mixed results from an early trial of its experimental Huntington’s disease treatment AMT-130.
UniQure hit a high with Hemgenix, which last year became the first gene therapy approved in the U.S. for hemophilia B. The company licensed the drug to Australian drugmaker CSL Behring in 2020 for $450 million, providing the former the cash to continue developing AMT-130, among other programs.
As it seeks the same success it had with Hemgenix, the company expects to start clinical trials for several other gene therapies over the next year, including two next quarter.
“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,” Matt Kapusta, UniQure’s CEO, said in a statement.
AMT-130, the company’s lead therapy, is designed to silence the malfunctioning gene responsible for the “huntingtin” protein, which in its mutated form builds up and damages nerve cells.
In UniQure’s Phase 1/2 trial, mutant huntingtin levels in trial participants declined by half in results collected one year after treatment. But data released in June showed those levels had rebounded by the two-year mark.
More data from the AMT-130 study is expected in the fourth quarter. UniQure said it plans to meet with federal regulators next year to discuss data from U.S. and European studies of the treatment.
The cuts announced Thursday include the closure of a lab in Lexington, Massachusetts, and discontinuation of an Parkinson’s drug candidate, among other unnamed programs. UniQure will still develop drugs for forms of epilepsy, ALS and Fabry disease.
Among the employees departing UniQure is chief scientific officer, Ricardo Dolmetsch, a former Novartis executive who joined the company in 2020. He will stay on as a consultant through the end of the year.