NEW YORK – UniQure on Tuesday said it has received permission from the US Food and Drug Administration to begin studying the gene therapy AMT-260 in patients with refractory mesial temporal lobe epilepsy (MTLE).
Having received FDA clearance of its investigational new drug application for AMT-260, Amsterdam-based UniQure said it expects to begin screening MTLE patients for a first-in-human Phase I/IIa trial in the fourth quarter.
AMT-260 is a one-time gene therapy consisting of an adeno-associated virus serotype 9 vector that delivers two engineered microRNAs. The miRNAs are designed to degrade the GRIK2 gene and suppress its aberrant expression of GLUK2, the glutamate receptor subtype believed to trigger seizures in patients with the chronic neurologic disorder.
The trial that UniQure is readying to conduct in the US will have two parts: a multicenter, open-label portion that will involve a dozen patients across two dosing cohorts and a randomized-controlled trial portion that provides proof of concept of the gene therapy's activity.
"The clearance of the IND for AMT-260 is an important achievement in advancing our pipeline and is our next program to enter clinical development in an area of high unmet medical need," Walid Abi-Saab, UniQure's chief medical officer, said in a statement. UniQure previously led development of Hemgenix (etranacogene dezaparvovec), a gene therapy for hemophilia B, which received FDA approval in 2022 and is being commercialized by CSL Behring.