The first-in-human Phase I/IIa clinical trial will be conducted in the United States and consist of two parts. The first part is a multicenter, open-label trial with two dosing cohorts of six patients each to assess safety, tolerability, and first signs for efficacy of AMT-260 in patients with refractory MTLE. The second part is expected to be a randomized, controlled trial to generate proof of concept (POC) data. Patient enrollment in the trial is expected to begin in third quarter of 2024.
Temporal Lobe Epilepsy (TLE)
The clinical trial of uniQure’s gene therapy candidate for the treatment of mesial temporal lobe epilepsy (MTLE) is expected to initiate in 2024.
Our gene therapy candidate AMT-260 is an AAV9 gene therapy product that locally delivers miRNA silencing technology to target the GRIK2 gene and suppress aberrantly expressed GluK2 containing kainate receptors. The therapeutic goal is to lower the expression of GluK2 containing kainate receptors, which are believed to trigger epilepsy when aberrantly expressed in the epileptic hippocampus. AMT-260 represents a novel potential one-time administered approach to treating refractory MTLE.
Temporal lobe epilepsy is a chronic neurologic disorder and is the most common form of focal epilepsy, with more than 600,000 individuals suffering from the disorder in the United States. Approximately 80% of all temporal lobe epilepsy cases are mesial, which involves the medial or internal structures. The majority of MTLE cases are refractory to anti-seizure medications, which severely limits treatment options.
In September 2023, we announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for AMT-260. There are few treatment options for patients who have refectory MTLE, and we are pleased to soon begin the clinical investigation of this one-time administered gene therapy approach as a potential new treatment.