Phase I/IIa Clinical Trial of AMT-260

CT-AMT-260–01 Study: A Multi-center, Phase 1/2a, First-in-human (FIH) Study Investigating the Safety, Tolerability, and Efficacy of AMT-260 in Adults with Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE) Administered via Magnetic Resonance Imaging (MRI)-guided Convection-enhanced Delivery (CED)

The GenTLE Phase I/IIa study is designed to investigate the safety, tolerability and efficacy of AMT-260 in adults with unilateral mesial temporal lobe epilepsy (MTLE). The study name, GenTLE, is short for Gene Therapy for Temporal Lobe Epilepsy. Investigational study sites are currently screening adult patients in the United States for participation.

You may have heard about ways that scientists are trying to create gene therapies to treat various diseases. AMT-260 is an experimental (not approved by any health authority) gene therapy candidate that is being investigated to determine its safety and tolerability profile and its potential to reduce seizure frequency in people with MTLE. AMT-260 is intended to be administered once in a person’s lifetime, although this is not certain at this time. AMT-260 has undergone preclinical laboratory testing, including studies in animals, and has now received an authorization from the U.S. Food and Drug Administration (FDA) to be tested in people.

AMT-260 is an investigational gene therapy candidate that is composed of a viral capsid (AAV9) that carries a genetic code. This code carries information (called micro-RNA) that is believed to be able to reduce the production of a particular receptor responsible for generating unwanted electric currents in the brain and causing seizures. The study will investigate if, when AMT-260 is delivered into a part of the brain called the hippocampus, the micro-RNA can reduce seizure generation in the treated area. The therapeutic goal is to sustainably lower the expression of a particular receptor type, which is believed to cause these seizures.

We hope to learn more about whether a single administration of AMT-260 is safe and well tolerated, and whether it may lead to a reduction in the frequency of seizures.

Participation in this study does not prevent you from pursuing other treatment options. such as anti-seizure medications, or from considering resection surgery in the future, each in consultation with your treating physician.

You can discuss the impact of participating in research studies on your future treatment options with your doctor or a study investigator.

There is no cost associated with the administration of the study drug or any procedures that are required to be performed as a part of the study. You may still be responsible for routine care costs, such as doctor visits, treatments, or tests that are not part of the study. In addition, uniQure provides a service that will help coordinate and either pay for, or reimburse, participants and caregivers for qualified for travel, lodging and other associated expenses for attending the study visits, up to certain established monetary limits.