Recent Updates
Recently added Catalysts

AAV9-hSyn1-miGRIK2

Phase 1

Mesial Temporal Lobe Epilepsy | Gene therapy | Neurology |uniQure N.V.|Last Updated: Jun 2, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06063850AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe EpilepsyPHASE1 RECRUITING 12Jun 12, 2024Dec 1, 2031Jun 2, 202618 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.
1 year

Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.

Secondary Endpoints
To evaluate first signs of efficacy of AMT-260.
1 year
To evaluate the biodistribution properties of AMT-260.
1 year
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AMT-260EXPERIMENTALCohort 1: AMT-260 starting dose (1.0x 10E12 gc/mL). Cohort 2: AMT-260 adapted dose (6.0x 10E11 gc/mL or 3.0x 10E12 gc/mL).
Interventions
NameTypeDescription
AAV9-hSyn1-miGRIK2GENETICAMT-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. Intervention will be a one-time intracerebral administration of AMT-260.
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites18

Inclusion Criteria: * Diagnosis of unilateral refractory MTLE * History of seizures with an average of ≥ 2 documented focal impaired awareness seizures or focal to bilateral tonic-clonic seizures per 30-day period over the 3 month Retrospective Period. * On a stable type and dose regimen of up to a...

Countries:United States
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWJun 2, 2026NCT06063850lastUpdatePostDate: changed
LOWJun 2, 2026NCT06063850lastUpdatePostDate: changed
LOWJun 2, 2026NCT06063850lastUpdatePostDate: changed
LOWMay 26, 2026NCT06063850primaryCompletionDate: changed
LOWMay 24, 2026NCT06063850studyFirstPostDate: changed