Pipeline

iQ-007 is a first-in-class, orally available small molecule designed to enhance physiological glutamate clearance through positive allosteric modulation of EAAT2.

EAAT2 is primarily expressed by astrocytes and is responsible for the majority of synaptic glutamate clearance in the brain. By enhancing EAAT2 function, iQ-007 aims to restore glutamate balance and reduce excitotoxic stress without blocking glutamate receptors.

iQ-007 has demonstrated robust preclinical efficacy across multiple seizure and epilepsy models, including drug-resistant paradigms, and is supported by peer-reviewed publications in Journal of Medicinal Chemistry and Annals of Neurology. The compound has also completed evaluation through the NIH Epilepsy Therapy Screening Program.

In parallel with advancing iQ-007 in epilepsy as the first clinical validation path, iQure is continuing to build its Parkinson’s disease expansion strategy, based on EAAT2’s role in glutamate homeostasis and preclinical work exploring glutamate-driven circuit dysfunction.

EAAT2 dysfunction and astrocyte-mediated glutamate imbalance are increasingly recognized across CNS disorders where neuronal overstimulation and excitotoxic stress contribute to disease biology. 

This growing body of evidence supports iQure’s rationale to expand beyond epilepsy into selected CNS indications where impaired glutamate clearance remains therapeutically underexplored, with Parkinson’s disease as the first neurodegenerative focus and ALS and migraine as additional mechanism-driven opportunities.

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