Pipeline
iQure is advancing a focused pipeline of small molecules that restore glutamate balance by modulating EAAT2, the brain’s main glutamate transporter. Building on the success of iQ-007, we are developing next-generation compounds tailored to disease-specific mechanisms in epilepsy, neurodegeneration, and chronic pain.
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Asset Disease Area Ph1 Discovery Lead identification Preclinical iQ-007 Epilepsy, AD, DS, PD iQ-032 Pain Undisclosed Pain Undisclosed ALS ASSET MOA INDICATION TYPE STAGE iQ-007 EAAT2 PAM positive allosteric modulator (PAM) Epilepsy
Alzheimer’s Disease and other neurodegenerative disorders
Small Molecule Phase 1
iQ-007 | First-in-class glutamate reuptake enhancer
iQ-007 breaks the excitotoxic cycle by restoring glutamate balance - and thereby breaks the seizure cycle in epilepsy.
It enhances glutamate uptake 2.5-fold, counteracting the toxic accumulation of glutamate that drives hyperexcitability, neuronal damage, and inflammation. While epilepsy is the lead clinical application, excitotoxicity is also implicated in other CNS disorders - including Alzheimer’s Disease, Parkinson’s Disease, ALS, and pain - where disruptions in glutamate regulation and astrocyte function have been observed.By enhancing EAAT2 activity, iQ-007 restores the brain’s natural capacity to clear excess glutamate - offering a disease-modifying approach not only in epilepsy, but also in neurodegenerative and neuroinflammatory conditions where excitotoxicity plays a central role.
iQ-007 received FDA Orphan Drug Designation in Dravet’s Syndrome
iQ-007 is participating in the NIH ETSP program
Scientific publications2024 Annals of Neurology
Enhancement of Glutamate Uptake as Novel Antiseizure Approach: Preclinical Proof of Concept
DOI: 10.1002/ana.27124
2024 Nature Reviews
New epilepsy therapiesin development
https://doi.org/10.1038/s41573-024-00981-w
2023 Journal of Medicinal Chemistry
Discovery of (R)‑N‑Benzyl-2-(2,5-dioxopyrrolidin-1-yl)propenamide [(R)-AS‑1], a Novel Orally Bioavailable EAAT2 Modulator with Druglike Properties and Potent Antiseizure Activity In Vivo
https://doi.org/10.1021/acs.jmedchem.2c00534 -
Targeting excitotoxicity across CNS diseases
EAAT2 dysfunction and astrocyte-mediated glutamate imbalance are increasingly recognized as drivers across major CNS indications. This growing body of evidence supports our rationale to expand beyond epilepsy into neurodegenerative and neuroinflammatory diseases where excitotoxicity remains unaddressed.
Read more about our science >>
PublicationsAlzheimer’s Disease
Reference: DOI 10.1021/acs.jmedchem.2c01572, 2023
Parkinson’s Disease
Reference: DOI 10.3389/fnagi.2022.952368, 2022
Neuroinflammation
Reference: DOI 10.3390/biom12040597, 2022
