iQure Pharma Inc.
iQure Pharma is a clinical-stage biotech company focused on developing therapies for CNS disorders by restoring glutamate homeostasis. Their approach targets astrocytic functions, specifically enhancing the glutamate transporter EAAT2, to break the cycle of excitotoxicity that drives neuronal damage across various CNS conditions. Their lead asset, iQ-007, is a first-in-class, orally available small molecule in Phase 1 clinical development for treatment-resistant epilepsy, with potential applications in neurodegeneration and pain. The company collaborates with academic and industry partners, and is supported by investors including Ventura BioMed, OKG Capital, and others.
Industries
Nr. of Employees
small (1-50)
iQure Pharma Inc.
475 Wall Street, Princeton, NJ 08540, USA
Products
Clinical-stage EAAT2 positive allosteric modulator (lead compound, Phase 1)
Orally bioavailable small-molecule positive allosteric modulator of EAAT2 designed to enhance glutamate uptake (reported ~2.5-fold increase in uptake in preclinical assays); advanced into Phase 1 first-in-human studies for epilepsy and being evaluated across other CNS indications.
Preclinical EAAT2-targeted small-molecule series for pain and neurodegeneration
Next-generation small-molecule programs targeting EAAT2 tailored to disease-specific mechanisms in pain, ALS, and neurodegeneration (preclinical stages: discovery, lead identification, preclinical testing).
Clinical-stage EAAT2 positive allosteric modulator (lead compound, Phase 1)
Orally bioavailable small-molecule positive allosteric modulator of EAAT2 designed to enhance glutamate uptake (reported ~2.5-fold increase in uptake in preclinical assays); advanced into Phase 1 first-in-human studies for epilepsy and being evaluated across other CNS indications.
Preclinical EAAT2-targeted small-molecule series for pain and neurodegeneration
Next-generation small-molecule programs targeting EAAT2 tailored to disease-specific mechanisms in pain, ALS, and neurodegeneration (preclinical stages: discovery, lead identification, preclinical testing).
Services
Collaborative preclinical and translational research partnerships with academic and industry groups to advance EAAT2-modulating small molecules.
Collaborative preclinical and translational research partnerships with academic and industry groups to advance EAAT2-modulating small molecules.
Expertise Areas
- CNS drug discovery
- Astrocyte-targeted therapeutics (glutamate homeostasis)
- Preclinical pharmacology and translational models
- Small-molecule medicinal chemistry
Key Technologies
- Positive allosteric modulation of membrane transporters (EAAT2)
- Small-molecule medicinal chemistry
- Oral drug formulation
- In vivo epilepsy models
News & Updates
iQure Pharma Appoints Former UCB Chief Medical Officer and Head of Development, Prof. Iris Loew-Friedrich, MD, as Clinical and R&D Advisor
July 2, 2025: iQure Pharma appoints Prof. Iris Loew-Friedrich as Clinical and R&D Advisor.
iQure Pharma Receives Approval for iQ-007 Phase 1 First in Human Study
March 12, 2025: Approval for Phase 1 study of iQ-007.
iQure Pharma’s iQ-007 Receives FDA Orphan Drug Designation for Dravet’s Syndrome
January 23, 2025: FDA orphan drug designation for iQ-007.
iQure Pharma Secures $4M Funding – Advancing Phase 1 Clinical Trial for iQ-007
January 7, 2025: Secured funding to advance clinical trials.
iQ-007 Preclinical Stage Complete — Phase 1 Preparations Targeted for Q1 2025
October 8, 2024: Preclinical stage completed, Phase 1 preparations underway.
iQ-007 Zebrafish Screening Reveals Positive Results in Other Neurodegenerative Diseases
June 20, 2024: Positive screening results.
iQure Pharma Appoints Former UCB Chief Medical Officer and Head of Development, Prof. Iris Loew-Friedrich, MD, as Clinical and R&D Advisor
July 2, 2025: iQure Pharma appoints Prof. Iris Loew-Friedrich as Clinical and R&D Advisor.
iQure Pharma Receives Approval for iQ-007 Phase 1 First in Human Study
March 12, 2025: Approval for Phase 1 study of iQ-007.
iQure Pharma’s iQ-007 Receives FDA Orphan Drug Designation for Dravet’s Syndrome
January 23, 2025: FDA orphan drug designation for iQ-007.
iQure Pharma Secures $4M Funding – Advancing Phase 1 Clinical Trial for iQ-007
January 7, 2025: Secured funding to advance clinical trials.
iQ-007 Preclinical Stage Complete — Phase 1 Preparations Targeted for Q1 2025
October 8, 2024: Preclinical stage completed, Phase 1 preparations underway.
iQ-007 Zebrafish Screening Reveals Positive Results in Other Neurodegenerative Diseases
June 20, 2024: Positive screening results.