iQure Pharma Receives Approval for iQ-007 Phase 1 First in Human Study

Princeton, NJ, March 12, 2025 ─ iQure Pharma Inc. (iQure), a biotechnology company focused on the development of novel therapies for neurological conditions, has received IRB (Institutional Review Board) approval for the first entry-into-human Phase 1 study with its lead asset iQ-007 in Healthy Volunteers. iQ-007, iQure’s flagship compound, is a novel therapeutic candidate under development to treat epilepsy and other neurodegenerative conditions.

iQ-007 represents a first-in-class EAAT2 Positive Allosteric Modulator (PAM), designed to clear toxic levels of glutamate from the synaptic cleft. Excess glutamate is a key driver of neurotoxicity and neurodegeneration in conditions such as epilepsy, Alzheimer’s Disease and Parkinson’s Disease or Pain.

In March 2025, iQure secured formal approval from the Human Research Ethics Committee (HREC) in Australia, for the iQ-007 Phase 1 study, IQ-007-CL-001. The study is a randomized, double-blind, placebo-controlled, single and multiple ascending dose study to assess the safety, tolerability and pharmacokinetics of oral iQ-007 in healthy male and female adult participants. Anticipated start for the study is targeted for April, with results expected in Q4 2025.

Pawel Zolnierczyk, iQure Pharma CEO: “We are delighted to secure approval to start clinical development of our lead asset, iQ-007. iQure Pharma is proud to be the first to enter clinical development with a new potential medication with a mechanism of action focused on the uptake of glutamate via EAAT2 PAM. Preclinical efficacy data indicate a highly promising outlook for iQ-007 in neurodegeneration including epilepsy, Alzheimer’s Disease, Parkinson’s Disease, ALS and pain. Data from Phase 1 will help to assess iQ-007’s safety and pharmacokinetic profile, which will allow us to reach the next milestone and optimize further development toward efficacy studies with iQ-007.”

Henk de Wilde, Chief Development Officer, added, “This significant milestone marks the first entry of an EAAT2 modulator into the clinical phase and will give us the opportunity to explore the extraordinary mechanism of action of iQ-007 in humans.”

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About iQure Pharma

iQure Pharma, a global biotech firm headquartered in the US, is focused on the development of new therapeutics for CNS, including neuropathic pain, epilepsy and other neurodegenerative diseases. iQure researchers, clinicians, and pharmaceutical professionals work closely with academic partners, industry experts, and patient organizations to build medically and socially beneficial therapies.