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iQ-007 Considered as First-in-Class Therapeutic Agent in Prestigious Journal of Medicinal Chemistry

International team of scientists share research that demonstrates the effectiveness of iQ-007 and its unique mechanism of action

Princeton, NJ, September 8, 2022 ─ iQure Pharma Inc. (iQure), a Princeton-based global biotech company, has announced that research into one of its lead drug candidates, iQ-007 ((R)-AS-1), has been published in the Journal of Medicinal Chemistry, a peer-reviewed journal, published by the American Chemical Society. The research, Discovery of (R)-N-Benzyl-2-(2,5-dioxopyrrolidin-1-yl)propanamide [(R)-AS-1], a Novel Orally Bioavailable EAAT2 Modulator with Drug-like Properties and Potent Antiseizure Activity In Vivo discusses iQ-007’s unique mechanism of action and suggests iQ-007 as a First-in-Class EAAT2 Positive Allosteric Modulator (PAM).

The research manuscript is co-authored by an international team of researchers, including Andréia C. K. Fontana, (Drexel University, Philadelphia, US), Christa E. Müller (Bonn University, DE), Ivet Bahar (University of Pittsburgh, US), R. Kaminski and K. Kaminski (Jagiellonian University, PL).

“This publication is a milestone event for iQ-007, confirming its unique mechanism of action and its potential as a new therapeutic,” said iQure R&D Director, Henk de Wilde. “Our internal efficacy data in neuropathic pain and the epilepsy data discussed in this paper, confirm iQ-007’s potential as a First-in-Class therapeutic candidate for more than one indication. We are now progressing with development of iQ-007 towards IND and subsequent clinical testing.”

“The iQure team would like to thank all researchers for their respective contributions to the publication,” added Henk de Wilde. “This international effort, further supported by iQ-007 participation in the NIH ETSP program, helps to translate this exciting discovery into a new therapy, helping patients in urgent need of novel, effective and safe, pain and epilepsy treatments.”

About Chronic Pain

Chronic pain is one of the most common chronic conditions in the United States. In 2019, 20.4% of adults experienced chronic pain and 7.4% had high-impact chronic pain that severely limited daily life or work activities. An estimated twenty million Americans experience some form of peripheral neuropathy, one of the most severe forms of chronic pain.

About Epilepsy

Epilepsy affects nearly 50 million people worldwide, making it one of the most common neurological diseases globally, according to WHO. Progress in epilepsy research has led to the approval and marketing authorization of more than 30 antiseizure drugs (ASDs) over recent decades. Yet, despite this unquestionable therapeutic success, approximately one-third of epilepsy patients still experience uncontrolled and debilitating seizures. Epilepsy, and especially drug-resistant epilepsy, remains an unmet clinical need affecting as many as 20%-40% of the epileptic population, corresponding to more than 400,000 patients in the US alone.

About iQure Pharma

A global biotech firm headquartered in Princeton, New Jersey, iQure Pharma 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. For more information about supporting iQure initiatives, contact iQure CFO Dr. Anna Rzewuska at


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