iQure Pharma's iQ-007 Shows Promise in Preclinical Development for Neurological Disorders

Key Insights

• iQure Pharma's iQ-007, a first-in-class small molecule, has completed preclinical development for drug-resistant epilepsy, offering a novel therapeutic approach.
• The molecule functions as a positive allosteric modulator of the EAAT2 glutamate transporter, crucial for managing excess glutamate levels and preventing excitotoxicity.
• Researchers suggest iQ-007 could have broader applications for treating neurological, neurodegenerative, and psychiatric conditions, including Alzheimer's and Parkinson's.

A novel molecule, iQ-007, developed by Polish researchers and commercialized by iQure Pharma, has shown promising preclinical results for treating drug-resistant epilepsy and potentially other neurological disorders. Clinical trials are slated to begin next year, marking a significant milestone in its development.

Novel Mechanism of Action

iQ-007 is a first-in-class, small molecule positive allosteric modulator of the EAAT2 glutamate transporter. EAAT2, primarily expressed in the central nervous system, plays a critical role in removing excess glutamate from synaptic spaces, thereby mitigating the risk of neurotoxicity. "This molecule represents a novel therapeutic approach," said Kamiński, a researcher involved in the project. "By modulating EAAT2, it helps manage excess glutamate levels, which is crucial in preventing excitotoxicity, a process linked to many neurological disorders."

Broad Therapeutic Potential

Beyond epilepsy, researchers believe iQ-007 holds therapeutic potential for a range of neurological, neurodegenerative, and psychiatric conditions where glutamate regulation is key. These include Alzheimer's disease, Parkinson's disease, Huntington's disease, multiple sclerosis, amyotrophic lateral sclerosis, ischemic stroke, pain, schizophrenia, depression, anxiety, and even addiction. The ability to modulate glutamate levels could offer a new avenue for addressing these complex and often debilitating conditions.

Collaborative Development and Commercialization

The development of iQ-007 has been a collaborative effort involving research groups from Poland and several international institutions, including the Medical University of Warsaw, the Institute of Pharmacology PAN, Harvard Medical School, the University of Oslo, and Drexel University College of Medicine. In 2020, the molecule was commercialized through the Jagiellonian University's Technology Transfer Center, CITTRU, and licensed to iQure Pharma, a U.S.-based biotech company. iQure Pharma has partnered with Jagiellonian researchers to complete the preclinical phase and advance the molecule towards clinical trials.