The iQ-007 molecule, developed at the Faculty of Pharmacy of the Jagiellonian University Medical College, is entering a crucial phase of clinical trials! American biotechnology company iQure Pharma Inc. has received approval from the Australian Human Research Ethics Committee (HREC) to conduct the first phase of clinical trials involving healthy volunteers.

The trials, which will begin in April 2025, aim to assess the safety of iQ-007 in adults. This is another step in the development of this promising molecule, which may find future applications in the treatment of drug-resistant epilepsy and neurodegenerative diseases.

Prof. Krzysztof Kamiński’s team from the Department of Pharmaceutical Chemistry at the Faculty of Pharmacy JU CM has been working on the development of iQ-007 for many years. In 2020, the molecule was commercialized by the CITTRU Technology Transfer Center and licensed to iQure Pharma Inc. In October 2024, iQ-007 successfully completed the preclinical phase of research, and in January 2025, the U.S. Food and Drug Administration (FDA) granted it orphan drug status for the treatment of Dravet syndrome.

“Approval for a ‘first-in-human’ trial is a crucial stage in the development of any drug,” emphasizes Prof. Kamiński. “This confirms the favorable safety profile of iQ-007, obtained in preclinical studies. The first phase of clinical trials will allow us to assess safety in humans and determine the pharmacokinetic profile, which is essential for planning further stages of research.”

The study will be randomized, double-blind, and placebo-controlled. The results are expected in the fourth quarter of 2025.