Ildong Pharm Group's pulmonary fibrosis drug candidate wins US orphan drug designation

IL21120033, a new drug candidate being developed by iLeadBMS, a drug development arm of the Ildong Pharmaceutical Group, received an Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for treating idiopathic pulmonary fibrosis (IPF).

IL21120033 is a small molecule-based antifibrotic therapy that acts on the chemokine receptor CXCR7. CXCR7 is a protein that plays a key role in the induction of fibrosis and inflammation in living tissues and binds to the chemokine ligand CXCL12 to regulate tissue repair, angiogenesis, and fibrosis.

The new drug is highly selective as a CXCR7 agonist and exerts anti-inflammatory and antifibrotic effects by removing the pro-inflammatory factor CXCL12. In preclinical studies, it was confirmed to selectively act on CXCR7 without cross-reacting with other chemokine receptors and showed excellent pharmacokinetic properties when administered orally.

In an animal model of pulmonary fibrosis, IL21120033 produced a dose-dependent improvement in the Ashcroft score, a measure of fibrosis severity, and was superior to existing therapies. In addition, IL21120033 showed little weight loss, a major side effect of existing therapies, in several animal experiments, so it is expected to be safe.

“Preclinical studies have consistently confirmed the anti-fibrosis efficacy of IL21120033,” iLeadBMS’ Chief Scientific Officer Lee Yoon-seok said. “As the FDA’s orphan drug designation recognizes the potential for new drug development, we will accelerate further development, including safety evaluation and clinical trial plan approval.”