Ildong Pharmaceutical's Airid BMS Pulmonary Fibrosis New Drug Designated as Orphan Drug by US FDA

Superior Anti-Fibrotic Efficacy Demonstrated Compared to Existing Standard Treatments

iLeadBMS, a new drug research and development company of Ildong Pharmaceutical Group, announced on the 13th that its first-in-class innovative drug candidate 'IL21120033' has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for idiopathic pulmonary fibrosis (IPF).

FDA orphan drugs are designated for diseases or conditions affecting fewer than 200,000 people in the U.S., where it is expected that development costs and revenues will not be recovered within 7 years after FDA approval or during development. When designated as an orphan drug, benefits such as a 50% tax credit on research and development (R&D) expenses, clinical development grants, and approximately a 25% tax credit (ODTC) on clinical trial costs for the indication in the U.S. are provided. Additionally, a 7-year market exclusivity period is granted post-approval.

IL21120033 is a small molecule-based anti-fibrotic drug candidate that acts on CXCR7 (C-X-C chemokine receptor 7), a receptor of chemokines involved in immune-related signal transduction, which is closely related to tissue fibrosis and inflammation induction.

CXCR7 is a key mediator in signal transduction involved in inflammation. It selectively binds to the chemokine receptor ligand CXCL12 (C-X-C motif chemokine ligand 12) and regulates various signaling pathways related to tissue repair, angiogenesis, and fibrosis.

IL21120033 is a CXCR7 agonist drug. It has high binding selectivity to CXCR7 and exhibits anti-inflammatory and anti-fibrotic effects by removing the pro-inflammatory factor CXCL12 inside cells.

According to preclinical studies, IL21120033 showed high selectivity for CXCR7 without binding to other chemokine receptors and demonstrated ideal pharmacokinetic properties upon oral administration.

In particular, in a bleomycin-induced pulmonary fibrosis animal model, IL21120033 dose-dependently improved the 'Ashcroft score,' an indicator used to measure pulmonary fibrosis. It showed superior anti-fibrotic efficacy compared to existing standard treatments. The Ashcroft score is a standardized numerical scale used to quantify the degree of pulmonary fibrosis in histological samples.

Furthermore, in various animal experiments, side effects such as weight loss observed with existing treatments were either absent or minimal, suggesting that safety requirements are expected to be met, according to the company.

Yoon Seok Lee, Chief Scientific Officer (CSO) of iLeadBMS, stated, "Through our research, we have consistently confirmed the anti-fibrotic efficacy of IL21120033. With the FDA's orphan drug designation recognizing the value and potential of this new drug candidate, we plan to accelerate subsequent clinical development tasks such as Good Laboratory Practice (GLP) safety evaluations and Investigational New Drug (IND) application submissions."