US-based early-stage pharmaceutical company EydisBio Inc announced on Wednesday that it has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for EYD-001, the company's highly selective, orally bioavailable TAK1 inhibitor intended for the treatment of systemic sclerosis.

The company's preclinical data published in 2023, in partnership with Dr John Varga, MD and his ScleroLab research group at the University of Michigan, indicated that EYD-001 (formerly called HS-276) reduced both dermal thickening and p-TAK1 expression in the lungs of a bleomycin-induced mouse model of systemic sclerosis. Treatment with EYD-001 in patient-derived skin fibroblasts significantly decreased mRNA expression of fibroinflammatory genes and blocked TGF beta-mediated increases in fibrotic protein expression.

Dr Tim Haystead, EydisBio founder and president, said: "We are excited to receive this designation from the FDA, which underscores the potential of our TAK1 inhibitor to make a meaningful impact on the lives of patients suffering from systemic sclerosis. This recognition highlights the innovative nature of our research and the dedication of our team to advancing treatments for rare diseases. It also strengthens our commitment to bringing EYD-001 to systemic sclerosis patients as swiftly and safely as possible."