EydisBio Receives FDA Orphan Drug Designation for TAK1 Inhibitor for the Treatment of Systemic Sclerosis

DURHAM, N.C., Sept. 25, 2024 /PRNewswire/ — EydisBio, Inc. is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to EYD-001, its highly selective and potent, orally bioavailable TAK1 inhibitor for the treatment of systemic sclerosis.

EydisBio is an early-stage pharmaceutical company leveraging a novel approach to treating various autoimmune and inflammatory diseases, including rare diseases.

Systemic sclerosis is a rare and debilitating autoimmune disease characterized by fibrosis of the skin and internal organs, leading to significant morbidity and mortality. Central to the disease’s pathogenesis is the transforming growth factor-β (TGF-β) pathway, with TAK1 playing a pivotal role. Activation of this pathway triggers inflammation and fibrosis, the hallmark features of systemic sclerosis, and novel inhibition of this pathway via TAK1 represents a promising approach to disease treatment.

EydisBio’s preclinical data published last year, in collaboration with Dr. John Varga, MD and his ScleroLab research group at the University of Michigan, showed that EYD-001 (formerly known as HS-276) significantly reduced both dermal thickening and p-TAK1 expression in the lungs of a bleomycin-induced mouse model of systemic sclerosis. Moreover, in patient-derived skin fibroblasts, treatment with EYD-001 significantly reduced mRNA expression of fibroinflammatory genes and blocked TGFβ-mediated increases in fibrotic protein expression.

The FDA’s Orphan Drug Designation recognizes the potential of EydisBio’s TAK1 inhibitor to address this unmet medical need, highlighting the importance of these findings and the promise of EYD-001 as a novel therapeutic option for patients suffering from systemic sclerosis.

“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,” said Dr. Tim Haystead, Founder and President of EydisBio. “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.”

The Orphan Drug Designation is a significant milestone for any company, especially for those developing treatments for rare diseases affecting fewer than 200,000 people in the United States. This designation offers several key benefits, including seven years of market exclusivity upon approval, which protects the product from direct competition. Companies also receive tax credits for clinical trial costs, alleviating the financial burden of drug development. Additionally, the FDA provides essential support in clinical trial design and waives prescription drug user fees, streamlining the development process. These incentives collectively enable companies to bring innovative treatments to market more efficiently, ultimately benefiting patients with rare conditions.

EydisBio is committed to advancing the development of its TAK1 inhibitor program and plans to initiate clinical trials in the near future. The company will continue to work closely with the FDA to bring this promising therapy to patients affected by systemic sclerosis as quickly as possible.

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