EydisBio receives FDA orphan drug designation for TAK1 inhibitor for the treatment of systemic sclerosis

DURHAM, NC, 25 September 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 a young pharmaceutical company pursuing a novel approach to the treatment of various autoimmune and inflammatory diseases, including rare diseases. For more information about the company, its development programs and groundbreaking research, visit www.eydisbio.com.

Systemic sclerosis is a rare and debilitating autoimmune disease characterized by fibrosis of the skin and internal organs, resulting in significant morbidity and mortality. Central to the pathogenesis of the disease is the transforming growth factor-β (TGF-β) signaling pathway, with TAK1 playing a critical 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 treat the disease.

Preclinical data from EydisBio published last year in collaboration with Dr. John VargaMD and his ScleroLab research group at the University of Michiganshowed that EYD-001 (formerly known as HS-276) significantly reduced both skin thickening and p-TAK1 expression in the lungs of a bleomycin-induced mouse model of systemic sclerosis. Furthermore, EYD-001 treatment in patient skin fibroblasts 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 and underscores the importance of these findings and the potential of EYD-001 as a novel therapeutic option for patients suffering from systemic sclerosis.

“We are very pleased with this FDA approval, which underscores the potential of our TAK1 inhibitor to significantly improve the lives of patients with systemic sclerosis,” said Dr. Tim HaysteadFounder and President of EydisBio. “This recognition underscores the innovative nature of our research and our team's commitment to advancing treatments for rare diseases. It also reinforces our commitment to bring EYD-001 to patients with systemic sclerosis as quickly and safely as possible.”

Orphan drug status is an important milestone for any company, especially those developing treatments for rare diseases that affect fewer than 200,000 people. the United States. This designation offers several important benefits, including seven years of market exclusivity after approval, which protects the product from direct competition. Companies also receive tax credits for clinical trial costs, reducing the financial burden of drug development. In addition, the FDA provides important assistance in clinical trial design and waives prescription drug use fees, streamlining the development process. These incentives allow companies to bring innovative treatments to market more efficiently, ultimately benefiting patients with rare diseases.

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 make this promising therapy available to patients with systemic sclerosis as quickly as possible.

Media contact: Robert Freeze, [email protected]

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