EydisBio’s systemic sclerosis treatment receives FDA orphan drug designation

Durham-based EydisBio Inc. has received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for EYD-001, a treatment for systemic sclerosis. This rare and debilitating autoimmune disease involves hardening and tightening of the skin.

EydisBio is a Duke University spin-out leveraging a novel approach to treating various autoimmune and inflammatory diseases — including rare diseases — which have significant needs for safer and more effective treatments. EYD-001 is a highly selective and potent, orally bioavailable TAK1 inhibitor. 

"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 Tim Haystead, Ph.D., founder and president of EydisBio and professor of pharmacology and cancer biology at Duke University. "This recognition highlights the innovative nature of our research and the dedication of our team to advancing treatments for rare diseases."

The Orphan Drug Designation program provides special status to drugs and biologics designed for the safe and effective treatment, diagnosis, or prevention of rare diseases and disorders. EydisBio plans to leverage the benefits associated with orphan drug status by utilizing the designation’s expedited regulatory pathways to shorten the development and approval timelines, potentially bringing the therapy to market more quickly. 

The company also plans to take advantage of orphan drug financial incentives, including tax credits and grants, to support clinical trials and reduce overall development costs. The seven-year market exclusivity that comes with Orphan Drug Designation will help give EYD-001 a competitive edge, ensuring that the treatment remains viable and accessible to patients. 

Growth and innovation

EydisBio was founded in 2016 by Haystead to develop potent and selective small molecules for autoimmune and inflammatory diseases. In 2019, the company gained momentum after a successful application for a $100,000 Biotechnology Innovation Grant (BIG) from the North Carolina Biotechnology Center to advance EYD-001 as a therapy for rheumatoid arthritis. The BIG program supported full-time faculty scientists at N.C. universities in assessing the commercial viability of life science inventions.

EydisBio has since secured approximately $6.7 million in non-dilutive funding, primarily from NIH SBIR/STTR grants, supporting drug development research related to various conditions, including rheumatoid arthritis and Alzheimer's disease. Central to EydisBio's success is Haystead's innovative drug discovery platform, which enables the precise targeting of disease-driving proteins and the identification of highly selective compounds with a large therapeutic window.

“We have a unique approach to drug development in that we are science-driven and patient-focused,” said Haystead. “We primarily focus where there are serious unmet patient needs first, and then try to develop drugs which can revolutionize the treatment landscapes and improve the standard of care.”  

Earlier in 2024, EydisBio received a $250,000 NCBiotech loan, which has been critical to advancing its TAK1 inhibitor program by supporting the optimization of EYD-001 analogs for safe and effective drug development. Robert Freeze, the company’s vice president of business development, noted that NCBiotech has also facilitated important contacts and provided forums for valuable feedback. 

“We're excited to see this important systemic sclerosis therapy progressing toward clinical trials,” said Kyle Bartholomew, Director of Investments at NCBiotech. “NCBiotech is pleased to have supported moving this research forward, and the Orphan Drug Designation will further accelerate its path to reaching the patients who need it.”

Next steps for systemic sclerosis treatment

Preclinical data published last year in collaboration with John Varga, MD, and his ScleroLab research group at the University of Michigan, showed that EYD-001 significantly reduced dermal thickening and fibrotic markers in a mouse model of systemic sclerosis and decreased fibrotic gene expression in patient-derived skin cells.

EydisBio recently received a Phase II STTR grant from the NIH to test EYD-001 in mouse models, assess safety in bacterial and fungal models, and complete IND-enabling studies. The project, conducted in collaboration with Varga, will pave the way for raising capital for an IND and Phase I clinical trial that includes healthy volunteers and systemic sclerosis patients.