Trefoil Therapeutics announced it will collaborate with the National Institutes of Health’s (NIH) National Center for Advancing Translational Sciences (NCATS) to complete the investigational new drug enabling activities for their lead compound, TTHX1114, toward a treatment for Fuchs endothelial corneal dystrophy (FECD).
Approximately 70,000 people suffer from FECD, which can lead to severe vision loss and for which there currently is no FDA-approved pharmaceutical therapy. Trefoil is developing TTHX1114 as potentially the first medical therapy for treatment of FECD, which is the most common driver of corneal transplantation.
“Fuchs dystrophy and other corneal endothelial diseases are potentially debilitating conditions, with limited nonsurgical treatment options available to patients today. For many patients, the long-term prognosis may require some form of corneal transplant surgery. Having a therapeutic option such as the Trefoil compound may significantly expand our treatment options for these serious conditions," Richard Abbott, MD, Clinical Professor of Ophthalmology and holder of the Thomas W. Boyden Endowed Chair in Ophthalmology at University of California, San Francisco, said in a company news release. "This collaboration with NCATS provides a high-quality interaction with a group of scientists experienced at bringing forward drugs that provide much-needed therapies for rare diseases to clinical testing in patients.”
“Trefoil appreciates the opportunity to participate in the NCATS Therapeutics for Rare and Neglected Diseases (TRND) program, which will significantly accelerate the development of this compound,” Trefoil CEO David Eveleth, PhD, said in the news release. “We know from speaking with patients and physicians just how significant the unmet medical need is, and now through the TRND program we have a path forward to filing an IND with the intention of entering human clinical trials quickly thereafter. Our goal is to transform the treatment of Fuchs dystrophy by giving patients a pharmaceutical option to regenerate the corneal endothelium and avoid surgery.”
Applicants to the TRND program are selected through a competitive process from an evaluation of the most promising proposals for these prestigious public-private partnerships. Once chosen for the program, the company and NCATS enter into a Collaborative Research and Development Agreement (CRADA) to set forth the activities that NCATS researchers will undertake to move the compound forward through the preclinical phase, including development of plans for clinical trials and submission of an IND application to the FDA.
TTHX1114 is an engineered FGF-1 (eFGF-1) developed by Trefoil co-founder Dr. Michael Blaber, Professor of Biomedical Sciences at the Florida State University College of Medicine. The eFGF-1 platform represents an innovative approach to improving the properties of FGFs as drugs, yielding novel FGF-1s, which have demonstrated superior pharmacodynamic and pharmaceutical properties compared to the naturally occurring FGF-1s in animal models of tissue healing.