Eloxx submits IND to FDA for ZKN-013 for treatment of RDEB

Eloxx Pharmaceuticals announced the submission of an Investigational New Drug, or IND, application with the FDA for ZKN-013 for the treatment of recessive Dystrophic Epidermolysis Bullosa, or RDEB, with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene. Recent preclinical results demonstrated read-through activity of ZKN-013 in multiple COL7 genotypes across multiple RDEB patient derived fibroblasts and keratinocytes. Read-through activity resulted in up to an 18-fold increase in full-length COL VII protein levels. Prolonged treatment with ZKN-013 further increased COL VII protein levels. Functionality of the restored full-length COL VII protein was confirmed. These results have been accepted for presentation at an upcoming medical conference. An additional IND filing for ZKN-013 for the treatment of familial adenomatous polyposis is planned in the first half of 2023. FAP, a rare inherited disease with no approved drug therapies, is characterized by proliferation of colon polyps. Eloxx is targeting a subset of patients that have nonsense mutations in the Adenomatous Polyposis Coli gene that is truncated in these patients.