Eloxx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application to initiate a single ascending dose (SAD) clinical trial in healthy volunteers for ZKN-013 for the potential treatment of recessive Dystrophic Epidermolysis Bullosa (RDEB) with nonsense mutations. RDEB is a rare skin disease characterized by mutations in Collagen7 gene. Further SAD and multiple ascending dose (MAD) testing is expected to be conducted following the completion of the planned dose cohorts in the SAD study and discussion with the FDA.

The MAD testing could potentially include RDEB patients given the strong benefit/risk in patients cited by the FDA. ZKN-013 has demonstrated robust functional preclinical activity in RDEB and JEB patient cells and in APCmin (multiple intestinal neoplasia) mice. Eloxx also plans to develop ZKN-013 for the treatment of FAP patients with nonsense mutations characterized by proliferation of colon polyps and progression to colon cancer.

In January 2023, Eloxx published its preclinical results that showed treatment with ZKN-013 demonstrated a decrease in intestinal polyps and adenomas, resulting in increased survival.