Drug Farm, a clinical-stage biopharmaceutical company, has announced a partnership with the U.S. National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) to develop DF-003, an oral drug targeting the disease-causing mutant alpha kinase-1 (ALPK1), which is responsible for ROSAH syndrome. This agreement focuses on advancing DF-003, a first-in-class ALPK1 inhibitor, through clinical trials to assess its safety, pharmacokinetics, and efficacy in treating ROSAH syndrome.
ROSAH syndrome is a rare, autosomal dominant autoinflammatory genetic disease characterized by symptoms including retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache. The disease is caused by mutations in the ALPK1 gene, leading to a range of symptoms such as progressive visual decline, optic disc elevation, uveitis, and retinal nerve degeneration. Patients often experience inflammatory features like low-grade fevers, arthralgia, headaches, and elevated levels of inflammatory cytokines.
DF-003 has shown preclinical efficacy in a transgenic ROSAH mouse model and has completed Phase 1 evaluation in healthy subjects. The upcoming clinical trial, sponsored by Drug Farm in collaboration with NIAID, aims to further evaluate DF-003's potential as a therapeutic option for ROSAH syndrome patients. This partnership represents a crucial advancement in the search for effective treatments for this debilitating condition.
