Drug Farm, a private biotechnology company with operations in Albany, New York, and Shanghai, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to DF-003, a small molecule ALPK1 inhibitor, for ROSAH syndrome treatment.
The designation provides incentives including tax credits, fee waivers, and seven-year US market exclusivity.
DF-003 completed a Phase I trial in healthy volunteers (NCT05997641) and is currently enrolling patients with ROSAH syndrome in a Phase Ib trial (NCT06395285) evaluating safety, pharmacokinetics, pharmacodynamic biomarkers, and efficacy parameters. Drug Farm developed DF-003 internally using its IDInVivo platform, which combines genetic screening and artificial intelligence to identify targets in living animals with intact immune systems.
ROSAH syndrome (Retinal dystrophy, Optic nerve edema, Splenomegaly, Anhidrosis, and Headache) is a rare, autosomal dominant autoinflammatory disease caused by activating mutations in the ALPK1 gene. Symptoms typically present in childhood or early adulthood and include progressive visual loss, systemic inflammation, and elevated pro-inflammatory cytokines. No disease-modifying therapies are currently approved for this indication.
DF-003 is described as a first-in-class ALPK1 inhibitor designed to directly inhibit the activity of disease-causing ALPK1 variants. ALPK1 has attracted limited therapeutic competition to date; no other ALPK1-targeted therapies in clinical development for ROSAH syndrome were identified in publicly available databases. The absence of competing clinical-stage programs targeting this kinase positions DF-003 without direct comparators in this space. Drug Farm has also noted preclinical activity for DF-003 in models of heart and kidney disease, suggesting the company may pursue additional indications for the molecule beyond ROSAH syndrome.
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