Biotechnology company Drug Farm on Thursday announced favourable safety and pharmacokinetic results from a completed Phase 1 clinical trial of DF-003, an immune-modulating ALPK1 inhibitor targeting ROSAH syndrome.

The randomised, placebo-controlled, double-blinded trial involved 48 healthy volunteers and was designed to evaluate the safety, tolerability and pharmacokinetics of DF-003. The study included single ascending doses ranging from 3 mg to 150 mg, and a 14-day regimen of 50 mg daily.

No serious adverse events were reported, and treatment emergent adverse events were comparable between DF-003 and placebo groups. No participants required dose modifications or interruptions.

Pharmacokinetic data indicated dose-proportional exposure, supporting the continued development of DF-003 as a once-daily oral therapy.

The results were presented at the American Society for Clinical Pharmacology & Therapeutics meeting in Washington, DC.

DF-003 is being developed to treat ROSAH syndrome, a rare autoinflammatory genetic disease caused by mutations in the ALPK1 gene.