The US Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application of Drug Farm enabling the commencement of a Phase Ib clinical trial of DF-003 in patients with ROSAH syndrome.

An autosomal dominant autoinflammatory ailment, ROSAH is characterised by retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache.

This trial aims to evaluate the safety, pharmacokinetics, and efficacy of the oral ALPK1 selective kinase inhibitor in individuals affected by this condition.

DF-003 is a potent and highly selective inhibitor of the alpha-kinase 1 (ALPK1) mutation, which is responsible for causing ROSAH syndrome.

Preclinical studies have shown significant activity of DF-003 in a ROSAH mouse model, supporting the progression to human trials.

DF-003 has received a rare pediatric disease designation from the FDA in January, highlighting the drug’s potential in addressing the needs of paediatric patients with ROSAH syndrome.

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The drug has already undergone a Phase I trial assessing its safety and pharmacokinetics in healthy subjects.

Results indicated that a once-a-day oral dosing regimen could be feasible for the upcoming trial with ROSAH patients.

Drug Farm CEO Dr Henri Lichenstein said: “We are thrilled with the rapidity that Drug Farm has executed on the DF-003 programme as our mission is to develop a safe, targeted therapy for ROSAH patients.

“Drug Farm preclinical data also supports the use of DF-003 as a uniquely differentiated candidate for cardio-renal disease and we are excited about the prospects of our drug in this patient population as well.”