Preclinical studies have shown significant activity of DF-003 in a ROSAH mouse model, supporting the progression to human trials. Credit: David Gyung / Shutterstock.com.

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.

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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.

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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.”