Drug Farm Gets Green Light from FDA to Initiate Phase 1b Trial of DF-003 in ROSAH Syndrome Patients

The FDA has cleared Drug Farm's investigational new drug (IND) application of DF-003, a first-in-class, oral, potent, highly selective alpha-kinase 1 (ALPK1) inhibitor for clinical evaluation in ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome patients. The trial will assess the safety, pharmacokinetics and efficacy of DF-003 in ROSAH patients.

DF-003 is a first-in-class, potent, highly selective inhibitor of the gain-of-function ALPK1 mutation responsible for causing ROSAH syndrome. Drug Farm has demonstrated significant preclinical activity for DF-003 in a ROSAH mouse model of disease and has received a Rare Pediatric Designation from FDA. Furthermore, DF-003 has completed phase 1 evaluation in healthy subjects with safety and pharmacokinetics supporting a once-a-day oral dosing regimen in the upcoming ROSAH patient trial.

“Until now, treatments for ROSAH have addressed the symptoms, but not the genetic root cause of disease. We have developed a precision drug that inhibits mutant ALPK1 and may stop progression of disease in patients afflicted with ROSAH,” said Jeysen Yogaratnam, MB.BCh, MRCSEd, PhD, Chief Medical Officer, Drug Farm.