Published in Pipeline

Drug Farm announces IND clearance by FDA enabling phase 1b initiation for ALPK1 selective kinase inhibitor, DF-003 in ROSAH syndrome patients

Drug Farm announced today the U.S. Food and Drug Administration (FDA) has cleared the 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 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.

“We are delighted to offer a promising new therapeutic with the potential to improve ocular and systemic outcomes in ROSAH syndrome,” said Lloyd Williams, MD, PhD, director of Global Ophthalmology, Duke University. “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 Dr. Jeysen Yogaratnam, chief medical officer, Drug Farm. “We are thrilled with the rapidity that Drug Farm has executed on the DF-003 program 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,” said Dr. Henri Lichenstein, CEO, Drug Farm.

About Drug Farm

Drug Farm is a private biotechnology Company developing innovative treatments targeting innate immunity for hepatitis B, heart and kidney diseases, and ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. Drug Farm's unique IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. IDInVivo technology allows the direct assessment of gene targets in living animals with intact immune systems. Using the IDInVivo platform, Drug Farm has identified novel innate immunity pathways and targets and is now rapidly advancing multiple first-in-class drug candidates into clinical development. For more information please visit: