Drug Farm Receives FDA IND Clearance for First-in-Class ALPK1 Inhibitor DF-003 in ROSAH Syndrome
Key Insights
Drug Farm (search) announced FDA clearance of its IND application for DF-003, a first-in-class oral ALPK1 (search) inhibitor targeting the genetic root cause of ROSAH syndrome (search).
The Phase 1b trial will evaluate safety, pharmacokinetics, and efficacy of DF-003 in ROSAH patients, with the drug having already completed Phase 1 testing in healthy subjects.
DF-003 has received FDA Rare Pediatric Designation and demonstrated significant preclinical activity in ROSAH mouse models, supporting once-daily oral dosing.
Drug Farm (search) announced that the U.S. Food and Drug Administration has cleared the Investigational New Drug application for DF-003, a first-in-class, oral, potent, highly selective alpha-kinase 1 (ALPK1 (search)) inhibitor for clinical evaluation in ROSAH syndrome (search) patients. The upcoming Phase 1b trial will assess the safety, pharmacokinetics and efficacy of DF-003 in ROSAH patients.
Targeting the Genetic Root Cause
DF-003 represents a precision medicine approach to treating ROSAH syndrome (search) by specifically targeting the gain-of-function ALPK1 (search) mutation responsible for causing the disease. Unlike existing treatments that only address symptoms, DF-003 aims to halt disease progression by inhibiting the mutant ALPK1 protein directly.
"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 (search) and may stop progression of disease in patients afflicted with ROSAH," said Dr. Jeysen Yogaratnam, Chief Medical Officer at Drug Farm (search).
Strong Preclinical Foundation and Regulatory Support
Drug Farm (search) has demonstrated significant preclinical activity for DF-003 in a ROSAH mouse model of disease. The FDA has granted DF-003 a Rare Pediatric Designation, recognizing the drug's potential significance for this underserved patient population.
The drug has already completed Phase 1 evaluation in healthy subjects, with safety and pharmacokinetics data supporting a once-a-day oral dosing regimen for the upcoming ROSAH patient trial.
Clinical Significance and Broader Applications
"We are delighted to offer a promising new therapeutic with the potential to improve ocular and systemic outcomes in ROSAH syndrome (search)," said Lloyd Williams, M.D., Ph.D., Director of Global Ophthalmology at Duke University (search).
Beyond ROSAH syndrome (search), Drug Farm (search)'s preclinical data also supports the potential use of DF-003 as a differentiated candidate for cardio-renal disease (search). "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 of Drug Farm.
About ROSAH Syndrome and Drug Farm's Platform
ROSAH syndrome (search) is characterized by retinal dystrophy (search), optic nerve edema (search), splenomegaly (search), anhidrosis (search) and headache (search). Drug Farm (search) is a private biotechnology company developing innovative treatments targeting innate immunity for hepatitis B (search), heart and kidney diseases, and ROSAH syndrome.
The company's proprietary IDInVivo platform combines breakthrough technologies in genetics and AI to discover new treatments. This technology allows the direct assessment of gene targets in living animals with intact immune systems, enabling Drug Farm (search) to identify novel innate immunity pathways and targets and rapidly advance multiple first-in-class drug candidates into clinical development.