Drug Farm announced today that the U.S. Food and Drug Administration (FDA) has given the green light to the Investigational New Drug (IND) application for DF-003. This oral alpha-kinase 1 (ALPK1) inhibitor will undergo clinical trials in ROSAH syndrome patients to evaluate its safety, pharmacokinetics, and efficacy.

DF-003 is a groundbreaking inhibitor designed to target the gain-of-function ALPK1 mutation responsible for ROSAH syndrome. It has shown significant activity in preclinical studies using a ROSAH mouse model and has received a Rare Pediatric Disease Designation from the FDA. Phase 1 trials conducted on healthy subjects have demonstrated its safety, supporting its once-daily oral dosing regimen for the upcoming ROSAH patient trials.

“We are excited to introduce a promising new therapy with the potential to improve both ocular and systemic outcomes for ROSAH syndrome,” stated Lloyd Williams, M.D., Ph.D., Director of Global Ophthalmology at Duke University. “Existing treatments have only managed symptoms, not the genetic cause. Our precision drug targets the mutant ALPK1, potentially halting disease progression,” added Dr. Jeysen Yogaratnam, Chief Medical Officer at Drug Farm. “We are proud of the swift progress in developing DF-003, as our goal is to provide a safe, targeted therapy for ROSAH patients. Additionally, our preclinical data suggest DF-003 could be a distinctive candidate for treating cardio-renal diseases, expanding its potential impact,” said Dr. Henri Lichenstein, CEO of Drug Farm.

 

Source: businesswire.com

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