• Therapeutic Cataract & Refractive
  • Lens Technology
  • Glasses
  • Ptosis
  • Comprehensive Eye Exams
  • AMD
  • COVID-19
  • DME
  • Ocular Surface Disease
  • Optic Relief
  • Geographic Atrophy
  • Cornea
  • Conjunctivitis
  • Myopia
  • Presbyopia
  • Allergy
  • Nutrition
  • Pediatrics
  • Retina
  • Cataract
  • Contact Lenses
  • Lid and Lash
  • Dry Eye
  • Glaucoma
  • Refractive Surgery
  • Comanagement
  • Blepharitis
  • OCT
  • Patient Care
  • Diabetic Eye Disease
  • Technology

Drug Farm receives FDA approval of IND application for DF-003 and proceeds with Phase 1b trial for patients with ROSAH syndrome


DF-003 is a first-in-class, oral, potent, highly selective alpha-kinase 1 (ALPK1), an inhibitor believed to be responsible for causing ROSAH syndrome.

Woman holding pill and glass of water while sitting down Image Credit: AdobeStock/NewAfrica

Image Credit: AdobeStock/NewAfrica

Biotechnology company Drug Farm has announced the US FDA’s approval of its Investigational New Drug application of DF-003 for patients with retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache (ROSAH) syndrome. DF-033 is a first-in-class, oral, potent, highly selective alpha-kinase 1 (ALPK1) inhibitor, according to a news release. The trial will assess the safety, pharmacokinetics, and efficacy of DF-003 in treating ROSAH syndrome.1

“We are delighted to offer a promising new therapeutic with the potential to improve ocular and systemic outcomes in ROSAH syndrome,” said Lloyd Williams, M.D., Ph.D., director of global ophthalmology at Duke University, in the release.

The ALPK1 mutation is responsible for causing ROSAH syndrome, according to the release. “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, MBBCh, MRCSEd, PhD, MBA, chief medical officer at Drug Farm, in the release.

Additionally, preclinical data for DF-003 in a ROSAH mouse model of the disease found to be significant and received a Rare Pediatric Designation from the FDA. DF-003 also completed a Phase 1 evaluation in subjects with safety and pharmacokinetics, supporting a once-a-day oral dosing regiment in the upcoming ROSAH patient trial.1

“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 Henri Lichenstein, PhD, CEO of Drug Farm, in the release.

For the upcoming Phase 1b trial, potential participants will undergo a 49 day screening period to determine eligibility for entry. Patients must be considered healthy in exclusion of a ROSAH syndrome diagnosis, which includes healthy cardiac conduction and function, in order to be included in the study, among other factors. Those then enrolled in the study will be administered DF-003 orally once daily for 28 days, or 4 weeks. Loading doses of 140 mg will be administered for the first 3 days of the trial, then followed by a maintenance dose of 45 mg once daily starting on day 4 and continuing through day 28.2

  1. Drug Farm announces IND clearance by US FDA enabling Phase 1b initiation for ALPK1 selective kinase inhibitor, DF-003 in ROSAH syndrome patients. News release. Drug Farm. May 17, 2024. Accessed May 20, 2024. https://drug-farm.com/news
  2. ClinicalTrials.gov. Evaluating the safety and tolerability of multiple doses of orally administered DF-003 in ROSAH syndrome patients. Shanghai Yao Yuan Biotechnology. NCT06395285. Last updated May 2, 2024. Accessed May 20, 2024. https://clinicaltrials.gov/study/NCT06395285?cond=ROSAH&rank=1
Related Videos
Adam Alexander, OD, chats with Optometry Times about his AOA e-poster presentation on Miebo
Nazlee Zebardast, MD, MSc, overviews her ARVO 2024 presentations on glaucoma and polygenic risk scores
Edmund Tsui, MD, details what insights swept-source anterior segment OCT images may give to determining eye inflammation
Michael Chaglasian, OD, details success of new OCT device at the ARVO 2024 meeting
Emily Chew, MD, outlines her lecture that landed her the Proctor Award at ARVO 2024
Deborah Ferrington details a session on mitochondrial maintenance and retinal health in patients with AMD at ARVO 2024
Dr. John Sheppard discusses results from trials testing the efficacy of eye drops to treat dry eye disease
Kelsey Roelofs, MD, details the best methods to identify thyroid eye disease at CIME 2024
© 2024 MJH Life Sciences

All rights reserved.