ALBANY, N.Y. & SHANGHAI–(BUSINESS WIRE)–Drug Farm, a clinical-stage biopharmaceutical company advancing two novel small molecule drugs for ROSAH syndrome and hepatitis B, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to DF-003, its investigational, first-in-class ALPK1 inhibitor, for the treatment of ROSAH syndrome, a rare, autosomal dominant autoinflammatory disease with no approved therapies.
Fast Track designation is designed to facilitate the development and expedite the review of drugs intended to treat serious or life-threatening conditions and address unmet medical needs. The designation enables more frequent interactions with the FDA and allows eligibility for rolling review and, if relevant criteria are met, priority review.
DF-003 is a structurally novel, orally administered small-molecule inhibitor that potently and selectively inhibits disease-causing mutant ALPK1, including the ROSAH-associated ALPK1 T237M variant. Preclinical studies demonstrated that DF-003 crosses the blood–retina and blood–brain barriers and significantly suppresses inflammatory cytokines and disease-associated phenotypes in a mouse model of ROSAH syndrome (1). These findings supported the advancement of DF-003 into clinical development.
“We have designed DF-003 to directly target the genetic root cause of ROSAH syndrome,” said Henri Lichenstein, Ph.D., Chief Executive Officer of Drug Farm. “The FDA’s Fast Track designation recognizes the serious nature of this disease and the urgent need for targeted, disease-modifying therapies, and it supports our efforts to accelerate development of DF-003 for patients.”
DF-003 is the first ALPK1 inhibitor to successfully complete a first-in-human Phase 1 study and has now entered a Phase 1b clinical trial in patients with ROSAH syndrome (NCT06395285), where it is being evaluated for safety, pharmacokinetics, pharmacodynamics and efficacy. Clinical data from the trial will be reported at meetings in 2026.
“I am encouraged by the progress of DF-003 and the FDA’s decision to grant Fast Track designation,” said Dr. John Grigg, Professor of Clinical and Experimental Ophthalmology, Save Sight Institute Faculty of Medicine and Health at The University of Sydney. “Patients with ROSAH syndrome currently have no approved treatment options, and therapies that can modify disease progression have the potential to meaningfully preserve vision and improve quality of life.”
- Fan J, et al. Discovery of a selective alpha-kinase 1 inhibitor for the rare genetic disease ROSAH syndrome, Nature Communications 2025 Sep 9;16(1):8251. doi: 10.1038/s41467-025-63731-5.PMID: 40925900
About DF-003
DF-003 is a proprietary, first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and variants of ALPK1 which cause ROSAH syndrome. DF-003 has therapeutic potential for ROSAH syndrome, as well as heart and kidney diseases, as the drug has shown efficacy in preclinical models of these indications. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in normal healthy volunteers and is now accruing patients with ROSAH syndrome in a Phase 1b trial (NCT06395285).
About ROSAH Syndrome
ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease caused by activating mutations in the ALPK1 gene. The disease is characterized by progressive visual loss, optic nerve and retinal pathology, and systemic inflammatory manifestations, including elevated pro-inflammatory cytokines. Symptoms often begin in childhood or early adulthood, and there are currently no approved disease-modifying therapies for ROSAH syndrome.
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 syndrome. Drug Farm’s unique IDInVivo platform combines breakthrough technologies in genetics and artificial intelligence to identify and validate novel drug targets directly in living animals with intact immune systems. Using this platform, Drug Farm is advancing multiple first-in-class drug candidates into clinical development.
For more information, please visit: https://www.drug-farm.com
Contacts
United States:
Henri Lichenstein, Ph.D.
Chief Executive Officer
Email: [email protected]
China:
Tony Xu, Ph.D.
Co-founder and Chief Operating Officer
Email: [email protected]
