Partnership focuses on DF-003, an oral drug that targets disease-causing mutant alpha kinase-1 (ALPK1), the genetic cause of ROSAH syndrome

Drug Farm, a clinical-stage biopharmaceutical company developing innovative treatments targeting innate immunity, announced today the signing of a Cooperative Research and Development Agreement (CRADA) with the U.S. National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) to develop a precision targeted drug, DF-003 for the treatment of ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome. DF-003 is a first-in-class highly selective ALPK1 inhibitor with nanomolar potency that can cross the blood-retina barrier and has demonstrated preclinical efficacy in a transgenic ROSAH mouse model. DF-003 has completed Phase 1 evaluation in healthy subjects and will be evaluated in patients with ROSAH syndrome. Under the terms of the agreement, Drug Farm will sponsor and partner with NIAID on a clinical trial (NCT06395285) to assess the safety, pharmacokinetics and efficacy of DF-003 in people with ROSAH. “NIAID is a leader in ROSAH syndrome research and we are excited about partnering with NIAID as a first step towards developing a precision targeted drug that has potential for providing therapeutic benefit to people with ROSAH syndrome,” said Dr. Henri Lichenstein, Chief Executive Officer at Drug Farm.

About ROSAH Syndrome

ROSAH (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis and headache) syndrome is a rare, autosomal dominant autoinflammatory genetic disease named according to the characteristic symptoms exhibited by affected patients (1, 2). Disease-causing mutations in ALPK1 lead to ROSAH Syndrome. The most common presenting symptom is a progressive decline in visual acuity that typically begins before 20 years of age, with ophthalmologic examination often revealing optic disc elevation, uveitis, and retinal nerve degeneration (2, 3). Most ROSAH patients also exhibit inflammatory features such as non-infectious low-grade fevers, arthralgia, headaches, and persistently elevated levels of inflammatory cytokines including tumor necrosis factor-α (TNFα), interleukin-6 (IL-6), and IL-1β (3).

1. Tantravahi SK, et al. An inherited disorder with splenomegaly, cytopenias, and vision loss. Am J Med Genet A. 2012;158(3):475-81.

2. Williams LB, et al. ALPK1 missense pathogenic variant in five families leads to ROSAH syndrome, an ocular multisystem autosomal dominant disorder. Genet Med. 2019;21(9):2103-15.

3. Kozycki CT, et al. Gain-of-function mutations in ALPK1 cause an NF-κB-mediated autoinflammatory disease: functional assessment, clinical phenotyping and disease course of patients with ROSAH syndrome. Ann Rheum Dis. 2022;81(10):1453-64.

About DF-003

DF-003 is a first-in-class drug developed by Drug Farm that inhibits the activity of ALPK1 and variants of ALPK1 that cause ROSAH syndrome. DF-003 has completed a Phase 1 clinical trial (NCT05997641) in normal healthy volunteers.

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: https://www.drug-farm.com.

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