Trial will evaluate DF-003, a first-in-class ALPK1 inhibitor that specifically targets the genetic root cause of ROSAH syndrome

Drug Farm, a private biotechnology company utilizing genetics and artificial intelligence technologies to discover and develop innovative, immune-modulating therapies, today announced that the first human participants have been dosed in a clinical trial (NCT06395285) of DF-003 in patients with ROSAH syndrome.

DF-003 is a first-in-class, immune-modulating alpha-kinase 1 (ALPK1) inhibitor that targets ALPK1 mutants causing ROSAH syndrome. The trial is evaluating the safety, pharmacokinetics and efficacy of orally administered DF-003 at sites in the USA and Australia.

“We are happy to offer patients with ROSAH syndrome the opportunity to participate in a clinical trial using a new precision-targeted drug to treat the root cause of their disease,” said Dr. John Grigg, Professor of Clinical and Experimental Ophthalmology, Save Sight Institute Faculty of Medicine and Health at The University of Sydney.

“The initiation of this trial is a critical step forward in our goal to provide a therapeutic option in disease indications with unmet medical need that are mediated by excessive ALPK1 activation,” said Henri Lichenstein, PhD, Chief Executive Officer of Drug Farm. “We are optimistic about the potential of DF-003 to make a meaningful difference in the lives of patients with ROSAH, and together with our collaborators, are committed to thoroughly investigating the safety and efficacy of DF-003 in patients with atherosclerotic cardiovascular disease as well.”

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 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 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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