Drug Farm Reports Data on Safety and Pharmacokinetics from Phase 1 First-in-Human Trial of DF-003 in Healthy Volunteers

Business Wire

29-05-2025

ALBANY, N.Y. & SHANGHAI--(BUSINESS WIRE)--Drug Farm, a private biotechnology company utilizing genetics and artificial intelligence technologies to discover and develop innovative, immune-modulating therapies, today announced positive results from a recently completed Phase 1 study (NCT05997641) of DF-003 in healthy volunteers. The data were presented at the American Society for Clinical Pharmacology & Therapeutics (ASCPT) meeting in Washington, DC. DF-003 is a first-in-class, immune-modulating alpha-kinase 1 (ALPK1) inhibitor that targets the root cause of the rare genetic disease, ROSAH syndrome.
This randomized, placebo-controlled, double-blinded study evaluated the safety, tolerability and pharmacokinetics of DF-003 in forty-eight healthy volunteers. In the first portion of the trial, single ascending doses were orally administered in five cohorts (randomized 3:1; DF-003: placebo) ranging from 3 mg up to 150 mg. In the second portion of the trial, 50 mg of DF-003 was orally administered daily for fourteen consecutive days in one cohort (randomized 3:1; DF-003: placebo) in eight healthy volunteers. The primary objective of the study was to evaluate the safety and tolerability of DF-003, as well as its pharmacokinetic properties.
Key Results
Safety: DF-003 was safe at all doses tested with no serious adverse events described. Rates of treatment emergent adverse events (TEAEs) were similar between active and placebo-treated participants, and no TEAEs required dose modification or interruption.
Pharmacokinetics: The pharmacokinetic profile of DF-003 was largely dose proportional and the data support additional clinical trials as a once-daily, orally administered drug.
'We are happy to share the results from our Phase 1 study that demonstrate the excellent safety of DF-003,' said Neil Solomons, MD, Head, Clinical Development at Drug Farm. 'We are also pleased that the pharmacokinetic profile of DF-003 leads to trough concentrations in blood that are consistent with efficacy in preclinical models of ROSAH syndrome and cardio-renal disease. The dose ranges explored in this Phase 1 trial will be used in our upcoming proof of concept trials in ROSAH syndrome and cardio-renal disease patient populations.'
'This is a major inflection point for Drug Farm in our quest to advance DF-003, a first-in-class, immune-modulating drug that precision targets the disease-causing mutations of ROSAH syndrome, as well as the excessive activation of the ALPK1 pathway found in cardio-renal disease,' said Henri Lichenstein, PhD, Chief Executive Officer at Drug Farm. 'There are no approved drugs for ROSAH syndrome, and we are excited about the potential of DF-003 to save sight and to ameliorate other debilitating inflammatory symptoms associated with this disease.'
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 and cardio-renal disease as the drug has shown efficacy in preclinical models of these disease 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.