Drug Farm Announces Collaboration with NIH to Co-Develop DF-003, a Precision Therapy for ROSAH Syndrome
Drug Farm
Innovative Therapies Researcher for Infectious and Immune Diseases
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DrugRong Circle learned that: On December 20, 2024, the collaboration between Drug Farm and the National Institutes of Health (NIH) is based on Drug Farm's original new drug (First-in-class) ALPK1 kinase inhibitor, DF-003. DF-003 is characterized by oral administration, high potency, and high selectivity, capable of inhibiting ALPK1 and ALPK1 mutant activity. Gain-of-function mutations in these variants can lead to ROSAH syndrome.
Drug Farm, a clinical-stage biopharmaceutical company, is dedicated to developing innovative therapies targeting innate immunity. It has signed a Cooperative Research and Development Agreement (CRADA) with the National Institute of Allergy and Infectious Diseases (NIAID), part of the U.S. National Institutes of Health, to develop the targeted drug DF-003 for the precise treatment of ROSAH (Retinal Dystrophy, Optic Nerve Edema, Splenomegaly, Anhidrosis, and Headache) syndrome. DF-003, a highly selective and potent ALPK1 inhibitor, can cross the blood-retinal 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 assessed in patients with ROSAH syndrome. Under the terms of the CRADA, Drug Farm will act as the sponsor, while the NIAID will provide funding. Both parties will collaborate to advance the clinical trial (NCT06395285) to evaluate the safety, pharmacokinetics, and efficacy of DF-003 in ROSAH patients.
"The National Institute of Allergy and Infectious Diseases (NIAID) of the U.S. National Institutes of Health is a leader in ROSAH syndrome research, and its previous natural history study of the disease has recruited the largest cohort of ROSAH subjects. We are very pleased to collaborate with NIAID this time to evaluate the efficacy of DF-003 as a novel therapy for ROSAH. This is the first step in developing a precision-targeted drug that could provide a tangible treatment option for patients with ROSAH syndrome," said Dr. Xu Cong, co-founder and Chief Operating Officer of Drug Farm.
ROSAH (Retinal dystrophy, Optic nerve edema, Splenomegaly, Anhidrosis, and Headache) syndrome is a rare autosomal dominant genetic disorder named according to the characteristic symptoms exhibited by affected patients.1，2Pathogenic mutations in ALPK1 lead to ROSAH syndrome. The most common initial symptom is progressive vision loss, typically beginning before the age of 20. Ophthalmic examinations often reveal optic disc elevation, uveitis, and retinal neurodegeneration.2、3Most ROSAH patients also exhibit inflammatory characteristics, such as non-infectious low-grade fever, arthralgia, headache, 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.
DF-003 is an original innovative First-in-class drug developed by Drug Farm. It can inhibit ALPK1 and the activity of ALPK1 mutants that cause ROSAH syndrome. DF-003 has completed Phase 1 clinical trials (NCT05997641) in normal healthy volunteers.
Drug Farm is a biotechnology company dedicated to the discovery of targets and the development of original new drugs (first-in-class), focusing on pioneering innovative therapies for autoimmune diseases such as hepatitis B, heart and kidney diseases, and ROSAH syndrome (retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache). Drug Farm's unique IDInVivo+ platform combines breakthrough technologies in genetics and artificial intelligence to discover new treatments, enabling direct selection of gene targets in animal models with intact immune systems. Using the IDInVivo+ platform, Drug Farm has discovered multiple novel innate immune pathways and targets, and is rapidly advancing several original new drugs into clinical development.
For more information, please visit: https://www.drug-farm.com
