▎Edited by the WuXi AppTec content team
Today (May 17), Drug Farm announced that the U.S. FDA has approved the Investigational New Drug (IND) application for DF-003. According to the Drug Farm press release,DF-003 is a "first-in-class" potential ALPK1 kinase inhibitor with the characteristics of oral administration, high potency, and high selectivity, and will be used for clinical testing in ROSAH syndrome patients.. This trial will evaluate the safety, pharmacokinetics, and efficacy of DF-003 in patients with this rare disease.
ROSAH (Retinal dystrophy, Optic nerve edema, Splenomegaly, Anhidrosis, and Headache) syndrome is a rare autosomal dominant hereditary autoinflammatory disease named after the characteristic symptoms exhibited by affected patients. The disease is caused by gain-of-function mutations in ALPK1. The most common symptom is progressive vision loss, typically beginning before the age of 20. Ophthalmologic examinations usually reveal optic disc elevation, uveitis, and retinal neurodegeneration. Most ROSAH patients also exhibit inflammatory features 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.DF-003 is a potential "first-in-class" new drug developed by Drug Farm based on its own target discovery platform IDInVivo+ and AI-driven medicinal chemistry platform Medchem5. It can inhibit ALPK1 and mutant ALPK1 activity, whose hyperactive mutations lead to ROSAH syndrome. In preclinical studies using the ROSAH mouse disease model, DF-003 demonstrated significant activity.In January this year, DF-003 received the FDA's Rare Pediatric Disease Designation (RPDD). In addition, DF-003 has completed Phase 1 clinical evaluation in healthy subjects, and its excellent safety and pharmacokinetic data support a once-daily oral dosing regimen in ROSAH patient trials.Dr. Lloyd Williams, Director of Global Ophthalmology at Duke University in the United States, commented: "We are delighted that DF-003 offers a promising new therapy with the potential to improve both ocular and systemic symptoms in patients with ROSAH syndrome."Dr. Jeysen Yogaratnam, Chief Medical Officer of Drug Farm, pointed out: "So far, the treatment for ROSAH syndrome has only been alleviating symptoms and has not addressed the genetic root cause of the disease. We have developed DF-003, a precision therapeutic drug that can inhibitALPK1Mutation, with the potential to halt disease progression in ROSAH syndrome patients."Drug Farm CEO Dr. Henri Lichenstein commented, "The rapid progress of the company's DF-003 program is exciting, and our mission is to develop a safe targeted therapy for ROSAH patients. At the same time, existing preclinical data supports the use of DF-003 in treating cardio-renal diseases, and we are also excited about the prospects of DF-003 in this patient population."[1] Drug Farm's DF-003 Receives FDA Rare Pediatric Disease Designation (RPDD) for the Treatment of ROSAH Syndrome.Retrieved Jan 16, 2024, from https://mp.weixin.qq.com/s/D3v5etUj2qhAh_3evGvN-Q
This article was compiled and edited by the WuXi AppTec content team based on publicly available information. Individuals are welcome to share it on their social media circles. For authorization to repost, please contact us via a message on the "MedView" WeChat Official Account. For other collaboration inquiries, please contact wuxi_media@wuxiapptec.com.
Disclaimer: The content team of WuXi AppTec focuses on introducing the research progress in global biopharmaceuticals and health. This article is for information exchange only, and the views expressed in the article do not represent the position of WuXi AppTec, nor does it mean that WuXi AppTec supports or opposes the views in the article. This article is not a recommendation for treatment plans. If you need guidance on treatment options, please visit a正规 hospital.
