Drug Farm's ALPK1 Inhibitor DF-003 Granted FDA Rare Pediatric Disease Designation for ROSAH Syndrome

Jan 17, 2024 16:56 CST Updated 16:56

Drug Farm

Innovative Therapies Researcher for Infectious and Immune Diseases

▎Edited by the WuXi AppTec content team

On January 16, Drug Farm announced that the U.S. FDA has granted the companyALPK1 Inhibitor New DrugDF-003Pediatric Rare Disease Qualification, used for treatmentROSAH（Retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and headache）SyndromePatient. Public information shows that DF-003 has been approved for clinical trials by the FDA in July 2023.

ROSAHSyndromeIt is a rare autosomal dominant hereditary autoinflammatory disease, named according to the characteristic symptoms exhibited by affected patients.. The disease isByALPK1Caused by highly active mutations in genetic functionThe most common symptom is progressive vision loss, typically beginning before the age of 20. Ophthalmic examination usually shows 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.。

According to the press release from Drug Farm, DF-003 is an orally administered, potent, and highly selective ALPK1 kinase inhibitor.ALPK1 is a key target in regulating inflammatory pathways in cardiac and renal pathology。DF-003 can inhibit ALPK1 and cause pathogenic effects in ROSAH patientsALPK1RelatedT237MDriver Mutation. The drug has shown promising results in animal models of heart and kidney diseases, as well as in the ROSAH transgenic mouse model.Significant Preclinical ActivityIn addition, DF-003 has good drug-like properties, including a lower risk of off-target effects and drug interactions, as well as a half-life suitable for once-daily dosing in humans.

ClinicalTrialsThe official website shows that DF-003 is undergoing a Phase 1 clinical trial to evaluate its safety and pharmacokinetics in healthy volunteers.

Drug Farm Chief Medical Officer Dr. Jeysen Yogaratnam commented: "Pediatric patients with ROSAH syndrome have significant unmet clinical needs, with limited treatment options for vision loss. The Rare Pediatric Disease designation recognizes the clinical necessity of addressing the severe and debilitating complications of this rare disease and supports our goal of developing DF-003 as the first targeted therapy to treat patients with ROSAH syndrome."

References:

[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

[2] Drug Farm's ALPK1 Inhibitor DF-003 Successfully Completes First Subject Dosing in Phase I Clinical Trial. Retrieved Sep 19 , 2023, from https://mp.weixin.qq.com/s/QrB8sDWfc3tOSW8yZn449g

[3] Approved! U.S. FDA Approves Drug Farm's First-in-class ALPK1 Kinase Inhibitor DF-003 Clinical IND, Phase I Clinical Trial to Begin Soon. Retrieved Jul 3, 2023, from https://mp.weixin.qq.com/s/BzaPGGk4x9sq2A2-8m9IaA

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