Dimerix has reached a significant milestone in its Phase III ACTION3 clinical trial for DMX-200, a treatment for the rare kidney disease focal segmental glomerulosclerosis (FSGS). The company has recruited its first paediatric patient for the trial at a specialist site in Manchester, UK.
The ACTION3 trial, which is examining the efficacy and safety of DMX-200 in treating FSGS, will involve 19 specialist paediatric kidney centres across the UK, USA, Mexico, and Argentina. These sites will focus on recruiting patients aged 12-17 to gather data supporting the potential marketing approval of DMX-200 for paediatric use.
A Key Development for Paediatric Kidney Disease Treatment
Dimerix aims to collect sufficient data to secure marketing approval for DMX-200 in children over the age of one. The company plans to enroll around 170 patients in the global trial, with approximately 19 of those sites dedicated to paediatric participants. The paediatric cohort will be randomised to receive either a placebo or DMX-200 (120mg twice daily) and will be followed for two years.
The dosage for paediatric patients will match the adult dose, which has already been established for the ongoing adult cohort. The Independent Data Monitoring Committee (IDMC) has reviewed the safety margin for DMX-200 and confirmed that the trial can proceed with the adult dose for children.
Tackling FSGS: A Rare and Devastating Kidney Disease
FSGS is a serious kidney disorder that damages the kidney’s filtering units, leading to permanent kidney failure and often requiring dialysis or a transplant. Currently, there are no drugs specifically approved to treat FSGS, creating a major gap in treatment options.
DMX-200 has received Orphan Drug Designation in the US and Europe, as well as the Innovative Licensing and Access Pathway (ILAP) designation in the UK, due to its potential to address this unmet medical need.
FSGS is one of the leading causes of kidney failure in children, accounting for 20% of nephrotic syndrome cases in paediatric patients. If successful, DMX-200 could provide a much-needed treatment option for this vulnerable population.
Expansion of DMX-200’s Approval to Paediatric Populations
Should the trial prove successful for the paediatric cohort, Dimerix intends to expand DMX-200’s approval to include children in key markets such as the US and Europe. The company’s paediatric development plan aims to meet the regulatory requirements of the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to secure approval for children aged one and older.
The study design includes in silico modelling and extrapolation of paediatric data to support a confirmatory open-label study for younger children aged one to 11. An interim analysis is scheduled for August 2025, which will review data from the first 144 patients in the trial.
Next Steps for ACTION3 and Dimerix’s Vision
The ACTION3 trial, which stands for Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis, is a multi-centre, double-blind, placebo-controlled study. It evaluates the effectiveness of DMX-200 in combination with angiotensin II receptor blockers (ARBs) in FSGS patients.
The trial has successfully passed its first interim analysis, demonstrating positive results with proteinuria efficacy. Dimerix anticipates further data collection and analysis points in August 2025, which will be critical in determining the drug’s potential for regulatory approval.
Dimerix’s chief medical officer, Dr. David Fuller, highlighted the significance of recruiting the first paediatric patient, emphasizing that the trial is entering an exciting phase. The company is optimistic about the progress toward full recruitment and the upcoming interim analysis.
The recruitment of the first paediatric patient is a crucial step in Dimerix’s goal to offer a new treatment option for children suffering from FSGS and expand the potential for DMX-200’s approval in both paediatric and adult populations worldwide.
Related Topics
