Dimerix Limited has achieved a significant milestone in its ACTION3 Phase 3 clinical trial for DMX-200, a drug targeting the rare kidney disease focal segmental glomerulosclerosis (FSGS), with the opening of its first pediatric site in Mexico. This allows for the active recruitment of adolescent patients into the study, marking a crucial step in addressing a significant unmet need in pediatric kidney disease.
Pediatric Site Activation and Dosage Confirmation
The newly activated clinical site specializes in pediatric kidney disease. This advancement follows confirmation from the Independent Data Monitoring Committee (IDMC), the US FDA, and the EMA, that the dosage of DMX-200 for adolescent patients (12-17 years) will be the same as that for adults (120 mg twice daily). This decision was based on a review of interim safety and pharmacokinetic data from the adult cohort of the ACTION3 trial.
FSGS is a leading cause of kidney failure in children, accounting for 20% of Nephrotic Syndrome cases in pediatric patients. The ACTION3 trial plans to include approximately 15 specialist pediatric kidney centers across the UK, US, Mexico, Brazil, and Argentina, aiming to recruit around 22 adolescent patients with FSGS.
Trial Design and Potential Market Expansion
Adolescent patients in the ACTION3 Phase 3 clinical trial will be randomized to receive either DMX-200 or a placebo, with blinding maintained throughout the two-year study period. Successful outcomes in the pediatric cohort could pave the way for DMX-200's approval and marketing to adolescents in key regions, including the US and Europe, contingent on adult approval.
Recruitment and Financial Stability
Currently, 116 of the targeted 144 patients for Part 2 of the study have been randomized, keeping recruitment on schedule. Dimerix anticipates an increase in recruitment rates with the activation of approximately 100 new trial sites, bringing the total to around 170 globally. The company's financial position remains robust, with $22 million in cash reserves as of the end of the June quarter, supplemented by an expected $8 million R&D tax incentive cash rebate and potential proceeds from option exercises.
Appointment of Dr. Howard Trachtman
To further support the pediatric focus of the ACTION3 study, Dimerix has appointed Dr. Howard Trachtman, an expert pediatric nephrologist, to its medical advisory board. Dr. Trachtman's extensive experience, including his roles as chief of pediatric nephrology at Cohen Children’s Medical Center and NYU Langone Health, and his involvement in numerous clinical trials in FSGS, will be invaluable to the study. He has co-authored over 195 peer-reviewed articles on kidney disease in the past 15 years.
Expert Commentary
Dr. David Fuller, Chief Medical Officer at Dimerix, emphasized the significance of this milestone, stating, "The initiation of the first paediatric site in Mexico marks a new milestone for the ACTION3 study." Dr. Trachtman added that FSGS remains a significant unmet need with poor outcomes in both children and adults, noting, "There are still no approved therapies to delay or prevent progression to kidney failure... Based on data to date, it is a drug candidate that is certainly worth testing in all patients with FSGS."
