FDA Confirms Inclusion of Paediatrics in ACTION3 Study of DMX-200 for FSGS

Highlights:

• FDA confirms inclusion of adolescent children (12-17 years old) in ACTION3 global Phase 3 FSGS study is appropriate, increasing the total addressable market for DMX-200 if approved
• FSGS is one of the leading causes of kidney failure in children, with 20% of all child nephrotic syndrome cases caused by FSGS
• Dimerix is also in discussion with the European Medicines Agency (EMA) regarding a Paediatric Investigation Plan (PIP), which is a regulatory requirement prior to seeking market approval in Europe, the outcome of which is expected in calendar year 2023
• 90 adult patients currently recruited in ACTION3 phase 3 pivotal clinical trial of DMX-200 in the treatment of focal segmental glomerulosclerosis (‘FSGS’) kidney disease
• Part 1 interim analysis, which will assess proteinuria reduction of the first 72 patients on DMX-200 versus placebo at week 35, is anticipated in the latter half of calendar year 2023
• All activated sites will continue to recruit suitable patients for Part 2 of the ACTION3 trial
• Total global FSGS market was valued at US$12.6 billion in 2022 with a CAGR of 8.2%, driven by approximately 220,000 FSGS sufferers across the 7 major markets and premium orphan drug pricing
• DMX-200 has previously received Orphan Drug Designation with the FDA, EMA and UK, allowing potential fast track of commercialisation if successful