Posted: 6 May 2024
Dimerix Limited (ASX: DXB), a clinical-stage biopharmaceutical company with late-stage clinical assets, today announced that the United Kingdom (UK) Medicines and Healthcare products Regulatory Agency (MHRA) has accepted its Paediatric Investigation Plan (PIP) for the development of DMX-200 for focal segmental glomerulosclerosis (FSGS). The Company’s ACTION Phase 3 study will now be opened up to include children from ages 12-17 in the UK.
A successful clinical trial in paediatrics would allow Dimerix to file for an extension to any market authorisation (MAA) in UK, thereby potentially expanding market access in a paediatric population where no treatment for FSGS exists and associated healthcare costs are high. The approved PIP in the UK aligns with the previously approved European EMA PIP2 and the US FDA’s advice1. FSGS is one of the leading causes of End Stage Renal Disease (ESRD) in children and is associated with up to 20% of all new cases of Nephrotic Syndrome in children each year.4
A PIP in the UK is a mandatory development plan aimed at ensuring that the necessary data are obtained to support the registration and use of new medicines for children in the UK. The PIP addresses the entire paediatric development program for DMX-200 in FSGS and provides a clear framework for the development and registration of DMX-200 for paediatric patients with FSGS in the UK. This positive and final opinion from the MHRA ensures that the Company’s clinical studies, including its ACTION Phase 3 clinical study of DMX-200 in FSGS, will be run in accordance with the MHRA’s expectations to allow for potential future product approval in children should it be successful.
The Company’s UK PIP covers all studies needed for registration of DMX-200 in the paediatric population and no safety concerns were raised. The safety package was accepted, and no further nonclinical studies are required. Paediatric patients from 12 to 17 years of age will now be included in the current ACTION Phase 3 study for patients with FSGS in the UK. In addition, in silico modelling, simulation and extrapolation of paediatric data from the ACTION Phase 3 study will also be used to support a confirmatory small open-label study in paediatric patients from 1 to 11 years of age.
The Phase 3 study, which is titled “Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis”, or ACTION3 for short, is a pivotal (Phase 3), multi-centre, randomised, double-blind, placebo-controlled study of the efficacy and safety of DMX200 in patients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB). Once the ARB dose is stable, patients will be randomized to receive either DMX200 (120 mg capsule twice daily) or placebo. All patients in the study are also eligible for enrolment in an open-label extension study for a further 2 years of treatment with DMX-200.
Further information about the study can be found on ClinicalTrials.gov (Study Identifier: NCT05183646) or Australian New Zealand Clinical Trials Registry (ANZCTR) (Study Identifier ACTRN12622000066785).
