Posted: 4 July 2023
Dimerix Limited (ASX: DXB), a biopharmaceutical company with a Phase 3 clinical study in inflammatory disease, today confirmed that the Chinese regulatory agency, National Medical Products Administration (NMPA) has provided a written response on the proposed Focal Segmental Glomerulosclerosis (FSGS) Phase 3 study design. The NMPA reviewed a dossier that summarised Dimerix’ proposed Phase 3 clinical program for FSGS and its supporting data in the form of non-clinical studies, the manufacturing and process controls and all existing Phase 1 and Phase 2 renal clinical data accumulated to date. The written response from the NMPA acknowledged that the overall ACTION3 design was acceptable for China, and that Dimerix may not be required to conduct any further clinical studies before proceeding with the ACTION3 study in the Chinese population.
“With over 100,000 patients estimated to be diagnosed in China3, and no approved treatments, there is very high unmet medical need for new therapeutic options for this disease, which makes studies like this one critical. This formal response from the NMPA is the first step towards ensuring that the proposed FSGS development program meets with Chinese regulatory expectations for clinical trial and marketing approval, in addition to that feedback we have already received from the US and Europe.” Dr Nina Webster, CEO & Managing Director, Dimerix
The ACTION3 Phase 3 trial is actively recruiting across clinical sites globally, including in Taiwan and Hong Kong. Once patients have successfully completed the background medication stabilisation period and subsequent re-screening, they are then randomised to receive either drug or placebo. The trial continues to recruit patients for any screen failure, drop out or do not comply with the clinical trial protocol and to support Part 2 of the trial. Those patients who fail the inclusion/exclusion criteria during this screening and stabilisation process are not dosed.
The single Phase 3 trial in FSGS patients has two interim analysis points built in that are designed to capture evidence of proteinuria and kidney function (eGFR slope) during the trial, aimed at generating sufficient evidence to support potential accelerated marketing approval. Part 1 interim analysis of the trial data will conclude once 72 patients have completed 35 weeks treatment, anticipated Q1 2024.
The Phase 3 trial, 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), multicentre, randomised, double-blind, placebo-controlled trial of the efficacy and safety of DMX-200 in patients with FSGS who are receiving a stable dose of an angiotensin II receptor blocker (ARB). Once the ARB dose is stable, patients, aged 18 to 80 years (broadening to 12 to 80 years following first successful interim analysis4), will be randomized to receive either DMX-200 (120 mg capsule twice daily) or placebo.
About FSGS
FSGS is a rare disease that attacks the kidney’s filtering units, where blood is cleaned (called the ‘glomeruli’), causing irreversible scarring. This leads to permanent kidney damage and eventual endstage failure of the organ, requiring dialysis or transplantation. For those diagnosed with FSGS the prognosis is not good. The average time from a diagnosis of FSGS to the onset of complete kidney failure is only five years and it affects both adults and children.5 For those who are fortunate enough to receive a kidney transplant, approximately 60% will get re-occurring FSGS in the transplanted kidney.6 At this time, there are no drugs specifically approved for FSGS anywhere in the world, so the treatment options and prognosis are poor.
FSGS is a billion-dollar plus market: the number of people with FSGS in the US alone is just over 80,000,1 and across the seven major markets about 220,000.2 The illness has a global compound annual growth rate of 8%, with over 5,400 new cases diagnosed in the US alone each year.1 Because there is no effective treatment, Dimerix has received Orphan Drug Designation for DMX-200 in both the US and Europe for FSGS.7 Orphan Drug Designation is granted to support the development of products for rare diseases and qualifies Dimerix for various development incentives including: seven years (FDA) and ten years (EMA) of market exclusivity if regulatory approval is received, exemption from certain application fees, and a fast-tracked regulatory pathway to approval. Dimerix reported positive Phase 2a data in FSGS patients in July 2020.
