BLINDED ASSESSMENT OF ACTION3 STATISTICAL ASSUMPTIONS COMPLETE
BLINDED ASSESSMENT OF ACTION3 STATISTICAL ASSUMPTIONS COMPLETE |
| [27-April-2026] |
Key Highlights:
Study Progress:
MELBOURNE, Australia, April 28, 2026 /PRNewswire/ -- Dimerix Limited (ASX: DXB), a biopharmaceutical company with a Phase 3 clinical asset in kidney disease, today announced that the blinded review of ACTION3 Phase 3 study data to confirm statistical assumptions of the primary endpoint has been successfully completed. This blinded review confirms that the ACTION3 study remains appropriately statistically powered to detect the treatment effect for the proteinuria primary endpoint. Having the ACTION3 Phase 3 study with >90% statistical power for its primary endpoint means that if DMX-200 continues to reduce proteinuria in trial patients as anticipated, then there is a >90% chance that the study will successfully show a statistically significant proteinuria treatment effect at the trial's conclusion. Evolving FSGS landscape knowledge guides decision making In parallel to the blinded review, Dimerix and its commercial partners assessed the changes to the Focal Segmental Glomerulosclerosis (FSGS) landscape and potential endpoints since initiation of the ACTION3 study in 2022, all of which support proteinuria as the primary endpoint. Specifically, the PARASOL working group outcomes provided evidence of proteinuria as a candidate surrogate endpoint for FSGS; FDA provided positive feedback to Dimerix confirming that the proteinuria endpoint is appropriate for the full approval of DMX-200; and, earlier this month, FDA approved a new treatment for FSGS based on proteinuria as an endpoint. A key finding from PARASOL working group analysis was that proteinuria required smaller sample sizes and demonstrated less variability than eGFR for FSGS clinical trials and could therefore reduce risk for potential downstream marketing approval in the US. Collectively, the PARASOL working group analysis, the National Registry of Rare Kidney Diseases UK (RaDaR) analyses,[4] Kaiser Permanente analyses,[5] third party FSGS study data, and FSGS key opinion leaders all suggest that measuring proteinuria change from baseline is a far more statistically achievable endpoint for DMX‑200 than eGFR endpoints and may substantially reduce the risk for traditional approval. Given that any application for accelerated approval based on proteinuria would likely require that the ACTION3 use an eGFR-based confirmatory endpoint, which would increase costs, lengthen study timeline and necessitate the splitting of alpha,[6] potentially reducing the probability of success for the final endpoint, Dimerix and its commercial partners have concluded that conducting an interim analysis required for an Accelerated Approval application would not now be the most strategic and optimal course for the trial. Proteinuria as primary endpoint may substantially reduce ACTION3 execution risk The FDA has previously confirmed that the proposed primary endpoint of percent reduction in proteinuria compared to placebo is suitable to support traditional approval of DMX-200 via the 505(b)(1) pathway, should the findings of the ACTION3 be positive, with change in eGFR as a secondary endpoint. "The understanding of the FSGS disease and appropriate surrogate clinical endpoints has evolved significantly since the initiation of the ACTION3 study and justified a comprehensive review of all considerations. The conclusion of this review has resulted in a strategic decision being made and is a good outcome for Dimerix and our partners, as it provides much needed clarity and insights on the DMX-200 regulatory pathway in our major commercial territories. By focussing on proteinuria, it significantly reduces the clinical and commercial risk for traditional approval. Given the successful blinded interim futility analysis in 2024, we know that DMX-200 was performing better than placebo in reducing proteinuria at that point in time, and with seven independent data monitoring committee reviews completed, we are confident that DMX-200 is well tolerated. The outcome of this review and the decision to proceed to completion of ACTION3 to support traditional approval helps us strengthen the probability of ACTION3 success and ultimately deliver a potential new therapy for patients with FSGS while also reducing risk and adding value for Dimerix shareholders and partners." Dr David Fuller, Chief Medical Officer, Dimerix "The move to having proteinuria as the ACTION3 primary endpoint for traditional approval increases the likelihood of a successful marketing approval and avoids the very real risks associated with use of eGFR endpoints. The required additional activities, cost and increased risk associated with accelerated approval would not be value generating in our view." Dr Jeff Castelli, Chief Development Officer, Amicus Therapeutics About FSGS Phase 3 Study The ACTION3 Phase 3 study is a pivotal Phase 3, multi-centre, randomised, double-blind, placebo-controlled study of the efficacy and safety of DMX-200 in patients with FSGS who are receiving a stable dose of a blood pressure medication known as an angiotensin II receptor blocker (ARB). Once the ARB dose is stable, patients are then randomised to receive either DMX-200 (120 mg capsule, twice daily) or placebo for a 2-year treatment period. The single Phase 3 trial in FSGS patients is designed to capture evidence of proteinuria reduction and kidney function (eGFR slope) during the trial, aimed at generating sufficient evidence to support marketing approval. 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). For further information, please visit our website at www.dimerix.com or contact:
Follow Dimerix on LinkedIn and X Authorised for lodgement by the Board of Dimerix —END— About Dimerix Limited DMX-200 is a chemokine receptor (CCR2) antagonist administered to patients already receiving an angiotensin II type I receptor (AT1R) blocker, the standard of care treatment for hypertension and kidney disease. DMX-200 is protected by granted patents in various territories until 2032, with patent applications submitted globally that may extend patent protection to 2045, in addition to Orphan Drug Designation granted in the United States, Europe, UK and Japan[7]. For more information, please visit the company's website at www.dimerix.com and follow on X and LinkedIn. About FSGS FSGS is a rare, serious kidney disorder characterised by progressive scarring (sclerosis) in parts of the glomeruli—the kidney's filtering units. This scarring leads to proteinuria, progressive loss of kidney function, and often end-stage renal disease. FSGS is increasingly understood to have an inflammatory component, with monocyte and macrophage activation contributing to glomerular injury. In the United States, more than 40,000 people are estimated to be living with FSGS, including both adults and children.[8] There are no therapies specifically approved for FSGS in the U.S., and disease management relies on non-specific immunosuppressive and supportive therapies. In patients with progressive or treatment-resistant FSGS, the average time from diagnosis to end-stage kidney disease can be as short as five years. Even among those who undergo kidney transplantation, disease recurrence occurs in up to 60% of cases,[9] underscoring the urgent need for new, disease-modifying treatments. Dimerix Forward Looking Statement This release includes forward-looking statements that are subject to risks and uncertainties. Although management believes that the expectations reflected in the forward-looking statements are reasonable at this time, Dimerix can give no assurance that these expectations will prove to be correct. Readers are cautioned not to place undue reliance on forward-looking statements. Actual results could differ materially from those anticipated. Reasons may include risks associated with drug development and manufacture, risks inherent in the regulatory processes, delays in clinical trials, results of clinical trials, contractual risks, risks associated with patent protection, future capital needs or other general risks or factors, including but not limited to those factors outlined in the most recent Dimerix Limited Annual Report. References
SOURCE Dimerix | ||||||||||||||
Company Codes: Australia:DXB |
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