Dimerix’s PARASOL Analysis Validates Proteinuria Endpoints at 104 Weeks
Dimerix’s latest PARASOL collaboration analysis reinforces proteinuria as a pivotal endpoint in their Phase 3 FSGS trial, setting the stage for FDA discussions on accelerated approval pathways.
- PARASOL data supports proteinuria endpoints at 104 weeks for ACTION3 study
- Potential link found between 12-month proteinuria levels and kidney failure risk
- Dimerix to engage FDA on accelerated approval strategies using early endpoints
- Blinded analysis of ACTION3 data pending to confirm statistical assumptions
- Collaboration with Amicus Therapeutics continues to shape US regulatory approach
Context of the PARASOL Collaboration
Dimerix Limited, an Australian biopharmaceutical company focused on kidney diseases, has announced the receipt of final data analysis from the PARASOL collaboration. This analysis, based on observational data from major renal registries, was designed to validate the use of proteinuria as a primary endpoint in their pivotal Phase 3 clinical trial, ACTION3, targeting focal segmental glomerulosclerosis (FSGS), a rare and serious kidney disorder.
The PARASOL collaboration’s findings align closely with earlier 2024 analyses, reinforcing the scientific rationale behind using proteinuria measurements at 104 weeks (two years) as a meaningful indicator of treatment efficacy. Proteinuria, the presence of excess protein in urine, is a key marker of kidney damage and disease progression in FSGS patients.
Implications for Regulatory Approval
One of the most significant outcomes from the PARASOL data is the observed potential relationship between proteinuria levels at 12 months and the subsequent risk of kidney failure. This insight opens the door for discussions with the US Food and Drug Administration (FDA) about the possibility of using earlier proteinuria endpoints to support accelerated approval pathways. Such pathways could allow Dimerix to bring their drug candidate, DMX-200, to market sooner, pending confirmatory data on kidney function over 24 months.
Dimerix and its US partner, Amicus Therapeutics, plan to engage with the FDA in the coming months to seek feedback on these findings before completing the blinded analysis of the ACTION3 trial data. This strategic approach aims to align on both traditional and accelerated approval endpoints, potentially expediting regulatory decisions.
The ACTION3 Study and Future Prospects
The ACTION3 trial is a randomized, double-blind, placebo-controlled Phase 3 study evaluating DMX-200 in FSGS patients already receiving standard angiotensin II receptor blocker therapy. The trial includes interim analyses designed to capture changes in proteinuria and kidney function, with the ultimate goal of generating robust evidence to support marketing approval.
While the detailed results of the PARASOL analysis remain confidential due to commercial sensitivities, the consistency of these findings with prior data provides a strong foundation for Dimerix’s regulatory strategy. The company’s leadership expressed optimism about the collaborative efforts and the statistical rigor underpinning the analysis, highlighting the role of Dr Abigail Smith from Northwestern University.
Looking beyond the US, Dimerix is also exploring alternative approval pathways in other key territories, working closely with commercial partners to maximize the potential reach of DMX-200.
Bottom Line?
Dimerix’s PARASOL data strengthens its regulatory case, but FDA feedback and blinded trial results will be pivotal next steps.
Questions in the middle?
- Will the FDA accept 12-month proteinuria endpoints for accelerated approval?
- How will the blinded ACTION3 data influence the final regulatory submission?
- What are the prospects for DMX-200 approval outside the US market?
