Exclusive US License Deal Puts Dimerix’s Kidney Drug on Path to FDA Approval

• Exclusive US commercialization rights for DMX-200 licensed to Amicus Therapeutics
• Dimerix receives $30 million upfront plus up to $560 million in milestone payments
• FDA aligns on proteinuria as primary endpoint for DMX-200 approval in FSGS
• DMX-200 Phase 3 ACTION3 trial progressing with full enrollment expected by end 2025
• Amicus responsible for US regulatory submission and commercialization costs

Strategic US Licensing Deal

Dimerix Limited (ASX: DXB), a clinical-stage biopharmaceutical company focused on rare kidney diseases, has taken a significant step forward by entering into an exclusive license agreement with Amicus Therapeutics (Nasdaq: FOLD) for the US commercialization of its lead drug candidate, DMX-200. This deal grants Amicus exclusive rights to develop and market DMX-200 in the United States for all indications, including Focal Segmental Glomerulosclerosis (FSGS), a rare and often fatal kidney disease with no FDA-approved therapies.

The agreement includes a substantial upfront payment of US$30 million (approximately AU$48 million) to Dimerix, alongside potential milestone payments totaling up to US$560 million (around AU$892 million), plus tiered royalties on net US sales. This infusion of capital and partnership with a seasoned rare disease specialist marks a pivotal moment for Dimerix’s clinical and commercial ambitions.

Clinical Progress and Regulatory Alignment

DMX-200 is currently in a pivotal Phase 3 trial named ACTION3, designed to evaluate its efficacy in treating FSGS patients who are already on standard angiotensin II receptor blocker therapy. Interim results released earlier in 2024 showed promising reductions in proteinuria, a key marker of kidney damage, with no significant safety concerns.

Importantly, Dimerix recently completed a Type C meeting with the FDA, securing agreement that proteinuria will serve as the primary endpoint for traditional marketing approval. This regulatory alignment is crucial, as it provides a clear pathway toward potential US approval and commercialization.

Division of Responsibilities and Future Prospects

Under the terms of the deal, Dimerix will continue to fund and conduct the ACTION3 trial, while Amicus will take charge of regulatory submissions, maintenance of the US regulatory dossier, and all commercialization activities within the US market. The companies will collaborate through a Joint Steering Committee to ensure coordinated development and commercialization strategies.

Beyond FSGS, Amicus holds exclusive rights to develop DMX-200 for additional indications in the US, potentially expanding the drug’s market reach. Dimerix retains all rights outside the US, preserving its global development and commercial opportunities.

Market and Patient Impact

FSGS affects an estimated 40,000 people in the US alone, with limited treatment options and a high risk of progression to end-stage kidney disease. The urgent need for effective therapies makes DMX-200’s progress particularly noteworthy. Amicus’s expertise in rare diseases and established commercial infrastructure could accelerate patient access to this innovative treatment.

Both companies expressed optimism about the partnership. Dr Nina Webster, CEO of Dimerix, highlighted the alignment of expertise and resources, while Amicus CEO Bradley Campbell emphasized their commitment to delivering transformative medicines to rare disease communities.

Bottom Line?

This deal not only validates DMX-200’s potential but also sets the stage for a high-stakes race to address a critical unmet need in kidney disease.

Questions in the middle?

• Will the ACTION3 trial meet its primary endpoint and trigger milestone payments?
• How will Amicus’s commercialization strategy impact DMX-200’s market penetration in the US?
• What are the prospects and timelines for expanding DMX-200 into additional indications?