Cyclo Therapeutics Community Update | June 18, 2025
- ANPDF
- 7 days ago
- 2 min read
June 18, 2025 – Newark, NJ
Rafael Holdings has announced that an independent Data Monitoring Committee (DMC) has completed its scheduled 48-week interim review of the Phase 3 TransportNPC™ trial for Trappsol® Cyclo™, a treatment candidate for Niemann‑Pick Disease Type C1 (NPC1), a rare and progressive neurodegenerative disorder [1].
🎯 Key Takeaways:
DMC Verdict: The trial has been approved to continue, with no safety concerns or futility signals identified in the interim data [1].
Safety Profile: Trappsol® Cyclo™, an intravenous formulation of hydroxypropyl‑beta‑cyclodextrin, continues to be well-tolerated, with adverse events consistent with previous Phase 1/2 and extension studies [1].
Next Milestone: The study progresses toward its 52-week endpoint, with topline results expected in mid‑2025 [1].
🔬 Trial Overview:
TransportNPC™ is a randomised, double-blind, placebo-controlled, multi-centre Phase 3 study enrolling both children and adults with confirmed NPC1. It is designed to assess whether Trappsol® Cyclo™ can slow disease progression and improve patient outcomes [1].
📊 What This Means:
Continued trial progression indicates promising interim performance, reassuring stakeholders and patient communities alike [2].
The study’s advancement supports future regulatory submission for a condition with significant unmet medical need [2].
Context & Broader Momentum
Merger strengthened focus: In March 2025, Rafael Holdings completed a strategic merger with Cyclo Therapeutics, acquiring Trappsol® Cyclo™ as its lead clinical asset [3].
Capital raised: A $25 million rights offering closed in early June 2025, securing necessary funding for the trial’s completion and potential commercial preparation [4].
What’s Next?
Final 52‑week data is expected in mid‑2025, covering both safety and efficacy.
Positive results could support future regulatory filings for the first disease-modifying NPC1 therapy.
The company’s ongoing momentum may lead to a significant milestone for the rare disease community [2].
To join our fight, as well as fund vital research for a cure, support the Australian NPC Disease Foundation in its goals of "Research. Cure. Persevere."
Ways to support NP-C in Australia:
Shop at our online NPC store
Fundraise your way! Check our free resources and inspo online.
References:
Rafael Holdings. (2025, June 18). Rafael Holdings Announces Continuation of its Phase 3 Study for the Treatment of Niemann-Pick Disease Type C1 (NPC1) Following Independent Data Monitoring Committee (DMC) Review of Prespecified 48-Week Interim Analysis. https://rafaelholdings.irpass.com
MarketScreener. (2025, June 12). Rafael Holdings: Financial and Clinical Pipeline Update. https://www.marketscreener.com
GlobeNewswire. (2025, March 26). Rafael Holdings Completes Merger with Cyclo Therapeutics Following Shareholder Approvals. https://www.globenewswire.com
GlobeNewswire. (2025, June 11). Rafael Holdings Reports Third Quarter Fiscal 2025 Financial Results. https://www.globenewswire.com
Comments