Encouraging Results in Patients Under Three Years Old
Cyclo Therapeutics, Inc. (Nasdaq: CYTH), a clinical-stage biotechnology company dedicated to advancing treatments for rare diseases, has announced promising preliminary data from its ongoing Phase 3 TransportNPC™ open-label sub-study. These findings were presented at the 21st Annual WORLDSymposium™ 2025 in San Diego, California.
The study focuses on evaluating the efficacy of Trappsol® Cyclo™ in Niemann-Pick Disease Type C1 (NPC1), a rare genetic disorder that causes cholesterol accumulation in cells, leading to severe health complications. TransportNPC™ is the largest and most comprehensive controlled study for NPC1 to date, covering a global patient population.
Key Findings
- After 24 weeks, 87% (7 out of 8 patients) demonstrated either stabilization or improvement on the Clinical Global Impression – Change (CGI-C) scale.
- After 48 weeks, 86% (6 out of 7 patients) continued to show stabilization or improvement.
- The treatment has shown a consistent clearance of lipids both centrally and systemically, confirming prior findings from a Phase 1 study in adult patients.
- Clinical improvements were most notable in patients with mild to moderate NPC1 severity at baseline.
- The adverse event (AE) profile remains in line with previous studies, with the majority (77%) of AEs being mild and only one severe AE recorded. No serious adverse events (SAEs) were considered related to the study drug.
Implications for NPC1 Treatment
NPC1 primarily affects young children, progressively impairing neurological and systemic functions. The ability to stabilize or even improve symptoms in patients under three years old is a significant breakthrough. Early intervention with Trappsol® Cyclo™ could potentially slow disease progression and improve quality of life for patients and their families.
Dr. Ronen Spiegel, a leading researcher in the study, emphasized the potential impact: “Slowing down disease progression through cholesterol mobilization is crucial for patients with established NPC1. These early findings reinforce Trappsol® Cyclo™’s potential as a transformative treatment.”
Next Steps and Future Outlook
Cyclo Therapeutics is on track to release topline data from the 48-week interim analysis of 104 enrolled patients in H1 2025. If results meet statistical significance, the company plans to seek regulatory approval from both the FDA and EMA, potentially making Trappsol® Cyclo™ an approved treatment option for NPC1.
CEO N. Scott Fine reaffirmed the company’s commitment: “The data continues to show the potential of Trappsol® Cyclo™ to address a devastating disease with significant unmet medical need. We eagerly await the interim analysis and hope to bring a much-needed therapy to the NPC community.”
For more details on the TransportNPC™ Phase 3 study, visit ClinicalTrials.gov and reference identifier NCT04860960.
