Rafael Holdings Announces Abstracts Accepted for Presentation at ICIEM 2025

Rafael Holdings subsidiary Cyclo Therapeutics has had two abstracts selected for presentation at the 15th International Congress of Inborn Errors of Metabolism (ICIEM) for its program evaluating Trappsol Cyclo for Niemann-Pick Disease Type C1 treatment. The abstracts focus on efficacy and safety data from 4 clinical studies and an ongoing expanded access program.

Rafael Holdings' subsidiary, Cyclo Therapeutics, has made significant strides in the development of Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1 (NPC1). The company recently had two abstracts selected for presentation at the 15th International Congress of Inborn Errors of Metabolism (ICIEM), highlighting the efficacy and safety data from four clinical studies and an ongoing expanded access program [1].

The abstracts focus on the therapeutic potential of Trappsol Cyclo, a cyclic heptasaccharide derivative, which has shown promise in preclinical studies for its ability to normalize intracellular cholesterol trafficking in NPC1 patients. The studies presented at ICIEM provide a comprehensive overview of the compound's effectiveness and safety profile in clinical settings.

One of the key findings from the studies is the potential of Trappsol Cyclo to improve survival rates in NPC1 model mice. The compound has demonstrated the ability to normalize intracellular cholesterol trafficking and reduce auditory dysfunction in healthy wild-type mice, indicating its potential to mitigate the neurological features of NPC1 [2].

However, the studies also highlight the importance of the diameter of the cyclodextrin (CD) ring in both therapeutic efficacy and ototoxicity. While Trappsol Cyclo has shown negligible ototoxicity, its efficacy is dependent on the ability of the CD ring to accommodate unesterified cholesterol (UC) molecules. The results suggest that the diameter of the CD ring is a critical factor in determining the therapeutic effects of these compounds [3].

The ongoing expanded access program is also a significant development, providing real-world evidence of Trappsol Cyclo's potential in treating NPC1 patients. This program allows for the compassionate use of the drug in patients who are not eligible for clinical trials, further validating its therapeutic potential [4].

In conclusion, Rafael Holdings' Cyclo Therapeutics is making significant progress in the development of Trappsol Cyclo for NPC1 treatment. The data presented at ICIEM underscores the compound's potential to improve survival rates and mitigate the neurological features of the disease. As the company continues to gather more data through clinical studies and expanded access programs, it is poised to make a significant impact on the treatment landscape for NPC1.

References:
[1] https://www.nature.com/articles/s41598-025-15599-0
[2] Cholesterol mobilization regulates dendritic cell maturation and the immunogenic response to cancer Article 21 January 2025
[3] The cholesterol transporter NPC1 is essential for epigenetic regulation and maturation of oligodendrocyte lineage cells Article Open access 05 July 2023
[4] Exploring the role of cyclodextrins as a cholesterol scavenger: a molecular dynamics investigation of conformational changes and thermodynamics Article Open access 08 December 2023