(RTTNews) - Cyclo Therapeutics, Inc. (CYTH) on Friday announced promising preliminary data from its ongoing Phase 3 TransportNPC study, evaluating Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1 or NPC1 in pediatric patients under the age of 3.
The study is the first to provide data on the treatment of this age group, a critical patient population suffering from a rare and progressive neurodegenerative disease.
The results show that a significant majority of patients in the study demonstrated stabilization or improvement in clinical assessments, suggesting the potential for Trappsol Cyclo to slow disease progression when administered early in the disease course.
Key Findings from the Preliminary Data:
-- At 24 weeks, 87 percent of patients showed stabilization or improvement on the Clinical Global Impression - Change or CGI-C scale.
-- At 48 weeks, 86 percent of patients demonstrated stabilization or improvement in the CGI-C scale.
-- Clearance of lipids was consistently observed both centrally and systemically, with the treatment showing promise for addressing the visceral aspects of NPC1.
-- Adverse events or AEs were generally mild or moderate, consistent with findings from earlier studies, with 77% of events being mild and 22 percent moderate.
-- Of 107 reported AEs, only 1 severe adverse event was recorded, and no serious adverse events or SAEs were deemed related to the study drug.
The safety profile of Trappsol Cyclo aligns with previous studies, with the majority of AEs being mild or moderate in nature.
Importantly, the treatment was well tolerated, and no SAEs were linked to the study drug.
These results build on the growing body of data supporting Trappsol® Cyclo as a potentially effective treatment for NPC1, particularly in its early stages. The company continues to collect data from the study, with topline results expected in H1 2025.
Cyclo Therapeutics plans to submit marketing applications for Trappsol Cyclo based on the 48-week interim data, pending statistical significance.
The ongoing study represents a key milestone in the development of treatments for NPC1, a rare genetic disease with no approved therapies.
The company remains optimistic about the potential of Trappsol Cyclo to provide a much-needed treatment option for patients with NPC1 and will continue to monitor the efficacy and safety of the treatment through its ongoing trials.
Cyclo Therapeutics is also progressing with regulatory submissions for the drug in multiple regions, including the U.S. and EU, and remains committed to advancing treatments for rare and challenging diseases.
Currently, CYTH is trading at $0.98 up by 28.57%.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
