Positive data reported for Trappsol Cyclo in patients 3 years and under with NPC1

Positive data reported for Trappsol Cyclo in patients 3 years and under with NPC1 | Image Credit: © ธนากร บัวพรหม - © ธนากร บัวพรหม - stock.adobe.com.

Trappsol Cyclo from Cyclo Therapeutics has demonstrated positive preliminary data from a phase 3 open-label sub-study among patients aged 3 years or younger with Niemann-Pick Disease Type C1 (NPC1), according to a press release from the company.¹

Results, presented at the 21 Annual WORLDSymposium in San Diego, California, are from the phase 3 global TransportNPC (NCT04860960) in patients aged 3 years and older. In addition, data from the sub-study, which enrolled 10 patients aged newborns to 3 years of age, was also presented.^1,2

The sub-study, which includes the youngest age subsets, also targets the visceral aspects of the disease. Doing this "may achieve its most optimal results when administered early in the disease course, thus having the potential of a preventative effect in overall symptom development," wrote Cyclo Therapeutics in the announcement.¹

A pair of patients in the sub-study discontinued the study after 48 weeks because of caregiver decision.

At baseline, sub-study patients had a mixture of mild to severe disease as determined by the Clinical Global Impression - Severity (CGI-S) scale. The company found that clinical improvements appeared to be best in patients with clinically mild to severe disease.

7 of 8 patients who reached 24 weeks, and 6 of 7 patients who reached 48 weeks, demonstrated stabilization or improvement in Clinical Global Impression - Change (CGI-C).

The profile for adverse effects is in line with prior findings from previous studies, including a double-blind phase 3 study running in parallel irrespective of age and disease severity.

"The preliminary findings from the TransportNPC sub-study are encouraging and further underscore the potential of Trappsol Cyclo to address the significant unmet medical need in the treatment of NPC," said Ronen Spiegel, MD, in a statement.

Spiegel is a clinical associate professor, director of Pediatric B Department, and Director Center of Rare Disease at Emek Medical Center. Spiegel also administered an oral presentation titled "Trappsol Cyclo: Open Label Treatment in the TransportNPC™ Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1."

"Slowing down disease progression through cholesterol mobilization is an important consideration for patients with established disease, who can expect to experience neurodegeneration without treatment," Spiegel added. "We look forward to gathering additional data from this ongoing study and establishing the effect of Trappsol Cyclo on progress of NPC.”

The ongoing TransportNPC study is a randomized, placebo-controlled, double-blind, parallel group, and multicenter study evaluating the safety, tolerability, and efficacy of 2000 mg / kg doses of Trappsol Cyclo in patients 3 years and older. It is administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1.

The rare, genetic disease causes cholesterol accumulation in cells, which leads to dysfunction, primarily of the liver, lung, spleen, brain, and can cause premature death. The 96-week study includes a 48-week comparative interim analysis.

According to Cyclo, if interim data at the 48-week mark meet statistical significance, the company intends to submit marketing applications for approval with the FDA and EMA.

"We continue to be encouraged by the data demonstrated by Trappsol Cyclo in the treatment of NPC," stated N. Scott Fine, CEO, Cyclo Therapeutics. "The growing body of data from our TransportNPC study and sub-study continue to demonstrate the potential of Trappsol Cyclo to address a devastating disease with significant unmet need. We look forward to our interim data readout from the TransportNPC study, which is expected in H1 2025 and ultimately providing a much needed treatment option for patients."

References:

1. Cyclo Therapeutics presents positive preliminary data from ongoing phase 3 TransportNPC open-label sub-study in patients (< 3 years old). Cyclo Therapeutics. Press release. February 7, 2025. Accessed February 7, 2025. https://www.businesswire.com/news/home/20250207991863/en/Cyclo-Therapeutics-Presents-Positive-Preliminary-Data-from-Ongoing-Phase-3-TransportNPC%E2%84%A2-Open-Label-Sub-Study-in-Patients-3-Years-Old

2. Phase 3 study to evaluate intravenous Trappsol Cyclo in pediatric and adult patients with Niemann-Pick Disease Type C1 (TransportNPC). ClinicalTrials.gov. Updated June 4, 2024. Accessed February 7, 2025.