Trappsol Cyclo Demonstrates Significant Benefit in Prespecified NPC Analysis

Rafael Holdings reported topline results from the pivotal Phase 3 TransportNPC study, evaluating Trappsol Cyclo in patients with Niemann-Pick Disease Type C (NPC).

Niemann-Pick Disease Type C (NPC) is a rare, progressive genetic disorder affecting neurological function and other organ systems, associated with significant disability and shortened survival.

Trappsol Cyclo, an investigational intravenous hydroxypropyl-ß-cyclodextrin, is being developed for the treatment of NPC. The Phase 3 study enrolled 94 patients aged 3 to 70 years, who received Trappsol Cyclo or placebo every two weeks for 96 weeks.

Trappsol Cyclo demonstrated a directionally favorable treatment effect on the primary endpoint, showing a 64% reduction in disease progression versus placebo at Week 96; however, the result did not reach statistical significance (p=0.19).

In a prespecified analysis of 78 patients receiving background miglustat and/or leucine, Trappsol Cyclo demonstrated a statistically significant 71% reduction in disease progression versus placebo at Week 96 (p=0.046).

Trappsol Cyclo was generally well tolerated over the 96-week treatment period, with no new safety signals observed. Treatment-emergent adverse events occurred in 93.8% of patients receiving Trappsol Cyclo, compared with 100% in the placebo group.

A separate analysis comparing infantile-onset NPC patients treated with Trappsol Cyclo against matched external controls from the International Niemann-Pick Disease Registry showed an 85% reduction in mortality risk.

Rafael indicated it remains on track to submit a New Drug Application (NDA) for Trappsol Cyclo to the FDA in the fourth quarter of 2026.

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