Rafael drug misses primary endpoint in rare disease trial

NEWARK, N.J. - Rafael Holdings Inc. reported that its experimental drug Trappsol Cyclo did not meet the primary goal in a Phase 3 trial for a rare genetic disease, Niemann-Pick Disease Type C. The TransportNPC study included 94 patients aged 3 to 70, who received either Trappsol Cyclo or a placebo for a year. The trial assessed the 4-domain NPC Clinical Severity Scale at the trial's end.

While Trappsol Cyclo showed a favorable direction, it failed to show statistical significance (p=0.19). However, when comparing patients on background medications, Trappsol Cyclo demonstrated a statistically significant effect (p=0.046). Serious side effects were reported more frequently in the treatment group (35.9%) compared to the placebo group (16.7%).

Hearing issues were also more common in the treatment group (15.6%) than the placebo group (13.3%). Rafael Holdings plans to submit a New Drug Application to the FDA in late 2026.