Rafael Holdings Falls 32% Premarket as Phase 3 NPC Trial Misses Statistical Significance

Rafael Holdings (RFL) shares plunged 31.68% to $1.38 in premarket trading on September 30, 2026, after the company reported that its Phase 3 TransportNPC study of Trappsol Cyclo in Niemann-Pick Disease Type C missed statistical significance on the primary endpoint. The trial enrolled 94 patients aged 3 to 70 who received Trappsol Cyclo or placebo every two weeks for 96 weeks. The drug achieved a 64% reduction in disease progression versus placebo, but the result was not statistically significant (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 (p=0.046). A separate comparison of infantile-onset patients against external registry controls showed an 85% reduction in mortality risk. Adverse events occurred in 93.8% of Trappsol Cyclo patients versus 100% for placebo, with no new safety signals. Rafael said it remains on track to submit an NDA to the FDA in the fourth quarter of 2026. RFL closed Tuesday at $2.03, up 0.50%, and has traded between $1.12 and $2.33 over the past year.