Rafael Holdings, Inc. announced topline efficacy and safety data from the pivotal Phase 3 TransportNPC study (CTD-TCNPC-301) evaluating Trappsol Cyclo (intravenous hydroxypropyl-beta-cyclodextrin) in patients with Niemann-Pick Disease Type C (NPC). The study enrolled 94 patients, ranging in age from 3 to 70 years, who were randomized to receive 2000 mg/kg of Trappsol Cyclo intravenously every two weeks for a 96-week period or placebo.

The primary endpoint measured the change in the 4-domain NPC Clinical Severity Scale (4DNPCSS) at Week 96. The data showed a directionally favorable treatment effect, indicating a 64% slowing of disease progression, but the result did not achieve statistical significance (p=0.19).

A prespecified analysis of the 83% of the study population receiving background miglustat and/or leucine therapy demonstrated a statistically significant and clinically meaningful treatment benefit. In this subgroup, Trappsol Cyclo indicated a 71% slowing of disease progression compared to placebo (p=0.046).

Regarding safety, the treatment was well tolerated over the 96-week period. Treatment-emergent adverse events (TEAEs) occurred in 93.8% of the Trappsol Cyclo group and 100% of the placebo group. Serious TEAEs occurred more frequently with Trappsol Cyclo (35.9%) than placebo (16.7%), though treatment-related serious events were reported at similar rates (3.1% vs. 3.3%). One treatment-related event of bilateral deafness was reported.

Rafael Holdings also presented a separate overall survival analysis comparing patients with infantile-onset NPC treated with Trappsol Cyclo to external controls from the International Niemann-Pick Disease Registry. The analysis indicated an 85% reduction in the risk of mortality compared to matched INPDR controls (HR=0.154; p=0.044). A secondary analysis with expanded historical controls showed a 94% reduction in the risk of mortality (HR=0.057; p=0.0008).

Based on a previously announced pre-NDA meeting with the FDA, the Company remains on track to submit a New Drug Application (NDA) for Trappsol Cyclo in the fourth quarter of 2026. The Company plans to host an investor conference call and webcast on September 30, 2026, at 8:30 a.m. ET.