Rafael Holdings Announces Topline Results from the Pivotal Phase 3 TransportNPC™ Study of Trappsol® Cyclo™ in Niemann-Pick Disease Type C (NPC)
Globe Newswire•30/09/2026•07:00 ET
0
Key Highlights
- ➤Primary endpoint missed significance: 4DNPCSS difference −0.81 points, p=0.19
- ➤Background therapy analysis significant: 71% slower progression, difference −1.11 points, p=0.046
- ➤Survival analysis showed 85% lower mortality risk versus INPDR controls, HR=0.154
- ➤Rafael Holdings (RFL) remains on track for Q4 2026 NDA submission
- ➤Trappsol Cyclo safety profile generally tolerated; serious TEAEs occurred in 35.9% versus 16.7% placebo
Expert Statements
Karen Mullen, Chief Medical Officer of Rafael Holdings
“The Phase 3 TransportNPC study is the most comprehensive trial in NPC conducted to date. We believe the totality of the data shared today provide a compelling rationale for the continued advancement of Trappsol Cyclo and its potential to offer clinically meaningful treatment benefit for those living with this devastating disease. We remain steadfast in our efforts to advance Trappsol Cyclo and are deeply grateful to the clinical investigators, patients, and caregivers whose courage and commitment were vital to completing this study.”
Caroline Hastings, Professor of Pediatric Hematology and Neurology at UCSF Benioff Children’s Hospital Oakland
“NPC is a devastating, heterogeneous disease, and preserving function and stabilizing symptoms are critical. As a clinician who has cared for these patients for many years, I am encouraged by the clinically meaningful TransportNPC results. The study was rigorously conducted, and its findings align with the two prior trials for Trappsol Cyclo across a decade of development. I am hopeful that these data can move us closer to delivering a much-needed therapeutic option for families facing profound unmet need.”
Trappsol(®) Cyclo™ did not achieve statistical significance but demonstrated a directionally favorable treatment effect versus placebo on the primary endpoint of change in the 4-domain NPC Clinical Severity Scale (4DNPCSS) at Week 96
A prespecified analysis in patients receiving background miglustat and/or leucine therapy, representing approximately 83% of the study population, showed a statistically significant effect on the 4DNPCSS
Get started
Create a free account to read the full story.