Rafael Holdings announces Phase 3 TransportNPC study completion; NDA submission targeted H2 2026

Rafael Holdings, Inc.

Last patient completed final 96-week visit in pivotal Phase 3 TransportNPC study for Trappsol Cyclo in Niemann-Pick Disease Type C.
Topline data from main cohort expected to be announced in second half of calendar 2026.
Pre-NDA meeting with FDA completed; NDA submission expected in second half of 2026.
FDA acknowledged US Expanded Access Program (EAP) can proceed.
Pediatric sub-study (0-3 years) data: 80% of patients improved or stable with no serious related adverse events.

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