The company has had a pre-NDA meeting with the FDA and expects to submit an NDA in second half of calendar 2026

The company has received acknowledgement from the FDA that our US Expanded Access Program (EAP) can proceed

NEWARK, N.J., June 10, 2026 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE:RFL) today announced the last patient has completed the final 96-week study visit in the pivotal Phase 3 TransportNPC™ study evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C (NPC). With 94 patients studied across 27 sites in 13 countries, the TransportNPC™ study is the most comprehensive, controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC. Additionally, 10 patients were enrolled in a single-arm sub-study per the adopted Pediatric Investigational Plan (PIP) treating newborns to 3 years of age. Topline data from the main study cohort is expected to be known and announced in the second half of calendar 2026.

Data presented at WORLDSymposium 2026 from the single arm pediatric sub-study in patients ages zero to three, continues to highlight the potential for Trappsol® Cyclo™ as a therapeutic option in NPC with 80% of the sub-study patients' CGI scores showing improvements or remaining stable with no serious adverse events considered to be related to the study drug.