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Daily updates
- Rafael Holdings reported Phase 3 TransportNPC results for Trappsol Cyclo in Niemann-Pick disease type C, with the primary 4DNPCSS endpoint not reaching statistical significance despite a 64% reduction in disease progression versus placebo.Sep 30, 2026
- Rafael Holdings reported Phase 3 TransportNPC results for Trappsol Cyclo in Niemann-Pick disease type C, with the primary 4DNPCSS endpoint not reaching statistical significance despite a 64% reduction in disease progression versus placebo.Sep 30, 2026
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News
- Rafael Holdings reports Phase 3 TransportNPC results for Trappsol Cyclo in Niemann-Pick disease type C, showing slower disease progression despite missing the primary 4DNPCSS endpoint; NDA filing remains planned for Q4 2026Sep 30, 2026
- Rafael Holdings completed the last patient visit in the pivotal Phase 3 TransportNPC study of Trappsol CycloJun 10, 2026
- Rafael Holdings signed a patent licensing agreement with MIT for small molecules targeting myelination improvement in ApoE4-positive Alzheimer's diseaseApr 22, 2026
- Rafael Holdings announces presentation of preliminary data from ongoing Phase 3 TransportNPC™ open-label, single-arm sub-study in patients under 3 years oldSep 16, 2025
- Rafael Holdings continues Phase 3 study for Niemann-Pick Disease Type C1 after independent data reviewJun 18, 2025