Rafael Holdings completes Phase 3 NPC trial, plans NDA submission in H2 2026

Rafael Holdings completed enrollment in its pivotal Phase 3 TransportNPC trial for Niemann-Pick disease Type C and expects to submit an NDA in the second half of 2026 following a pre-NDA FDA meeting. The company reported Q3 net loss of $4.2 million with $30.5 million in cash, positioning itself to transition toward commercial-stage operations in a high-unmet-need market.