Rafael's Trappsol Cyclo Misses Primary Endpoint, Targets 2026 FDA Filing
Rafael Holdings reported top-line results from its pivotal Phase III TransportNPC study of Trappsol Cyclo for Niemann-Pick Disease Type C, noting the study missed its primary endpoint. The company said it remains on track to submit a New Drug Application to the FDA in the fourth quarter of 2026.
Why it matters — The failure of the pivotal Phase III trial to meet its primary endpoint is a significant setback for Rafael's lead drug candidate and raises uncertainty around its development and regulatory path.