Rafael's Trappsol Cyclo misses Phase III primary endpoint but shows subgroup benefit

Rafael's Trappsol Cyclo missed the primary endpoint in its Phase III trial for Niemann-Pick disease type C but showed significant benefit in a key subgroup already receiving background treatment. Rafael plans to submit a New Drug Application to the US FDA in Q4 2026, relying on this and supportive data despite the overall trial miss.

This Wire brief sits within Fusion42's coverage of Drug Discovery.