Rafael Reports Phase 3 Trappsol Cyclo Data In NPC

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(RTTNews) - Wednesday, Rafael Holdings (RFL) reported topline results from the pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo in patients with Niemann-Pick Disease Type C (NPC).

Trappsol Cyclo is an investigational intravenous hydroxypropyl-ss-cyclodextrin being developed for the treatment of NPC, a rare and progressive genetic disorder.

The study showed a directionally favorable treatment effect on the primary endpoint, with Trappsol Cyclo slowing disease progression by 64% at Week 96 compared with placebo, although the result did not reach statistical significance.

In a prespecified analysis of patients receiving background miglustat and/or leucine, Trappsol Cyclo showed a statistically significant 71% reduction in disease progression compared with placebo.

Rafael said Trappsol Cyclo was well tolerated, with no new safety signals observed. The company remains on track to submit a New Drug Application (NDA) to the FDA in the fourth quarter of 2026.

Rafael shares closed on Tuesday at $2.03, up 0.50%. In the Premarket trading, shares are down 34.17% at $1.32.