Rafael Holdings stock tumbles 25% as drug trial misses goal

Investing.com -- Rafael Holdings Inc Class B (NYSE: RFL) shares fell 25% in premarket trading Wednesday after the company's Phase 3 trial of Trappsol Cyclo for Niemann-Pick Disease Type C failed to meet its primary endpoint.

The TransportNPC study, which enrolled 94 patients, showed Trappsol Cyclo did not achieve statistical significance on the primary endpoint of change in the 4-domain NPC Clinical Severity Scale at Week 96. The drug demonstrated a directionally favorable treatment effect versus placebo, slowing disease progression by 64%, but the result missed statistical significance with a p-value of 0.19.

However, a prespecified analysis in patients receiving background miglustat and/or leucine therapy, representing approximately 83% of the study population, showed a statistically significant effect. In this subgroup of 78 patients, Trappsol Cyclo slowed disease progression by 71% compared with placebo at Week 96, with a p-value of 0.046.

The drug demonstrated a well-tolerated safety profile consistent with previous studies, with no new safety signals observed. Treatment-emergent adverse events were reported in 93.8% of patients receiving Trappsol Cyclo versus 100% in the placebo group. Serious treatment-related adverse events occurred at similar rates between groups at 3.1% and 3.3%, respectively.

Rafael Holdings also conducted a separate overall survival analysis comparing patients with infantile-onset NPC treated with Trappsol Cyclo with matched external controls from the International Niemann-Pick Disease Registry. The analysis indicated treatment was associated with an 85% reduction in the risk of mortality compared with matched controls.

Based on its previously announced pre-NDA meeting with the FDA, the company remains on track to submit a New Drug Application in the fourth quarter of 2026.

Original Article