Rafael's Trappsol Cyclo Shows Significant Benefit In NPC Prespecified Analysis
(RTTNews) - 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).
NPC is a rare, progressive genetic disorder that can affect neurological function and other organ systems and is associated with significant disability and shortened survival.
Trappsol Cyclo is an investigational intravenous hydroxypropyl-ß-cyclodextrin being developed for the treatment of NPC. The Phase 3 study enrolled 94 patients aged 3 to 70 years who received Trappsol Cyclo or placebo every two weeks for 96 weeks.
Trappsol Cyclo demonstrated a directionally favorable treatment effect on the primary endpoint, with a 64% reduction in disease progression compared with placebo at Week 96. However, the result did not reach statistical significance (p=0.19).
In a prespecified analysis of 78 patients receiving background miglustat and/or leucine, Trappsol Cyclo showed a statistically significant 71% reduction in disease progression compared with placebo at Week 96 (p=0.046).
Trappsol Cyclo was generally well tolerated over the 96-week treatment period, with no new safety signals observed. Treatment-emergent adverse events occurred in 93.8% of patients receiving Trappsol Cyclo compared with 100% for placebo.
A separate analysis comparing infantile-onset NPC patients treated with Trappsol Cyclo with matched external controls from the International Niemann-Pick Disease Registry showed an 85% reduction in mortality risk.
Rafael said it remains on track to submit a New Drug Application (NDA) for Trappsol Cyclo to the FDA in the fourth quarter of 2026.
RFL has traded between $1.12 and $ 2.33 over the past year. Rafael's shares closed on Tuesday at $2.03, up 0.50%. In the Premarket trading, shares are down 31.68% at $1.38.
