Rafael (RFL) investor relations material

Strategic focus and pipeline

• Dedicated to developing Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC), with additional interests in medical devices and real estate.

• Lead program is in late-stage clinical development, targeting both systemic and neurological NPC manifestations.

• Trappsol® Cyclo™ has Orphan Drug, Fast Track, and Rare Pediatric Disease designations in the US and EU.

• Company is positioned to redefine the NPC treatment paradigm with a foundational therapy.

Clinical development and trial progress

• Phase 3 Transport NPC trial fully enrolled; last patient last visit (LPLV) completed June 2026.

• NDA submission and topline data expected in the second half of 2026.

• Interim analysis at 48 weeks led to continuation to 96-week final analysis; independent DMC recommended study continuation.

• Substudy for newborns to age 3 is ongoing, with all patients receiving Trappsol® Cyclo™.

Efficacy and safety data

• Phase 1/2 studies showed Trappsol® Cyclo™ is well-tolerated, with most patients stabilizing or improving on key clinical endpoints.

• Long-term extension study suggests durable effect and slowed disease progression.

• Safety profile consistent across studies; most adverse events mild to moderate and unrelated to treatment.

• Hearing loss and infusion reactions are monitored, with most hearing issues resolving or stabilizing.