Welcome to our dedicated page for Rafael Holdings news (Ticker: RFL), a resource for investors and traders seeking the latest updates and insights on Rafael Holdings stock.
Rafael Holdings, Inc. reports developments as a biotechnology company focused on pharmaceutical development and interests in clinical-stage and early-stage pharmaceutical and medical device companies. Its lead program is Trappsol® Cyclo™, a hydroxypropyl-beta-cyclodextrin product candidate evaluated for Niemann-Pick Disease Type C1, with additional cyclodextrin-related work in neurodegenerative disease.
Recurring company news covers clinical development of Cyclo Therapeutics programs, scientific presentations, patent and licensing activity, quarterly and annual financial results, and governance changes. Updates also address the company’s consolidated expenses following the Cyclo Therapeutics acquisition and its portfolio interests in healthcare and medical-device development.
Cyclo Therapeutics has completed enrollment for its Phase 3 TransportNPC™ trial, the largest study of its kind for Niemann-Pick Disease Type C1 (NPC1). The study includes 104 patients and aims to evaluate the efficacy of Trappsol® Cyclo™ in treating systemic and neurological symptoms of NPC1.
Topline data from the 48-week interim analysis is expected in the first half of 2025. Positive results could lead to the submission of a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the EMA in the second half of 2025.
The trial also features a substudy for newborns to 3-year-olds, targeting early-stage interventions. The company has received Orphan Drug Designation in both the U.S. and EU, Fast Track and Rare Pediatric Disease designations in the U.S., which could qualify them for a Priority Review Voucher.
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