Rafael Holdings, Inc. (RFL), a clinical and early-stage biopharmaceutical company, announced that its last patient has completed their final 96-week study visit in the pivotal Phase 3 trial, TransportNPC, which evaluated Trappsol Cyclo for the treatment of Niemann-Pick disease type C (NPC).
Company Profile
Rafael Holdings is focused on the development and commercialization of therapies for oncology and rare diseases. The company through its subsidiary Cyclo Therapeutics advances a portfolio of preclinical and clinical candidates to address unmet medical needs.
Niemann-Pick Disease Type C (NPC)
It is a rare, fatal, and progressive genetic disorder caused by a defect in the NPC1 protein. This condition is characterized by the accumulation of cholesterol and lipids in the cells of major organs, ultimately leading to cell and tissue dysfunction and death.
Currently, it is diagnosed in more than 9,000 individuals across over 80 countries, with approximately 400 affected in the U.S. According to the company, NPC1 affects approximately 1 in 100,000 live births globally with about 35 new cases reported each year in the U.S.
Trappsol Cyclo and Trial Details
Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin) is designed to mobilize and transport accumulated cholesterol from lysosomes and endosomes, thereby addressing the cholesterol trafficking defect associated with NPC1 without replacing it.
The TransportNPC is a phase 3 trial that enrolled a total of 94 patients in over 27 sites across 13 countries. The company expects to announce its top-line results from the main study cohort in the second half of calendar 2026.
Additionally, after a pre-NDA meeting with the FDA, the company expects to submit the NDA in the second half of calendar 2026.
Rafael is currently trading at $1.88, down 2.59%.
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June 12, 2026 17:14 ET Major central bank action was the focus this week in economic news. The European Central Bank became the first major central bank to move in response to the rising inflationary pressures in the backdrop of the conflict in the Middle East. In North America, the U.S. inflation and trade data as well as Canada’s central bank decision gained attention. The Chinese trade data was the main news in Asia.