Rafael Holdings Announces Presentation of Preliminary Data from Ongoing Phase 3 TransportNPC™ Open-Label, Single-Arm Sub-Study in Patients Less Than 3 Years Old
Rafael Holdings, Inc. Class B (RFL)
NASDAQ:AMEX Investor Relations:
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Company Research
Source: GlobeNewswire
First data on treatment of NPC1 with Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) in this age group over a period of 48 weeks At 48 weeks of the sub-study, 7 of 9 patients1 showed stabilization or improvement in CGI-S score Data presented at the15th International Congress of Inborn Errors of Metabolism (ICIEM) NEWARK, N.J., Sept. 16, 2025 (GLOBE NEWSWIRE) -- Rafael Holdings, Inc. (NYSE: RFL; NYSE American: RFL-WT) announced today that data from Cyclo Therapeutics’ Phase 3 TransportNPC™ open-label, single-arm sub-study evaluating Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin) for Niemann-Pick Disease Type C1 (“NPC1”) were presented at the 15th International Congress of Inborn Errors of Metabolism (ICIEM). The Company’s ongoing TransportNPC™ study is a randomized, double-blind, placebo-controlled, parallel group, multicenter study designed to evaluate the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol® Cyclo™ administered intravenously and standard o
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News
- Rafael (NYSE:RFL) had its "sell (e+)" rating reaffirmed by analysts at <a style="font-weight:bold" href="https://weissratings.com/">Weiss Ratings</a>.MarketBeat
- Rafael Holdings Reports Fourth Quarter and Full Year Fiscal 2025 Financial ResultsGlobeNewswire
- Rafael Holdings Mourns the Passing of Lead Independent Director, Stephen Greenberg, and Announces the Appointment of Alan Grayson to its Board of DirectorsGlobeNewswire
- Rafael (NYSE:RFL) was downgraded by analysts at <a style="font-weight:bold" href="https://www.wallstreetzen.com/newsletters/zen-ratings?ticker=RFL&utm_campaign=getratings&utm_medium=online&utm_source=MarketBeat">Wall Street Zen</a> from MarketBeat
- Rafael (NYSE:RFL) was downgraded by analysts at <a style="font-weight:bold" href="https://www.wallstreetzen.com/newsletters/zen-ratings?ticker=RFL&utm_campaign=getratings&utm_medium=online&utm_source=MarketBeat">Wall Street Zen</a> from MarketBeat
RFL
Sec Filings
- 12/2/25 - Form 8-K
- 11/18/25 - Form DEF
- 10/31/25 - Form 3
- RFL's page on the SEC website