On December 11, 2025, Rafael Holdings, Inc. (RFL) disclosed financial results and clinical trial updates in an SEC 8-K filing. The filing reported a net loss of $9.8 million for the first quarter of fiscal 2026, which ended on October 31, 2025, and revealed that the Data Monitoring Committee (DMC) recommended continuing the Phase 3 TransportNPC study for Trappsol Cyclo after reviewing 48-week data.

The company's $9.8 million net loss for the quarter compares to a $9.0 million net loss for the same period in the previous fiscal year. According to the filing, research and development expenses increased to $7.5 million from $1.3 million year-over-year, while general and administrative expenses rose to $2.8 million from $2.5 million. Rafael Holdings reported cash and cash equivalents of $45.5 million as of October 31, 2025.

The Phase 3 TransportNPC study evaluates Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1 (NPC1), a rare and fatal genetic disease. The Data Monitoring Committee reviewed prespecified safety and efficacy data at 48 weeks and recommended the study continue without modification, according to the company's disclosure.

"We remain pleased with the progress of our pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1, which the Data Monitoring Committee (DMC) recommended continuing after their review of prespecified safety and efficacy data at 48 weeks. We believe that Trappsol Cyclo could provide an important new treatment option for patients suffering from this rare and fatal genetic disease." — Howard Jonas, Chief Executive Officer, Executive Chairman and Chairman of the Board of Rafael Holdings

The filing noted that Rafael Holdings acquired Cyclo Therapeutics on March 1, 2025, which brought the Trappsol Cyclo program into its portfolio. The company identified risks related to the potential, safety, and efficacy of product candidates, regulatory and clinical progress, and the potential of its internal cancer metabolism research programs.