RFL News & Events

Rafael announced that data from Cyclo Therapeutics' Phase 3 TransportNPC open-label, single-arm sub-study evaluating Trappsol Cyclo for Niemann-Pick Disease Type C1 were presented at the 15th International Congress of Inborn Errors of Metabolism, ICIEM. Summary Of Data Presented In Dr. Staretz-Chacham's Poster. At baseline, sub-study patients had a mixture of very mild to severe disease based on the Clinical Global Impression - Severity scale; Seven of nine patients who have reached 48 weeks participation in the study had an outcome of stabilization or improvement in CGI-S, with three patients showing improvement, and two patients showing deterioration of their CGI-S score; Adverse Event profile appears to be consistent with prior findings from earlier studies, and from the larger Phase 3 TransportNPC study that is irrespective of age and disease severity; As of May 14, 2025, there were 146 AEs, with the majority reported as mild or moderate, 3 reported as severe and the remainder reported as Serious Adverse Events; and No SAEs were considered by the principal investigators as related to or possibly related to study drug.

Rafael Holdings announced that its subsidiary Cyclo Therapeutics' 96-week pivotal phase 3 TransportNPC study evaluating intravenous Trappsol Cyclo for the potential treatment of Niemann-Pick Disease Type C1 will continue based on the independent Data Monitoring Committee review of safety and efficacy data at the prespecified 48-week interim analysis. Additionally, the Food and Drug Administration has accepted the statistical analysis plan for the TransportNPC study. These developments underscore the company's continuing commitment to advancing its lead investigational candidate through late-stage clinical development to support global regulatory and commercial readiness.

Reports Q3 revenue $362,000 vs $336,000 last year. "We are pleased to have completed our merger with Cyclo Therapeutics and look forward to reporting the topline data from the 48-week interim analysis of the pivotal Phase 3 TransportNPC study evaluating Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1 anticipated later this month," said Howard Jonas, Chief Executive Officer, Executive Chairman and Chairman of the Board of Rafael Holdings. Jonas added, "We have enhanced our financial position with the closing of a $25 million rights offering earlier this month which will support advancing this potential new treatment option for patients suffering from this rare genetic disease."

Rafael Holdings announced revised subscription rights in connection with its previously announced rights offering. Holders of record as of May 9, 2025 will be eligible to participate in the rights offering. To be considered a Holder of record on the Record Date, prospective Holders must complete open market purchases by May 8, 2025. Former holders of Cyclo Therapeutics, Inc. who did not hold shares in street name and have not submitted their letter of transmittal to Equiniti Trust Company and wish to participate in the rights offering, should send their letter of transmittal to Equiniti and complete the steps to exchange their Cyclo common stock for Class B common stock of the Company as soon as possible, as they must be record holders of Class B common stock prior to the Record Date in order to participate in the rights offering. Under the proposed rights offering, Rafael Holdings intends to distribute one non-transferable subscription right for each share of Class B Common Stock or Class A Common Stock or share of Class B Common Stock purchasable on exercise of Public Warrants, in each case, held as of the Record Date. Each subscription right will entitle the Holder to purchase 0.603 of a share of Class B Common Stock at a subscription price of $1.28 per share. Subscription rights may only be exercised in whole numbers, and fractional shares will not be issued. The number of shares that may be purchased by each holder of subscription rights will be rounded down to the nearest whole number. Howard Jonas, the Company's Executive Chairman and Chairman of the Board will enter into a Standby Purchase Agreement with the Company pursuant to which he will purchase from the Company, in a private placement, any shares of Class B Common Stock not subscribed for in the proposed rights offering for the same subscription price payable by holders electing to exercise the subscription rights in the proposed rights offering. The rights offering will be subject to certain conditions, and Rafael Holdings reserves the right to terminate the rights offering at any time prior to its expiration date. The subscription rights are non-transferable and may only be exercised during the subscription period, from May 13, 2025, through 5:00 PM ET on May 29, 2025, unless extended or terminated earlier by Rafael Holdings. The expected calendar for the rights offering is as follows: May 8, 2025 Ownership Day - Shares and/or Public Warrants must be acquired by this date to be considered a holder of record on May 9, 2025. May 9, 2025: Record Date. May 13, 2025: Subscription Period Begins. May 29, 2025: Subscription Period Ends at 5:00 PM ET.