Company on track for topline data from the 48-week interim analysis of 104 enrolled patients in TransportNPC\u2122 in H1 2025<\/i><\/p>\n
GAINESVILLE, Fla.–(BUSINESS WIRE<\/a>)–Cyclo Therapeutics, Inc.<\/a> (Nasdaq: CYTH) (\u201cCyclo Therapeutics\u201d or the \u201cCompany\u201d), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with rare and challenging diseases, today announced the presentation of data from its ongoing Pivotal Phase 3 global study (TransportNPC\u2122) evaluating Trappsol\u00ae <\/sup>Cyclo\u2122 for Niemann-Pick Disease Type C1 (\u201cNPC1\u201d) at the 21st<\/sup> Annual WORLDSymposium<\/i>\u2122<\/a> in San Diego, CA.<\/p>\n \nThe TransportNPC\u2122 study is the most comprehensive, controlled pivotal study regarding patient size, global footprint, duration and clinical outcomes of an investigational therapy for NPC1. The study completed enrollment in May 2024. Additionally, the Company enrolled ten (10) patients in its single-arm sub-study per their adopted Pediatric Investigational Plan (PIP) treating newborns to 3 years of age. Two patients terminated the sub-study after 48 weeks (caregiver decision). The sub-study is evaluating Trappsol\u00ae<\/sup> Cyclo\u2122 in the youngest age subsets as it targets also the visceral aspects of the disease and may achieve its most optimal results when administered early in the disease course, thus having the potential of a preventative effect in overall symptom development. The sub-study is being conducted in countries outside of the United States per the Company\u2019s adopted PIP.<\/p>\n \nAs part of the Annual WORLDSymposium\u2122<\/i>, Ronen Spiegel, MD, Clinical Associate Professor, Director of Pediatric B Department, and Director Center of Rare Disease, at Emek Medical Center gave an oral presentation titled, \u201cTrappsol\u00ae<\/sup> Cyclo\u2122: Open Label Treatment in the TransportNPC\u2122 Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1.\u201d<\/i><\/p>\n Key Data Highlights<\/b><\/p>\n \nDr. Spiegel commented, \u201cThe preliminary findings from the TransportNPC\u2122 sub-study are encouraging and further underscore the potential of Trappsol\u00ae<\/sup> Cyclo\u2122 to address the significant unmet medical need in the treatment of NPC. Slowing down disease progression through cholesterol mobilization is an important consideration for patients with established disease, who can expect to experience neurodegeneration without treatment. We look forward to gathering additional data from this ongoing study and establishing the effect of Trappsol\u00ae<\/sup> Cyclo\u2122 on progress of NPC.\u201d<\/p>\n \n\u201cWe continue to be encouraged by the data demonstrated by Trappsol\u00ae<\/sup> Cyclo\u2122 in the treatment of NPC. The growing body of data from our TransportNPC\u2122 study and sub-study continue to demonstrate the potential of Trappsol\u00ae<\/sup> Cyclo\u2122 to address a devastating disease with significant unmet need. We look forward to our interim data readout from the TransportNPC\u2122 study, which is expected in H1 2025 and ultimately providing a much needed treatment option for patients,\u201d commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics.<\/p>\n \nThe Company\u2019s ongoing TransportNPC\u2122 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\u00ae<\/sup> Cyclo\u2122 administered intravenously and standard of care (SOC), compared to placebo administered intravenously and SOC alone, in patients with NPC1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction primarily of the liver, lung, spleen and brain and premature death. The study duration is a 96-week study, with a 48-week comparative interim analysis. Should the 48-week interim data meet statistical significance, the Company, in alignment with the FDA and EMA, intends to submit marketing applications for approval to market Trappsol\u00ae<\/sup> Cyclo\u2122 based on the 48-week interim data.<\/p>\n \nIn addition to the oral presentation given by Dr. Spiegel, the Company had two posters presented by Dr. Caroline Hastings, Pediatric hematologist oncologist, Director of Neuro-oncology, and Professor of Pediatrics at UCSF Benioff Children\u2019s Hospital Oakland titled, \u201cTrappsol\u00ae<\/sup> Cyclo\u2122 (HP\u03b2CD) for the Long-Term Treatment of Niemann-Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program,\u201d<\/i> and \u201cTrappsol\u00ae <\/sup>Cyclo\u2122 and NPC: Efficacy Shown Across Individual 5D Domains and Utilization of Future Assessment Tools to Demonstrate Clinically Relevant Outcomes.\u201d <\/i>Dr. Spiegel also presented the poster titled, \u201cTrappsol\u00ae<\/sup> Cyclo\u2122: Open Label Treatment in the TransportNPC\u2122 Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1<\/i>.\u201d The presented posters are available on the Company\u2019s website here<\/a>.<\/p>\n \nFor more information about the Company\u2019s TransportNPC\u2122<\/a> pivotal Phase 3 study, visit www.ClinicalTrials.gov<\/a> and reference identifier NCT04860960<\/a>.<\/p>\n \nCyclo Therapeutics received Orphan Drug Designation for Trappsol\u00ae<\/sup> Cyclo\u2122 to treat NPC1 in both the U.S. and EU and Fast Track and Rare Pediatric Disease Designations in the U.S. The Rare Pediatric Disease Designation is one of the chief requirements for sponsors to receive a Priority Review Voucher in the U.S. upon marketing authorization.<\/p>\n About <\/i>WORLDSymposium\u2122<\/i><\/b><\/p>\n \nWORLDSymposium\u2122<\/i> is an annual research conference dedicated to lysosomal diseases. WORLD is an acronym that stands for We\u2019re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium\u2122<\/i> has grown to an international research conference that attracts over 2,000 participants from more than 50 countries around the globe. For more information, please visit: worldsymposia.org<\/a>.<\/p>\n About Niemann-Pick Disease Type C1 (NPC)<\/i><\/b><\/p>\n \nNPC is a rare genetic disease affecting 1 in 100,000 live births globally. Approximately 95% of individuals with NPC have mutations in the NPC1 gene and 5% have mutations in the NPC2 gene. NPC affects nearly every cell in the body due to a deficiency in either the NPC1 or NPC2 protein, which are required for the transport and processing of cholesterol within the cell. As cholesterol accumulates within cells, NPC causes symptoms that affect the brain, liver, spleen, lungs, and other organs and often leads to premature death.<\/p>\n About Cyclo Therapeutics<\/i><\/b><\/p>\n \nCyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with rare and challenging diseases. The Company\u2019s Trappsol\u00ae<\/sup> Cyclo\u2122, an orphan drug designated product in the United States and Europe, is the subject of four formal clinical trials for Niemann-Pick Disease Type C1, a rare and fatal genetic disease, (NCT02939547<\/a>, NCT02912793<\/a>, NCT03893071<\/a> and NCT04860960<\/a>). The Company is conducting a Phase 2b clinical trial using Trappsol\u00ae<\/sup> Cyclo\u2122 intravenously in early Alzheimer\u2019s disease (NCT05607615<\/a>) based on encouraging data from an Expanded Access program for Alzheimer\u2019s disease (NCT03624842<\/a>). Additional indications for the active ingredient in Trappsol\u00ae<\/sup> Cyclo\u2122 are in development. For additional information, visit the Company\u2019s website: www.cyclotherapeutics.com<\/a>.<\/p>\n \nOn August 21, 2024, Cyclo Therapeutics entered into an Agreement and Plan of Merger (as amended, the \u201cMerger Agreement\u201d), by and among the Company; Rafael Holdings, Inc., a Delaware corporation (\u201cRafael\u201d); Tandem Therapeutics, Inc., a Nevada corporation and a wholly-owned subsidiary of Rafael (\u201cFirst Merger Sub\u201d); and Tandem Therapeutics, LLC, a Nevada limited liability company and a wholly-owned subsidiary of Rafael (\u201cSecond Merger Sub\u201d), pursuant to which: (i) First Merger Sub will merge with and into the Company, with the Company being the surviving entity (the \u201cFirst Merger\u201d), and (ii) immediately following the First Merger, the Company will merge with and into Second Merger Sub, with Second Merger Sub being the surviving entity of the subsequent merger (the \u201cSecond Merger\u201d and together with the First Merger, the \u201cMerger\u201d). The Merger is subject to the satisfaction or waiver of several conditions set forth in the Merger Agreement and the approval of the Company\u2019s and Rafael\u2019s respective stockholders. Rafael is the holder of approximately 39.5% of the Company\u2019s outstanding common stock, par value $0.0001 per share (the \u201cCommon Stock\u201d).<\/p>\n Safe Harbor Statement<\/i><\/b><\/p>\n \nThis press release contains \u201cforward-looking statements\u201d about the company\u2019s current expectations about future results, performance, prospects and opportunities. Statements that are not historical facts, such as \u201canticipates,\u201d \u201cbelieves\u201d and \u201cexpects\u201d or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company\u2019s future performance include the company\u2019s ability to complete the merger transaction with Rafael Holdings Inc., success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company\u2019s biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company\u2019s filings with the Securities and Exchange Commission, including, but not limited to, the company\u2019s reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20250207991863\/en\/<\/a><\/span><\/p>\n\n
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