Cyclo Therapeutics Presents Positive Preliminary Data from Ongoing Phase 3 TransportNPC™ Open-Label Sub-Study in Patients (< 3 Years Old)
First data in NPC1 on treatment in this age group over a period of 48 weeks
At 24 weeks of the sub-study, 7 of 8 patients (87%) and at 48 weeks of the sub-study, 6 of 7 patients (86%) show stabilization or improvement in Clinical Global Impression – Change (CGI-C) Scale
Data presented at the 21st Annual WORLDSymposium™ 2025
Company on track for topline data from the 48-week interim analysis of 104 enrolled patients in TransportNPC™ in H1 2025
GAINESVILLE, Fla.–(BUSINESS WIRE)–Cyclo Therapeutics, Inc. (Nasdaq: CYTH) (“Cyclo Therapeutics” or the “Company”), 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™) evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (“NPC1”) at the 21st Annual WORLDSymposium™ in San Diego, CA.
The TransportNPC™ 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® Cyclo™ 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’s adopted PIP.
As part of the Annual WORLDSymposium™, 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, “Trappsol® Cyclo™: Open Label Treatment in the TransportNPC™ Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1.”
Key Data Highlights
- Clearance of lipids centrally and systemically were consistently demonstrated in a completed 14-week Phase 1 study in adult patients with NPC;
- At baseline, sub-study patients had a mixture of very mild to severe disease based on the Clinical Global Impression – Severity (CGI-S) scale. Clinical improvements seem to be best in patients with clinically mild to moderate disease;
- 7 of 8 patients who have reached 24 weeks and 6 of 7 who have reached 48 weeks show stabilization or improvement in CGI-C;
- Adverse Effect (AE) profile is in line with prior findings from earlier studies, and from a double-blind Phase 3 study running in parallel irrespective of age and disease severity;
- As of December 2024, AEs are limited (107), majority are mild (77%) or moderate (22%) and 1 AE is severe; with only 1 (mild grade) considered possibly related to or possibly related to study drug; and
- Of all 107 AEs, 19 were reported as SAEs. No SAEs were considered as related to or possibly related to study drug.
Dr. Spiegel commented, “The preliminary findings from the TransportNPC™ sub-study are encouraging and further underscore the potential of Trappsol® Cyclo™ 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® Cyclo™ on progress of NPC.”
“We continue to be encouraged by the data demonstrated by Trappsol® Cyclo™ in the treatment of NPC. The growing body of data from our TransportNPC™ study and sub-study continue to demonstrate the potential of Trappsol® Cyclo™ to address a devastating disease with significant unmet need. We look forward to our interim data readout from the TransportNPC™ study, which is expected in H1 2025 and ultimately providing a much needed treatment option for patients,” commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics.
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 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® Cyclo™ based on the 48-week interim data.
In 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’s Hospital Oakland titled, “Trappsol® Cyclo™ (HPβCD) for the Long-Term Treatment of Niemann-Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program,” and “Trappsol® Cyclo™ and NPC: Efficacy Shown Across Individual 5D Domains and Utilization of Future Assessment Tools to Demonstrate Clinically Relevant Outcomes.” Dr. Spiegel also presented the poster titled, “Trappsol® Cyclo™: Open Label Treatment in the TransportNPC™ Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1.” The presented posters are available on the Company’s website here.
For more information about the Company’s TransportNPC™ pivotal Phase 3 study, visit www.ClinicalTrials.gov and reference identifier NCT04860960.
Cyclo Therapeutics received Orphan Drug Designation for Trappsol® Cyclo™ 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.
About WORLDSymposium™
WORLDSymposium™ is an annual research conference dedicated to lysosomal diseases. WORLD is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLDSymposium™ 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.
About Niemann-Pick Disease Type C1 (NPC)
NPC 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.
About Cyclo Therapeutics
Cyclo 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’s Trappsol® Cyclo™, 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, NCT02912793, NCT03893071 and NCT04860960). The Company is conducting a Phase 2b clinical trial using Trappsol® Cyclo™ intravenously in early Alzheimer’s disease (NCT05607615) based on encouraging data from an Expanded Access program for Alzheimer’s disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the Company’s website: www.cyclotherapeutics.com.
On August 21, 2024, Cyclo Therapeutics entered into an Agreement and Plan of Merger (as amended, the “Merger Agreement”), by and among the Company; Rafael Holdings, Inc., a Delaware corporation (“Rafael”); Tandem Therapeutics, Inc., a Nevada corporation and a wholly-owned subsidiary of Rafael (“First Merger Sub”); and Tandem Therapeutics, LLC, a Nevada limited liability company and a wholly-owned subsidiary of Rafael (“Second Merger Sub”), pursuant to which: (i) First Merger Sub will merge with and into the Company, with the Company being the surviving entity (the “First Merger”), 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 “Second Merger” and together with the First Merger, the “Merger”). The Merger is subject to the satisfaction or waiver of several conditions set forth in the Merger Agreement and the approval of the Company’s and Rafael’s respective stockholders. Rafael is the holder of approximately 39.5% of the Company’s outstanding common stock, par value $0.0001 per share (the “Common Stock”).
Safe Harbor Statement
This press release contains “forward-looking statements” about the company’s current expectations about future results, performance, prospects and opportunities. Statements that are not historical facts, such as “anticipates,” “believes” and “expects” 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’s future performance include the company’s 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’s 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’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s 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.
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