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
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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version on businesswire.com: https://www.businesswire.com/news/home/20250207991863/en/
Investor Contact: JTC Team, LLC Jenene Thomas (908)
824-0775 CYTH@jtcir.com
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