Cyclo Therapeutics Selected to Present Overview of Pivotal Phase 3 Study for Lead Candidate, Trappsol® Cyclo™, at WORLDSymposium 2022
February 08 2022 - 8:05AM
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 diseases, today announced the
poster presentation of its ongoing pivotal Phase 3 study,
TransportNPC™ at the 18th Annual WORLDSymposium 2022, a leading
medical and scientific conference for professionals working to
advance understanding and treatments for lysosomal storage
diseases, including Niemann-Pick Disease Type C (“NPC”), being held
virtually and in San Diego, CA, February 7–11, 2022.
The ePoster titled, “TransportNPC: a Phase 3 global trial of
Trappsol® Cyclo™ administered intravenously to patients with
Niemann-Pick Disease Type C1 (NPC1),” will be presented by Lise
Lund Kjems, MD, PhD, Chief Medical Officer of Cyclo Therapeutics in
the Clinical Applications Abstracts session on Wednesday, February
9, 2022 from 3:00 – 5:00 PM PST.
“Our pivotal Phase 3 TransportNPC™ clinical trial was designed
in close collaboration with regulatory agencies, Key Opinion
Leaders, patient advocacy groups and patient communities in order
to best enable a potentially positive outcome. With the severity of
this disease and the limited treatment options currently available
to patients, there is a significant unmet need for a product that
can treat the systemic and neurologic manifestations of NPC. We
continue to be encouraged by the potential of Trappsol® Cyclo™ and
are leveraging this critical product, its mechanism of action and
route of administration with the hope of providing a much-needed
clinical benefit to the NPC patient population. We are dedicated to
progressing this trial and look forward to the new insights it will
provide,” commented Dr. Lise Lund Kjems.
The Company’s ongoing pivotal Phase 3 study, TransportNPC™, 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.
The Phase 3 study intends to enroll at least 93 pediatric (age 3
years and older) and adult patients with NPC1 in at least 23 study
centers in 9 countries. Eligible patients will be randomized 2:1 to
receive either Trappsol® Cyclo™ or a placebo. Randomization will
not be constrained based on patient age, nor will patient
enrollment be gated by patient age. The study duration is 96 weeks
and includes an interim analysis at 48 weeks.
This study has dual primary objectives. For the U.S. and
countries following FDA guidance, the primary objective is to
evaluate the efficacy of Trappsol® Cyclo™ versus placebo using a
4-Domain Niemann-Pick Disease Type C Severity Scale (4D-NPC-SS
[Ambulation, Fine Motor, Speech and Swallow]) composite score at
Weeks 48 and 96. For the EU and countries following EMA guidance,
the primary objective is to evaluate the efficacy of Trappsol®
Cyclo™ versus placebo using the 5-Domain Niemann-Pick Disease Type
C Severity Scale (5D-NPC- SS) composite score (Ambulation, Fine
Motor, Speech, Swallow, and Cognition) at Weeks 48 and 96.
As previously announced, the Company received a positive opinion
from the Paediatric Committee (PDCO) of the EMA and agreement on
its Paediatric Investigation Plan (PIP) for Trappsol® Cyclo™. The
PIP opinion from PDCO endorsed the clinical program to evaluate the
safety, tolerability and efficacy of Trappsol® Cyclo™ in patients
from 3 to less than 18 years of age with NPC Type C1, and in
addition, to include a single-arm sub-study of patients from birth
to less than 3 years of age with NPC Type C1 irrespective of
symptoms to evaluate safety and to obtain descriptive data on
global disease severity and the response to Trappsol® Cyclo™. The
substudy in patients from birth to less than 3 years of age will
only be conducted in the EU and countries following EMA
guidelines.
Cyclo Therapeutics recently formed a Global Steering Committee
(GSC) comprised of Key Opinion Leaders and experts on lysosomal
storage diseases to guide the pivotal Phase 3 global clinical
development program of Trappsol® Cyclo™ for the treatment of
NPC.
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.
For more information about the Company’s TransportNPC™ pivotal
Phase 3 study, visit www.ClinicalTrials.gov and reference
identifier NCT04860960.
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
2000 participants from more than 50 countries around the globe. For
more information, please visit: worldsymposia.org.
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 suffering from
disease. 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 C, a rare and
fatal genetic disease, (www.ClinicalTrials.gov NCT02939547,
NCT02912793, NCT03893071 and NCT04860960). The Company is planning
an early phase clinical trial using Trappsol® Cyclo™ intravenously
in Alzheimer’s Disease based on encouraging data from an Expanded
Access program for late-onset 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.
Safe Harbor Statement
This press release contains “forward-looking statements” about
the company’s current expectations about future results,
performance, prospects and opportunities, including, without
limitation, statements regarding the satisfaction of closing
conditions relating to the offering and the anticipated use of
proceeds from the offering. 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 obtain additional capital to
expand operations as planned, 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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Investor Contact: JTC Team, LLC Jenene Thomas (833)
475-8247 CYTH@jtcir.com
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