Global clinical protocol agreed upon by US Food
and Drug Administration (FDA) and European Medicines Agency
(EMA)
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
commencement of its pivotal Phase 3 study (“TransportNPC”)
evaluating Trappsol® Cyclo™, a proprietary formulation of
hydroxypropyl beta cyclodextrin, delivered intravenously, for the
treatment of Niemann-Pick Disease Type C1 (NPC1). The TransportNPC
study has the regulatory and IRB approval required to commence
patient enrollment, and site activation is underway.
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, lung and other organs and often leads to
premature death.
“The start of patient enrollment in our pivotal Phase 3 study is
an important milestone for both the NPC community and Cyclo
Therapeutics. We are grateful for the hard work on the part of so
many in getting us to the point of enrollment. We are excited to
move forward with speed, and remain deeply committed to advancing
our NPC clinical program forward with the goal of bringing a safe
and effective treatment to NPC patients, families and physicians,”
said N. Scott Fine, Chief Executive Officer of Cyclo
Therapeutics.
The TransportNPC study is a randomized, double-blind,
placebo-controlled, parallel group, multicenter study designed to
evaluate the safety, tolerability, and efficacy of 2000 mg/kg of
Trappsol® Cyclo™ administered intravenously and standard of care
(SOC) compared to placebo administered intravenously and SOC in
patients with NPC1. The TransportNPC study intends to enroll at
least 93 pediatric (age 3 to less than 18 years) and adult patients
with NPC1 in at least 23 study centers in 9 countries. Eligible
patients will be randomized 2:1 to receive Trappsol® Cyclo™ or
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.
The Company expects to report topline results from the interim
analysis in the first half (H1) of 2023.
This study has dual primary objectives based on regulatory
preferences. For the US and countries following FDA guidance, the
primary objective is to evaluate the mean change from baseline to
48 weeks or 96 weeks as measured by improvement in Trappsol® Cyclo™
versus placebo using a 4-Domain modified Niemann-Pick Disease Type
C Severity Scale (4D-NPC-SS [Ambulation, Fine Motor, Speech and
Swallow]) composite score. For the EU and countries following EMA
guidance, the primary objective is to evaluate the mean change from
baseline to 48 weeks or 96 weeks as measured by improvement in
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).
“The initiation of this clinical trial is an important advance
in research and a new opportunity for hope for the NPC community,
building upon data that has been accumulated over many years
supporting the development of this promising therapy to treat NPC,”
added Caroline Hastings, MD, UCSF Benioff Children’s Hospital
Oakland, California, and Principal Investigator. “We applaud all
the families and researchers who have made this opportunity
possible, and we look forward to the new insights that will be
available as this Phase 3 trial advances in the months ahead.”
“The launch of the Phase 3 pivotal program for Trappsol® Cyclo™
is a significant milestone for the NPC community,” commented Joslyn
Crowe, Executive Director of the National Niemann-Pick Disease
Foundation. “With the heterogeneity of the disease, we hope that
patients will one day have multiple options for treatment regimens
that will be effective to meet individual patient needs.”
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 pediatric
patients of all ages. The Phase 3 study will consist of a main
study cohort that includes pediatric patients with NPC1 from age 3
to less than 18 years, and in addition, a single-arm sub-study of
patients from birth to less than 3 years of age with NPC1
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 be conducted in the EU, and countries following
EMA guidelines, and others as approved by local regulatory
authorities.
“We are incredibly pleased with the strides the NPC program
continues to make, and we remain optimistic as Trappsol® Cyclo™
advances into the final phase of clinical development. Having met
all of the primary endpoints of our Phase 1 and Phase 1/2 studies
showing favorable safety and promising trends in efficacy with
Trappsol® Cyclo™, we believe that Cyclo Therapeutics is
well-positioned to address the systemic and neurologic
manifestations of NPC, and ultimately, provide hope to people
living with NPC and their families,” added Gerry Cox, MD, PhD,
Acting Chief Medical Officer of the Company.
Cyclo Therapeutics has received Orphan Drug Designation for
Trappsol® Cyclo™ to treat NPC in both the US and EU, and
additionally, Fast Track and Rare Pediatric Disease Designations in
the US. The Rare Pediatric Disease Designation is one of the chief
requirements for sponsors to receive a Priority Review Voucher in
the US upon marketing authorization.
For more information about the pivotal Phase 3 study, visit
www.ClinicalTrials.gov and reference identifier NCT04860960.
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 three
ongoing 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210617005132/en/
Investor Contact: JTC Team, LLC Jenene Thomas (833)
475-8247 CYTH@jtcir.com
Cyclo Therapeutics (NASDAQ:CYTH)
Historical Stock Chart
From Mar 2024 to Apr 2024
Cyclo Therapeutics (NASDAQ:CYTH)
Historical Stock Chart
From Apr 2023 to Apr 2024