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Patient enrollment on track to commence in
Q2 2021
Global clinical protocol agreed with US Food
and Drug Administration (FDA) and European Medicines Agency (EMA)
Dual primary objectives agreed; FDA 4-Domain
Niemann-Pick disease Type C-Severity Scale (4D-NPC-SS) and EMA 5D-NPC-SS
April 27, 2021 09:05
AM Eastern Daylight Time
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 suffering from diseases, today
announced that it has posted its protocol on www.ClinicalTrials.gov and is preparing to commence its
pivotal Phase 3 study evaluating Trappsol®Cyclo™, a proprietary formulation of
hydroxypropyl beta cyclodextrin, used intravenously, in the treatment of
Niemann-Pick Disease type C1 (NPC1). Both the FDA and EMA are in agreement
with the Company’s pivotal Phase 3 protocol and have given clearance to
proceed with the study.
NPC is a rare
genetic disease affecting 1 in 100,000 live births globally. NPC affects
every cell in the body due to a defect in the NPC1 protein which is
responsible for cholesterol processing in the cell. As cholesterol
accumulates in cells, NPC causes symptoms in the brain, liver, spleen, lung
and other organs and often leads to premature death. There are currently no
approved drug therapies for NPC in the United States and only one approved
therapy in Europe.
“Over the recent
months, we have worked diligently with both FDA and EMA to ensure our
pivotal Phase 3 study has the potential for the best outcome possible with
the criteria and endpoints necessary to achieve success. We are excited to
share this study information with NPC patients, families and physicians as
we work toward enrollment,” said N. Scott Fine, Chief Executive Officer of
Cyclo Therapeutics.
“Equipped with
promising clinical data that Trappsol®Cyclo™ may be a safe and effective
treatment for both systemic and neurologic manifestations of NPC, we are
optimistic moving into this final phase of development, and are excited to
be a step closer to bringing an approved treatment to people living with
this devastating disease,” added Sharon H. Hrynkow, PhD, Chief Scientific
Officer, Senior Vice President for Medical Affairs of the Company.
The pivotal Phase 3
study will be 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 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 investigational drug 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.
This study has dual
primary objectives. For the US 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
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 enables sponsors to receive a
Priority Review Voucher for another investigational drug upon marketing
authorization of the Rare Pediatric Disease drug in the US.
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 and NCT03893071). 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.
Investor Contact:
JTC Team, LLC
Jenene Thomas
(833) 475-8247
[email protected]
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