Cyclo Therapeutics Announces New Positive Safety and Efficacy Data from Ongoing Phase 1 Open-Label Extension Study of Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1
July 30 2021 - 8:05AM
Business Wire
- Data seen to-date in the ongoing Phase 1
extension study provide additional support for the capacity of
Trappsol® Cyclo™ to stabilize disease progression with home-based
intravenous infusions in Niemann-Pick Disease Type C1 (“NPC”)
- Favorable safety profile of Trappsol® Cyclo™
administered intravenously for more than 2 years
- Pivotal Phase 3 study (“TransportNPC™”)
currently open for patient enrollment
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 new
data from its Phase 1 extension study evaluating Trappsol® Cyclo™
for the treatment of Niemann-Pick Disease Type C1 (“NPC”), a rare,
progressive and fatal genetic disorder characterized by abnormal
accumulation of cholesterol in cells. These data are being
presented at the National Niemann Pick Disease Foundation Family
Support & Medical Conference being held virtually July
29-August 1, 2021, and at the International Niemann Pick Disease
Foundation biennial meeting held on August 1, 2021, also being held
virtually.
Trappsol® Cyclo™ is a proprietary formulation of hydroxypropyl
beta cyclodextrin which has an affinity for cholesterol. In
multiple clinical studies, Trappsol® Cyclo™ has shown encouraging
results to normalize the transportation of cholesterol in
cells.
“We are incredibly pleased with the continued trends of safety
and efficacy being demonstrated by Trappsol® Cyclo™ in the
treatment of NPC. We are grateful for the opportunity to present
these data to patients and their families and hope to provide
continued encouragement as we work diligently to treat the systemic
and neurologic manifestations of NPC,” commented Sharon Hrynkow,
PhD, the Company’s Chief Scientific Officer and Senior Vice
President for Medical Affairs.
“We remain focused on expeditiously advancing this clinical
program toward potential market approval and believe strongly that
Trappsol® Cyclo™ has enormous potential to meet the significant
unmet needs for NPC patients and their families,” stated N. Scott
Fine, CEO and Executive Director of Cyclo Therapeutics.
All 8 eligible patients (U.S. residents) enrolled in the
Extension Protocol, which supports IV infusions in the home setting
under the care of a healthcare professional. Initial efficacy
evaluation in September 2020 showed disease stabilization with
notable clinical improvements in some patients.
The first patient was dosed in the Open-Label extension study in
May 2019 and the last patient enrolled was in February 2020. The
Company previously reported data on the Phase 1 extension study in
January 2021 based on the initial efficacy results at a data
cut-off of September 2020. The results summarized below have a data
cut-off of July 2021.
- The safety profile of Trappsol® Cyclo™ continues to be
favorable, with no Adverse Events attributed to drug.
- Efficacy as measured with the 17-Domain NPC Severity Scale in 8
patients from baseline (Phase 1) through most recent data (Phase 1
plus Extension data) show a trend of stability for patients, with
the mean length of exposure of 25 months. The 17-Domain scale
evaluates 9 major disease features and 8 minor disease features.
Disease features using this tool showed worsening in hearing in 3
patients and improvement in hearing in one patient: worsening in
eye movements in four patients, worsening in swallow in one patient
and improvement in swallow in three patients, improvement in memory
in one patient, with other disease features of the 17-Domain scale
stable.
- Using a sub-set of the 17-Domain NPC Severity Scale, the
5-Domain NPC Severity Scale which measures disease features most
important to patients’ quality of life, per NPC patient assessments
(Speech, Swallow, Fine Motor skills, Ambulation and Cognition), a
trend in disease stabilization is observed.
- An average of worsening of 0.4 pts per year was observed for
all patients with all available data. This is significant as a
worsening of 1.4 pts per year would be expected for NPC patients
based on published calculations (Cortina-Borja et al, 2018).
- 3 patients were stable overall, i.e. no change in their
5-Domain score (with expected changes of 1.3, 2.7 and 2.1 pts based
on time on Trappsol® Cyclo™ since baseline dosing); 1 patient
improved (actual change -1 and expected change 2.2), and 3 worsened
but at a slower rate than expected without intervention (actual
change 2 versus expected 4.4; actual change 4 versus expected 4.8,
actual change 1 versus expected 3.5) and 1 worsened beyond expected
(actual change 4 versus expected change 2.9)
- One patient added Miglustat to their treatment program after 1
year on the extension protocol, with no change to the 5-Domain
score.
- Dose level does not appear to be a factor in the positive data
reported here but given that the sample size is limited it is not
possible to interpret dose definitively. The patient who improved
overall in the 5-Domain NPC Severity Score received the 1500 mg/kg
dose, while the patient who worsened overall also received the 1500
mg/kg dose. Of the 3 patients who were stable overall, 2 received
the 1500 mg/kg dose and 2 received the 2500 mg/kg dose.
These findings provide additional support for the capacity of
Trappsol® Cyclo™ to stabilize disease progression in NPC1.
Trappsol® Cyclo™ is currently being evaluated in the pivotal
Phase 3 study, TransportNPC™, for the treatment of NPC1. As
previously announced, Cyclo Therapeutics received Orphan Drug
Designation for Trappsol® Cyclo™ to treat NPC1 in both the US and
EU and 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 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, lung, 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 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.
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Investor Contact: JTC Team, LLC Jenene Thomas (833)
475-8247 CYTH@jtcir.com
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