Home-based intravenous infusions of Trappsol® Cyclo™ for up
to one year show improvement in disease features or disease
stabilization
Cyclo Therapeutics, Inc. (NasdaqCM:CYTH) (NasdaqCM:CYTHW), a
clinical stage biotechnology company developing a cyclodextrin
platform for the treatment of neurodegenerative diseases, including
its lead candidate Trappsol® Cyclo™ for the treatment of
Niemann-Pick Disease Type C (NPC), today announced initial data
from its “Open-Label Study of Long-Term Safety and Efficacy of
Intravenous Trappsol® Cyclo™ (HPBCD) in Niemann-Pick Disease Type
C,” NCT03893071. Eight patients living in the US who completed the
Phase I trial (NCT02939547) were eligible for the extension
program. All eight enrolled.
The first patient was dosed in the Open-Label extension study in
May 2019 and the last patient enrolled was in February 2020. The
initial efficacy results presented below have a data cut-off of
September 2020.
All patients included in this analysis demonstrated disease
stability or improvement as measured by the 17-domain NPC Severity
Score scale (NPC-SS, Yanjinin et al, 2010). The NPC-SS measures
primarily neurologic features of the disease, including ambulation,
fine motor skills, ability to speak and swallow, and cognition.
Without intervention, over the course of one year, NPC patients
would be expected to worsen by one to two points using this
scale.
During this Open-Label Study, patients receive intravenous
infusions of Trappsol® Cyclo™ in their homes under the care of
healthcare professionals every two weeks, with regular assessments
for NPC-SS at UCSF Benioff Children’s Hospital Oakland under the
care of Dr. Caroline Hastings and Dr. Benny Liu, Co-Principal
Investigators.
Of the patients who received Trappsol® Cyclo ™ at the 1500 mg/kg
body weight dose level, four patients remained stable in terms of
their NPC-SS and two patients improved:
Patient 1 had a baseline NPC-SS of 24; and the same score 10
months later.
Patient 2 had a baseline NPC-SS of 18, with the same score 4
months later and again 6 months after the second assessment.
Patient 3 had a baseline NPC-SS of 11 and the same score 7
months later.
Patient 4 had a baseline NPC-SS of 12 and the same score 6
months later.
Patient 5 had a baseline NPC-SS of 16, a score of 15 five months
later (improvement of 1 point), and a score of 13 eight months
later (representing improvement by total 3 points).
Patient 6 had a baseline NPC-SS of 12 and a score of 10 eight
months later (an improvement of 2 points).
Of the patients in the 2500 mg/kg dose group, both improved
overall:
Patient 7 had a baseline NPC-SS of 18, followed by a score of 20
four months later (a worsening of 2 points) and a score of 15 six
months later (a total improvement of 3 points compared to baseline
during the 10-month assessment period).
And, Patient 8 had a baseline NPC-SS of 12 and a score of 8 five
months later (an improvement of 4 points).
“These preliminary data provide additional signals of efficacy
for our drug as a treatment for NPC, a progressive and debilitating
disease often leading to premature death. The fact that patients
either stabilized or improved during periods up to one year is
consistent with data emerging from our Phase I/II in Europe and
Israel,” said Cyclo Therapeutics CEO and Director, N. Scott Fine.
“These extension protocol data provide additional compelling
support for the efficacy of our drug in this patient population,
and as we launch our pivotal Phase 3 trial in early 2021.”
The safety profile was favorable with 16 Adverse Events (AEs) in
total between May 2019 and September 2020, all mild (Grade 1 and 2)
and none related to the study drug, per the treating physician.
“NPC is a progressive, devastating disorder without approved
therapies in the United States. The overall safety profile of
Trappsol® Cyclo™ is reassuring in that the drug is well tolerated,
with adverse events in the mild and moderate range of severity. In
addition, the clinical trajectory of the participants suggests
stabilization while on therapy. These results offer the exciting
promise of an effect on the progression of symptoms in NPC, a
notable breakthrough for this condition,” said Loren Pena, MD, PhD,
Associate Professor, Department of Pediatrics, University of
Cincinnati College of Medicine and Attending Physician, Division of
Human Genetics, Cincinnati Children’s Hospital, who is not
associated with the study.
Cyclo Therapeutics has authorization from FDA to begin
enrollment in its phase 3 trial and plans to enroll the first
patient early in 2021.
About Cyclo Therapeutics:
Cyclo Therapeutics, Inc. is a clinical-stage biotechnology
company that develops cyclodextrin-based products for the treatment
of Niemann-Pick Disease Type C and Alzheimer’s 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, (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
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version on businesswire.com: https://www.businesswire.com/news/home/20210105005179/en/
Investor/Media Contact: Jeffrey L. Tate, Ph.D., COO Cyclo
Therapeutics, Inc. jeff@cyclodex.com +1 (386) 418-8060
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