Cyclo Therapeutics Announces Formation of Global Steering Committee Comprised of Leading Experts to Advise on the Global Phase 3 Clinical Development Program for Trappsol® Cyclo™ in Niemann-Pick Disease Type C
February 03 2022 - 8:05AM
Business Wire
– Company’s pivotal Phase 3 study,
TransportNPC™, ongoing with global site activation and patient
enrollment underway
– Data seen to-date provide support for the
capacity of Trappsol® Cyclo™ to stabilize disease progression with
home-based intravenous infusions in NPC
– Trappsol® Cyclo™ demonstrates acceptable
safety profile, administered intravenously, for more than 2
years
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
formation of a Global Steering Committee (GSC) to guide the pivotal
Phase 3 global clinical development program of Trappsol® Cyclo™ for
the treatment of Niemann-Pick Disease Type C (NPC). As the Global
Principal Investigator for the TransportNPC™ study, Caroline
Hastings, MD serves as the senior scientific and clinical expert
for the trial and will also chair the GSC.
“Dr. Caroline Hastings, global principal investigator for
TransportNPC™ and chair of the GSC, has been instrumental in
assembling this high caliber Global Steering Committee with
representation of renowned Key Opinion Leaders and clinical experts
in NPC. It is another testimony of our commitment to serve the NPC
community and deliver on the unmet medical needs. I feel humbled
and privileged to be working with this outstanding group of
professionals who are committed to advance science and clinical
trials that can bring hope and treatment benefits to so many
patients and their families,” commented Lise Kjems, MD, PhD, Chief
Medical Officer of Cyclo Therapeutics.
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.
Dr. Hastings, Global Principal Investigator for the
TransportNPC™ trial and member of Cyclo Therapeutics’ Scientific
Advisory Board added, “I am very grateful by the overwhelmingly
positive responses as I reached out to fellow scientists and
physicians to invite them to join the Global Steering Committee. I
am honored to be working alongside these wonderful colleagues with
outstanding knowledge and expertise and who represent the excellent
investigators taking part in the TransportNPC™ trial. Together, we
have a very unique opportunity to further refine the scientific
strategy for Trappsol® Cyclo™ and help drive this important program
toward potential approval.”
“NPC is a devastating neurodegenerative disease that needs more
effective therapies. Given the clinical course and progressive
nature of this disease, novel therapeutic strategies with the
potential for disease modifying effects are necessary. The
TransportNPC™ trial is unique as it is designed to demonstrate the
long-term clinical benefits and potential for disease
modification,” commented Professor Roberto Giugliani, MD, PhD.
“I have been caring for patients with NPC for more than 25
years. These patients urgently need better treatment options that
will better halt the cruel, neurodegenerative course that this
disease takes. In this study with cyclodextrin intravenously, I see
an opportunity to improve the therapeutic offer,” added Dr. Eugen
Mengel.
The members of the TransportNPC™ Global Steering Committee
are:
- Professor Caroline Hastings, MD is the Chair of the
Phase 3 Trappsol® Cyclo™ Program Steering Committee, and the Global
Principal Investigator for the Company’s ongoing TransportNPC™
study evaluating Trappsol® Cyclo™ for the treatment of NPC. Dr.
Hastings currently serves as the Pediatric hematologist oncologist,
Director of Neuro-oncology, and Professor of Pediatrics at UCSF
Benioff Children’s Hospital Oakland and is an advisor to U.S. and
Australian NPC Advocacy organizations and to physicians globally on
NPC. She has been practicing in the field of Pediatric Hematology
Oncology since 1992 and has served as the director of the
fellowship program at the Children’s Hospital & Research Center
Oakland since 1996. She has devoted herself to her patients and to
fostering education in this specialty. Her academic interests
include tumors of the brain and spinal cord, relapsed acute
lymphoblastic leukemia, and lysosomal storage diseases including
Niemann Pick Type C disease.
- Professor Mark Walterfang MBBS Hons, PhD, FRANZCP is a
consultant psychiatrist at the Adult Mental Health Rehabilitation
Unit at Sunshine Hospital and a consultant neuropsychiatrist at the
Royal Melbourne Hospital Neuropsychiatry Unit. Dr. Walterfang has
been involved in the development in a number of clinical tools for
use in psychiatric patients in the areas of cognition and
behavioral observation, and their validation in a variety of
medical, neurological and psychiatric settings since 2000. His
continuing research involves the neuroimaging and neuropsychiatric
investigation of neurometabolic disorders including phenylketonuria
and NPC, and in shape analysis of cortical and subcortical regions
in neurodegenerative disorders. He has published over 170
Medline-indexed scientific papers, and recently contributed a new
chapter on the Neuropsychiatry of Neurometabolic and Neuroendocrine
Disorders to the world's most respected reference text in
psychiatry, Kaplan and Sadock's "Comprehensive Textbook of
Psychiatry".
- Professor Maurizio Scarpa, MD, PhD, Paediatrician
is the Founder and President of the Brains for Brain Foundation, a
Pan-European task force on brain and neurodegenerative diseases. He
is Professor of Paediatrics at the Department of Women’s and
Children’s Health, University of Padova, Italy and the Director,
Coordinating Center for Rare Diseases, Udine University Hospital,
Udine, Italy. Professor Scarpa has extensive expertise as a basic
scientist in genetics and biotechnology, and as a clinician in the
diagnosis and treatment of paediatric rare disorders,
neurometabolic diseases in particular. He is especially interested
in developing innovative health approaches for the diagnosis and
the treatment of metabolic inherited diseases. Professor Scarpa has
published about 140 international peer reviewed clinical and
scientific papers, book chapters and reviews. Professor Scarpa is
the Coordinator of the European Reference Network for Inherited
Metabolic Diseases (MetabERN).
- Dr. Eugen Mengel is the principal investigator, and the
founder and CEO of the SphinCS GmbH & SphinCS Lyso non-profit
UG. Dr. Mengel has dedicated himself to research in the field of
lysosomal diseases and has already significantly contributed to the
advancement of this field of study. He is the co-author of 129
peer-reviewed publications with focuses on Gaucher disease, NPC and
Pompe disease and is an active member of the Society for the Study
of Inborn Errors of Metabolism (SSIEM), European Study Group on
Lysosomal Diseases (ESGLD) and European Working Group on Gaucher
Disease (EWGGD).
- Orna Staretz-Chacham, MD is a neonatologist, metabolic
specialist and senior lecturer at the Faculty of Health Sciences,
Ben-Gurion University of the Negev, Israel. For years Dr.
Staretz-Chacham has worked in the field of inherited metabolic
disorders with a focus on neonate and leads the Center for Rare
Disease in Soroka Medical Center in since 2021. Among the many
critical contributions stemming from her research is
characterization of the pulmonary pathologies in Niemann-Pick C1
and most recently in lysosomal patients with COVID-19. Dr.
Staretz-Chacham is regarded as one of most prominent clinical
experts in the clinical pulmonary presentations in patients with
NPC. Dr. Staretz-Chacham has been involved in clinical trials with
Trappsol® Cyclo™ from early on and has significant experience in
treating patients with NPC. Her commitment to clinical research is
evidenced by the more than 40 published articles in peer-reviewed
journals in the field of inherited metabolic diseases.
- Loren DM Pena, MD, PhD is a Clinical Geneticist,
Division of Human Genetics, and Associate Professor, Department of
Pediatrics at Cincinnati Children’s Hospital Medical Center. As a
clinical geneticist, Dr. Pena works with children who have rare and
orphan diseases, lysosomal storage disorders and metabolic
disorders, and also works in neurogenetics and gene discovery. Dr.
Pena has made noteworthy contributions to the field through her
research as a lead faculty member for clinical trials in genetics
and leading several clinical trials for rare conditions.
- Professor Roberto Giugliani, MD, PhD is a medical
geneticist with specialization in inherited metabolic diseases. He
is Full Professor at Department of Genetics of the Federal
University of Rio Grande do Sul and active member of the Medical
Genetics Service of HCPA (University hospital), Brazil. He is also
Coordinator of the Brazilian Institute of Population Medical
Genetics, Editor-in-Chief of the Journal of Inborn Errors of
Metabolism and Screening, Chairman of the Latin American School of
Human and Medical Genetics, Member of Brazilian Academy of
Sciences, Head of Rare Diseases of DASA/GeneOne, and co-founder of
House of Rares. He is the past President of the Latin American
Society of Inborn Errors of Metabolism and Newborn Screening, the
Latin American Network of Human Genetics (RELAGH), and the
Brazilian Society of Medical Genetics. Professor Giugliani’s main
interests are concentrated in screening, diagnosis, and treatment
of IEM, having supervised the training of over 100 MSc/PhDs, and
authoring of over 500 scientific papers.
- Professor Dr. Fatih Ezgü is a Professor of
Pediatrics and Head of the Department of Pediatric Genetics at Gazi
University, Faculty of Medicine, Department of Pediatrics in
Ankara, Turkey. He is also on faculty at the Department of
Pediatric Metabolic Disorders at the same institution. Professor
Ezgü has worked in the field of inborn metabolic and genetic
diseases for 21 years and has published over 100 articles in
peer-reviewed journals. His primary interests are lysosomal
disorders, mitochondrial disorders, clinical dysmorphology and
genetic and metabolic bone disease and novel treatments for genetic
disorders. Dr. Ezgü is the vice chairman of the Phase 1 Clinical
Trials Center for Genetic Diseases at Gazi University.
For more information about the Company’s TransportNPC™ pivotal
Phase 3 study, visit www.ClinicalTrials.gov and reference
identifier NCT04860960.
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.
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
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the company’s current expectations about future results,
performance, prospects and opportunities, including, without
limitation, statements regarding the satisfaction of closing
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proceeds from the offering. Statements that are not historical
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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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JTC Team, LLC Jenene Thomas (833) 475-8247 CYTH@jtcir.com
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