Cyclo Therapeutics Appoints Caroline Hastings, M.D. as Global Principal Investigator for Ongoing TransportNPC™ Study Evaluating Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease
December 09 2021 - 8:05AM
Business Wire
– Dr. Hastings is a world-renowned Key Opinion
Leader for Niemann-Pick Disease Type C1 and the first physician in
the U.S. to use cyclodextrins for treatment in NPC, compassionate
use
– Site activation ongoing and currently
enrolling patients in pivotal Phase 3 study, TransportNPC™
– Data seen to-date provide support for the
capacity of Trappsol® Cyclo™ to stabilize disease progression with
home-based intravenous infusions in NPC and demonstrate acceptable
safety profile of Trappsol® Cyclo™ 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
appointment of Caroline Hastings, M.D. as Global Principal
Investigator (“PI”) for its pivotal Phase 3 study
(“TransportNPC™”), which commenced in June 2021, evaluating
Trappsol® Cyclo™, the Company’s proprietary formulation of
hydroxypropyl beta cyclodextrin, for the treatment of Niemann-Pick
Disease Type C1 (“NPC”), a rare, progressive and fatal genetic
disorder.
Dr. Caroline Hastings 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. 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.
“Dr. Hastings is a key voice and advocate within the NPC
community and has dedicated her clinical career to serving this
community. We are honored to expand her role as our Global
Principal Investigator for our pivotal study and help drive this
important program forward toward potential approval. As a pioneer
in intravenous administration of cyclodextrins for NPC treatment
and with a wealth of knowledge and expertise, we believe she brings
a great deal of value to not only this program, but to other
investigators in the trial and the patient community,” commented
Lise Lund Kjems, MD, PhD, Chief Medical Officer of Cyclo
Therapeutics.
As the Global Principal Investigator for the TransportNPC™
study, Dr Hastings will serve as the senior scientific and clinical
expert for the trial, be a key resource for participating
investigators and Cyclo Therapeutics, as well as provide clinical
perspective in health authority interactions.
The Company’s pivotal TransportNPC™ Phase 3 study 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.
“Patients and families within the NPC community continue to be
faced with significant unmet needs. The clinical data to date shows
that Trappsol® Cyclo™ reaches the central nervous system (CNS) and
positively affects CNS biomarkers, when given intravenously. This
fuels my hope that these needs can be met. I am truly humbled to
serve as Global PI for this potentially catalytic program for the
NPC community. I am dedicated to advancing TransportNPC™ towards
completion and Trappsol® Cyclo™ towards potential approval for the
treatment of NPC,” added Dr. Hastings, Global Principal
Investigator for the TransportNPC™ trial and member of Cyclo
Therapeutics’ Scientific Advisory Board.
As previously announced, the Company also 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 in the
randomized study, and in addition, to include a single-arm
open-label 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 sub-study in patients from birth
to less than 3 years of age will only be conducted in the EU and
countries following EMA guidelines.
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
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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version on businesswire.com: https://www.businesswire.com/news/home/20211209005150/en/
Investor Contact: JTC Team, LLC Jenene Thomas (833)
475-8247 CYTH@jtcir.com
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