NPC1

Orphan designation: Adeno-associated viral vector serotype 9 containing the human NPC1 gene Treatment of Niemann-Pick's disease, type C, 13/10/2023 Positive

Retrieved on: 
Sunday, February 4, 2024

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Adeno-associated viral vector serotype 9 containing the human NPC1 gene
    - Intended use
    - Treatment of Niemann-Pick's disease, type C
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2843
    - Date of designation
    - Sponsor
    FGK Representative Service GmbH
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Cyclo Therapeutics, Inc. Completes Merger with Applied Molecular Transport Inc.

Retrieved on: 
Wednesday, December 27, 2023

The combined company will now operate under the name “Cyclo Therapeutics, Inc.” and will continue trading on The Nasdaq Capital Market under the ticker symbol “CYTH”.

Key Points: 
  • The combined company will now operate under the name “Cyclo Therapeutics, Inc.” and will continue trading on The Nasdaq Capital Market under the ticker symbol “CYTH”.
  • Cyclo Therapeutics’ primary focus will continue to be on advancing its pivotal Phase 3 global study (TransportNPC™) evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1).
  • “The completion of this merger with Applied Molecular Transport represents a significant milestone and bolsters our confidence and excitement for the future of Cyclo Therapeutics.
  • With the merger now closed and following our recent positive Type C meeting with the FDA, fundamentally the Company has never been stronger,” commented N. Scott Fine, CEO of Cyclo Therapeutics.

Cyclo Therapeutics Announces Positive Outcome from Type C Meeting with the U.S. FDA Discussing Trappsol® Cyclo™ Clinical Program for the Treatment of Niemann-Pick Disease Type C1

Retrieved on: 
Monday, December 18, 2023

N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, added, “We view our Type C meeting with FDA as a catalytic and transformational milestone for our ongoing pivotal Phase 3 NPC program.

Key Points: 
  • N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, added, “We view our Type C meeting with FDA as a catalytic and transformational milestone for our ongoing pivotal Phase 3 NPC program.
  • 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.

Cyclo Therapeutics, Inc. and Applied Molecular Transport Inc. Enter into a Definitive Merger Agreement

Retrieved on: 
Thursday, September 21, 2023

Following the closing of the Merger, the combined company will operate under the name “Cyclo Therapeutics, Inc.” and will continue trading on The Nasdaq Capital Market under the ticker symbol “CYTH”.

Key Points: 
  • Following the closing of the Merger, the combined company will operate under the name “Cyclo Therapeutics, Inc.” and will continue trading on The Nasdaq Capital Market under the ticker symbol “CYTH”.
  • The combined company will focus on advancing Cyclo Therapeutics’ pivotal Phase 3 global study (TransportNPC™) evaluating Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1).
  • “We are extremely pleased to enter into this merger agreement with Applied Molecular Transport,” commented N. Scott Fine, CEO of Cyclo Therapeutics.
  • Cyclo Therapeutics has continued to demonstrate its commitment to developing life-changing medicines and execution across clinical and regulatory fronts.

CENTOGENE Biodatabank Reveals Unique Genetic Variants in World's Largest Niemann-Pick Type C1 Disease Cohort

Retrieved on: 
Wednesday, July 12, 2023

NPC1 is a rare and severe autosomal recessive disorder, characterized by a range of neurovisceral clinical manifestations and a fatal outcome with no effective treatment to date.

Key Points: 
  • NPC1 is a rare and severe autosomal recessive disorder, characterized by a range of neurovisceral clinical manifestations and a fatal outcome with no effective treatment to date.
  • To gain a deeper understanding of the disease, researchers carried out an analysis using the CENTOGENE Biodatabank, which included clinical, genetic, and biomarker data from NPC1 patients from the past 15 years.
  • “The publication of this landmark study marks a pivotal moment in our understanding of Niemann-Pick type C1 disease patients,” said Professor Peter Bauer, CENTOGENE’s Chief Medical and Genomic Officer.
  • “By assembling the world's largest NPC1 cohort, we have gained unprecedented insights that will fuel further research, clinical development, and ultimately, improved patient outcomes.”
    To read the full study, visit: https://link.centogene.com/npc1-publication

Cyclo Therapeutics Announces Publication of Positive Data from Phase 2 Clinical Study of Trappsol® Cyclo™ for the Treatment of Niemann-Pick Disease Type C1

Retrieved on: 
Thursday, June 29, 2023

“The data seen to-date provide support for the capacity of Trappsol® Cyclo™ to alter the disease on a biochemical level, including in the central nervous system, and improve clinical signs and symptoms of NPC.

Key Points: 
  • “The data seen to-date provide support for the capacity of Trappsol® Cyclo™ to alter the disease on a biochemical level, including in the central nervous system, and improve clinical signs and symptoms of NPC.
  • In these Phase 2 data, all three doses of Trappsol® Cyclo™ were well-tolerated overall with efficacy across multiple clinical endpoints of intravenous (IV) administration of Trappsol® Cyclo™.
  • Cyclo Therapeutics continues to advance enrollment in its ongoing pivotal Phase 3 study, TransportNPC™ .
  • N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, commented, “We first provided this investigational therapy to address symptoms of NPC back in 2009.

International Niemann-Pick Disease Registry and Mandos Health announce new research collaboration using real-world data to evaluate the treatment effects of VTS-270 to manage Niemann-Pick type C1

Retrieved on: 
Friday, June 16, 2023

The International Niemann-Pick Disease Registry (INPDR) and biopharmaceutical company Mandos Health today announce a new collaboration giving Mandos Health controlled access to registry information via INPDR’s research “Gateway” platform.

Key Points: 
  • The International Niemann-Pick Disease Registry (INPDR) and biopharmaceutical company Mandos Health today announce a new collaboration giving Mandos Health controlled access to registry information via INPDR’s research “Gateway” platform.
  • This partnership will provide Mandos Health with real-world evidence that will directly inform the development of its Niemann-Pick type C1 (NPC1) investigational product, VTS-270.
  • INPDR is the largest active database about the diagnosis, management, and progression of the devastating inherited lysosomal storage disorder known as Niemann-Pick type C1.
  • This collaboration with Mandos Health demonstrates that INPDR is serving the Niemann-Pick community in the way it was intended.

Cyclo Therapeutics Provides Business Update and Reports Full Year 2022 Financial Results

Retrieved on: 
Monday, March 20, 2023

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 reported its financial results for the full year 2022 and provided a business update.

Key Points: 
  • 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 reported its financial results for the full year 2022 and provided a business update.
  • Further we continue to have encouraging strategic discussions across the industry,” commented N. Scott Fine, CEO of Cyclo Therapeutics.
  • 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.
  • Enrolled patients will be randomized across three study arms: 500 mg/kg or 1000 mg/kg of Trappsol® Cyclo™ and placebo.

Cyclo Therapeutics Provides Clinical Program Update and Highlights Recent Achievements

Retrieved on: 
Wednesday, January 11, 2023

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 provided updates for the Company’s Trappsol® Cyclo™ clinical program and provided a summary of key achievements in 2022, focused on our clinical portfolio of two neurodegenerative diseases with major unmet medical needs.

Key Points: 
  • 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 provided updates for the Company’s Trappsol® Cyclo™ clinical program and provided a summary of key achievements in 2022, focused on our clinical portfolio of two neurodegenerative diseases with major unmet medical needs.
  • We recently announced the commencement of patient enrollment and dosing in our early AD clinical study and are dedicated to building momentum for that program.
  • For more information about the Company’s Trappsol® Cyclo™ clinical program for the treatment of NPC1, visit www.ClinicalTrials.gov and reference identifiers NCT02939547 , NCT02912793 , NCT03893071 and NCT04860960 .
  • For more information about the Company’s Trappsol® Cyclo™ clinical program for the treatment of early AD and Expanded Access program, visit www.ClinicalTrials.gov and reference identifiers NCT05607615 and NCT03624842 , respectively.

Cyclo Therapeutics Announces First Patient Dosed in Phase 2b Study of Trappsol® Cyclo™ for the Treatment of Early Alzheimer’s Disease

Retrieved on: 
Monday, January 9, 2023

“The start of this study is a significant milestone for our Company and the Alzheimer’s disease community.

Key Points: 
  • “The start of this study is a significant milestone for our Company and the Alzheimer’s disease community.
  • The Phase 2b study is a U.S. multicenter, randomized, placebo-controlled, double-blind, parallel group, 6-month study evaluating the safety, tolerability, and potential efficacy of monthly Trappsol® Cyclo™ infusions in patients with early Alzheimer’s disease.
  • Enrolled patients will be randomized across three study arms: 500 mg/kg or 1000 mg/kg of Trappsol® Cyclo™ and placebo.
  • The study will consist of a screening period, treatment period up to 24 weeks, and a safety follow-up period.