Cyclo Therapeutics Recognizes Niemann-Pick Disease Awareness Month Highlighting Need for Broader Awareness and Support for Research to Find a Treatment

Oct 19, 2021

Company engaged in ongoing initiatives to help raise awareness and provide information on research and support for patients and families

Cyclo Therapeutics, Inc. (Nasdaq: CYTH), a clinical stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with diseases, today joins with the global Niemann-Pick Disease community in recognizing Niemann-Pick Disease Awareness Month, an initiative to build broader awareness of the impact this disease has on patients, families, and caregivers across the globe.

“As a company dedicated to advancing research to develop a treatment for Niemann-Pick Disease Type C (NPC), we are honored to commemorate Niemann-Pick Disease Awareness Month and join with the entire community in reinforcing the critical need for broader disease awareness, more services for families and caregivers, and support for research that can lead to the first approved therapy in the U.S. for a community in desperate need of treatment options,” said Scott Fine, CEO of Cyclo Therapeutics. “We also acknowledge the many leaders from the NPC community who have joined in efforts to share their stories and have made the important decision to participate in clinical research.”

NPC is a rare, progressive and fatal genetic disorder characterized by abnormal accumulation of cholesterol in cells. Symptoms of the disease include enlarged liver or spleen, difficulties with motor skills, speech, swallowing and feeding as well as cognitive impairment.

In honor of Niemann-Pick Disease Awareness Month, Cyclo Therapeutics will be engaging in several initiatives to help raise awareness and provide information on research and support for patients and families. These include:

  • Participation in an informational webinar in collaboration with the National Niemann-Pick Disease Foundation (NNPDF) to provide an update on the Company’s pivotal Phase 3 clinical trial, TransportNPC™, that is currently enrolling patients at treatment centers in the U.S. and around the world. A replay of this event is available at
  • The launch of, a new website with information about the disease and perspectives from patients and others about living with NPC.
  • A community newsletter with updates on progress in the Company’s research efforts.

“We have heard from so many patients and families about how much they need a treatment to be available for NPC, and Niemann-Pick Disease Awareness Month is an important time to renew our focus on moving promising research forward,” said Lisa Kjems, MD, PhD, the Company’s Chief Medical Officer. “We are very grateful to all of the patients and families who are supporting our efforts to develop a treatment for NPC. Their bravery and dedication inspire our work every day.”

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 four formal clinical trials for Niemann-Pick Disease Type C, a rare and fatal genetic disease, ( 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:

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Investor Contact:


Jenene Thomas

(833) 475-8247

Trappsol® Cyclo™ is not currently approved for any indication.

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