Benzinga | Nov 16, 2021 08:10AM EST

Cyclo Therapeutics Submits Investigational New Drug Application To FDA To Advance Trappsol Cyclo In Phase 2 Study For Treatment Of Alzheimer's Disease

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 it has submitted its initial investigational new drug ("IND") application with the U.S. Food and Drug Administration ("FDA") for a Phase 2 study of Trappsol(r) Cyclo(tm) for the treatment of early Alzheimer's Disease (AD).

"We are excited to have submitted our Initial IND to evaluate Trappsol(r) Cyclo(tm) for the treatment of Alzheimer's Disease. The feedback and recommendations received from our Type B Meeting with the FDA have provided valuable input that we believe positions us for success in advancing this asset. We have taken another step towards commencing our Phase 2 study and, importantly, bringing a much-needed treatment option to patients and families," commented Michael Lisjak, Chief Regulatory Officer, Senior Vice President for Business Development of Cyclo Therapeutics.

Trappsol(r) Cyclo(tm) is a proprietary formulation of hydroxypropyl beta cyclodextrin and in multiple clinical studies has shown encouraging results to effectively manage the transportation of cholesterol. Many of the known risk factors for AD are associated with cholesterol metabolism. Cholesterol imbalance in AD patients is well known, and significant research exists suggesting these imbalances are responsible for A? and tau accumulation. Furthermore, neurons, because of their high metabolic demands, experience an increased level of oxidative stress. Oxidative stress has also been linked to abnormal cholesterol accumulation and processing. AD shares features with NPC-1, a neurovisceral, genetic disease in which cholesterol accumulates in lysosomes, including progressive decline in cognitive ability, amyloid beta plaques in the CNS, and increased levels of tau in the cerebrospinal fluid (CSF). Cyclo Therapeutics is currently testing the same investigational Trappsol(r) Cyclo(tm) drug in clinical trials for the treatment of Niemann-Pick Disease Type C1 (NPC-1). Taking the place of the defective NPC-1 protein, Trappsol(r) Cyclo(tm), with its cyclic structure, facilitates the transport of accumulated cholesterol out of cellular lysosomes so it can be further processed and excreted out of cells.

"There remain significant unmet needs within the Alzheimer's Disease community, which we believe Trappsol(r) Cyclo(tm) is well-positioned to address. We are committed to advancing this important program as quickly and efficiently as possible following feedback from FDA in the coming weeks," added N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics.