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

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 publication of positive data from its Phase 1/2 clinical trial, which demonstrated promising safety and efficacy results for Trappsol Cyclo in the treatment of Niemann-Pick Disease Type C1, a rare, genetic disease causing cholesterol accumulation in cells, leading to dysfunction of the liver, lung, spleen and brain and premature death. The manuscript titled, “Long-term administration of intravenous Trappsol Cyclo (HPβCD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick Disease Type C1: Results of an international 48-week Phase I/II trial,” were published in the official journal of Molecular Genetics and Metabolism Reports.

“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. I am pleased with the progress we’ve made and remain committed to exploring the potential of Trappsol Cyclo as a potential life-changing medicine for the NPC community,” commented Caroline Hastings, MD, author of the published manuscript, Chair of the Company’s Phase 3 Trappsol Cyclo Program Steering Committee and Global Principal Investigator for the Company’s ongoing TransportNPC study evaluating Trappsol Cyclo for the treatment of NPC.

Cyclo Therapeutics continues to advance enrollment in its ongoing pivotal Phase 3 study, TransportNPC. The Phase 3 study intends to enroll approximately 93 pediatric and adult patients (age 3 years and older) with NPC1 and is now active in 12 countries. Enrollment is expected to be completed by the end of this year.

N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics, commented, “We first provided this investigational therapy to address symptoms of NPC back in 2009. This was a compassionate use program begun in the US and several other countries. Our formal clinical study began in 2015 and our commitment to this community has only intensified. We look forward to completing enrollment and advancing this important program rapidly on behalf of this deserving community.”