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- Pivotal Phase 3 global study (TransportNPC\u2122) evaluating Trappsol\u00ae<\/sup> Cyclo\u2122 for Niemann-Pick Disease Type C1 (NPC1) projected 40% enrolled by the end of March; Company on track to complete enrollment by year end 2023<\/i><\/li>\n<\/ul>\n
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- Phase 2b study of Trappsol\u00ae<\/sup> Cyclo\u2122 for the treatment of early Alzheimer\u2019s Disease (AD) enrollment and dosing ongoing<\/i><\/li>\n<\/ul>\n
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- Completed financing with single Institutional Investor in January 2023 with net proceeds to the Company of $3.7 million<\/i><\/li>\n<\/ul>\n
GAINESVILLE, Fla.–(BUSINESS WIRE<\/a>)–Cyclo Therapeutics, Inc.<\/a> (Nasdaq: CYTH) (\u201cCyclo Therapeutics\u201d or the \u201cCompany\u201d), 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.\n<\/p>\n
\n\u201cWe continue to be extremely active and engaged with the NPC patient and scientific communities and believe these valuable interactions underpin the significant progress we have seen with the advancement of our pivotal Phase 3 program. Importantly, this momentum gives us the line of sight necessary to confirm our goal of completing enrollment before year end, a monumental milestone for the Company. Further we continue to have encouraging strategic discussions across the industry,\u201d commented N. Scott Fine, CEO of Cyclo Therapeutics. \u201cI am extremely proud of our entire team. As we think about the remainder of 2023, we believe this will be a breakthrough year for our company, driving value for all stakeholders. We continue to be cognizant of the markets but believe we are well positioned strategically. We have not been affected by the recent activity in the Banking Sector, and our commitment to driving our programs forward remains unwavering.\u201d\n<\/p>\n
Recent Pipeline Highlights<\/b><\/p>\n
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- \nProgressed patient enrollment to 40% complete in the ongoing TransportNPC\u2122 study completed and dosing ongoing across multiple regions;\n<\/li>\n
- \nInitiated patient dosing in Phase 2b Study of Trappsol\u00ae<\/sup> Cyclo\u2122 for the treatment of early Alzheimer\u2019s Disease; and\n<\/li>\n
- \nPresented poster titled \u201cTransportNPC: A Phase 3 Global Trial of Trappsol\u00ae<\/sup> Cyclo\u2122 Administered Intravenously to Patients with Niemann-Pick Disease Type C1 (NPC1),\u201d<\/i> at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting.\n<\/li>\n<\/ul>\n
Trappsol\u00ae<\/sup> Cyclo\u2122 Clinical Program Update<\/b><\/p>\n
\nTrappsol\u00ae<\/sup> Cyclo\u2122 is the Company\u2019s proprietary formulation of hydroxypropyl beta cyclodextrin, used intravenously (IV) and currently in development for the treatment of NPC, a rare genetic disorder causing cholesterol accumulation in lysosomes of cells, organ dysfunction and premature death, and early Alzheimer\u2019s Disease (AD), where disrupted lipid pathways play a key role in the etiology and disease progression.\n<\/p>\n
Niemann-Pick Disease Type C1 Development Program<\/i><\/p>\n
\nThe Company continues to advance enrollment in its ongoing pivotal Phase 3 study, TransportNPC\u2122. 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\u00ae<\/sup> Cyclo\u2122 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.\n<\/p>\n
\nTo date, the Company remains on track for expected completion of enrollment by year end 2023.\n<\/p>\n
\nCyclo Therapeutics received Orphan Drug Designation for Trappsol\u00ae<\/sup> Cyclo\u2122 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.\n<\/p>\n
\nFor more information about the Company\u2019s Trappsol\u00ae<\/sup> Cyclo\u2122 clinical program for the treatment of NPC1, visit www.ClinicalTrials.gov<\/a> and reference identifiers NCT02939547<\/a>, NCT02912793<\/a>, NCT03893071<\/a> and NCT04860960<\/a>.\n<\/p>\n
Alzheimer\u2019s Disease Development Program<\/i><\/p>\n
\nThe Company\u2019s ongoing 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\u00ae<\/sup> Cyclo\u2122 infusions in patients with early Alzheimer\u2019s disease. The study will enroll approximately 120 patients who have shown evidence of progressive cognitive decline in the last year as determined by serial cognitive test scores, if available, or patient or informant\/caregiver\/study partner report as documented by the Investigator. Enrolled patients will be randomized across three study arms: 500 mg\/kg or 1000 mg\/kg of Trappsol\u00ae<\/sup> Cyclo\u2122 and placebo. The study will consist of a screening period, treatment period up to 24 weeks, and a safety follow-up period.\n<\/p>\n
\nFor more information about the Company\u2019s Trappsol\u00ae<\/sup> Cyclo\u2122 clinical program for the treatment of early AD and Expanded Access program, visit www.ClinicalTrials.gov<\/a> and reference identifiers NCT05607615<\/a> and NCT03624842<\/a>, respectively.\n<\/p>\n
Summary of Financial Results for the Full Year 2022<\/b><\/p>\n
\nNet loss for the year ended December 31, 2022 was approximately $15.5 million. Research and development expenses decreased 2% to $9.0 million for the year ended December 31, 2022, from $9.2 million for the year ended December 31, 2021. The changes in research and development expenses relate to the timing of startup costs in our clinical programs. We expect research and development costs to increase in 2023 as we continue to seek regulatory approval for the use of Trappsol\u00ae<\/sup> Cyclo\u2122 in the treatment of NPC and Alzheimer\u2019s disease.\n<\/p>\n
\nThe Company ended the year with approximately $1.5 million of cash. Pro-Forma year end cash of $5.2 million post the financing completed on with single institutional investor in January of 2023.\n<\/p>\n
About Cyclo Therapeutics<\/i><\/b><\/p>\n
\nCyclo Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing life-changing medicines through science and innovation for patients and families living with disease. The Company\u2019s Trappsol\u00ae<\/sup> Cyclo\u2122, 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<\/a>NCT02939547<\/a>, NCT02912793<\/a>, NCT03893071<\/a> and NCT04860960<\/a>). The Company is conducting a Phase 2b clinical trial using Trappsol\u00ae<\/sup> Cyclo\u2122 intravenously in early Alzheimer\u2019s disease (NCT05607615<\/a>) based on encouraging data from an Expanded Access program for Alzheimer\u2019s disease (NCT03624842<\/a>). Additional indications for the active ingredient in Trappsol\u00ae<\/sup> Cyclo\u2122 are in development. For additional information, visit the Company\u2019s website: www.cyclotherapeutics.com<\/a>.\n<\/p>\n
Safe Harbor Statement<\/i><\/b><\/p>\n
\nThis press release contains \u201cforward-looking statements\u201d about the company\u2019s 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 \u201canticipates,\u201d \u201cbelieves\u201d and \u201cexpects\u201d 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\u2019s future performance include the company\u2019s 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\u2019s 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\u2019s filings with the Securities and Exchange Commission, including, but not limited to, the company\u2019s 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.\n<\/p>\n
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View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20230320005157\/en\/<\/a><\/span><\/p>\n
- \nPresented poster titled \u201cTransportNPC: A Phase 3 Global Trial of Trappsol\u00ae<\/sup> Cyclo\u2122 Administered Intravenously to Patients with Niemann-Pick Disease Type C1 (NPC1),\u201d<\/i> at the Society for Inherited Metabolic Disorders (SIMD) 44th Annual Meeting.\n<\/li>\n<\/ul>\n
- Completed financing with single Institutional Investor in January 2023 with net proceeds to the Company of $3.7 million<\/i><\/li>\n<\/ul>\n
- Phase 2b study of Trappsol\u00ae<\/sup> Cyclo\u2122 for the treatment of early Alzheimer\u2019s Disease (AD) enrollment and dosing ongoing<\/i><\/li>\n<\/ul>\n