Niemann-Pick Diseases

Continued progress of pivotal Phase 3 study (TransportNPC™) evaluating Trappsol® Cyclo™ for the treatment of Niemann-Pick Disease Type C1 (NPC1) toward 48-week comparative interim analysis data readout, planned for Q1 2025 Aligned development strategy for Trappsol® Cyclo™ with FDA to enable a patient population lacking available therapies to have earlier access to a potentially efficacious product with an acceptable safety pro > Ended the year with approximately $9.2 million cash to fund operations; continued efforts to bolster cash position and extend runway through clinical and regulatory milestones GAINESVILLE, Fla.--(BUSINESS WIRE)-- 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 2023 and provided a business update. “We made significant progress over the course of the past year. We continue to be extremely active with the NPC patient and scientific communities and believe these valuable interactions underscore the progress we have seen with the advancement of our TransportNPC study,” commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics. “Following the completion of the merger with Applied Molecular Transport, we are poised to continue building momentum and achieving the value-driving milestones ahead. Importantly, supported with the positive feedback from FDA and alignment on our strategy moving forward, we remain focused on generating the appropriate data required to successfully drive our TransportNPC study across the finish line and the potential to provide a much-needed treatment option for the NPC community. We are grateful for the continued support of our investors, including Rafael Holdings and other longtime shareholders, who have bolstered our cash runway and helped position us to achieve our mission and continue our work to advance the clinical development of Trappsol® Cyclo™.” William Conkling, Chief Executive Officer of Rafael Holdings, Inc., added, “We are pleased with the continued clinical progress and operational execution and look forward to the continued advancement of those efforts in 2024.” Recent Highlights Granted U.S. patent covering use of Trappsol® Cyclo™ for treatment of Alzheimer's Disease from the U.S. Patent and Trademark Office; Completed merger with Applied Molecular Transport Inc to focus on advancing Cyclo Therapeutics’ pivotal Phase 3 global study, TransportNPC™, evaluating Trappsol® Cyclo™ for NPC; Dr. Caroline Hastings, Chair of the Company’s Phase 3 Trappsol® Cyclo™ program steering committee and Global Principal Investigator for the Company’s ongoing TransportNPC™ study presented at the 20th Annual WORLDSymposium™; and Announced 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. Strengthened Balance Sheet with early exercise of warrants by board members, management and a significant shareholder resulting in bolstered cash proceeds of $2.4 million. The Investors include Rafael Holdings, Inc., a significant shareholder of the Company, several directors of the Company, Company management, and their affiliates. Summary of Financial Results for the Full Year 2023 Net loss for the year ended December 31, 2023 was approximately $20.1 million. Research and development expenses increased 58% to $14.2 million for the year ended December 31, 2023, from $9.0 million for the year ended December 31, 2022. The changes in research and development expenses resulted from the increased activity in the Company’s Phase 3 study of Trappsol® Cyclo™ for the treatment of NPC. The Company ended the year with approximately $9.2 million of cash. 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 living with 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 C1, a rare and fatal genetic disease, ( NCT02939547, NCT02912793, NCT03893071 and NCT04860960). The Company is conducting a Phase 2b clinical trial using Trappsol® Cyclo™ intravenously in early Alzheimer’s disease (NCT05607615) based on encouraging data from an Expanded Access program for Alzheimer’s disease (NCT03624842). Additional indications for the active ingredient in Trappsol® Cyclo™ are in development. For additional information, visit the Company’s website: . Safe Harbor Statement This press release contains “forward-looking statements” about the company’s 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 “anticipates,” “believes” and “expects” 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’s future performance include the company’s 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’s 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’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s 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.

GAINESVILLE, Fla.--( BUSINESS WIRE )-- 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 United States Patent and Trademark Office (USPTO) has granted U.S. Patent No. 11,925,659 titled, “ Methods for Treating Alzheimer’s Disease ,” with claims that cover the use of Trappsol ® Cyclo™ for the prevention and treatment of early onset Alzheimer’s disease. “There continues to be a significant unmet need for therapeutic solutions to treat early onset Alzheimer’s disease. We are continuing to build momentum in our early AD clinical study evaluating Trappsol ® Cyclo™ and continue to believe in its potential to address the unmet medical need that patients, families and physicians are facing. We are pleased to add this now issued patent and remain committed to bolstering our patent estate covering Trappsol ® Cyclo™,” commented N. Scott Fine, Chief Executive Officer of Cyclo Therapeutics. 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 living with 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 C1, a rare and fatal genetic disease, ( www.ClinicalTrials.gov NCT02939547 , NCT02912793 , NCT03893071 and NCT04860960 ). The Company is conducting a Phase 2b clinical trial using Trappsol ® Cyclo™ intravenously in early Alzheimer’s disease ( NCT05607615 ) based on encouraging data from an Expanded Access program for Alzheimer’s disease ( NCT03624842 ). Additional indications for the active ingredient in Trappsol ® Cyclo™ are in development. For additional information, visit the Company’s website: www.cyclotherapeutics.com . Safe Harbor Statement This press release contains “forward-looking statements” about the company’s 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 “anticipates,” “believes” and “expects” 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’s future performance include the company’s 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’s 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’s filings with the Securities and Exchange Commission, including, but not limited to, the company’s 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.

Azafaros announces completion of 12-week Phase 2 RAINBOW study evaluating lead asset nizubaglustat in rare disease patients Major milestone reached in bringing new treatment options to patients and their families. Leiden, The Netherlands, March 12, 2024 – Azafaros B.V. today announced the completion of its 12-week Phase 2 clinical study, RAINBOW (Phase 2 RAINBOW studyNCT05758922). The randomized, double-blind, placebo-controlled study, conducted in Brazil, involved 13 patients from the age of 12 years who are affected by GM2 gangliosidosis or Niemann-Pick disease type C (NPC). The aim of the RAINBOW study is to determine the safety, pharmacodynamics, and pharmacokinetics of two different doses of nizubaglustat in patients, in order to identify the target dosage for Azafaros’ planned Phase 3 pivotal studies. The main part of the study is now complete, and patients are entering the extension phase, in which all of them will be given the study drug. Top line results of the RAINBOW study are expected to be announced in Q2 2024 and presented to the scientific community later this year. Prof. Dr. Roberto Giugliani, Chief of the Medical Genetics Clinical Research Group at the Hospital de Clinicas de Porto Alegre and Lead Principal Investigator for the study, said: “This is an important milestone because nizubaglustat has a dual mode of action that represents a leap forward from other agents. This dual activity targets some of the fundamental biology of the diseases with high potency and it might offer promise in pivotal studies.” Chris Freitag, Chief Medical Officer at Azafaros, said: “This is a major milestone on our journey to provide patients and their families with a potentially transformative treatment. With the RAINBOW data, we will be able to identify the optimal dose for a pediatric population in our Phase 3 efficacy study, which we plan to initiate as soon as the dose is determined, and the required approvals are in place. We are looking forward to presenting the data from RAINBOW to the scientific community. Azafaros is grateful to the patients and their families who made the decision to participate in this study.” About nizubaglustat Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NPC). Nizubaglustat has received the following designations and support: United States Food and Drug Administration (FDA) Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC. Orphan Drug Designations (ODD) for GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC. Fast Track Designation and IND clearance for GM1 and GM2 gangliosidoses. European Medicines Agency (EMA) Orphan Medicinal Product Designation (OMPD) for the treatment of GM2 gangliosidosis. UK Medicines and Healthcare Products Regulatory Agency (MHRA) Innovation Passport for the treatment of GM1 and GM2 gangliosidoses. About GM1 and GM2 Gangliosidoses GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous system (CNS), resulting in progressive and severe neurological impairment and early death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available. About Niemann-Pick Disease Type C (NPC) Niemann-Pick disease type C (NPC) is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease can happen throughout the lifespan of an affected individual, from prenatal life through adulthood. About Azafaros Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms, a compound library from Leiden University, and led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by a syndicate of leading Dutch and Swiss investors including Forbion, BioGeneration Ventures, BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital. Media ICR Consilium Amber Fennell, Ashley Tapp +44 (0)20 3709 5700 Azafaros@consilium-comms.com