Niemann-Pick Diseases

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 its abstracts have been accepted for oral and poster presentation at the 21 st Annual WORLD Symposium ™ being held February 3-7, 2025 in San Diego, CA. Details of the presentations are as follows: Oral Presentation: Title: Trappsol ® Cyclo™: Open Label Treatment in the TransportNPC™ Sub-Study in Patients Under the Age of 3 Diagnosed with Niemann-Pick Disease Type C1 Presenter: Ronen Spiegel, MD, Clinical Associate Professor, Director of Pediatric B Department, and Co-Director Center of Rare Disease at Emek Medical Center Session: Clinical Applications Date and Time: Thursday, February 6, 2025 at 1:30 PM PT Poster Presentations: Title: Trappsol ® Cyclo™ (HPβCD) for the Long-Term Treatment of Niemann-Pick Type C1: Efficacy and Safety Data from 4 Clinical Studies and the Ongoing Expanded Access Program Presenter: Dr. Caroline Hastings, Pediatric hematologist oncologist, Director of Neuro-oncology, and Professor of Pediatrics at UCSF Benioff Children’s Hospital Oakland Session: Clinical Applications & Rapid-Fire – Poster Session III Date and Time: Thursday, February 6, 2025 from 3:30 – 5:30 PM PT Title: Trappsol ® Cyclo™ and NPC: Efficacy Shown Across Individual 5D Domains and Utilization of Future Assessment Tools to Demonstrate Clinically Relevant Outcomes Presenter: Dr. Caroline Hastings, Pediatric hematologist oncologist, Director of Neuro-oncology, and Professor of Pediatrics at UCSF Benioff Children’s Hospital Oakland Session: Clinical Applications & Rapid-Fire – Poster Session III Date and Time: Thursday, February 6, 2025 from 3:30 – 5:30 PM PT For more information, please visit the conference website worldsymposia.org . About WORLD Symposium™ WORLD Symposium™ is an annual research conference dedicated to lysosomal diseases. WORLD is an acronym that stands for We’re Organizing Research on Lysosomal Diseases. Since its inception as a small group of passionate researchers in 2002, WORLD Symposium™ has grown to an international research conference that attracts over 2000 participants from more than 50 countries around the globe. For more information, please visit: worldsymposia.org . 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: 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.

LEIDEN, Netherlands--( BUSINESS WIRE )--Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that it will present at J.P. Morgan’s 43 rd Annual Healthcare Conference on Thursday, January 16, 2025. The company’s presentation will begin at 8:30 a.m. PT/11:30 am (Eastern Time). The presentation will focus on the company’s lead product, nizubaglustat, a potential treatment for rare lysosomal storage disorders with neurological involvement including GM1/GM2 gangliosidoses and Niemann Pick type C (NPC). Positive topline data reported earlier this year from the company’s successful Phase 2 study investigating nizubaglustat in GM2 and NPC patients demonstrated that the compound had a positive safety profile. Preliminary improvements or stabilization of clinical endpoints were observed in the majority of patients, highlighting encouraging early efficacy trends for the compound. Azafaros is preparing to initiate Phase 3 studies with nizubaglustat in GM1/GM2 gangliosidoses and NPC in Q2, 2025. 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 GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC. Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC European Medicines Agency (EMA) Orphan Medicinal Product Designation (OMPD) for the treatment of for GM1 and GM2 gangliosidosis 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 premature 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 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 using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is 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 (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital.

Leiden, Netherlands, 09 January 2025 – Azafaros, a company focused on developing treatments for the unmet needs of patients with rare lysosomal storage disorders, today announced that it will present at J.P. Morgan’s 43rd Annual Healthcare Conference on Thursday, January 16, 2025. The company’s presentation will begin at 8:30 a.m. PT/11:30 am (Eastern Time). The presentation will focus on the company’s lead product, nizubaglustat, a potential treatment for rare lysosomal storage disorders with neurological involvement including GM1/GM2 gangliosidoses and Niemann Pick type C (NPC). Positive topline data reported earlier this year from the company’s successful Phase 2 study investigating nizubaglustat in GM2 and NPC patients demonstrated that the compound had a positive safety profile. Preliminary improvements or stabilization of clinical endpoints were observed in the majority of patients, highlighting encouraging early efficacy trends for the compound. Azafaros is preparing to initiate Phase 3 studies with nizubaglustat in GM1/GM2 gangliosidoses and NPC in Q2, 2025. 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 GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC. Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC European Medicines Agency (EMA) Orphan Medicinal Product Designation (OMPD) for the treatment of for GM1 and GM2 gangliosidosis 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 premature 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 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 using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is 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 (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital. For further information: Azafaros B.V. Email: info@azafaros.com www.azafaros.com