Trappsol Cyclo (hydroxypropyl beta cyclodextrin) / Rafael Holdings, University of the Witwatersrand - LARVOL DELTA

Cyclo Therapeutics’ TransportNPC Phase 3 clinical trial for Trappsol Cyclo for the treatment of Niemann-Pick Disease Type C1, a rare and fatal genetic disease, is continuing at the recommendation of the Data Monitoring Committee (DMC) following their review of prespecified safety and efficacy date at 48 weeks (GlobeNewswire)

Trial status • Rare Diseases

Rafael Holdings, Inc. Announces Revised Subscription Rights in Connection With its Previously Announced $25 Million Rights Offering to Support Its Commitment to the Development and Potential Launch of Trappsol Cyclo (Rafael Pharma Press Release) - "Rafael Holdings, Inc....announced revised subscription rights in connection with its previously announced rights offering....Under the proposed rights offering, Rafael Holdings intends to distribute one (1) non-transferable subscription right for each share of Class B Common Stock or Class A Common Stock or share of Class B Common Stock purchasable on exercise of Public Warrants, in each case, held as of the Record Date. Each subscription right will entitle the Holder to purchase 0.603 of a share of Class B Common Stock at a subscription price of $1.28 per share. Subscription rights may only be exercised in whole numbers, and fractional shares will not be issued."

Commercial • Rare Diseases

Rafael Holdings, Inc. Announces Proposed Terms of a $25 Million Rights Offering to Support Its Commitment to the Development and Potential Launch of Trappsol Cyclo (GlobeNewswire) - "Rafael Holdings...today announced a rights offering, including key dates and terms. The planned offering is designed to provide existing stockholders and holders of our public warrants ('Holders') the opportunity to purchase additional shares of Rafael Holdings’ Class B common stock, par value $0.01 per share (“Class B Common Stock”), subject to the terms outlined below for an aggregate offering of $25 million. The funds provide the Company additional capital for the potential launch of Trappsol Cyclo in the event of a positive interim result from the 48-week interim analysis of the TransportNPC™ Phase 3 clinical trial in Niemann-Pick Disease Type C1 ('NPC1')."

Financing • Rare Diseases

Rafael Holdings Reports Second Quarter Fiscal 2025 Financial Results (GlobeNewswire) - "...'We believe that Trappsol Cyclo has the potential to improve the lives of patients suffering from this rare genetic disease and topline data from the 48-week interim analysis of 104 enrolled patients is expected in the middle of 2025'."

P3 data: top line • Genetic Disorders • Rare Diseases

Cyclo Therapeutics Announces Notice of Decision to Grant European Patent Application for Treatment of Alzheimer’s Disease from European Patent Office (GlobeNewswire) - "Cyclo Therapeutics, Inc...today announced that the Company received a notice from the European Patent Office of a decision to grant European patent application No. 19805439.7 titled, 'Methods for Treating Alzheimer’s Disease' under European patent number 3873604B. The notice states that mention of the decision will be published on August 21, 2024 in European Patent Bulletin 24/34 and will take effect on that date...'With the desperate need for innovation and therapeutic solutions to treat early onset Alzheimer’s disease, we remain hopeful in the potential for Cyclo Therapeutics’ Trappsol Cyclo to play a significant role in the treatment armamentarium.'"

Patent • Alzheimer's Disease • CNS Disorders

TransportNPC: Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (clinicaltrials.gov) - P3 | N=94 | Active, not recruiting | Sponsor: Cyclo Therapeutics, Inc. | Recruiting ➔ Active, not recruiting | Trial completion date: Dec 2025 ➔ Jun 2026 | Trial primary completion date: Dec 2023 ➔ Jun 2025

Enrollment closed • Trial completion date • Trial primary completion date • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics

Cyclo Therapeutics Announces Allowance of Patent from European Patent Office for Trappsol Cyclo for Alzheimer’s Disease (Businesswire) - "Cyclo Therapeutics, Inc...announced that the Company received a Communication from the European Patent Office (EPO) regarding Cyclo Therapeutics’ European Patent Application No.19805439.7 titled, 'Methods for Treating Alzheimer’s Disease.' The EPO Communication stated that '[t]he newly filed claims are considered to be allowable' and that 'the applicant is therefore requested to bring the description into conformity with these claims'."

Patent • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Launches Phase 2b Study for Early Alzheimer’s Disease Drug (Healio) - "'[This] investigational drug shows promise in AD, based on the drug's ability to stabilize disease progression in an AD patient,' the trial's principal investigator...Martin R. Farlow, MD, said in the release. 'Notably, the ability of Trappsol Cyclo to gain access to the cerebrospinal fluid/brain and impact CSF tau and serum 24Shydroxycholesterol when intravenously administered are most impressive and represent an exciting avenue for therapeutic development.'"

Media quote

Cyclo Therapeutics Announces First Patient Dosed in Phase 2b Study of Trappsol Cyclo for the Treatment of Early Alzheimer’s Disease (Businesswire) - “Cyclo Therapeutics, Inc…announced the first patient has been enrolled and dosed in its Phase 2b study of Trappsol® Cyclo™ for the treatment of early Alzheimer’s disease (AD), targeting the disrupted lipid pathways and the reduction of amyloid beta and tau….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.”

Trial status • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Reports Third Quarter 2022 Financial Results and Highlights Recent Achievements (Businesswire) - “Research and development expenses increased 55% to approximately $2.9 million for the three months ended September 30, 2022, from $1.8 million for the three months ended September 30, 2021. The increase in research and development expense in the more recent period was related to the timing of startup, site initiation and enrollment costs in our clinical programs. The Company expects research and development to further increase in 2022 as we continue to seek regulatory approval for the use of Trappsol^® Cyclo™ in the treatment of NPC and Alzheimer’s disease.”

Commercial • Alzheimer's Disease • CNS Disorders

EAD501: A 6-Month Study to Evaluate the Safety & Potential Efficacy of Trappsol Cyclo in Patients With Early Alzheimer's Disease (clinicaltrials.gov) - P2 | N=90 | Recruiting | Sponsor: Cyclo Therapeutics, Inc.

New P2 trial • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Commences Phase 2b Study of Trappsol Cyclo for the Treatment of Early Alzheimer’s Disease (Businesswire) - "Cyclo Therapeutics, Inc...announced the commencement of its Phase 2b study of Trappsol® Cyclo™ for the treatment of early Alzheimer’s disease (AD), targeting the reduction of amyloid beta and tau. The Phase 2b study has the regulatory and IRB approval required to commence patient enrollment, with site activation now underway....Enrolled patients will be randomized across three study arms: 500 mg/kg or 1000 mg/kg of Trappsol® Cyclo™ and Placebo. The study will consist of a screening period, treatment period up to 24 weeks, and a safety follow-up period."

Enrollment status • Trial status • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Provides Business Update and Reports Second Quarter 2022 Financial Results (Businesswire) - "On track to commence Phase 2 study of Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease before year end...Research and development expenses decreased 29% to approximately $1.9 million for the three months ended June 30, 2022, from $2.6 million for the three months ended June 30, 2021....The Company expects research and development to further increase in 2022 as we continue to seek regulatory approval for the use of Trappsol® Cyclo™ in the treatment of NPC and Alzheimer’s disease."

Commercial • New P2 trial • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Announces Formation of Global Steering Committee Comprised of Leading Experts to Advise on the Global Phase 3 Clinical Development Program for Trappsol Cyclo in Niemann-Pick Disease Type C (Businesswire) - "'NPC is a devastating neurodegenerative disease that needs more effective therapies. Given the clinical course and progressive nature of this disease, novel therapeutic strategies with the potential for disease modifying effects are necessary. The TransportNPC trial is unique as it is designed to demonstrate the long-term clinical benefits and potential for disease modification,' commented Professor Roberto Giugliani, MD, PhD."

Media quote

Cyclo Therapeutics Provides Business Update and Reports First Quarter 2022 Financial Results (Businesswire) - “Cyclo Therapeutics is also planning to evaluate Trappsol® Cyclo™ for the treatment of Alzheimer’s Disease, targeting the reduction of amyloid beta and tau….The Company expects to commence this Phase 2 study and enroll patients in 2022.”

Enrollment status • New P2 trial • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics to Participate in the Virtual Investor Alzheimer’s Disease Spotlight Event (Businesswire) - “Cyclo Therapeutics, Inc…announced it will participate in the Virtual Investor Alzheimer’s Disease Spotlight event on Tuesday, April 5, 2022 at 1:00 PM ET….As part of the event, Cyclo Therapeutics will discuss its development program of Trappsol^® Cyclo™ for the treatment of Alzheimer’s disease. The Company recently received clearance of its investigational new drug (‘IND’) application from the U.S. Food and Drug Administration (‘FDA’) to proceed with its Phase 2 study of Trappsol^® Cyclo™ for the treatment of early Alzheimer’s disease.”

Clinical • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Enters Research and Collaboration Agreement with Wits Commercial Enterprise (Pty) Ltd to Explore Potential Applications of Trappsol Cyclo to Expand Pipeline (Businesswire) - “Cyclo Therapeutics, Inc…announced that it has entered into a research and collaboration agreement with the University of the Witwatersrand, Johannesburg (Wits) represented by Wits Commercial Enterprise (Pty) Ltd...Under the terms of the collaboration agreement, Wits and Cyclo Therapeutics plan to develop and coordinate projects or activities to further scientific advancement of the Company’s proprietary platform technology, Trappsol® Cyclo™…‘We have established proof-of-concept and compelling data to-date with Trappsol^® Cyclo™ and currently have a Phase 3 study in Niemann-Pick Disease Type C underway, and are preparing to advance our Phase 2 study in Alzheimer’s disease.’”

Licensing / partnership • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Receives IND Clearance from the U.S. FDA to Advance its Phase 2 Study of Trappsol® Cyclo for the Treatment of Alzheimer’s Disease (Businesswire) - "Cyclo Therapeutics, Inc....today announced it has received notification that its study may proceed from the U.S. Food and Drug Administration ('FDA') for its initial investigational new drug ('IND') application for a Phase 2 study of Trappsol® Cyclo™ for the treatment of early Alzheimer’s disease."

IND • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics jumps 18% after filing IND application for Trappsol Cyclo in Alzheimer’s Disease (SeekingAlpha) - "Cyclo Therapeutics (NASDAQ:CYTH) soars 18% premarket after submitting its initial investigational new drug (IND) application to the FDA for a Phase 2 study of Trappsol Cyclo for the treatment of early Alzheimer’s Disease (AD)."

IND • Stock price • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics Submits Investigational New Drug Application to U.S. FDA to Advance Trappsol Cyclo in Phase 2 Study for Treatment of Alzheimer’s Disease (Businesswire) - “Cyclo Therapeutics, Inc…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® Cyclo™ for the treatment of early Alzheimer’s Disease (AD).”

IND • Alzheimer's Disease • CNS Disorders

Cyclo Therapeutics to Participate in the 2021 Virtual Cyclodextrin Conference Hosted by CycloLab (Businesswire) - “Cyclo Therapeutics, Inc…announced it will participate in the 2021 Virtual Cyclodextrin Conference hosted by CycloLab, 'Cyclodextrins as active ingredients in the 21st century,' taking place on October 11, 2021. The event will focus on the use of cyclodextrins as active ingredients both from a therapeutic/industrial and chemistry point of view. As part of the event, Sharon H. Hrynkow, PhD, Chief Scientific Officer, Senior Vice President of Medical Affairs of Cyclo Therapeutics will participate in an interactive, moderated round-table discussion with several key opinion leaders in the field and address the questions of the attendees….By the end of 2021, Cyclo Therapeutics will submit an IND to FDA for a Phase 2 trial with Trappsol® Cyclo™ in Alzheimer’s disease...”

IND • Review • Alzheimer's Disease • CNS Disorders • Endocrine Disorders • Genetic Disorders • Metabolic Disorders • Rare Diseases

Cyclo Therapeutics Provides Business Update and Reports Second Quarter 2021 Financial Results (Businesswire) - “Research and development expenses increased 54% to $2.6 million for the three months ended June 30, 2021, from $1.7 million for the three months ended June 30, 2020. The increase in research and development expense is due to increased activity in the Company’s international clinical program and U.S. clinical trials. The Company expects research and development costs to further increase in 2021 as we continue to seek regulatory approval for the use of Trappsol® Cyclo™ in the treatment of NPC and Alzheimer’s disease.”

Commercial • Alzheimer's Disease • CNS Disorders

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients (clinicaltrials.gov) - P1/2; N=12; Completed; Sponsor: Cyclo Therapeutics, Inc.; Active, not recruiting ➔ Completed

Trial completion • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders

Cyclo Therapeutics Commences Commercial-Scale Manufacturing for Trappsol Cyclo (Businesswire) - “Cyclo Therapeutics…announced that it has commenced the commercial-scale production of batches of Trappsol® Cyclo™, the Company’s proprietary formulation of hydroxypropyl beta cyclodextrin…‘Additionally, the resulting drug product inventory will be used to support TransportNPC™, our…upcoming Phase 2 study for our Alzheimer’s disease asset, and our active compassionate use program’.”

Commercial • Alzheimer's Disease • CNS Disorders

TransportNPC: Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 (clinicaltrials.gov) - P3; N=93; Recruiting; Sponsor: Cyclo Therapeutics, Inc.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Frontotemporal Lobar Degeneration • Genetic Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics