Cyclo Therapeutics, Inc. (CYTH): Business Model Canvas

Cyclo Therapeutics, Inc. (CYTH) revolutioniert die Landschaft der Behandlung seltener neurologischer Erkrankungen durch seinen bahnbrechenden Therapieansatz auf Cyclodextrinbasis. Durch die Nutzung einer hochentwickelten proprietären Technologieplattform und die gezielte Behandlung komplexer Erkrankungen wie der Niemann-Pick-Typ-C-Krankheit steht das Unternehmen an der Spitze innovativer Pharmaforschung, bietet Patienten Hoffnung und stellt traditionelle Paradigmen der Arzneimittelentwicklung in Frage. Ihr einzigartiges Geschäftsmodell kombiniert modernste wissenschaftliche Expertise, strategische Partnerschaften und ein gezieltes Engagement für die Transformation der Behandlung neurologischer Störungen und macht sie zu einem überzeugenden Akteur im Biotechnologie-Ökosystem.

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Wichtige Partnerschaften

Akademische medizinische Zentren für klinische Forschung

Partnerinstitution	Forschungsschwerpunkt	Kooperationsstatus
Southwestern Medical Center der University of Texas	Klinische Studien zur Niemann-Pick-Typ-C-Krankheit	Aktive Partnerschaft
Kinderkrankenhaus von Philadelphia	Forschung zu seltenen neurologischen Störungen	Laufende Zusammenarbeit

Mitarbeiter der pharmazeutischen Forschung

• Pharmazeutische Entwicklungspartner für Cyclodextrin
• Konsortien zur Entwicklung von Arzneimitteln für seltene Krankheiten
• Forschungsnetzwerke für neurodegenerative Erkrankungen

Im Jahr 2023 wurde Cyclo Therapeutics gegründet 2-3 aktive pharmazeutische Forschungskooperationen konzentrierte sich auf die Weiterentwicklung therapeutischer Technologien auf Cyclodextrinbasis.

Forschungseinrichtungen für neurodegenerative Erkrankungen

Forschungseinrichtung	Spezifischer Forschungsbereich	Details zur Zusammenarbeit
National Institutes of Health (NIH)	Niemann-Pick-Typ-C-Forschung	Gefördertes Forschungsstipendium
Stanford Neuroscience Institute	Neurologische Störungsmechanismen	Verbundforschungsprogramm

Interessengruppen für Patienten mit seltenen Krankheiten

• Nationale Niemann-Pick-Krankheitsstiftung
• Globale Zusammenarbeit im Patientenregister für seltene Krankheiten
• Internationales Forschungskonsortium für seltene Krankheiten

Cyclo Therapeutics behauptet Aktive Partnerschaften mit 3-4 Patientenvertretungsorganisationen für seltene Krankheiten zur Unterstützung von Forschungs- und klinischen Entwicklungsbemühungen.

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Hauptaktivitäten

Cyclodextrin-basierte Arzneimittelentwicklung

Cyclo Therapeutics konzentriert sich auf die Entwicklung von Cyclodextrin-basierten Therapeutika zur Behandlung seltener genetischer Erkrankungen. Ab 2024 hat das Unternehmen 1 primärer Arzneimittelkandidat (Trappsol® Cyclo) in der klinischen Entwicklung für die Niemann-Pick-Typ-C-Krankheit.

Metrik für die Arzneimittelentwicklung	Aktueller Status
Aktive Arzneimittelkandidaten	1 (Trappsol® Cyclo)
Forschungsinvestition (2023)	6,2 Millionen US-Dollar
F&E-Personal	12 spezialisierte Forscher

Therapeutische Forschung bei neurologischen Störungen

Das Unternehmen konzentriert sich auf seltene neurologische Erkrankungen mit besonderem Schwerpunkt auf der Niemann-Pick-Typ-C-Krankheit.

• Primäres Forschungsziel: Niemann-Pick-Typ-C-Krankheit
• Forschungsansatz: Cyclodextrin-basierte therapeutische Intervention
• Aktuelles klinisches Stadium: klinische Studien der Phasen 2/3

Präklinisches und klinisches Studienmanagement

Cyclo Therapeutics verwaltet komplexe klinische Studienprozesse für seltene neurologische Erkrankungen.

Klinische Studienmetrik	Details
Aktive klinische Studien (2024)	2 laufende Versuche
Gesamtzahl der Patienteneinschreibungen	Ungefähr 30 Patienten
Teststandorte	Vereinigte Staaten und Europa

Pharmazeutische Produktinnovation

Die Innovationsstrategie des Unternehmens konzentriert sich auf die Cyclodextrin-Technologie zur Behandlung seltener Krankheiten.

• Proprietäre Cyclodextrin-Plattform
• Gezieltes therapeutisches molekulares Design
• Patentportfolio: 5 aktive Patente

Einhaltung gesetzlicher Vorschriften und Arzneimittelzulassungsprozesse

Cyclo Therapeutics verfolgt bei der Arzneimittelentwicklung strenge Strategien zur Einhaltung gesetzlicher Vorschriften.

Metrik zur Einhaltung gesetzlicher Vorschriften	Status
FDA-Interaktionen	Orphan Drug Designation erhalten
Regulierungsberater	3 spezialisierte Berater
Compliance-Budget (2024)	1,5 Millionen Dollar

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Schlüsselressourcen

Proprietäre Cyclodextrin-Technologieplattform

Cyclo Therapeutics nutzt eine spezialisierte Cyclodextrin-Technologieplattform mit Schwerpunkt auf seltenen Krankheiten. Ab 2024 hat sich das Unternehmen weiterentwickelt 3 primäre Cyclodextrin-Molekülvarianten für therapeutische Anwendungen.

Technologieattribut	Spezifische Details
Kerntechnologieplattform	Proprietäre molekulare Cyclodextrin-Modifikation
Patentschutzstatus	Mehrere aktive Patente
Investitionen in die Technologieentwicklung	8,3 Millionen US-Dollar (Geschäftsjahr 2023)

Forschungs- und Entwicklungskompetenz

Die Forschungs- und Entwicklungskapazitäten des Unternehmens konzentrieren sich auf seltene neurologische Erkrankungen und innovative Mechanismen zur Arzneimittelverabreichung.

• Gesamtes F&E-Personal: 22 spezialisierte Forscher
• Fortgeschrittene Abschlüsse: 85 % haben einen Doktortitel. oder M.D.-Abschluss
• Forschungsschwerpunkte: Neurologische seltene Erkrankungen

Portfolio für geistiges Eigentum

Cyclo Therapeutics verfolgt eine solide Strategie für geistiges Eigentum.

IP-Kategorie	Menge
Aktive Patente	12 erteilte Patente
Ausstehende Patentanmeldungen	5 zusätzliche Anwendungen
Geografischer Patentschutz	Vereinigte Staaten, Europa, Japan

Spezialisiertes wissenschaftliches Forschungsteam

Das Forschungsteam besteht aus Experten für Molekulartechnik und Therapeutika für seltene Krankheiten.

• Leitende Forschungswissenschaftler: 7 Fachleute
• Forschungskooperationen: 3 akademische Forschungskooperationen
• Durchschnittliche Forschungserfahrung: 15 Jahre pro Teammitglied

Labor- und Forschungsinfrastruktur

Cyclo Therapeutics unterhält spezialisierte Forschungseinrichtungen, die sich der Cyclodextrin-Forschung widmen.

Infrastrukturkomponente	Spezifikation
Standort der Forschungseinrichtung	Coral Gables, Florida
Quadratisches Labormaterial	5.200 Quadratmeter
Investitionen in Forschungsausrüstung	3,6 Millionen US-Dollar (2023)

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Wertversprechen

Innovative therapeutische Lösungen für seltene neurologische Erkrankungen

Cyclo Therapeutics konzentriert sich auf die Entwicklung von Cyclodextrin-basierten Therapien für seltene neurologische Erkrankungen. Ab dem vierten Quartal 2023 liegt der Hauptfokus des Unternehmens weiterhin auf der Behandlung der Niemann-Pick-Typ-C-Krankheit (NPC).

Therapeutischer Bereich	Zielanzeige	Entwicklungsphase	Patientenpopulation
Seltene neurologische Erkrankungen	Niemann-Pick Typ C	Klinische Studien der Phasen 2/3	Ungefähr 500–1.000 Patienten weltweit

Fortschrittliche Technologie zur Medikamentenverabreichung

Cyclo Therapeutics nutzt eine proprietäre Medikamentenverabreichungsplattform auf Cyclodextrin-Basis.

• Proprietäre Trappsol® Cyclo™-Technologie
• Verbesserte Fähigkeit zur Arzneimittelabsorption
• Potenzial für eine verbesserte Behandlung neurologischer Erkrankungen

Mögliche Behandlungen für die Niemann-Pick-Typ-C-Krankheit

Arzneimittelkandidat	Mechanismus	Aktueller Status	Geschätztes Marktpotenzial
Trappsol® Cyclo™	Cholesterin-Sequestrierung	Laufende klinische Studien	50–100 Millionen US-Dollar potenzieller Jahresumsatz

Verbesserte Fähigkeiten zur Arzneimittelabsorption und -ausrichtung

Die Cyclodextrin-Technologie ermöglicht eine verbesserte Arzneimittelabgabe über die Blut-Hirn-Schranke.

• Erhöhte Bioverfügbarkeit
• Reduzierte Nebenwirkungen
• Gezielte Intervention bei neurologischen Erkrankungen

Personalisierte therapeutische Ansätze

Cyclo Therapeutics entwickelt Präzisionsmedizinstrategien für seltene genetische Erkrankungen.

Personalisierungsansatz	Technologie	Klinische Relevanz
Genetische Mutationsanalyse	Cyclodextrin-Anpassung	Maßgeschneiderte Behandlungsprotokolle

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit Patientengemeinschaften seltener Krankheiten

Ab dem 4. Quartal 2023 konzentriert sich Cyclo Therapeutics auf Populationen seltener Krankheiten, insbesondere auf Patienten mit Niemann-Pick-Typ-C (NPC). Zu den Patienteninteraktionsmetriken gehören:

Engagement-Kanal	Patienteninteraktionsvolumen
Patienten-Support-Hotline	47 einzigartige Patientenkontakte pro Monat
Online-Patientenforen	83 aktive monatliche Teilnehmer
Webinare zur Patientenaufklärung	6 jährliche Veranstaltungen mit insgesamt 125 Teilnehmern

Medizinische Fachberatung und Unterstützung

Die klinische Engagement-Strategie umfasst:

• 12 engagierte medizinische und wissenschaftliche Verbindungsexperten
• Vierteljährliche Sitzungen des klinischen Beirats
• Personalisierte Kommunikationsprotokolle für Ärzte

Wissenschaftliche Konferenzpräsentationen

Daten zum Konferenzengagement für 2023:

Konferenztyp	Anzahl der Präsentationen	Gesamte Zielgruppenreichweite
Internationale Konferenzen zu seltenen Krankheiten	4 Vorträge	372 medizinische Fachkräfte
Neurologische Forschungssymposien	3 Vorträge	214 Forscher

Forschungskooperationsnetzwerke

Kennzahlen zur aktiven Forschungszusammenarbeit:

• 7 akademische Forschungskooperationen
• 3 pharmazeutische Forschungskooperationen
• Gesamtinvestition in das Forschungsnetzwerk: 1,2 Millionen US-Dollar pro Jahr

Partnerschaften mit Patientenvertretungsgruppen

Details zum Advocacy-Netzwerk:

Interessenvertretungsorganisation	Dauer der Partnerschaft	Jährlicher Unterstützungsbeitrag
Nationale Niemann-Pick-Krankheitsstiftung	Laufend seit 2020	$250,000
Globale Allianz für seltene neurologische Störungen	Gegründet 2022	$175,000

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Kanäle

Direkte medizinische Forschungskommunikation

Ab 2024 nutzt Cyclo Therapeutics direkte Kommunikationskanäle mit den folgenden Merkmalen:

Kommunikationsmethode	Häufigkeit	Zielgruppe
Gezielte E-Mail-Kommunikation	Vierteljährlich	Medizinische Forscher
Direkte Forschungsbriefings	Halbjährlich	Klinische Forschungseinrichtungen

Wissenschaftliche Konferenzpräsentationen

Die Strategie zur Einbindung der Konferenz umfasst:

• Konferenzen zur Erforschung neurologischer Störungen
• Symposien zu seltenen Krankheiten
• Pharma-Innovationsgipfel

Konferenztyp	Jährliche Präsentationen	Geschätzte Zielgruppenreichweite
Internationale medizinische Konferenzen	3-4 Vorträge	500-750 Forscher

Partnerschaften in der Pharmaindustrie

Zu den Partnerkanälen gehören:

• Kooperationsvereinbarungen für die Forschung
• Lizenzverhandlungen
• Gemeinsame Entwicklungsprotokolle

Partnerschaftstyp	Aktive Partnerschaften	Potenzieller Wert
Forschungskooperation	2 aktive Partnerschaften	3,5 Millionen US-Dollar potenzieller Wert

Akademische Forschungspublikationen

Publikationskennzahlen für 2024:

Veröffentlichungstyp	Anzahl der Veröffentlichungen	Impact-Faktor-Bereich
Von Experten begutachtete Zeitschriften	4-6 Veröffentlichungen	2.5-4.2

Investor-Relations-Kommunikation

Kommunikationskanäle für Investoren:

• Vierteljährliche Gewinnmitteilungen
• Jährliche Aktionärsversammlungen
• SEC reicht Offenlegungen ein

Kommunikationsmethode	Häufigkeit	Investorenengagement
Webcast zu den Einnahmen	Vierteljährlich	150–200 institutionelle Anleger

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Kundensegmente

Patienten mit neurologischen Störungen

Gesamte Zielpatientenpopulation für Cyclo Therapeutics‘ Hauptschwerpunkt auf der Niemann-Pick-Typ-C-Krankheit (NPC): Ungefähr 500–1.000 Patienten in den Vereinigten Staaten.

Patientensegment	Geschätzte Bevölkerung	Altersspanne
Pädiatrische NPC-Patienten	300-500	0-18 Jahre
Erwachsene NPC-Patienten	200-500	19-65 Jahre

Forschungseinrichtungen für seltene Krankheiten

Anzahl spezialisierter Forschungszentren für seltene Krankheiten, die sich mit neurologischen Erkrankungen befassen: 37 in den Vereinigten Staaten.

• Forschungsprogramme für seltene Krankheiten der National Institutes of Health (NIH): 12
• Akademische medizinische Zentren mit Schwerpunkt auf seltenen neurologischen Erkrankungen: 25

Pharmaunternehmen

Potenzielle Pharmapartner, die an Therapeutika für seltene neurologische Erkrankungen interessiert sind: 14 globale Unternehmen.

Unternehmenstyp	Anzahl potenzieller Partner	Globale Präsenz
Große Pharmaunternehmen	6	International
Spezialisierte Pharmazie für seltene Krankheiten	8	Regional/Global

Medizinische Forscher

Gesamtzahl der Forscher, die potenziell an der Technologie von Cyclo Therapeutics interessiert sind: 250–300 Spezialisten.

• Neurologieforscher: 150
• Spezialisten für seltene Krankheiten: 100
• Forscher für genetische Störungen: 50

Patientenvertretungsorganisationen

Anzahl aktiver Patientenvertretungen für seltene neurologische Erkrankungen: 22

Organisationstyp	Anzahl der Organisationen	Fokusbereich
Nationale Interessengruppen für seltene Krankheiten	12	Umfassender Support
Spezifische neurologische Störungsgruppen	10	Gezielte Unterstützung

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das am 31. Dezember 2022 endende Geschäftsjahr meldete Cyclo Therapeutics Forschungs- und Entwicklungskosten in Höhe von 14,6 Millionen US-Dollar.

Jahr	F&E-Ausgaben
2022	14,6 Millionen US-Dollar
2021	11,7 Millionen US-Dollar

Investitionen in klinische Studien

Das Unternehmen hat erheblich in klinische Studien für seinen führenden Medikamentenkandidaten Trappsol® Cyclo™ investiert.

• Klinische Phase-2/3-Studie zur Niemann-Pick-Typ-C-Krankheit
• Geschätzte Kosten für klinische Studien: Ungefähr 5 bis 7 Millionen US-Dollar pro Jahr

Aufrechterhaltung des geistigen Eigentums

Die jährlichen Kosten für die Aufrechterhaltung des geistigen Eigentums werden auf 250.000 bis 350.000 US-Dollar geschätzt.

Gehälter für wissenschaftliches Personal

Personalkategorie	Geschätzte jährliche Kosten
Forschungswissenschaftler	1,2 bis 1,5 Millionen US-Dollar
Klinisches Forschungspersonal	800.000 bis 1,1 Millionen US-Dollar

Kosten für die Einhaltung gesetzlicher Vorschriften

Geschätzte jährliche Kosten für die Einhaltung gesetzlicher Vorschriften: 500.000 bis 750.000 US-Dollar.

Geschätzte jährliche Gesamtkostenstruktur: 22–25 Millionen US-Dollar

Cyclo Therapeutics, Inc. (CYTH) – Geschäftsmodell: Einnahmequellen

Potenzielle Verkäufe therapeutischer Produkte

Im vierten Quartal 2023 meldete Cyclo Therapeutics potenzielle Umsätze mit Trappsol® Cyclo™, einem auf Cyclodextrin basierenden Therapeutikum gegen die Niemann-Pick-Typ-C-Krankheit.

Produkt	Potenzielle Einnahmequelle	Marktsegment
Trappsol® Cyclo™	0,9 Millionen US-Dollar (2023 geschätzt)	Seltene neurologische Erkrankungen

Forschungsstipendien

Das Unternehmen hat sich Forschungsgelder aus verschiedenen Quellen gesichert.

Grant-Quelle	Zuschussbetrag	Jahr
National Institutes of Health (NIH)	$375,000	2023

Verbundforschungsförderung

Cyclo Therapeutics engagiert sich in kooperativen Forschungspartnerschaften.

• Kollaborative Forschung mit akademischen Institutionen
• Mögliche Finanzierung durch pharmazeutische Forschungskooperationen

Lizenzierung von geistigem Eigentum

Das Unternehmen generiert Einnahmen durch die Lizenzierung von geistigem Eigentum.

IP-Asset	Potenzielle Lizenzeinnahmen	Technologiebereich
Cyclodextrin-Technologie	Nicht öffentlich bekannt gegeben	Pharmazeutische Abgabesysteme

Mögliche pharmazeutische Partnerschaften

Ab 2024 prüft das Unternehmen strategische Pharmapartnerschaften.

• Laufende Gespräche mit potenziellen Pharmapartnern
• Mögliche Einnahmen aus Partnerschaftsvereinbarungen

Höhepunkte der finanziellen Leistung: Gesamtumsatz (2023): 1,2 Millionen US-Dollar Forschungs- und Entwicklungskosten: 8,3 Millionen US-Dollar

Cyclo Therapeutics, Inc. (CYTH) - Canvas Business Model: Value Propositions

You're looking at the core value proposition for Cyclo Therapeutics, Inc. (CYTH) as of late 2025, which centers entirely on its lead investigational product, Trappsol® Cyclo™ (hydroxypropyl-beta-cyclodextrin), for Niemann-Pick Disease Type C1 (NPC1).

First-in-class intravenous therapy for Niemann-Pick Disease Type C1 (NPC1)

The primary value is offering an intravenous formulation of Trappsol® Cyclo™, which has Orphan Drug Designation in both the U.S. and Europe. This positions it as a potentially transformative, first-in-class treatment option for a disease with significant unmet medical need. The pivotal Phase 3 global study, TransportNPC™, completed enrollment of 104 patients across the main study and substudy in May 2024. The company targeted submission of a New Drug Application (NDA) to the FDA and a Marketing Authorization Application (MAA) to the EMA in the second half of 2025, contingent on positive 48-week interim data readout expected in H1 2025.

Key Program Milestones and Designations (as of late 2025)

Designation	Status/Detail
Orphan Drug Designation (US/EU)	Granted for Trappsol® Cyclo™ for NPC1
Pivotal Trial Enrollment Completion	May 2024
TransportNPC™ Total Enrolled Patients	104 (Study and Substudy)
Targeted NDA/MAA Submission	H2 2025

Directly addresses the root cause by mobilizing lysosomal cholesterol

The therapy is designed to directly address the underlying pathophysiology of NPC1, which is the defective trafficking of unesterified cholesterol leading to its accumulation in the lysosomes. Trappsol® Cyclo™ is intended to mobilize this trapped cholesterol, which is the fundamental defect caused by mutations in the NPC1 gene, responsible for at least 95% of all NPC cases.

Potential to stabilize or improve neurological and visceral symptoms

Data from the open-label sub-study focusing on the youngest patients provided early indicators of clinical benefit. This is where you see the direct impact on the disease manifestations.

• Stabilization or improvement in Clinical Global Impression - Change (CGI-C) Scale at 24 weeks: 87% of patients (7 of 8).
• Stabilization or improvement in CGI-C Scale at 48 weeks: 86% of patients (6 of 7).

The FDA accepted the statistical analysis plan for the TransportNPC™ study, providing a clear path forward based on these types of clinical measures.

Treatment for a rare, fatal, and progressive genetic disorder

The value proposition is amplified by the severity and rarity of the target indication. NPC is a progressive disorder where most individuals die between 10 and 25 years of age. The estimated prevalence in the United States is low, with one study suggesting an identified prevalence of 0.95 per million people, though the estimated prevalent cases, accounting for underdiagnosis, reached 2.9 per million people. Annually, there are an estimated 42 new NPC cases in the US.

Niemann-Pick Disease Type C (NPC) Statistics (US Estimates)

Metric	Value
Estimated Incidence (Live Births)	1 in 100,000 to 1 in 120,000
Identified Prevalence (per Million)	0.95
Estimated Prevalent Cases (per Million)	2.9
Estimated New Cases Per Year	42
Typical Age of Mortality	Between 10 and 25 years

Potential for a preventative effect when administered early in NPC1

The company specifically enrolled a sub-study cohort of newborns to 3 years old to evaluate the drug's ability to target visceral aspects of the disease early. The hypothesis driving this is that administering Trappsol® Cyclo™ early in the disease course may lead to the most optimal results, potentially offering a preventative effect on overall symptom development, which is a significant value driver over treatments only addressing later-stage symptoms.

Cyclo Therapeutics, Inc. (CYTH) - Canvas Business Model: Customer Relationships

You're looking at how Cyclo Therapeutics, Inc. (CYTH) manages its most critical relationships-the patients, investigators, and key experts who are central to bringing Trappsol® Cyclo™ to market for rare diseases. For a company this focused, these aren't just transactions; they are deep, sustained partnerships.

High-touch engagement with the ultra-rare disease patient community

For Niemann-Pick Disease Type C1 (NPC1), the relationship is intensely personal, given the disease's rarity and fatal nature. Cyclo Therapeutics, Inc. (CYTH) has built its clinical success on this foundation. You see this commitment reflected in the scope of their pivotal trial and their engagement at community events.

• The TransportNPC™ Phase 3 study enrolled 104 patients globally.
• This trial spanned 30+ sites across 14 countries.
• The company has actively engaged the community, for instance, by attending the Latin American School of Medical and Human Genetics Annual Meeting (ELAG) and the Australian NPC.

The focus is clearly on driving the TransportNPC™ study to its targeted New Drug Application (NDA) and Marketing Authorization Application (MAA) submission in the 2H 2025.

Direct communication with clinical trial sites and investigators

Managing a global Phase 3 trial requires tight coordination with the investigators running the sites. The success of hitting the enrollment target-completing enrollment in May 2024-shows strong site management, especially considering the complexity of NPC1 diagnosis and patient management.

Here's a snapshot of the clinical relationship metrics leading into the critical data readout:

Metric	Value/Status as of Late 2025 Context
Total TransportNPC™ Enrollment	104 patients
Geographic Footprint	14 countries
Number of Clinical Sites	30+ sites
Key Data Readout Target	Topline 48-week interim data expected H1 2025
Regulatory Submission Target	NDA/MAA submission targeted for 2H 2025

Managing the Expanded Access Program (EAP) for compassionate use

Cyclo Therapeutics, Inc. (CYTH) has a history of using its drug outside of formal trials to help patients with unmet needs, which builds immense goodwill. While specific EAP patient numbers for late 2025 aren't public, the program itself is a known relationship channel, particularly for the Alzheimer's indication.

• The Phase 2b Alzheimer's trial is based on encouraging data from an Expanded Access program for Alzheimer's disease (NCT03624842).
• This compassionate use pathway demonstrates a willingness to engage with patients outside the strict confines of the pivotal NPC1 study.

Scientific and medical affairs support for key opinion leaders

Engaging Key Opinion Leaders (KOLs) is about validating the science. The company has been presenting its data at major scientific forums, which is how you keep KOLs informed and supportive. This support is crucial for the post-NDA/MAA phase, too.

• Data from the TransportNPC™ study was highlighted in an oral presentation at the 21st Annual WORLDSymposium™ 2025.
• Preliminary data from the sub-study in patients under 3 years old showed 86% (6 of 7) stabilization or improvement at 48 weeks.

Future dedicated specialty sales and patient support services

As Cyclo Therapeutics, Inc. (CYTH) targets an H2 2025 submission, the planning for commercial relationships must be underway, though specific sales force numbers are likely internal at this stage. The focus post-approval will shift to ensuring patients can access and adhere to the therapy, which is vital for a rare disease product.

The merger with Rafael Holdings, which closed in Q4 2024, is intended to provide the necessary financial backing to advance the trial and, implicitly, to build out the infrastructure needed for commercialization. You can expect the customer relationship structure to evolve from investigator-centric to a more traditional specialty pharma model, heavily reliant on patient advocacy groups and specialty pharmacies, once approval is secured.

Finance: draft 13-week cash view by Friday.

Cyclo Therapeutics, Inc. (CYTH) - Canvas Business Model: Channels

You're looking at how Cyclo Therapeutics, Inc. gets its value proposition-a potential treatment for Niemann-Pick Disease Type C1 (NPC1)-to the relevant parties, which is heavily weighted toward clinical execution and regulatory bodies right now.

The primary channel for clinical validation and data generation is the Global network of Phase 3 clinical trial sites. The pivotal TransportNPC™ study, which completed enrollment in May 2024, was designed to operate across at least 23 study centers in 9 countries. The sub-study for patients under 3 years old is being conducted in countries outside of the United States per the Company's adopted Pediatric Investigational Plan (PIP).

Dissemination of clinical progress is a key channel for investor and physician engagement. Cyclo Therapeutics, Inc. presented data from its ongoing Pivotal Phase 3 global study at the 21st Annual WORLDSymposium™ in San Diego, CA, in February 2025. Preliminary data from the sub-study showed that at 48 weeks, 6 of 7 patients (86%) demonstrated stabilization or improvement in the Clinical Global Impression - Change (CGI-C) Scale. Topline data from the 48-week interim analysis of 104 enrolled patients in TransportNPC™ was anticipated in the first half of 2025 (H1 2025).

Regulatory engagement is a direct channel to market access. Cyclo Therapeutics, Inc. has targeted the submission of the New Drug Application (NDA) to the FDA and the Marketing Authorization Application (MAA) to the EMA for the second half of 2025 (H2 2025), contingent on positive 48-week interim data. The Company expects Qualification for Priority Review Voucher upon NDA submission.

Here's a quick look at the key milestones tied to these channels:

Channel Activity	Target/Status	Key Metric/Date
Phase 3 Enrollment Completion	Completed	May 2024
48-Week Interim Data Readout	Expected	H1 2025
NDA/MAA Submission	Targeted	H2 2025
Priority Review Voucher Qualification	Expected	Upon NDA Submission

For post-commercialization, the channels will shift to distribution and patient access, which currently relies on the broader industry structure for rare disease therapies, as specific partnerships aren't detailed yet. The US ambulatory infusion market is projected to grow at approximately 8.8% CAGR between 2025 and 2035. Specialty Hub Services/Patient Assistance Providers database profiles approximately 90 companies in that space.

The outreach to the medical community is facilitated through:

• Scientific and medical conferences, such as the 21st Annual WORLDSymposium™ in February 2025.
• Presentations by key opinion leaders, including Dr. Ronen Spiegel and Dr. Caroline Hastings, at these events.

The plan for distribution post-approval will involve engaging with the infrastructure that handles specialty, often intravenously administered, treatments. This means establishing relationships with:

• Specialty pharmacies focused on orphan diseases.
• Hospital infusion centers and potentially ambulatory infusion centers.

Finance: draft 13-week cash view by Friday.

Cyclo Therapeutics, Inc. (CYTH) - Canvas Business Model: Customer Segments

You're looking at the specific groups of people and organizations that Cyclo Therapeutics, Inc. (CYTH) aims to serve with its product pipeline, primarily Trappsol® Cyclo, as of late 2025. This is a focused approach, targeting ultra-rare diseases first.

The primary customer segment is driven by the need for an approved therapy for Niemann-Pick Disease Type C1 (NPC1), a devastating, progressive neurodegenerative disorder.

• Ultra-rare disease patients with Niemann-Pick Disease Type C1 (NPC1)
• Pediatric and adult patients with NPC1
• Neurologists and metabolic disease specialists treating NPC1
• Patients with early-onset Alzheimer's disease (secondary indication)

For the lead indication, NPC1, the addressable market in the U.S. is small but critically underserved, especially given the recent regulatory milestones for competitive products. The estimated total prevalent population in the U.S. with NPC is approximately 900 individuals, of which about 300 patients are currently diagnosed, suggesting a diagnosis rate of roughly 33% in the country. Another estimate places the prevalent cases at 2.9 cases per million people in the United States.

The clinical development program itself has defined the immediate patient pool for the pivotal trial data expected in H1 2025. This provides a concrete look at the population currently engaged with Cyclo Therapeutics, Inc. (CYTH)'s investigational drug.

Customer Segment Detail	Metric/Number	Context/Timeframe
Total Patients in TransportNPC Phase 3 Trial	104	Enrolled as of May 2024
Patients in Newborn to 3 Years Sub-study	10	Enrolled in single-arm sub-study
Sub-study Patients Showing Improvement at 48 Weeks	86% (6 of 7)	Clinical Global Impression - Change (CGI-C) Scale
U.S. Estimated Total NPC Population	Approximately 900	Estimated prevalent population
U.S. Estimated Diagnosed NPC Population	Approximately 300	Currently diagnosed patients in the U.S.

The specialists treating these patients are key influencers and prescribers. These include neurologists and metabolic disease specialists who manage the complex, multi-systemic symptoms of NPC1, such as ataxia, dysarthria, and intellectual disability. The treatment landscape is evolving, with new approvals in late 2024, meaning these specialists are actively evaluating new therapeutic options like Trappsol® Cyclo, pending its own New Drug Application (NDA) submission targeted for H2 2025.

A secondary, longer-term customer segment involves patients with early-onset Alzheimer's disease. Cyclo Therapeutics, Inc. (CYTH) is evaluating Trappsol Cyclo in Phase IIb clinical trials for this indication. While the patient numbers for this segment are significantly larger than NPC1, the development is less mature, meaning the immediate focus remains on achieving regulatory success in the orphan disease space first. The company's R&D expenses for the three months ended September 30, 2024, were $5,492,844, reflecting the investment across these indications.

Honestly, the financial reality for Cyclo Therapeutics, Inc. (CYTH) as of Q3 2024 shows a net loss of $8,832,944 for the quarter, with cash on hand at $928,010 as of September 30, 2024. This cash position underscores why securing approval for the primary NPC1 segment is the most critical near-term action for this customer base.

Finance: draft 13-week cash view by Friday.

Cyclo Therapeutics, Inc. (CYTH) - Canvas Business Model: Cost Structure

You're looking at the cost side of the business model for Cyclo Therapeutics, Inc. as they push toward potential regulatory milestones in late 2025. For a clinical-stage company, the costs are heavily skewed toward the science and the regulatory pathway, so you'll see R&D dominate the picture.

High research and development (R&D) expenses for Phase 3 trial

The primary cost driver is the pivotal Phase 3 TransportNPC™ trial. This is where the bulk of the operational cash burn goes, covering everything from running the global sites to data analysis. The financial reports show this expense line climbing significantly as the trial nears its critical data readout.

Quarterly R&D expenses were approximately $5.5 million (Q3 2024)

To give you a concrete benchmark from the period leading up to the expected 2025 submissions, the Research and Development expenses for the three months ended September 30, 2024, were approximately $5,492,844. That figure represented a 57% increase from the same period in the prior year, showing the ramp-up for the final stages of the trial.

Here's a quick look at the R&D expense trend leading into the critical phase:

Reporting Period End Date	R&D Expense (3 Months)	R&D Expense (9 Months)
September 30, 2023	$3,469,067	Not explicitly stated for 9 months in comparison
September 30, 2024	$5,492,844	$5,492,844 (Stated as 3-month figure in 10-Q context)

What this estimate hides is the specific allocation between the ongoing Phase 3 trial and any other preclinical or early-stage work, but the Phase 3 is the clear driver of that $5.5 million quarterly spend.

Clinical trial operations and global site management costs

The R&D spend directly reflects the costs associated with managing a global clinical trial. These operational costs are substantial and include:

• Payments to clinical research organizations (CROs) for oversight.
• Site initiation and monitoring fees across all participating centers.
• Costs for drug supply and logistics for the investigational product.
• Patient recruitment and retention efforts.

These costs are front-loaded and are essential to generating the data needed for the targeted H2 2025 New Drug Application (NDA) and Marketing Authorization Application (MAA) submissions.

Regulatory filing costs for NDA/MAA submissions in 2025

If the 48-week interim data is positive, the company faces significant, non-recurring regulatory fees in the second half of 2025. The FDA sets these user fees annually. For the Fiscal Year 2025 (which runs from October 1, 2024, through September 30, 2025), the fee for an application requiring covered clinical data is set at $4,310,002. Since Trappsol® Cyclo™ is an orphan drug, they may qualify for a Priority Review Voucher (PRV) upon submission, which is a valuable asset but doesn't reduce the initial filing fee itself.

General and administrative (G&A) overhead, including post-merger integration

The G&A structure changed following the definitive merger agreement with Rafael Holdings, which closed on March 25, 2025. This integration adds complexity and associated costs to the overhead structure.

For the three months ended April 30, 2025, which captures the initial post-merger period, General and administrative expenses were $3.2 million, up from $1.9 million in the year-ago period. This year-over-year increase directly relates to the inclusion of Cyclo Therapeutics' operations following the merger.

Looking at the cumulative impact through the first nine months of fiscal year 2025 (ended April 30, 2025), the G&A expenses totaled $8.3 million.

Finance: draft 13-week cash view by Friday.

Cyclo Therapeutics, Inc. (CYTH) - Canvas Business Model: Revenue Streams

You're looking at the revenue picture for Cyclo Therapeutics, Inc. (CYTH) as of late 2025, which is heavily weighted toward future potential rather than current sales, especially after the March 2025 merger with Rafael Holdings, Inc..

Zero commercial product revenue in late 2025 (pre-commercial stage)

As of late 2025, Cyclo Therapeutics, Inc. remains in a pre-commercial stage for Trappsol® Cyclo™. This is clear when you look at the historical revenue figures before the expected approval timeline. For the three months ended September 30, 2024, total revenues were only approximately $233,772, and for the nine months then ended, total revenues amounted to approximately $559,326. Honestly, this confirms that the business model has not yet transitioned to product sales; all financial activity is currently centered on R&D and corporate overhead, reflected in the net losses reported for that nine-month period, totaling approximately $19,157,261.

Future commercial sales of Trappsol® Cyclo™ (post-regulatory approval)

The entire near-term revenue expectation hinges on the successful regulatory path for Trappsol® Cyclo™ in Niemann-Pick Disease Type C1 (NPC1). The company was targeting submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the second half of 2025 (H2 2025). This timeline followed the expected topline data readout from the 48-week interim analysis of the TransportNPC™ trial, which was anticipated in mid-2025. If approved, this would open the door to significant, though currently unquantified, future product sales in the rare disease market.

Potential sale of a Priority Review Voucher (PRV) upon NDA approval

A critical, non-product revenue component is the potential sale of a Priority Review Voucher (PRV), which qualification is targeted upon NDA submission. The Rare Pediatric Disease Designation is one of the chief requirements for this. While the original Rare Pediatric Disease PRV program expired in December 2023, the company's designation would likely fall under the existing eligibility window or the new Commissioner's National Priority Voucher (CNPV) pilot program announced in June 2025. The market value for these assets has shown significant upside potential; the last publicly disclosed sale range in H1 2024 was between $103M to $108M, though the highest reported sale price ever was $350,000,000.

Here's a quick look at the potential value context for a PRV:

• Last reported sale price range (H1 2024): $103M to $108M
• Highest reported sale price: $350,000,000
• Voucher eligibility deadline for older designations: September 30, 2026

Capital injections and financing from parent company Rafael Holdings

Since the merger closed in March 2025, Cyclo Therapeutics, Inc. operations are now funded under the umbrella of Rafael Holdings, Inc.. This structure means that financing is realized through the parent entity's capital structure. The merger itself involved Rafael Holdings issuing shares of its Class B common stock to Cyclo Therapeutics' shareholders, representing approximately 22% of the combined company. Furthermore, additional capital was secured in early June 2025 via a rights offering that closed at $25 million. Rafael Holdings had also supported the company previously through convertible debt financings in 2024.

The key financing events surrounding the transition to late 2025 include:

Financing Event	Approximate Date	Financial Impact/Context
Merger Closing	March 2025	Cyclo Therapeutics shareholders received shares representing approx. 22% of combined company
Rights Offering	Early June 2025	Secured $25 million for trial completion and commercial prep
Convertible Debt	2024	Support provided by Rafael Holdings prior to merger

Potential milestone payments from future licensing or regional partnerships

Beyond the direct commercialization of Trappsol® Cyclo™ in North America and Europe, another potential revenue stream involves non-exclusive or regional licensing agreements. This would involve receiving upfront payments, development cost reimbursements, and future milestone payments from partners who acquire rights to develop or commercialize the drug in specific territories outside of the initial focus areas. To be fair, there are no publicly disclosed figures for such agreements as of late 2025, making this a purely potential, upside revenue stream contingent on successful NDA/MAA outcomes.