Cyclo Therapeutics, Inc. (CYTH): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

Cyclo Therapeutics, Inc. (CITH) está revolucionando el panorama del tratamiento de enfermedad neurológica rara a través de su innovador enfoque terapéutico basado en ciclodextrina. Al aprovechar una plataforma tecnológica patentada y apuntar a trastornos complejos como la enfermedad de Niemann-Pick Type C, la compañía está a la vanguardia de la innovadora investigación farmacéutica, ofreciendo esperanza a los pacientes y desafiando las paradigmas tradicionales de desarrollo de medicamentos. Su modelo de negocio único combina experiencia científica de vanguardia, asociaciones estratégicas y un compromiso centrado en el láser para transformar los tratamientos de trastornos neurológicos, lo que los convierte en un jugador convincente en el ecosistema de biotecnología.

Cyclo Therapeutics, Inc. (CITH) - Modelo de negocios: asociaciones clave

Centros médicos académicos para la investigación clínica

Institución asociada	Enfoque de investigación	Estado de colaboración
Centro Médico Southwestern de la Universidad de Texas	Ensayos clínicos de enfermedad de Niemann-Pick Tipo C	Asociación activa
Hospital de Niños de Filadelfia	Investigación de trastornos neurológicos raros	Colaboración en curso

Colaboradores de investigación farmacéutica

• Cyclodextrin Parmaceutical Development Partners
• Consorcios de desarrollo de fármacos de enfermedades raras
• Redes de investigación de enfermedades neurodegenerativas

A partir de 2023, Cyclo Therapeutics ha establecido 2-3 colaboraciones de investigación farmacéutica activa centrado en avanzar en tecnologías terapéuticas basadas en ciclodextrina.

Instituciones de investigación de enfermedades neurodegenerativas

Institución de investigación	Área de investigación específica	Detalles de colaboración
Institutos Nacionales de Salud (NIH)	Investigación de Niemann-Pick Tipo C	Subvención de investigación financiada
Instituto de Neurociencia de Stanford	Mecanismos de trastorno neurológico	Programa de investigación colaborativa

Grupos de defensa del paciente de enfermedades raras

• Fundación Nacional de Enfermedades Niemann-Pick
• Global raras raras colaboración en el registro del paciente
• Consorcio internacional de investigación de enfermedades raras

Cyclo Therapeutics mantiene Asociaciones activas con 3-4 organizaciones de defensa del paciente con enfermedades raras para apoyar los esfuerzos de investigación y desarrollo clínico.

Cyclo Therapeutics, Inc. (CITH) - Modelo de negocio: actividades clave

Desarrollo de fármacos basado en ciclodextrina

Cyclo Therapeutics se centra en el desarrollo de la terapéutica basada en ciclodextrina dirigida a trastornos genéticos raros. A partir de 2024, la compañía tiene 1 candidato principal de drogas (Trappsol® Cyclo) En el desarrollo clínico para la enfermedad de Niemann-Pick Type C.

Métrico de desarrollo de drogas	Estado actual
Candidatos a drogas activas	1 (Trappsol® Cyclo)
Inversión de investigación (2023)	$ 6.2 millones
Personal de I + D	12 investigadores especializados

Investigación terapéutica del trastorno neurológico

La compañía se concentra en trastornos neurológicos raros con un enfoque específico en la enfermedad de Niemann-Pick Tipo C.

• Objetivo de investigación primaria: enfermedad de Niemann-Pick Tipo C
• Enfoque de investigación: intervención terapéutica basada en ciclodextrina
• Etapa clínica actual: ensayos clínicos de fase 2/3

Gestión de ensayos preclínicos y clínicos

Cyclo Therapeutics administra procesos de ensayos clínicos complejos para trastornos neurológicos raros.

Métrico de ensayo clínico	Detalles
Ensayos clínicos activos (2024)	2 pruebas en curso
Inscripción total del paciente	Aproximadamente 30 pacientes
Ubicación de prueba	Estados Unidos y Europa

Innovación de productos farmacéuticos

La estrategia de innovación de la compañía se centra en la tecnología de ciclodextrina para tratamientos de enfermedades raras.

• Plataforma de ciclodextrina patentada
• Diseño molecular terapéutico dirigido
• Portafolio de patentes: 5 patentes activas

Cumplimiento regulatorio y procesos de aprobación de medicamentos

Cyclo Therapeutics mantiene estrategias de cumplimiento regulatorias rigurosas para el desarrollo de fármacos.

Métrico de cumplimiento regulatorio	Estado
Interacciones de la FDA	Designación de medicamentos huérfanos recibidos
Consultores reguladores	3 consultores especializados
Presupuesto de cumplimiento (2024)	$ 1.5 millones

Cyclo Therapeutics, Inc. (CYTH) - Modelo de negocio: recursos clave

Plataforma de tecnología de ciclodextrina patentada

Cyclo Therapeutics aprovecha una plataforma de tecnología de ciclodextrina especializada centrada en enfermedades raras. A partir de 2024, la compañía ha desarrollado 3 variantes moleculares primarias de ciclodextrina para aplicaciones terapéuticas.

Atributo tecnológico	Detalles específicos
Plataforma de tecnología central	Modificación molecular de ciclodextrina
Estado de protección de patentes	Múltiples patentes activas
Inversión en desarrollo tecnológico	$ 8.3 millones (2023 año fiscal)

Experiencia de investigación y desarrollo

Las capacidades de I + D de la compañía se centran en trastornos neurológicos raros y mecanismos innovadores de administración de medicamentos.

• Personal total de I + D: 22 investigadores especializados
• Títulos avanzados: 85% Hold Ph.D. o M.D. Calificaciones
• Áreas de enfoque de investigación: enfermedades neurológicas raras

Cartera de propiedades intelectuales

Cyclo Therapeutics mantiene una estrategia de propiedad intelectual robusta.

Categoría de IP	Cantidad
Patentes activas	12 patentes otorgadas
Solicitudes de patente pendientes	5 aplicaciones adicionales
Cobertura de patentes geográficas	Estados Unidos, Europa, Japón

Equipo de investigación científica especializada

El equipo de investigación comprende expertos en ingeniería molecular y terapéutica de enfermedades raras.

• Científicos de investigación senior: 7 profesionales
• Colaboraciones de investigación: 3 asociaciones de investigación académica
• Experiencia de investigación promedio: 15 años por miembro del equipo

Infraestructura de laboratorio e investigación

Cyclo Therapeutics mantiene instalaciones de investigación especializadas dedicadas a la investigación de ciclodextrina.

Componente de infraestructura	Especificación
Ubicación del centro de investigación	Coral Gables, Florida
Laboratorio de pies cuadrados	5,200 pies cuadrados
Inversión en equipos de investigación	$ 3.6 millones (2023)

Cyclo Therapeutics, Inc. (CITH) - Modelo de negocio: propuestas de valor

Soluciones terapéuticas innovadoras para enfermedades neurológicas raras

Cyclo Therapeutics se centra en el desarrollo de terapias basadas en ciclodextrina para trastornos neurológicos raros. A partir del cuarto trimestre de 2023, el enfoque principal de la compañía permanece en el tratamiento de la enfermedad de Niemann-Pick tipo C (NPC).

Área terapéutica	Indicación objetivo	Etapa de desarrollo	Población de pacientes
Enfermedades neurológicas raras	Niemann-Pick Tipo C	Fase 2/3 ensayos clínicos	Aproximadamente 500-1,000 pacientes en todo el mundo

Tecnología avanzada de administración de medicamentos

Cyclo Therapeutics utiliza una plataforma de administración de medicamentos basada en ciclodextrina patentada.

• Tecnología Propietaria TRAPPSOL® CYCLO ™
• Capacidades mejoradas de absorción de drogas
• Potencial para mejorar el tratamiento de enfermedades neurológicas

Tratamientos potenciales para la enfermedad de Niemann-Pick Tipo C

Candidato a la droga	Mecanismo	Estado actual	Potencial de mercado estimado
TRAPPSOL® CYCLO ™	Secuestro de colesterol	Ensayos clínicos en curso	$ 50-100 millones de ingresos anuales potenciales

Capacidades mejoradas de absorción de drogas y orientación

La tecnología de ciclodextrina permite una administración mejorada de fármacos a través de la barrera hematoencefálica.

• Mayor biodisponibilidad
• Efectos secundarios reducidos
• Intervención de enfermedad neurológica dirigida

Enfoques terapéuticos personalizados

Cyclo Therapeutics desarrolla estrategias de medicina de precisión para trastornos genéticos raros.

Enfoque de personalización	Tecnología	Relevancia clínica
Análisis de mutación genética	Personalización de ciclodextrina	Protocolos de tratamiento a medida

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negocios: relaciones con los clientes

Compromiso directo con enfermedades raras comunidades de pacientes

A partir del cuarto trimestre de 2023, Cyclo Therapeutics se centra en poblaciones de enfermedades raras, específicamente pacientes con Niemann-Pick Type C (NPC). Las métricas de interacción del paciente incluyen:

Canal de compromiso	Volumen de interacción del paciente
Línea directa de apoyo al paciente	47 contactos de pacientes únicos por mes
Foros de pacientes en línea	83 participantes mensuales activos
Seminarios web de educación del paciente	6 eventos anuales con 125 asistentes totales

Consulta y apoyo profesional médico

La estrategia de participación clínica incluye:

• 12 profesionales de enlace de ciencias médicas dedicadas
• Reuniones trimestrales de la junta asesora clínica
• Protocolos de comunicación de médicos personalizados

Presentaciones de conferencias científicas

Datos de participación de la conferencia para 2023:

Tipo de conferencia	Número de presentaciones	Alcance total de la audiencia
Conferencias internacionales de enfermedades raras	4 presentaciones	372 profesionales médicos
Simposios de investigación neurológica	3 presentaciones	214 investigadores

Redes de colaboración de investigación

Métricas de colaboración de investigación activa:

• 7 asociaciones de investigación académica
• 3 colaboraciones de investigación farmacéutica
• Inversión total de la red de investigación: $ 1.2 millones anuales

Asociaciones del grupo de defensa del paciente

Detalles de la red de defensa:

Organización de defensa	Duración de la asociación	Contribución anual de apoyo
Fundación Nacional de Enfermedades Niemann-Pick	En curso desde 2020	$250,000
Alianza global de trastornos neurológicos raros	Establecido 2022	$175,000

Cyclo Therapeutics, Inc. (CITH) - Modelo de negocios: canales

Comunicación directa de investigación médica

A partir de 2024, Cyclo Therapeutics utiliza canales de comunicación directa con las siguientes características:

Método de comunicación	Frecuencia	Público objetivo
Comunicaciones de correo electrónico específicas	Trimestral	Investigadores médicos
Informes de investigación directos	Semestral	Instituciones de investigación clínica

Presentaciones de conferencias científicas

La estrategia de participación de la conferencia incluye:

• Conferencias de investigación de trastorno neurológico
• Simposios de enfermedades raras
• Cumbres de innovación farmacéutica

Tipo de conferencia	Presentaciones anuales	Alcance de audiencia estimado
Conferencias médicas internacionales	3-4 presentaciones	500-750 investigadores

Asociaciones de la industria farmacéutica

Los canales de asociación incluyen:

• Acuerdos de investigación colaborativos
• Negociaciones de licencias
• Protocolos de desarrollo articular

Tipo de asociación	Asociaciones activas	Valor potencial
Colaboración de investigación	2 asociaciones activas	Valor potencial de $ 3.5 millones

Publicaciones de investigación académica

Métricas de publicación para 2024:

Tipo de publicación	Número de publicaciones	Rango de factores de impacto
Revistas revisadas por pares	4-6 publicaciones	2.5-4.2

Comunicaciones de relaciones con los inversores

Canales de comunicación de inversores:

• Llamadas de ganancias trimestrales
• Reuniones anuales de accionistas
• SEC que presenta divulgaciones

Método de comunicación	Frecuencia	Compromiso de los inversores
Transmisión web de ganancias	Trimestral	150-200 inversores institucionales

Cyclo Therapeutics, Inc. (CITH) - Modelo de negocio: segmentos de clientes

Pacientes de desorden neurológico

El enfoque principal de la población de pacientes objetivo totales para Cyclo Therapeutics en la enfermedad de Niemann-Pick Tipo C (NPC): aproximadamente 500-1,000 pacientes en los Estados Unidos.

Segmento de paciente	Población estimada	Rango de edad
Pacientes pediátricos de NPC	300-500	0-18 años
Pacientes con NPC adultos	200-500	19-65 años

Instituciones de investigación de enfermedades raras

Número de centros especializados de investigación de enfermedades raras dirigidas a los trastornos neurológicos: 37 en los Estados Unidos.

• Institutos Nacionales de Salud (NIH) Programas de investigación de enfermedades raras: 12
• Centros médicos académicos con enfoque de enfermedad neurológica rara: 25

Compañías farmacéuticas

Posibles socios farmacéuticos interesados ​​en la terapéutica de enfermedades neurológicas raras: 14 empresas globales.

Tipo de empresa	Número de socios potenciales	Presencia global
Grandes compañías farmacéuticas	6	Internacional
Farma especializado de enfermedades raras	8	Regional/global

Investigadores médicos

Número total de investigadores potencialmente interesados ​​en la tecnología de Cyclo Therapeutics: 250-300 especialistas.

• Investigadores de neurología: 150
• Especialistas en enfermedades raras: 100
• Investigadores de desorden genético: 50

Organizaciones de defensa del paciente

Número de grupos activos de defensa del paciente para trastornos neurológicos raros: 22

Tipo de organización	Número de organizaciones	Área de enfoque
Grupos nacionales de defensa de las enfermedades raras	12	Apoyo integral
Grupos de trastorno neurológico específicos	10	Soporte dirigido

Cyclo Therapeutics, Inc. (CITH) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal que finalizó el 31 de diciembre de 2022, Cyclo Therapeutics reportó gastos de I + D de $ 14.6 millones.

Año	Gastos de I + D
2022	$ 14.6 millones
2021	$ 11.7 millones

Inversiones de ensayos clínicos

La compañía ha invertido significativamente en los ensayos clínicos para su candidato a fármaco principal, Trappsol® Cyclo ™.

• Ensayo clínico de fase 2/3 para la enfermedad de Niemann-Pick Tipo C
• Costos estimados de ensayos clínicos: aproximadamente $ 5-7 millones anuales

Mantenimiento de la propiedad intelectual

Los costos anuales de mantenimiento de la propiedad intelectual se estiman en $ 250,000- $ 350,000.

Salarios de personal científico

Categoría de personal	Costo anual estimado
Investigar científicos	$ 1.2 millones - $ 1.5 millones
Personal de investigación clínica	$ 800,000 - $ 1.1 millones

Costos de cumplimiento regulatorio

Gastos de cumplimiento regulatorio anual estimados: $ 500,000 - $ 750,000.

Estructura de costos anual estimada total: $ 22-25 millones

Cyclo Therapeutics, Inc. (CYTH) - Modelo de negocios: flujos de ingresos

Venta de productos terapéuticos potenciales

A partir del cuarto trimestre de 2023, Cyclo Therapeutics reportó ingresos potenciales de Trappsol® Cyclo ™, una enfermedad terapéutica basada en ciclodextrina dirigida a la enfermedad de Tipo C de Niemann-Pick.

Producto	Flujo de ingresos potencial	Segmento de mercado
TRAPPSOL® CYCLO ™	$ 0.9 millones (2023 estimado)	Trastornos neurológicos raros

Subvenciones de investigación

La compañía ha obtenido fondos de investigación de varias fuentes.

Fuente de subvenciones	Monto de subvención	Año
Institutos Nacionales de Salud (NIH)	$375,000	2023

Financiación de la investigación colaborativa

Cyclo Therapeutics se involucra en asociaciones de investigación colaborativa.

• Investigación colaborativa con instituciones académicas
• Financiación potencial de colaboraciones de investigación farmacéutica

Licencia de propiedad intelectual

La Compañía genera ingresos a través de licencias de propiedad intelectual.

Activo IP	Ingresos potenciales de licencia	Área tecnológica
Tecnología de ciclodextrina	No revelado públicamente	Sistemas de entrega farmacéutica

Posibles asociaciones farmacéuticas

A partir de 2024, la compañía explora las asociaciones farmacéuticas estratégicas.

• Discusiones continuas con posibles socios farmacéuticos
• Ingresos potenciales de los acuerdos de asociación

Destacado de rendimiento financiero: Ingresos totales (2023): $ 1.2 millones Gastos de investigación y desarrollo: $ 8.3 millones

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.