Cyclo Therapeutics, Inc. (Cyth): Modelo de negócios Canvas [Jan-2025 Atualizado]

A Cyclo Therapeutics, Inc. (Cyth) está revolucionando o cenário do tratamento raro de doenças neurológicas por meio de sua abordagem terapêutica inovadora à base de ciclodextrina. Ao alavancar uma sofisticada plataforma de tecnologia proprietária e direcionar distúrbios complexos como a doença de Niemann-Pick tipo C, a empresa está na vanguarda da pesquisa farmacêutica inovadora, oferecendo esperança aos pacientes e desafiando os paradigmas tradicionais de desenvolvimento de medicamentos. Seu modelo de negócios exclusivo combina experiência científica de ponta, parcerias estratégicas e um compromisso focado a laser em transformar tratamentos de transtorno neurológico, tornando-os um participante atraente no ecossistema de biotecnologia.

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: Parcerias -chave

Centros Médicos Acadêmicos de Pesquisa Clínica

Instituição parceira	Foco na pesquisa	Status de colaboração
Centro Médico do Sudoeste da Universidade do Texas	Ensaios clínicos da doença do tipo C Niemann-Pick	Parceria ativa
Hospital Infantil da Filadélfia	Pesquisa de distúrbios neurológicos raros	Colaboração em andamento

Colaboradores de pesquisa farmacêutica

• Parceiros de Desenvolvimento Farmacêutico da Ciclodextrina
• Consórcio de Desenvolvimento de Medicamentos de Doenças Raras
• Redes de pesquisa de doenças neurodegenerativas

A partir de 2023, a Cyclo Therapeutics estabeleceu 2-3 Colaborações de pesquisa farmacêutica ativa focado no avanço das tecnologias terapêuticas à base de ciclodextrina.

Instituições de pesquisa de doenças neurodegenerativas

Instituição de pesquisa	Área de pesquisa específica	Detalhes da colaboração
Institutos Nacionais de Saúde (NIH)	Niemann-Pick Tipo C Research	Concessão de pesquisa financiada
Instituto de Neurociência de Stanford	Mecanismos de Transtorno Neurológico	Programa de pesquisa colaborativa

Grupos de defesa de pacientes com doenças raras

• Fundação Nacional de Doença Niemann-Pick
• Colaboração global de registro de pacientes com doenças raras
• Consórcio Internacional de Pesquisa de Doenças Raras

Cyclo Therapeutics mantém Parcerias ativas com 3-4 organizações de defesa de pacientes com doenças raras para apoiar os esforços de pesquisa e desenvolvimento clínico.

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: Atividades -chave

Desenvolvimento de medicamentos à base de ciclodextrina

A Cyclo Therapeutics se concentra no desenvolvimento de terapêuticas à base de ciclodextrina visando distúrbios genéticos raros. A partir de 2024, a empresa tem 1 candidato a medicamentos primários (Trappsol® Cyclo) no desenvolvimento clínico para a doença de Niemann-Pick tipo C.

Métrica de Desenvolvimento de Medicamentos	Status atual
Candidatos ativos de drogas	1 (Trappsol® Cyclo)
Investimento de pesquisa (2023)	US $ 6,2 milhões
Pessoal de P&D	12 pesquisadores especializados

Pesquisa terapêutica do distúrbio neurológico

A empresa se concentra em distúrbios neurológicos raros, com foco específico na doença de Niemann-Pick Tipo C.

• Alvo de pesquisa primária: doença de Niemann-Pick Tipo C
• Abordagem de pesquisa: intervenção terapêutica baseada em ciclodextrina
• Etapa clínica atual: Fase 2/3 de ensaios clínicos

Gerenciamento de ensaios pré -clínicos e clínicos

A Cyclo Therapeutics gerencia processos complexos de ensaio clínico para distúrbios neurológicos raros.

Métrica do ensaio clínico	Detalhes
Ensaios clínicos ativos (2024)	2 ensaios em andamento
Inscrição total do paciente	Aproximadamente 30 pacientes
Locais de teste	Estados Unidos e Europa

Inovação de produtos farmacêuticos

A estratégia de inovação da empresa centra -se em tecnologia de ciclodextrina para tratamentos de doenças raras.

• Plataforma de ciclodextrina proprietária
• Projeto molecular terapêutico direcionado
• Portfólio de patentes: 5 patentes ativas

Processos de conformidade regulatória e aprovação de medicamentos

A Cyclo Therapeutics mantém estratégias rigorosas de conformidade regulatória para o desenvolvimento de medicamentos.

Métrica de conformidade regulatória	Status
Interações FDA	Designação de medicamentos órfãos recebidos
Consultores regulatórios	3 consultores especializados
Orçamento de conformidade (2024)	US $ 1,5 milhão

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: Recursos -chave

Plataforma proprietária de tecnologia de ciclodextrina

A Cyclo Therapeutics aproveita uma plataforma especializada em tecnologia de ciclodextrina focada em doenças raras. A partir de 2024, a empresa desenvolveu 3 variantes moleculares primárias de ciclodextrina para aplicações terapêuticas.

Atributo de tecnologia	Detalhes específicos
Plataforma de tecnologia principal	Modificação molecular de ciclodextrina proprietária
Status de proteção de patentes	Múltiplas patentes ativas
Investimento em desenvolvimento de tecnologia	US $ 8,3 milhões (2023 ano fiscal)

Especialização em pesquisa e desenvolvimento

Os recursos de P&D da empresa estão centrados em distúrbios neurológicos raros e mecanismos inovadores de administração de medicamentos.

• Pessoal total de P&D: 22 pesquisadores especializados
• Graus avançados: 85% Hold Ph.D. ou qualificações de M.D.
• Áreas de foco de pesquisa: doenças raras neurológicas

Portfólio de propriedade intelectual

A Cyclo Therapeutics mantém uma estratégia de propriedade intelectual robusta.

Categoria IP	Quantidade
Patentes ativas	12 patentes concedidas
Pedidos de patente pendentes	5 aplicações adicionais
Cobertura de patente geográfica	Estados Unidos, Europa, Japão

Equipe de pesquisa científica especializada

A equipe de pesquisa compreende especialistas em engenharia molecular e terapêutica de doenças raras.

• Cientistas de pesquisa seniores: 7 profissionais
• Colaborações de pesquisa: 3 parcerias de pesquisa acadêmica
• Experiência média de pesquisa: 15 anos por membro da equipe

Infraestrutura de laboratório e pesquisa

A Cyclo Therapeutics mantém instalações de pesquisa especializadas dedicadas à pesquisa da ciclodextrina.

Componente de infraestrutura	Especificação
Localização da instalação de pesquisa	Coral Gables, Flórida
Metragem quadrada de laboratório	5.200 pés quadrados
Investimento em equipamentos de pesquisa	US $ 3,6 milhões (2023)

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: proposições de valor

Soluções terapêuticas inovadoras para doenças neurológicas raras

A Cyclo Therapeutics se concentra no desenvolvimento de terapias à base de ciclodextrina para distúrbios neurológicos raros. A partir do quarto trimestre 2023, o foco principal da empresa permanece no tratamento da doença de Niemann-Pick tipo C (NPC).

Área terapêutica	Indicação alvo	Estágio de desenvolvimento	População de pacientes
Doenças neurológicas raras	Niemann-pick tipo c	Fase 2/3 Ensaios Clínicos	Aproximadamente 500-1.000 pacientes em todo o mundo

Tecnologia avançada de entrega de medicamentos

A Cyclo Therapeutics utiliza a plataforma proprietária de entrega de medicamentos à base de ciclodextrina.

• Tecnologia proprietária Trappsol® Cyclo ™
• Capacidades aprimoradas de absorção de drogas
• Potencial para melhorar o tratamento de doenças neurológicas

Tratamentos em potencial para doença de Niemann-Pick Tipo C

Candidato a drogas	Mecanismo	Status atual	Potencial estimado de mercado
TRAPPSOL® Cyclo ™	Sequestro de colesterol	Ensaios clínicos em andamento	Receita anual potencial de US $ 50-100 milhões

Melhor absorção de drogas e capacidades de direcionamento

A tecnologia de ciclodextrina permite a entrega aprimorada de medicamentos através da barreira hematoencefálica.

• Aumento da biodisponibilidade
• Efeitos colaterais reduzidos
• Intervenção de doença neurológica direcionada

Abordagens terapêuticas personalizadas

A Cyclo Therapeutics desenvolve estratégias de medicina de precisão para distúrbios genéticos raros.

Abordagem de personalização	Tecnologia	Relevância clínica
Análise de mutação genética	Personalização da ciclodextrina	Protocolos de tratamento personalizado

Cyclo Therapeutics, Inc. (Cyth) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com comunidades de pacientes com doenças raras

A partir do quarto trimestre 2023, a Cyclo Therapeutics se concentra em populações de doenças raras, especificamente pacientes com Niemann-Pick tipo C (NPC). As métricas de interação do paciente incluem:

Canal de engajamento	Volume de interação do paciente
Apoio ao paciente Linha direta	47 contatos únicos de pacientes por mês
Fóruns de pacientes online	83 participantes mensais ativos
Webinars de educação do paciente	6 eventos anuais com 125 participantes no total

Consulta profissional médica e suporte

A estratégia de engajamento clínico inclui:

• 12 Profissionais dedicados de ligação médica
• Reuniões trimestrais do Conselho Consultivo Clínico
• Protocolos de comunicação médicos personalizados

Apresentações da conferência científica

Dados de envolvimento da conferência para 2023:

Tipo de conferência	Número de apresentações	Alcance total do público
Conferências de doenças raras internacionais	4 apresentações	372 profissionais médicos
Simpósios de pesquisa neurológica	3 apresentações	214 pesquisadores

Redes de colaboração de pesquisa

Métricas de colaboração de pesquisa ativa:

• 7 parcerias de pesquisa acadêmica
• 3 colaborações de pesquisa farmacêutica
• Investimento total da rede de pesquisa: US $ 1,2 milhão anualmente

Parcerias do Grupo de Advocacia dos Pacientes

Detalhes da rede de advocacia:

Organização de Advocacia	Duração da parceria	Contribuição de suporte anual
Fundação Nacional de Doença Niemann-Pick	Em andamento desde 2020	$250,000
Aliança Global de Distúrbios Neurológicos Raros	Estabelecido 2022	$175,000

Cyclo Therapeutics, Inc. (Cyth) - Modelo de Negócios: Canais

Comunicação direta de pesquisa médica

Em 2024, a Cyclo Therapeutics utiliza canais de comunicação direta com as seguintes características:

Método de comunicação	Freqüência	Público -alvo
Comunicações de email direcionadas	Trimestral	Pesquisadores médicos
Briefres de pesquisa direta	Semestral	Instituições de Pesquisa Clínica

Apresentações da conferência científica

A estratégia de envolvimento da conferência inclui:

• Conferências de Pesquisa de Transtorno Neurológico
• Simpósios de doenças raras
• Cúpulas de inovação farmacêutica

Tipo de conferência	Apresentações anuais	Alcance estimado do público
Conferências médicas internacionais	3-4 apresentações	500-750 pesquisadores

Parcerias da indústria farmacêutica

Os canais de parceria incluem:

• Acordos de pesquisa colaborativa
• Negociações de licenciamento
• Protocolos de desenvolvimento conjunto

Tipo de parceria	Parcerias ativas	Valor potencial
Colaboração de pesquisa	2 parcerias ativas	Valor potencial de US $ 3,5 milhões

Publicações de pesquisa acadêmica

Métricas de publicação para 2024:

Tipo de publicação	Número de publicações	Faixa de fatores de impacto
Revistas revisadas por pares	4-6 publicações	2.5-4.2

Comunicações de Relações com Investidores

Canais de comunicação de investidores:

• Chamadas de ganhos trimestrais
• Reuniões anuais de acionistas
• Sec Divulgações de arquivamento

Método de comunicação	Freqüência	Engajamento do investidor
Webcast de ganhos	Trimestral	150-200 investidores institucionais

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: segmentos de clientes

Pacientes com transtorno neurológico

População total de pacientes-alvo para o foco principal da Cyclo Therapeutics na doença de Niemann-Pick tipo C (NPC): aproximadamente 500-1.000 pacientes nos Estados Unidos.

Segmento de pacientes	População estimada	Faixa etária
Pacientes com NPC pediátricos	300-500	0-18 anos
Pacientes com NPC adultos	200-500	19-65 anos

Instituições de pesquisa de doenças raras

Número de centros especializados de pesquisa de doenças raras direcionadas a distúrbios neurológicos: 37 nos Estados Unidos.

• Institutos Nacionais de Saúde (NIH) Programas de pesquisa de doenças raras: 12
• Centros médicos acadêmicos com doenças neurológicas raras foco: 25

Empresas farmacêuticas

Parceiros farmacêuticos em potencial interessados ​​em terapêutica rara de doenças neurológicas: 14 empresas globais.

Tipo de empresa	Número de parceiros em potencial	Presença global
Grandes empresas farmacêuticas	6	Internacional
Doenças raras especializadas farmacêuticas	8	Regional/Global

Pesquisadores médicos

Número total de pesquisadores potencialmente interessados ​​na tecnologia da Cyclo Therapeutics: 250-300 especialistas.

• Pesquisadores de neurologia: 150
• Especialistas em doenças raras: 100
• Pesquisadores de Transtorno Genético: 50

Organizações de defesa de pacientes

Número de grupos ativos de defesa de pacientes para distúrbios neurológicos raros: 22

Tipo de organização	Número de organizações	Área de foco
Grupos Nacionais de Advocacia de Doenças Raras	12	Apoio abrangente
Grupos de transtorno neurológico específicos	10	Suporte direcionado

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o exercício fiscal encerrado em 31 de dezembro de 2022, a Cyclo Therapeutics registrou despesas de P&D de US $ 14,6 milhões.

Ano	Despesas de P&D
2022	US $ 14,6 milhões
2021	US $ 11,7 milhões

Investimentos de ensaios clínicos

A Companhia investiu significativamente em ensaios clínicos para seu candidato a medicamentos, Trappsol® Cyclo ™.

• Fase 2/3 Ensaio Clínico para Doença de Niemann-Pick Tipo C
• Custos estimados de ensaios clínicos: aproximadamente US $ 5-7 milhões anualmente

Manutenção da propriedade intelectual

Os custos anuais de manutenção da propriedade intelectual são estimados em US $ 250.000 a US $ 350.000.

Salários de pessoal científico

Categoria de pessoal	Custo anual estimado
Cientistas de pesquisa	US $ 1,2 milhão - US $ 1,5 milhão
Equipe de pesquisa clínica	$ 800.000 - US $ 1,1 milhão

Custos de conformidade regulatória

Despesas anuais estimadas de conformidade regulatória: US $ 500.000 - US $ 750.000.

Estrutura de custo anual estimada total: US $ 22-25 milhões

Cyclo Therapeutics, Inc. (Cyth) - Modelo de negócios: fluxos de receita

Vendas potenciais de produtos terapêuticos

A partir do quarto trimestre 2023, a Cyclo Therapeutics relatou receita potencial do Trappsol® Cyclo ™, uma doença terapêutica terapêutica à base de ciclodextrina Niemann-Pick Type C.

Produto	Fluxo de receita potencial	Segmento de mercado
TRAPPSOL® Cyclo ™	US $ 0,9 milhão (2023 estimado)	Distúrbios neurológicos raros

Bolsas de pesquisa

A empresa garantiu financiamento de pesquisa de várias fontes.

Fonte de concessão	Valor de concessão	Ano
Institutos Nacionais de Saúde (NIH)	$375,000	2023

Financiamento de pesquisa colaborativa

A Cyclo Therapeutics se envolve em parcerias de pesquisa colaborativa.

• Pesquisa colaborativa com instituições acadêmicas
• Financiamento potencial de colaborações de pesquisa farmacêutica

Propriedade intelectual de licenciamento

A empresa gera receita por meio de licenciamento de propriedade intelectual.

Ativo IP	Potencial receita de licenciamento	Área de tecnologia
Tecnologia da ciclodextrina	Não divulgado publicamente	Sistemas de entrega farmacêutica

Parcerias farmacêuticas em potencial

A partir de 2024, a empresa explora parcerias farmacêuticas estratégicas.

• Discussões em andamento com potenciais parceiros farmacêuticos
• Receita potencial de acordos de parceria

Destaques de desempenho financeiro: Receita total (2023): US $ 1,2 milhão Despesas de pesquisa e desenvolvimento: US $ 8,3 milhões

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.