Cyclo Therapeutics, Inc. (Cyth): Business Model Canvas [Jan-2025 Mis à jour]

Cyclo Therapeutics, Inc. (Cyth) révolutionne le paysage du traitement rares de la maladie neurologique par son approche thérapeutique révolutionnaire à base de cyclodextrine. En tirant parti d'une plate-forme technologique propriétaire sophistiquée et en ciblant les troubles complexes comme la maladie de Niemann-Pick de type C, l'entreprise est à l'avant-garde d'une recherche pharmaceutique innovante, offrant de l'espoir aux patients et contester les paradigmes traditionnels de développement de médicaments. Leur modèle commercial unique combine une expertise scientifique de pointe, des partenariats stratégiques et un engagement axé sur le laser à transformer les traitements des troubles neurologiques, ce qui en fait un acteur convaincant dans l'écosystème de la biotechnologie.

Cyclo Therapeutics, Inc. (Cyth) - Modèle commercial: partenariats clés

Centres médicaux académiques pour la recherche clinique

Institution partenaire	Focus de recherche	Statut de collaboration
Université du Texas Southwestern Medical Center	Niemann-Pick Type C Disease Clinical Trials	Partenariat actif
Hôpital pour enfants de Philadelphie	Recherche de troubles neurologiques rares	Collaboration continue

Collaborateurs de la recherche pharmaceutique

• Cyclodextrin Pharmaceutical Development Partners
• Consortiums de développement de médicaments rares
• Réseaux de recherche sur les maladies neurodégénératives

En 2023, le cyclo thérapeutique a établi 2-3 collaborations de recherche pharmaceutique active axé sur l'avancement des technologies thérapeutiques à base de cyclodextrine.

Institutions de recherche sur les maladies neurodégénératives

Institution de recherche	Domaine de recherche spécifique	Détails de collaboration
National Institutes of Health (NIH)	Recherche Niemann-Pick de type C	Subvention de recherche financée
Stanford Neuroscience Institute	Mécanismes de troubles neurologiques	Programme de recherche collaborative

Groupes de plaidoyer pour les patients atteints de maladies rares

• Fondation nationale de la maladie de Niemann-Pick
• Collaboration mondiale du registre des patients atteints de patients
• Consortium international de recherche sur les maladies rares

Cyclo Therapeutics maintient Partenariats actifs avec 3-4 organisations de défense des patients atteints de maladies rares pour soutenir les efforts de recherche et de développement clinique.

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: activités clés

Développement de médicaments à base de cyclodextrine

Cyclo Therapeutics se concentre sur le développement de la thérapeutique basée sur la cyclodextrine ciblant les troubles génétiques rares. Depuis 2024, la société a 1 candidat de médicament primaire (Trappsol® Cyclo) dans le développement clinique de la maladie de Niemann-Pick de type C.

Métrique de développement de médicaments	État actuel
Candidats à la drogue active	1 (Trappsol® Cyclo)
Investissement en recherche (2023)	6,2 millions de dollars
Personnel de R&D	12 chercheurs spécialisés

Recherche thérapeutique des troubles neurologiques

La société se concentre sur les troubles neurologiques rares avec un accent spécifique sur la maladie de Niemann-Pick de type C.

• Primal Research Cible: Niemann-Pick Type C
• Approche de la recherche: intervention thérapeutique à base de cyclodextrine
• Étape clinique actuelle: phase 2/3 essais cliniques

Gestion des essais précliniques et cliniques

Cyclo Therapeutics gère des processus d'essais cliniques complexes pour des troubles neurologiques rares.

Métrique d'essai clinique	Détails
Essais cliniques actifs (2024)	2 essais en cours
Inscription totale	Environ 30 patients
Lieux d'essai	États-Unis et Europe

Innovation de produit pharmaceutique

La stratégie d'innovation de l'entreprise se concentre sur la technologie de la cyclodextrine pour les traitements de maladies rares.

• Plate-forme de cyclodextrine propriétaire
• Conception moléculaire thérapeutique ciblée
• Portefeuille de brevets: 5 brevets actifs

Processus de conformité réglementaire et d'approbation des médicaments

Cyclo Therapeutics maintient des stratégies de conformité réglementaire rigoureuses pour le développement de médicaments.

Métrique de la conformité réglementaire	Statut
Interactions de la FDA	Désignation de médicaments orphelins reçus
Consultants réglementaires	3 consultants spécialisés
Budget de conformité (2024)	1,5 million de dollars

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: Ressources clés

Plateforme de technologie de cyclodextrine propriétaire

Le cyclo thérapeutique exploite une plate-forme technologique spécialisée de la cyclodextrine axée sur les maladies rares. Depuis 2024, la société a développé 3 variantes moléculaires de cyclodextrine primaires pour les applications thérapeutiques.

Attribut technologique	Détails spécifiques
Plateforme de technologie de base	Modification moléculaire de cyclodextrine propriétaire
Statut de protection des brevets	Multiples brevets actifs
Investissement de développement technologique	8,3 millions de dollars (2023 Exercice)

Expertise en recherche et développement

Les capacités de R&D de l'entreprise sont centrées sur des troubles neurologiques rares et des mécanismes innovants d'administration de médicaments.

• Personnel total de R&D: 22 chercheurs spécialisés
• Degrés avancés: 85% tiennent le doctorat. ou les qualifications de M.D.
• Recherchez les domaines d'intérêt: maladies rares neurologiques

Portefeuille de propriété intellectuelle

Cyclo Therapeutics maintient une solide stratégie de propriété intellectuelle.

Catégorie IP	Quantité
Brevets actifs	12 brevets accordés
Demandes de brevet en attente	5 applications supplémentaires
Couverture des brevets géographiques	États-Unis, Europe, Japon

Équipe de recherche scientifique spécialisée

L'équipe de recherche comprend des experts en génie moléculaire et en thérapeutiques rares.

• Chercheurs principaux: 7 professionnels
• Collaborations de recherche: 3 partenariats de recherche universitaire
• Expérience de recherche moyenne: 15 ans par membre de l'équipe

Infrastructure de laboratoire et de recherche

Cyclo Therapeutics maintient des installations de recherche spécialisées dédiées à la recherche sur la cyclodextrine.

Composant d'infrastructure	Spécification
Emplacement de l'installation de recherche	Coral Gables, Floride
Laboratoire en pieds carrés	5 200 pieds carrés
Investissement de l'équipement de recherche	3,6 millions de dollars (2023)

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: propositions de valeur

Solutions thérapeutiques innovantes pour les maladies neurologiques rares

Cyclo Therapeutics se concentre sur le développement de thérapies à base de cyclodextrine pour des troubles neurologiques rares. Depuis le quatrième trimestre 2023, l'objectif principal de l'entreprise reste sur le traitement de la maladie de Niemann-Pick de type C (NPC).

Zone thérapeutique	Indication cible	Étape de développement	Population de patients
Maladies neurologiques rares	Niemann-Pick Type C	Phase 2/3 essais cliniques	Environ 500 à 1 000 patients dans le monde

Technologie avancée de livraison de médicaments

Cyclo Therapeutics utilise la plate-forme de livraison de médicaments basée sur la cyclodextrine propriétaire.

• Technologie propriétaire de Trappsol® Cyclo ™
• Capacités d'absorption des médicaments améliorés
• Potentiel d'amélioration du traitement des maladies neurologiques

Traitements potentiels pour la maladie de Niemann-Pick de type C

Drogue	Mécanisme	État actuel	Potentiel de marché estimé
Trappsol® Cyclo ™	Séquestration du cholestérol	Essais cliniques en cours	50 à 100 millions de dollars de revenus annuels potentiels

Amélioration des capacités d'absorption et de ciblage des médicaments

La technologie de la cyclodextrine permet une administration accrue de médicaments à travers la barrière hémato-encéphalique.

• Biodisponibilité accrue
• Effets secondaires réduits
• Intervention ciblée des maladies neurologiques

Approches thérapeutiques personnalisées

Le cyclo thérapeutique développe des stratégies de médecine de précision pour les troubles génétiques rares.

Approche de personnalisation	Technologie	Pertinence clinique
Analyse de mutation génétique	Personnalisation de la cyclodextrine	Protocoles de traitement sur mesure

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: relations clients

Engagement direct avec les communautés de patients atteints de maladies rares

Depuis le Q4 2023, le cyclo-thérapeutique se concentre sur les populations de maladies rares, en particulier les patients Niemann-Pick de type C (NPC). Les mesures d'interaction des patients comprennent:

Canal de fiançailles	Volume d'interaction des patients
Hotline de soutien des patients	47 contacts uniques des patients par mois
Forums de patients en ligne	83 participants mensuels actifs
Webinaires de l'éducation des patients	6 événements annuels avec 125 participants au total

Consultation et soutien professionnel en médecine

La stratégie d'engagement clinique comprend:

• 12 professionnels de liaison en sciences médicales dédiées
• Réunions trimestrielles du conseil consultatif clinique
• Protocoles de communication des médecins personnalisés

Présentations de la conférence scientifique

Données d'engagement de la conférence pour 2023:

Type de conférence	Nombre de présentations	Total du public
Conférences internationales de maladies rares	4 présentations	372 professionnels de la santé
Symposiums de recherche neurologique	3 présentations	214 chercheurs

Réseaux de collaboration de recherche

Métriques de collaboration de recherche active:

• 7 partenariats de recherche académique
• 3 collaborations de recherche pharmaceutique
• Investissement total du réseau de recherche: 1,2 million de dollars par an

Partenariats du groupe de défense des patients

Détails du réseau de plaidoyer:

Organisation de plaidoyer	Durée du partenariat	Contribution de soutien annuelle
Fondation nationale de la maladie de Niemann-Pick	En cours depuis 2020	$250,000
Alliance mondiale des troubles neurologiques rares	Établi 2022	$175,000

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: canaux

Communication de recherche médicale directe

En 2024, Cyclo Therapeutics utilise des canaux de communication directs avec les caractéristiques suivantes:

Méthode de communication	Fréquence	Public cible
Communications par e-mail ciblées	Trimestriel	Chercheurs en médecine
Briefings de recherche directs	Semestriel	Institutions de recherche clinique

Présentations de la conférence scientifique

La stratégie d'engagement de la conférence comprend:

• Conférences de recherche sur les troubles neurologiques
• Symposiums de maladies rares
• Sommets de l'innovation pharmaceutique

Type de conférence	Présentations annuelles	Reach du public estimé
Conférences médicales internationales	3-4 présentations	500-750 chercheurs

Partenariats de l'industrie pharmaceutique

Les canaux de partenariat comprennent:

• Accords de recherche collaborative
• Négociations de licence
• Protocoles de développement conjoints

Type de partenariat	Partenariats actifs	Valeur potentielle
Collaboration de recherche	2 partenariats actifs	Valeur potentielle de 3,5 millions de dollars

Publications de recherche universitaire

Métriques de publication pour 2024:

Type de publication	Nombre de publications	Plage du facteur d'impact
Journaux évalués par des pairs	4-6 publications	2.5-4.2

Communications des relations avec les investisseurs

Canaux de communication des investisseurs:

• Appels de résultats trimestriels
• Réunions annuelles des actionnaires
• Divulgations de classement de la SEC

Méthode de communication	Fréquence	Engagement des investisseurs
Webdication sur les gains	Trimestriel	150-200 investisseurs institutionnels

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: segments de clientèle

Patients de troubles neurologiques

La population totale de patients cible pour les cyclo thérapeutiques axée sur la maladie de Niemann-Pick de type C (NPC): environ 500 à 1 000 patients aux États-Unis.

Segment des patients	Population estimée	Tranche d'âge
Patients atteints de PNJ pédiatrique	300-500	0-18 ans
Patients atteints de PNJ adulte	200-500	19-65 ans

Institutions de recherche de maladies rares

Nombre de centres de recherche sur les maladies rares spécialisées ciblant les troubles neurologiques: 37 aux États-Unis.

• Programmes de recherche sur les maladies rares du National Institutes of Health (NIH): 12
• Centres médicaux académiques avec des maladies neurologiques rares focus: 25

Sociétés pharmaceutiques

Partners pharmaceutiques potentiels intéressés par la thérapie rare des maladies neurologiques: 14 entreprises mondiales.

Type d'entreprise	Nombre de partenaires potentiels	Présence mondiale
Grandes sociétés pharmaceutiques	6	International
Maladies rares spécialisées pharmaceutique	8	Régional / global

Chercheurs en médecine

Nombre total de chercheurs potentiellement intéressés par la technologie de Cyclo Therapeutics: 250-300 spécialistes.

• Rechercheurs de neurologie: 150
• Spécialistes de maladies rares: 100
• Rechercheurs de troubles génétiques: 50

Organisations de défense des patients

Nombre de groupes de défense des patients actifs pour les troubles neurologiques rares: 22

Type d'organisation	Nombre d'organisations	Domaine de mise au point
Groupes de plaidoyer nationaux de maladies rares	12	Soutien complet
Groupes de troubles neurologiques spécifiques	10	Support ciblé

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice clos le 31 décembre 2022, Cyclo Therapeutics a déclaré des dépenses de R&D de 14,6 millions de dollars.

Année	Dépenses de R&D
2022	14,6 millions de dollars
2021	11,7 millions de dollars

Investissements d'essais cliniques

La société a investi considérablement dans des essais cliniques pour son candidat principal, TRAPPSOL® Cyclo ™.

• Phase 2/3 essai clinique pour la maladie de Niemann-Pick de type C
• Coût estimé des essais cliniques: environ 5 à 7 millions de dollars par an

Maintenance de la propriété intellectuelle

Les coûts annuels de maintenance de la propriété intellectuelle sont estimés de 250 000 $ à 350 000 $.

Salaires du personnel scientifique

Catégorie de personnel	Coût annuel estimé
Chercheur	1,2 million de dollars - 1,5 million de dollars
Personnel de recherche clinique	800 000 $ - 1,1 million de dollars

Coûts de conformité réglementaire

Dépenses de conformité réglementaire annuelles estimées: 500 000 $ - 750 000 $.

Structure totale des coûts annuels estimés: 22 à 25 millions de dollars

Cyclo Therapeutics, Inc. (Cyth) - Modèle d'entreprise: Strots de revenus

Ventes de produits thérapeutiques potentiels

Depuis le Q4 2023, Cyclo Therapeutics a signalé des revenus potentiels de Trappsol® Cyclo ™, un ciblage thérapeutique à base de cyclodextrine ciblant la maladie de Niemann-Pick de type C.

Produit	Stronce de revenus potentiel	Segment de marché
Trappsol® Cyclo ™	0,9 million de dollars (2023 estimé)	Troubles neurologiques rares

Subventions de recherche

La Société a obtenu un financement de recherche à partir de diverses sources.

Source d'octroi	Montant d'octroi	Année
National Institutes of Health (NIH)	$375,000	2023

Financement de recherche collaborative

Cyclo Therapeutics s'engage dans des partenariats de recherche collaborative.

• Recherche collaborative avec les établissements universitaires
• Financement potentiel des collaborations de recherche pharmaceutique

Licence de propriété intellectuelle

La société génère des revenus grâce à des licences de propriété intellectuelle.

Actif IP	Revenus de licence potentielle	Zone technologique
Technologie de cyclodextrine	Pas divulgué publiquement	Systèmes de livraison pharmaceutique

Partenariats pharmaceutiques potentiels

Depuis 2024, la société explore les partenariats pharmaceutiques stratégiques.

• Discussions en cours avec des partenaires pharmaceutiques potentiels
• Revenus potentiels des accords de partenariat

Points forts de la performance financière: Revenu total (2023): 1,2 million de dollars Dépenses de recherche et de développement: 8,3 millions de dollars

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.