Cyclo Therapeutics, Inc. (CYTH): Business Model Canvas [Dec-2025 Updated]

You're looking at Cyclo Therapeutics, Inc. (CYTH) right at the critical inflection point: a company pivoting from pure R&D to potential commercialization, banking on its pivotal Phase 3 trial data for Niemann-Pick Disease Type C1 (NPC1) to land an NDA/MAA submission in the second half of 2025. As an analyst who's seen this movie before, I can tell you the business model-heavily reliant on its partner, Rafael Holdings, for capital while burning through about $5.5 million quarterly in R&D as of Q3 2024-is entirely dependent on regulatory success and securing that future revenue stream, perhaps even via a Priority Review Voucher sale. This Canvas maps the entire structure, showing how they plan to move from zero commercial sales today to delivering a first-in-class therapy for an ultra-rare disease, so let's dive into the key resources and the massive risks baked into their 2026 launch timeline.

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

You're looking at the structure of Cyclo Therapeutics, Inc. (now a wholly owned subsidiary of Rafael Holdings, Inc. following the merger) as of late 2025. The key partnerships here are all about securing the capital, executing the critical clinical program, and preparing for potential commercialization of Trappsol® Cyclo™.

Rafael Holdings, Inc. as primary funding and strategic partner

Rafael Holdings, Inc. is the ultimate strategic owner, having completed the business combination with Cyclo Therapeutics on March 26, 2025. This partnership is the financial backbone. Rafael Holdings previously supported Cyclo Therapeutics with strategic investments starting in March 2023 and financing rounds in the fall of 2023. The financing arrangements leading up to the merger included convertible notes totaling $16 million since June 2024. Rafael Holdings had a reported market cap of $22.7 million as of February 2025.

The post-merger structure cemented this relationship. Rafael Holdings issued shares of its Class B common stock to Cyclo Therapeutics' shareholders, representing approximately 22% of the combined company, based on an exchange ratio of 0.3525. To ensure continued development, Rafael Holdings closed a significant $25 million rights offering in June 2025, which included a $21.0 million backstop commitment from the Jonas family. As of July 31, 2025, the combined entity reported cash and cash equivalents of $52.8 million. The combined company recorded a net loss attributable to Rafael Holdings of $30.5 million for the twelve months ended July 31, 2025.

Here's a quick look at the financial backing and structure:

Financial Metric/Event	Value/Date	Context
Merger Closing Date	March 26, 2025	Finalization of business combination.
Rafael Holdings Ownership Post-Merger (Approx.)	22% of combined company	Issued to former Cyclo Therapeutics shareholders.
Total Convertible Note Financing (Since June 2024)	$16 million	Financing prior to merger closing.
June 2025 Rights Offering Amount	$25 million	New capital raised post-merger.
Cash & Equivalents (As of July 31, 2025)	$52.8 million	Liquidity position for the combined entity.
R&D Expenses (12 Months Ended July 31, 2025)	$12.8 million	Includes spending at Cyclo Therapeutics post-acquisition.

Global Clinical Research Organizations (CROs) for trial execution

The execution of the pivotal TransportNPC™ Phase 3 trial relies heavily on external CRO expertise. Cyclo Therapeutics signed a Master Services Agreement with Worldwide Clinical Trials to serve as the lead CRO for its clinical programs. This partnership covers both the Niemann-Pick Disease Type C (NPC) and Alzheimer's Disease evaluations. The TransportNPC™ study, evaluating Trappsol® Cyclo™ for NPC1, reached a significant milestone with enrollment completion in May 2024. The study has 104 enrolled patients for the primary analysis.

The key near-term deliverable from this partnership was the 48-week interim analysis, which was expected in the middle of 2025 or H1 2025. Furthermore, Cyclo Therapeutics is conducting a Phase 2b clinical trial for early Alzheimer's disease (NCT05607615), also utilizing this CRO framework.

• TransportNPC™ Trial Enrollment Completion: May 2024.
• Number of Enrolled Patients for Interim Analysis: 104.
• Expected 48-Week Interim Analysis Readout: Mid-2025 / H1 2025.
• Primary CRO Partner: Worldwide Clinical Trials.

Key Opinion Leaders (KOLs) and clinical investigators worldwide

The clinical credibility of Trappsol® Cyclo™ is supported by presentations from leading medical experts at major international symposia. The TransportNPC™ study is a prospective, randomized, double-blind, placebo-controlled, multi-center therapeutic study. Data from the ongoing pivotal study and its sub-study were highlighted at the 21st Annual WORLDSymposium™ 2025 in February 2025. Key investigators presented findings, including Dr. Caroline Hastings and Ronen Spiegel, MD. Additional data was presented at ICIEM 2025. The sub-study in patients under 3 years old showed stabilization or improvement in Clinical Global Impression - Change (CGI-C) Scale for 87% (6 of 7 patients) at 48 weeks.

The engagement with KOLs is quantified by the presentation venues and the scope of the trials:

• WORLDSymposium™ Attendance (Global): Over 2000 participants from more than 50 countries.
• Sub-Study 48-Week CGI-C Stabilization/Improvement Rate: 86%.
• Number of Formal NPC1 Clinical Trials: Four.

Patient advocacy groups (e.g., NNPDF) for community support

Cyclo Therapeutics has expressed commitment to the Niemann-Pick Disease Type C community globally. The company's work is aimed at patients suffering from this rare, fatal genetic disease. While the NNPDF (National Niemann-Pick Disease Foundation) is a key group in this space, specific financial contributions or joint statistical metrics from 2025 are not detailed in the latest public filings reviewed. The partnership focus is on delivering trial results to the community.

Contract manufacturers for commercial-scale production of Trappsol® Cyclo™

The company has established its supply chain readiness. Cyclo Therapeutics commenced commercial-scale production of Trappsol® Cyclo™ batches in July 2021. This was done to support the Chemistry and Manufacturing Controls (CMC) section required by the FDA and to build inventory for clinical trials. The manufacturing process is described as robust, scalable, and validated. The resulting drug product inventory was specifically designated to support the ongoing pivotal Phase 3 study. Specific contract manufacturer names or current commercial production volumes for 2025 are not publicly itemized. Finance: draft 13-week cash view by Friday.

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

You're looking at the core engine driving Cyclo Therapeutics, Inc. right now-the activities that must fire perfectly to get Trappsol® Cyclo™ across the finish line for Niemann-Pick Disease Type C1 (NPC1) and keep the Alzheimer's program moving. It's all about execution on the clinical and regulatory fronts.

Executing the pivotal Phase 3 TransportNPC™ trial

The main focus is definitely the pivotal Phase 3 TransportNPC™ trial. This study completed enrollment back in May 2024. The key activity now is processing the data from the 104 enrolled patients. The company was on track for topline data from the 48-week interim analysis in the first half of 2025 (H1 2025). This is a 96-week, randomized, double-blind, placebo-controlled, multicenter study. The team is managing a global footprint, which adds complexity, but the structure is set.

Here's a quick look at the patient profile from the study and substudy:

Metric	Value	Context
Total Enrolled (Study + Substudy)	104	Patients in TransportNPC™ trial and substudy
Substudy Enrollment (Newborn to 3 years)	Ten (10)	Patients in the single-arm substudy
Average ASIS Score at Recruitment	1.04	Average severity score for enrolled patients
Average Age at Inclusion	20.2 years	Range was 3 - 65 years
Primary Endpoint (EU)	Change in 5D-NPC-CSS	Measures ambulation, motor, speech, swallow, cognition

If onboarding takes too long, the timeline slips. Anyway, the merger with Rafael Holdings closed on March 26, 2025, which should solidify resources for this crucial phase.

Preparing New Drug Application (NDA) and MAA submissions

The successful execution of the trial directly feeds into the next major activity: regulatory submissions. Cyclo Therapeutics, Inc. was targeting a 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). This is a huge push. Furthermore, the company expects qualification for a Priority Review Voucher (PRV) upon NDA submission.

• Achieve positive 48-week interim analysis data readout (Targeted H1 2025).
• Prepare and file NDA with the U.S. Food and Drug Administration (FDA).
• Prepare and file MAA with the European Medicines Agency (EMA).
• Secure the expected Priority Review Voucher upon approval.

Research and development for Trappsol® Cyclo™ in Alzheimer's disease (Phase 2b)

While NPC1 is the near-term focus, the R&D engine is running on the Alzheimer's disease (AD) asset. Cyclo Therapeutics, Inc. is conducting a Phase 2b clinical trial for early AD, which is designed to enroll approximately 120 patients. This study evaluates monthly infusions of Trappsol® Cyclo™ at doses of 500 mg/kg or 1000 mg/kg, compared to placebo, over a 6-month treatment period. The scientific activity here is targeting the reduction of amyloid beta and tau pathology. To fund this, Research and development expenses for the three months ended September 30, 2024, were approximately $5.5 million.

Securing and maintaining global Orphan Drug Designations (ODD)

Maintaining regulatory status is a key activity that de-risks the lead asset. Cyclo Therapeutics, Inc. has secured several important designations for Trappsol® Cyclo™ in treating NPC1. These designations are critical for market access and potential financial incentives.

• Orphan Drug Designation (ODD) secured in both the U.S. and EU.
• Fast Track Designation in the U.S..
• Rare Pediatric Disease Designation in the U.S., which is tied to the PRV qualification.

Managing intellectual property and patent portfolio expansion

Protecting the platform technology is non-negotiable. The company actively manages its intellectual property estate. A significant recent activity was the announcement of a decision to grant a European Patent Application for the treatment of Alzheimer's Disease in July 2024. Even earlier, in March 2024, Cyclo Therapeutics, Inc. announced the issuance of a U.S. Patent covering the use of Trappsol® Cyclo™ for Alzheimer's Disease treatment. This shows defintely an ongoing effort to build a strong patent estate around the core molecule.

Finance: draft 13-week cash view by Friday.

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

You're looking at the core assets that power Cyclo Therapeutics, Inc.'s value proposition. These aren't just line items; they are the patented science, regulatory advantages, and financial backing that underpin their entire operation as of late 2025.

Proprietary Trappsol® Cyclo™ (HPBCD) formulation

The foundational resource is the proprietary drug substance, Trappsol® Cyclo™, which is hydroxypropyl-beta-cyclodextrin (HPBCD). This molecule is central to their development pipeline for both Niemann-Pick Disease Type C1 (NPC1) and Alzheimer's disease. A key technical resource is the demonstrated ability of Trappsol® Cyclo™ to cross the blood-brain barrier following intravenous (IV) administration, as shown in completed Phase 1 and Phase 2 studies. The mechanism of action involves mobilizing cholesterol from late-stage endosomes and lysosomes, directly targeting the root cause of NPC1.

The formulation itself is a mixture of beta-cyclodextrin molecules substituted by hydroxypropyl groups. The Alzheimer's treatment patent specifies potential monthly dose amounts for the composition:

• Stabilization or improvement in Clinical Global Impression - Severity (CGI-S) score for sub-study patients at 48 weeks.

Orphan Drug Designation status in the US and EU

Cyclo Therapeutics, Inc. has secured significant regulatory advantages for Trappsol® Cyclo™ in its lead indication, Niemann-Pick Disease Type C1 (NPC1). This designation is a critical resource because it offers market exclusivity incentives and potential tax credits upon approval. The product is designated as an orphan drug in both major markets:

• United States designation for NPC1.
• Europe designation for NPC1.

This status is tied to the four formal clinical trials for NPC1: NCT02939547, NCT02912793, NCT03893071, and NCT04860960.

U.S. Patent No. 11,925,659 covering Alzheimer's disease treatment

Intellectual property provides a crucial barrier to entry for competitors. U.S. Patent No. 11,925,659, granted on March 14, 2024, specifically covers methods for treating early onset Alzheimer's disease using Trappsol® Cyclo™. This patent is active and is set to expire on December 27, 2040. The patent claims detail specific dosing regimens for the active ingredient, hydroxypropyl-beta-cyclodextrin, which can be administered in monthly amounts selected from a range of values.

The claimed monthly dose amounts are:

Dose Amount (mg/kg)	Administration Frequency
500	Monthly, Twice a Month, or Weekly
1,000	Monthly, Twice a Month, or Weekly
1,500	Monthly, Twice a Month, or Weekly
2,000	Monthly, Twice a Month, or Weekly
2,500	Monthly, Twice a Month, or Weekly
3,000	Monthly, Twice a Month, or Weekly

Capital funding and financial support from Rafael Holdings

The financial runway is heavily influenced by the strategic relationship with Rafael Holdings, Inc. The definitive merger agreement to combine the two companies closed in March 2025, effectively consolidating financial resources. Rafael Holdings, Inc. enhanced its financial position with a rights offering in June 2025.

Key financial figures related to this support, as reported by Rafael Holdings, Inc. for its fiscal year 2025:

• Rights offering closed on June 4, 2025, for $25 million.
• The Jonas family funded the backstop commitment within that offering for $21.0 million.
• Cash and cash equivalents for Rafael Holdings, Inc. stood at $52.8 million as of July 31, 2025.
• Research and development expenses for the twelve months ended July 31, 2025, were $12.8 million, reflecting the inclusion of Cyclo Therapeutics, Inc.'s spending post-acquisition.

This financial backing was intended to fund the TransportNPC™ clinical trial through its critical 48-week interim analysis. Rafael Holdings, Inc. had previously provided support via a $2.1 million private placement in March 2023 and subsequent convertible debt financings in 2024.

Clinical data from the comprehensive TransportNPC™ Phase 3 study

The pivotal Phase 3 global study, TransportNPC™, is a randomized, double-blind, placebo-controlled, multicenter study for NPC1, designed with a 96-week duration and a 48-week comparative interim analysis. Enrollment for the main study was completed in May 2024, with 104 patients enrolled across over 25 sites in 13 countries. The independent Data Monitoring Committee (DMC) reviewed the 48-week interim data in June 2025 and recommended the study continue for the full 96 weeks.

Data from the open-label sub-study in the youngest patients (< 3 years old) is also a key resource, as it suggests potential preventative effects when administered early. The sub-study enrolled ten (10) patients.

Here are the reported statistical outcomes from the sub-study data presented in September 2025:

Endpoint/Analysis Point	Patient Group Size	Result/Number
Patients Reached 48 Weeks (CGI-S)	Nine	Seven showed stabilization or improvement
Patients Showing Improvement (CGI-S) at 48 Weeks	Nine	Three
Patients Showing Deterioration (CGI-S) at 48 Weeks	Nine	Two
Total Adverse Events (AEs) as of May 14, 2025	N/A	146
Severe AEs Reported	N/A	3

The primary endpoints for the main TransportNPC study are the mean change in the 4-domain NPC Clinical Severity Scale (4D-NPC-CSS). The drug is administered intravenously at a dose of 2000 mg/kg every 2 weeks.

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.