Cyclo Therapeutics, Inc. (CYTH): History, Ownership, Mission, How It Works & Makes Money

When you look at Cyclo Therapeutics, Inc. (CYTH), are you seeing a small-cap biotech with a 52-week price change of nearly -50%, or a company on the cusp of a major breakthrough in a rare, fatal disease? This clinical-stage firm, with a market capitalization of just $23.59 million as of March 2025, is betting its future on Trappsol® Cyclo™-a proprietary formulation of hydroxypropyl beta cyclodextrin-to treat Niemann-Pick Disease Type C1 (NPC1).

The entire investment thesis hinges on the topline data from the pivotal Phase 3 TransportNPC™ trial, which is expected in the first half of 2025 and could trigger a New Drug Application (NDA) submission to the FDA in the second half of the year, so understanding their core mission and precarious financial runway-like the Q3 2024 net loss of approximately $8.8 million-is defintely critical right now.

Cyclo Therapeutics, Inc. (CYTH) History

You're looking at a company that has undergone a fundamental transformation, shifting from a specialty chemical supplier to a focused, clinical-stage biotechnology firm. That pivot is the real story here. Cyclo Therapeutics, Inc. (CYTH) didn't start in the rare disease space; it evolved there by capitalizing on a unique molecule, Trappsol® Cyclo™ (hydroxypropyl beta cyclodextrin), and committing to the high-risk, high-reward world of drug development.

Given Company's Founding Timeline

Year established

The company traces its roots back to 1990, when it was formed as a Specialty Fine Chemical business centered on cyclodextrins, which are complex sugar molecules used in various industries. This foundation in chemical manufacturing provided the core expertise that would later be applied to medicine.

Original location

The principal offices are located in Gainesville, Florida. While the company reincorporated from Florida to Nevada in November 2020 for corporate structure reasons, its operational and scientific base remains in Gainesville.

Founding team members

Specific names of the original 1990 founding team are not widely publicized, but the company's formation was driven by individuals with expertise in cyclodextrin technology and pharmaceutical applications. Key scientific and executive leadership in the modern biotech era includes CEO N. Scott Fine, and scientific leaders like Dr. Sharon H. Hrynkow and Dr. Caroline Hastings, who have been critical to advancing the clinical programs.

Initial capital/funding

Details on the initial 1990 capital are not public. However, the first reported funding round for the company, then CTD Holdings, Inc., was a $500K Post IPO round in February 2014, marking the start of its dedicated biotech funding. More recently, the company secured a $5M Post IPO funding round on June 5, 2023.

Given Company's Evolution Milestones

Given Company's Transformative Moments

The company's trajectory is defined by a few high-stakes decisions that turned a chemical supplier into a biotech player. Honestly, the most significant was the mid-2010s pivot to clinical development.

• The 2014 Biotech Pivot: The decision to expand beyond selling cyclodextrins to actually developing a proprietary drug, Trappsol® Cyclo™, for a rare disease like NPC was a massive capital and strategic commitment. This move shifted the entire risk profile of the business.
• Securing Orphan Drug Status: Gaining Orphan Drug Designation in the U.S. (2010) and Europe (2015) provided crucial incentives, including tax credits, fee waivers, and, most importantly, market exclusivity for 7 to 10 years post-approval. This exclusivity is the core of the future revenue model.
• The 2024 Merger with Rafael Holdings: This transaction, expected to close in late 2024, is a financial lifeline and a strategic consolidation. Rafael Holdings committed to funding the pivotal TransportNPC™ clinical trial through its 48-week interim analysis in 2025. This ensures the trial's completion, which is the only thing that matters right now.
• The 2025 Regulatory Gateway: The expected topline data readout in the first half of 2025 is the ultimate transformative moment. Positive data will trigger the NDA/MAA submissions in the second half of 2025, which would transition Cyclo Therapeutics from a clinical-stage company with 2023 revenue of $1.08 million and a net loss of $20.06 million into a commercial-stage pharmaceutical company.

You can see the financial pressure in the Q3 2024 net loss of $8.8 million, driven by R&D expenses of $5.5 million, which shows the cost of running a Phase 3 trial. The entire valuation hinges on the 2025 data. For a deeper look at the balance sheet implications of this development, you should read Breaking Down Cyclo Therapeutics, Inc. (CYTH) Financial Health: Key Insights for Investors.

Cyclo Therapeutics, Inc. (CYTH) Ownership Structure

The ownership structure of Cyclo Therapeutics, Inc. underwent a fundamental shift in the 2025 fiscal year, transitioning from a publicly traded entity to a wholly-owned subsidiary of Rafael Holdings, Inc. This means all strategic decisions and financial oversight now flow directly from the parent company, which is itself a publicly traded biotechnology holding company.

Cyclo Therapeutics, Inc.'s Current Status

Cyclo Therapeutics, Inc. (CYTH) is no longer an independent, NASDAQ-listed public company. The company completed a merger with its long-time backer, Rafael Holdings, Inc. (NYSE: RFL), on March 26, 2025, effectively becoming Cyclo Therapeutics, LLC. This acquisition transformed the company into a private, wholly-owned operating subsidiary of the publicly traded Rafael Holdings. This move consolidated the development of its lead clinical asset, Trappsol® Cyclo™, under one corporate umbrella. You can dig deeper into the transaction's implications here: Exploring Cyclo Therapeutics, Inc. (CYTH) Investor Profile: Who's Buying and Why?

The strategic rationale was simple: secure funding and resources to push the pivotal Phase 3 TransportNPC™ trial to its 48-week interim analysis, which is expected in mid-2025. Before the merger, Rafael Holdings already held a significant stake, owning approximately 39.5% of Cyclo Therapeutics' common stock in early March 2025. The merger streamlined governance and financial commitment, but it also means Cyclo Therapeutics' performance is now an internal metric for Rafael Holdings.

Cyclo Therapeutics, Inc.'s Ownership Breakdown

As of November 2025, the ownership breakdown of the operating entity, Cyclo Therapeutics, LLC, is straightforward because of the acquisition. The parent company, Rafael Holdings, Inc., holds all equity. Former Cyclo Therapeutics shareholders received shares of Rafael Holdings Class B common stock, representing approximately 22% of the combined parent company.

Cyclo Therapeutics, Inc.'s Leadership

The company's direction is steered by the executive team of the subsidiary, Cyclo Therapeutics, LLC, with ultimate oversight from the leadership of the parent, Rafael Holdings, Inc. Following the merger, the leadership of the parent company saw a transition in April 2025, with a renewed focus on the Trappsol® Cyclo™ program.

Here's the quick math: The subsidiary's team drives the clinical execution, but the parent company's CEO sets the capital allocation strategy. It's a dual-layer structure now.

• Howard Jonas: Executive Chairman and CEO of Rafael Holdings, Inc. (Parent Company). He assumed the CEO role in April 2025, leading the overall corporate strategy.
• John Goldberg, MD: Chief Medical Officer of Cyclo Therapeutics, LLC. He is critical to the ongoing TransportNPC™ Phase 3 trial, which is the company's primary value driver.
• Michael Lisjak: Chief Regulatory Officer and Senior Vice President for Business Development of Cyclo Therapeutics, LLC. He manages the regulatory pathway toward a potential New Drug Application (NDA) submission, targeted for the second half of 2025.
• Karen Mullen, FFPM: Interim Chief Medical Officer of Cyclo Therapeutics, LLC.

This structure ensures the clinical-stage asset, Trappsol® Cyclo™, has dedicated operational and regulatory leadership while benefiting from the financial backing and strategic direction of the larger, publicly traded parent company, Rafael Holdings. This defintely reduces the near-term risk of funding shortfalls.

Cyclo Therapeutics, Inc. (CYTH) Mission and Values

Cyclo Therapeutics' core purpose is to bring hope to families facing rare, devastating diseases by pioneering new, life-changing medicines. Their mission is deeply rooted in patient-focused drug development, which drives their clinical strategy for Niemann-Pick Type C disease (NPC).

Cyclo Therapeutics' Core Purpose

The company's commitment extends beyond typical biotech growth metrics, focusing instead on addressing areas of significant unmet medical need, a strategy that led to their merger with Rafael Holdings in March 2025. This strategic move was intended to solidify resources to deliver the results of their pivotal clinical trial.

Official Mission Statement

The mission statement clearly articulates a dedication to the human element of drug development, emphasizing the impact on both patients and their families.

• Dedicated to developing life-changing medicines through science and innovation for patients and families suffering from disease.

This mission is embodied in their lead asset, Trappsol Cyclo (hydroxypropyl beta cyclodextrin), which is an orphan drug designated product in the U.S. and Europe, specifically targeting the rare, fatal genetic disorder Niemann-Pick Disease Type C1 (NPC1). The urgency of this mission is reflected in their financial profile; for the fiscal year 2024, the company reported Annual Sales of approximately $1,080 K but an Annual Net Income of approximately -$20,060 K, showing the significant investment required for clinical-stage rare disease research.

Vision Statement

The vision statement maps their scientific work directly to a tangible, empathetic outcome for the people they serve. It's a clean one-liner that cuts right to the point.

• Providing hope through patient-focused drug development to improve quality of life.

To be fair, this vision is being tested in 2025 with the TransportNPC™ Phase 3 global study, which is fully enrolled. The results from the 48-week interim analysis are expected in the middle of 2025, which is a critical, near-term milestone for realizing this vision.

Cyclo Therapeutics' Core Values in Action

While a formal list of core values isn't always published, the company's actions and communications consistently highlight a few key operational values that defintely drive their strategy:

• Patient Focus: Prioritizing rare and fatal diseases where treatment options are scarce, such as NPC1.
• Scientific Innovation: Developing a cyclodextrin-based product, Trappsol Cyclo, to facilitate cholesterol transport in cells, effectively taking the place of the defective NPC1 protein.
• Commitment to Data: Actively presenting encouraging preliminary data from the ongoing Phase 3 study at major scientific symposia like the WORLDSymposium 2025.

The merger with Rafael Holdings in 2025, where Cyclo Therapeutics became a wholly-owned subsidiary, was a clear action to leverage a stronger balance sheet and management team to ensure the TransportNPC™ trial could be fully funded and completed, showing a deep commitment to the patient community over short-term market stability. Mission Statement, Vision, & Core Values of Cyclo Therapeutics, Inc. (CYTH). Finance: track the TransportNPC™ interim analysis release date by mid-2025 for its impact on the combined entity's valuation.

Cyclo Therapeutics, Inc. (CYTH) How It Works

Cyclo Therapeutics, Inc., now a wholly owned subsidiary of Rafael Holdings, Inc. as of March 2025, operates as a clinical-stage biopharmaceutical company focused on developing a proprietary drug to treat devastating neurodegenerative diseases. The company's entire value proposition hinges on its lead asset, Trappsol Cyclo, which is designed to fix a fundamental cellular transport problem at the root of Niemann-Pick Disease Type C1 (NPC1) and potentially other cholesterol-related disorders.

The core business model is straightforward: invest heavily in clinical trials to gain regulatory approval for an orphan drug, and then commercialize that drug to serve a high-unmet-need patient population.

Cyclo Therapeutics, Inc.'s Product/Service Portfolio

Cyclo Therapeutics, Inc.'s Operational Framework

The company's operations are almost entirely focused on clinical development and regulatory strategy, which is typical for a clinical-stage biotech. Their value creation process is a multi-stage, high-cost pipeline designed to move a molecule from the lab to market.

Here's the quick math: in a recent quarter, the company reported revenue of roughly $1.08 million, primarily from the sale of cyclodextrins, but a net income loss of approximately $8.83 million due to the enormous cost of research and development (R&D) for their trials. This loss is the cost of doing business when you're trying to bring a life-changing drug to market.

• R&D Execution: Manage the pivotal Phase 3 TransportNPC™ trial, which involves 104 enrolled patients across 23 sites in 9 countries, with a key 48-week interim analysis expected in the middle of 2025.
• Manufacturing & Supply: Oversee the third-party production of the proprietary Trappsol Cyclo formulation, ensuring a consistent and high-quality supply for ongoing clinical trials.
• Regulatory Strategy: Prepare for a potential New Drug Application (NDA) submission to the U.S. FDA and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) based on the 48-week interim data, should it demonstrate significance.
• Corporate Finance: Manage the capital structure and funding, now significantly supported by the resources of the parent company, Rafael Holdings, which is committed to funding the TransportNPC™ trial.

The merger with Rafael Holdings in March 2025 defintely solidified the financial runway for the critical Phase 3 trial.

Cyclo Therapeutics, Inc.'s Strategic Advantages

In the high-stakes world of rare disease drug development, an analyst looks for three things: a unique mechanism, regulatory protection, and a clear path to market. Cyclo Therapeutics has all three. Mission Statement, Vision, & Core Values of Cyclo Therapeutics, Inc. (CYTH).

• Unique Mechanism of Action: Trappsol Cyclo's active ingredient, HPβCD, is a cyclic oligosaccharide that physically sequesters and transports accumulated cholesterol out of the cell's lysosomes, bypassing the defective NPC1 protein. This direct approach is a powerful differentiator.
• Orphan Drug Exclusivity: The drug has Orphan Drug Designation in both the U.S. and Europe for NPC1, which provides significant regulatory incentives, including market exclusivity upon approval.
• Advanced Clinical Stage: The company is one of the most advanced in the NPC treatment space, with a fully enrolled pivotal Phase 3 trial (TransportNPC™). Positive preliminary data showed 86% of patients in the sub-study demonstrated stabilization or improvement at 48 weeks.
• Strong Gross Margin Potential: Despite current losses, the company maintains an impressive gross profit margin of over 91.24% on its core operations, indicating that once approved, the drug's revenue will be highly profitable.

Cyclo Therapeutics, Inc. (CYTH) How It Makes Money

Cyclo Therapeutics, Inc., following its merger with Rafael Holdings in March 2025, operates as a clinical-stage biotechnology company whose current financial engine is two-fold: selling specialized cyclodextrin chemicals for non-drug applications and, more critically, securing capital and grants to fund the development of its lead drug candidate, Trappsol® Cyclo™.

The company's long-term revenue model is entirely dependent on the successful regulatory approval and commercial launch of Trappsol® Cyclo™ for Niemann-Pick Disease Type C1 (NPC1), which would shift its revenue from minimal commercial sales to a high-value, low-volume orphan drug pricing structure.

Given Company's Revenue Breakdown

As a pre-commercial biotech, Cyclo Therapeutics' revenue is minimal and volatile, driven by its legacy business of selling cyclodextrins-the base chemical for its drug-and non-recurring grants. The trailing twelve months (TTM) revenue for the Cyclo Therapeutics business unit as of late 2024 was approximately $870.73K, a very small figure relative to its operational burn rate.

Here's the quick math: the bulk of the current revenue, roughly 90%, comes from the sale of its core cyclodextrin compounds to the pharmaceutical and nutritional industries. This commercial revenue stream has been decreasing, with a 52.82% year-over-year drop in Q3 2024 revenue. The remaining portion comes from grants and collaborations, which are inherently unpredictable. The real value driver is the Phase 3 trial, not these small commercial sales.

Business Economics

The entire economic fundamental of Cyclo Therapeutics hinges on the successful commercialization of Trappsol® Cyclo™ as an orphan drug (a drug for a rare disease affecting fewer than 200,000 people in the U.S.). This is a classic low-volume, high-price model.

• Orphan Drug Pricing: The median annual wholesale acquisition cost (WAC) for new orphan drugs for genetic disorders in the U.S. is approximately $218,872 per patient per year. Given the chronic nature of NPC1, the annual cost of Trappsol® Cyclo™ is expected to fall within this high-end range, a necessary price point to justify the significant research and development (R&D) investment for a small patient population.
• Market Size: Niemann-Pick Disease Type C1 is extremely rare, with an estimated prevalence of about 1 in 100,000. The commercial opportunity is small in patient count, but the high price per patient creates a viable market.
• Priority Review Voucher (PRV): The company has received Rare Pediatric Disease Designation, which qualifies it for a PRV upon FDA marketing approval. This voucher can be sold to a larger pharmaceutical company for a substantial, non-dilutive cash infusion, historically fetching well over $100 million. This is a crucial, near-term financial opportunity, defintely worth more than a year of drug sales at launch.

Given Company's Financial Performance

As of the most recent reporting periods in 2025, the company's financial performance reflects its status as a high-burn, clinical-stage entity focused on generating pivotal trial data, not profit. The merger with Rafael Holdings, completed in March 2025, was primarily a financial maneuver to secure capital for the final stages of the TransportNPC™ Phase 3 trial.

• Net Loss: For the full fiscal year ended July 31, 2025, the consolidated entity reported a net loss attributable to Rafael Holdings of approximately $30.5 million. This loss is directly tied to the high cost of running a global Phase 3 trial.
• R&D Expenses: Research and development expenses for the three months ended July 31, 2025 (Q4 Fiscal 2025), soared to $7.5 million. This massive increase is a direct result of consolidating Cyclo Therapeutics' clinical trial spending post-merger and reflects the intense investment required to complete the TransportNPC™ trial.
• Cash Position: As of January 31, 2025, the company reported cash and cash equivalents of approximately $48.3 million. This cash runway is the lifeblood of the business, funding the R&D burn until the anticipated New Drug Application (NDA) submission in the second half of 2025.
• Working Capital Risk: Prior to the merger, Cyclo Therapeutics faced severe liquidity issues, reporting a negative working capital of $15,463,491 as of September 30, 2024. The merger was the clear action taken to resolve this critical near-term risk.

The entire financial story right now is a race against the clock: burn the cash to get the data, file the NDA, and unlock the high-margin orphan drug revenue stream. You can dive deeper into the implications of this cash burn and the merger's impact here: Breaking Down Cyclo Therapeutics, Inc. (CYTH) Financial Health: Key Insights for Investors.

Cyclo Therapeutics, Inc. (CYTH) Market Position & Future Outlook

Cyclo Therapeutics, Inc. (CYTH) is at a critical inflection point in late 2025, transitioning from a clinical-stage entity to a potential commercial-stage rare disease player, provided its Phase 3 data is positive. The company's future hinges on the success of Trappsol Cyclo, a cyclodextrin-based therapy for Niemann-Pick Disease Type C1 (NPC1), which targets a rapidly expanding market projected to grow at a CAGR of up to 17.05% through 2035.

The strategic merger with Rafael Holdings, which completed in March 2025, has provided a new structure to fund the final steps of the pivotal TransportNPC™ trial. You're looking at a classic biotech high-risk, high-reward scenario: a pre-revenue company with a potential blockbuster drug submission on the near-term horizon.

Competitive Landscape

The Niemann-Pick Disease Type C (NPC) treatment market is ultra-rare but saw two new FDA approvals in late 2024, fundamentally changing the landscape as Cyclo Therapeutics prepares for its own submission.

Opportunities & Challenges

The most immediate opportunity is the readout of the Phase 3 data, but the company must also navigate severe liquidity risk.

Industry Position

As of late 2025, Cyclo Therapeutics is positioned as a high-potential, high-risk challenger in the ultra-rare disease space, specifically Niemann-Pick Disease Type C.

• The company's primary asset, Trappsol Cyclo, is a cholesterol-mobilizing agent, a mechanism of action that directly addresses the underlying pathology of cholesterol accumulation, differentiating it from the recently approved therapies.
• While the company is pre-revenue for its lead drug-projected FY 2025 revenue is only around $985.66K-its valuation is driven entirely by the Phase 3 outcome.
• The market's excitement is defintely tempered by the Q3 2024 net loss of approximately $8.8 million, which highlights the urgent need for a successful regulatory outcome to secure long-term financing.
• The merger with Rafael Holdings was a necessary step to consolidate resources and focus on the NPC program, but the fundamental challenge remains: moving from a research expense to a revenue generator.

To be fair, the market for NPC is small-about 1,000 diagnosed prevalent cases in the 7MM in 2024-but the high price point of orphan drugs makes it a multi-hundred-million-dollar opportunity for the first-to-market players. You need to weigh the clinical promise against the financial runway. You can read more about the company's backing at Exploring Cyclo Therapeutics, Inc. (CYTH) Investor Profile: Who's Buying and Why?

Next Step: Monitor the H1 2025 topline data release for the TransportNPC™ trial; this is the single most important catalyst for the stock in the near term.