Cyclo Therapeutics

What Cyclo Therapeutics does

Cyclo Therapeutics is a clinical-stage biotechnology company developing Trappsol Cyclo, a proprietary intravenous formulation of hydroxypropyl beta cyclodextrin (HPbCD), for the treatment of Niemann-Pick Disease Type C1 (NPC1) and early Alzheimer's Disease. The lead asset works by replacing the function of the defective NPC1 protein to mobilize accumulated cholesterol from cellular lysosomes, with demonstrated ability to cross the blood-brain barrier after IV administration in completed Phase 1 and Phase 2 studies. The company is headquartered in Alachua, Florida, has operated since 2017, and maintains a small team of 1-10 employees, with global clinical operations across 27+ sites in 13 countries. The pivotal TransportNPC Phase 3 program in NPC1 (patients aged 3 and older, with a pediatric substudy for newborns to 3 years) completed enrollment in May 2024 and last patient last visit in June 2026, with topline results expected in Q3 2026 and NDA submission planned for the second half of 2026. A parallel Phase 2b study is ongoing in early Alzheimer's Disease, supported by an exclusive licensing agreement with MIT covering U.S. Patent No. 12285440 for cyclodextrin use in ApoE4 carriers, and an Expanded Access Program supplies Trappsol Cyclo to NPC patients unable to enroll in trials.

Cyclo Therapeutics holds Orphan Drug Designation in both the US and EU for NPC1, plus FDA Fast Track and Rare Pediatric Disease Designations, with the latter qualifying the sponsor for a Priority Review Voucher upon approval. A competing therapy, Zevra's MIPLYFFA, was approved in 2024, modestly reducing the unmet-need framing in NPC1. The company is pre-revenue, with no approved products on the market, and revenue will be contingent on FDA/EMA approval and adoption via rare disease specialists and specialty pharmacy distribution. Following a $7.1 million private placement from Rafael Holdings in 2023, an all-stock merger with Applied Molecular Transport in December 2023 (Nasdaq: CYTH), and a definitive merger agreement in August 2024, Rafael Holdings completed its acquisition of Cyclo Therapeutics in March 2025, making Cyclo Therapeutics, LLC a wholly owned subsidiary of Rafael Holdings, Inc. (NYSE: RFL). Rafael issued shares representing approximately 22% of the combined company to former Cyclo shareholders at an exchange ratio of 0.3525.

Name

Cyclo Therapeutics

Legal name

Cyclo Therapeutics, LLC

Website

https://cyclotherapeutics.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

1–10 employees

Short description

Cyclo Therapeutics is a clinical-stage biotechnology company developing Trappsol Cyclo, a proprietary hydroxypropyl beta cyclodextrin therapy, for Niemann-Pick Disease Type C1 and early Alzheimer's Disease, targeting ultra-rare and neurological disease patient populations.

Ownership category

akta.pro rank

Product category

Rare disease therapeutics

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Scientific Research and Development Services (5417)

SIC

Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

akta.pro secondary industry

Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• Rare disease therapeutics
• Cyclodextrin drug development
• Neurological disorder treatments
• Niemann-Pick disease therapy
• Alzheimer's disease research

Headquarters

HQ city

Alachua

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations, Marketing or Sales

Revenue model

1. Pharmaceutical Product Revenue: Pre-revenue clinical stage biotechnology company. Revenue will be generated from sale of Trappsol Cyclo upon successful FDA/EMA regulatory approval for Niemann-Pick Disease Type C1 and potentially Alzheimer's Disease. The company holds Orphan Drug Designation in the US and EU, qualifying it for market exclusivity benefits.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels7 records

Core offering

Cyclo Therapeutics is a clinical-stage biotechnology company developing Trappsol Cyclo, a proprietary intravenous formulation of hydroxypropyl beta cyclodextrin (HPbCD) for the treatment of Niemann-Pick Disease Type C1 and early Alzheimer's Disease. The compound mobilizes accumulated cholesterol out of cellular lysosomes by substituting for the defective NPC1 protein, and has demonstrated blood-brain barrier crossing in clinical studies. The product pipeline is being advanced through the pivotal TransportNPC Phase 3 trial, a Phase 2b Alzheimer's trial, and an FDA-acknowledged Expanded Access Program.

Product overview

Cyclo Therapeutics is a clinical-stage biotechnology company with a single lead therapeutic asset, Trappsol Cyclo (hydroxypropyl beta cyclodextrin), being developed for multiple indications. The product pipeline includes: (1) Trappsol Cyclo as the core proprietary drug candidate; (2) the pivotal Phase 3 TransportNPC trial for Niemann-Pick Disease Type C1 in patients 3 years and older with a substudy for patients birth to 3 years; (3) a Phase 2b trial for early Alzheimer's Disease; and (4) an Expanded Access Program for patient access. Trappsol Cyclo is not currently approved for any indication.

Differentiator

Problem solved

Functional benefit

Products and services

• Trappsol Cyclo A proprietary intravenous formulation of hydroxypropyl beta cyclodextrin (HPbCD) being evaluated in multiple clinical trials for the potential treatment of Niemann-Pick Disease Type C1 (a rare, fatal, progressive genetic disorder) and Alzheimer's Disease. The drug facilitates transport of accumulated cholesterol out of cellular lysosomes by substituting for the defective NPC1 protein.
• TransportNPC Phase 3 Study A pivotal Phase 3 randomized, double-blind, placebo-controlled, multicenter study evaluating the safety, tolerability, and efficacy of 2,000 mg/kg doses of Trappsol Cyclo administered intravenously in patients aged 3 years and older with Niemann-Pick Disease Type C1. The 96-week study enrolled 94 patients across 13 countries with an additional 10-patient substudy for patients birth to 3 years of age.
• Phase 2b Early Alzheimer's Disease Study A Phase 2b clinical trial using Trappsol Cyclo intravenously in early Alzheimer's Disease, targeting reduction of amyloid beta and tau. The trial also includes patients carrying the ApoE4 genetic variant based on an exclusive MIT license (US Patent 12285440).
• Expanded Access Program A program providing Trappsol Cyclo to physicians for single patient expanded access use, currently focused on NPC patients who cannot participate in clinical trials. The company has received FDA acknowledgement for its US Expanded Access Program to proceed.

Quantifiable outcome

• 89% of patients met efficacy outcome measure of improvement in at least 2 domains of the 17-domain NPC Severity Scale in Phase 1/2 study
• +3 more outcomes

Named customers2 records

Segments2 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Four partnerships are on record, tiered core and minor.

• Massachusetts Institute of Technology (MIT)coreTechnology or Integration · 22 April 2026Exclusive licensing agreement with MIT covering U.S. Patent No. 12285440 for the use of cyclodextrins in treating patients with Alzheimer's Disease who carry the ApoE4 genetic mutation. The ApoE4 gene variant is estimated to be present in 50-70% of Alzheimer's patients and is a key risk factor in early-onset sporadic AD. This strengthens Cyclo's IP portfolio around Trappsol Cyclo for Alzheimer's applications.
• University of the Witwatersrand, JohannesburgminorStrategic or Co-development PartnerOngoing collaboration with University of the Witwatersrand, Johannesburg to advance Trappsol Cyclo Platform and explore pipeline expansion opportunities.
• National Niemann-Pick Disease Foundation (NNPDF)coreGTM or Marketing PartnerPatient advocacy partner providing support, connections and resources for NPC patients and families. Cyclo works closely with NNPDF to build awareness and bring solutions to reality. NNPDF serves as the US member organization of the International Niemann-Pick Disease Alliance.
• International Niemann-Pick Disease Alliance (INPDA)coreGTM or Marketing PartnerGlobal alliance of patient advocacy organizations working to support NPC patients and families. Cyclo collaborates with INPDA member organizations worldwide.

Scale indicators7 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Zevra Therapeutics: Zevra Therapeutics received FDA approval of MIPLYFFA (arimoclomol) for Niemann-Pick Disease Type C in 2024, making it the most direct NPC1 competitor to Trappsol Cyclo. Both companies target the same rare-disease population with disease-modifying therapies, and Zevra's first-mover position defines the competitive headwind Cyclo would face at launch.
• Orphazyme: Orphazyme developed arimoclomol for NPC (later acquired by Zevra) and was historically one of the most visible NPC-focused biotechs. Its trajectory — including financial difficulties and ultimate asset transfer — illustrates both the opportunity and the execution risk inherent in commercializing an NPC therapy.

Broad incumbents

• BioMarin Pharmaceutical: BioMarin is a large rare-disease pharmaceutical company with established global commercialization, regulatory, and patient-advocacy infrastructure for ultra-rare diseases. While it does not currently market an NPC therapy, it represents the strategic-acquisition profile for late-stage rare-disease assets like Trappsol Cyclo.
• Ultragenyx Pharmaceutical: Ultragenyx is a commercial-stage rare-disease biotech with a portfolio of approved therapies for ultra-rare genetic disorders. As a comparable rare-disease commercializer with global infrastructure, it represents both a peer in commercial-stage rare-disease execution and a potential strategic-acquirer profile for Trappsol Cyclo.
• Mallinckrodt Pharmaceuticals: Mallinckrodt (via its acquired Actelion portfolio) markets Zavesca (miglustat), an approved substrate-reduction therapy for NPC. As a large specialty pharmaceutical company with established rare-disease commercialization infrastructure, Mallinckrodt represents both an incumbent NPC competitor and a potential commercialization template for Cyclo's Trappsol Cyclo.
• Sage Therapeutics (a Biogen company): Sage Therapeutics, now part of Biogen, commercialized CNS therapies including Zurzuvae. As a CNS-focused pharmaceutical company with rare/neurological disease expertise and global commercial reach, Sage/Biogen is a relevant comparable for the longer-term commercial trajectory of an approved CNS specialty therapy like Trappsol Cyclo.

Emerging players

• Passage Bio: Passage Bio is a clinical-stage gene-therapy company focused on CNS disorders, including rare pediatric neurodegenerative conditions. While Passage uses a different modality (AAV gene therapy), the shared focus on rare pediatric CNS indications and the shared clinical-trial design considerations make it a relevant adjacent comparable.
• Intrabio: Intrabio is developing ibiglustat, a substrate-reduction therapy that has been evaluated in NPC1. As a smaller emerging player in the same NPC therapeutic space, Intrabio is directly comparable as another mechanism-of-action alternative to Trappsol Cyclo.
• Denali Therapeutics: Denali Therapeutics is a clinical-stage biotech developing therapies for neurodegenerative and lysosomal storage diseases, with an emphasis on BBB-penetrant therapeutics. The platform-overlap in CNS drug delivery and lysosomal biology makes Denali a relevant comparable for Cyclo's longer-term pipeline-expansion thesis.
• Sangamo Therapeutics: Sangamo develops CNS-directed genomic medicines, including programs for rare neurological diseases. While Sangamo's primary modality is zinc-finger gene regulation rather than small-molecule cyclodextrin, both companies operate in the rare-CNS therapeutic space and pursue similar regulatory pathways including Orphan Drug Designation.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/cyclodexpert
• Facebookfacebook.com/CycloTherapeutics
• LinkedInlinkedin.com/company/cyclotherapeutics

Compliance3 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles10 records

Funding overview

Funding rounds9 records

Investors1 record

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M&A1 record

Investments

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Cyclo Therapeutics