What Rafael Holdings does

Rafael Holdings, Inc. (NYSE: RFL) is a U.S.-domiciled, clinical-stage biotechnology company developing therapeutics for difficult-to-treat diseases. Following its March 2025 merger with Cyclo Therapeutics, Rafael has refocused on Trappsol® Cyclo™, a hydroxypropyl-beta-cyclodextrin (HPBCD) formulation that has received FDA Breakthrough Therapy and Fast Track designations for Niemann-Pick Disease Type C1 (NPC), a rare, fatal neurodegenerative disease with no approved treatments. The pivotal Phase 3 TransportNPC study enrolled 94 patients across 27 sites in 13 countries, completed last patient last visit in mid-2026, and is positioned to support an NDA submission in the second half of calendar 2026. Rafael also retains CPI-613®, an oncology compound from its earlier 2021 merger with Rafael Pharmaceuticals, which had been in Phase 3 trials for pancreatic cancer and acute myeloid leukemia.

The company's commercial model is contingent on regulatory approval: it has negligible current revenue and operates at an annual net loss of approximately $30 million (FY2025 net loss attributable to Rafael of $30.5M; Q1 FY2026 net loss of $9.8M), funded primarily by a 2021 private placement of $104.2M and a 2025 rights offering of ~$25M backstopped by CEO Howard Jonas and affiliates. Distribution is intended to be built out as a direct, specialty rare-disease commercialization capability once Trappsol Cyclo is approved. Rafael further extended its intellectual property footprint via an April 2026 exclusive license from MIT (U.S. Patent No. 12285440) covering cyclodextrin use in Alzheimer's Disease patients carrying the ApoE4 variant — a population estimated at 50-70% of AD patients — opening a substantially larger second indication for the same drug platform.

Organizationally, Rafael runs with 11-50 employees from its Newark, NJ headquarters (520 Broad Street) under CEO Howard Jonas, with David Polinsky (CFO) and a small executive team covering development, medical, commercial, and compliance functions. The board lost Lead Independent Director Stephen Greenberg in October 2025 and added former Congressman Alan Grayson. The company is publicly traded on NYSE under RFL with global clinical footprint but no current product sales.

Name

Rafael Holdings

Legal name

Rafael Holdings, Inc.

Website

https://rafaelholdings.com

Company type

Public

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Rafael Holdings (NYSE: RFL) is a clinical-stage biotechnology company developing Trappsol Cyclo, a cyclodextrin-based therapy for Niemann-Pick Disease Type C1, with planned NDA submission in H2 2026 and pipeline expansion into ApoE4 Alzheimer's Disease via an MIT exclusive license.

Ownership category

akta.pro rank

Product category

Biopharmaceutical Drug Development

NAICS

Pharmaceutical and Medicine Manufacturing (32541), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

akta.pro secondary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

Keywords

• Clinical stage biotechnology
• Rare disease therapeutics
• Oncology drug development
• Cyclodextrin drug delivery
• Orphan disease therapies

Headquarters

HQ city

Newark

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Others

Revenue model

1. Pharmaceutical product development and commercialization: Pre-revenue clinical stage biotechnology company focused on developing and commercializing novel therapeutics for rare diseases and oncology. Revenue will be generated from drug product sales upon regulatory approval.

Distribution channels1 record

Marketing channels1 record

Core offering

Rafael Holdings is a clinical-stage biotechnology company developing novel drug therapies for rare and difficult-to-treat diseases. Following its merger with Cyclo Therapeutics, its lead asset is Trappsol Cyclo (hydroxypropyl-beta-cyclodextrin) for Niemann-Pick Disease Type C1, currently in Phase 3 trials with NDA submission planned for H2 2026. The company also retains legacy oncology asset CPI-613 for pancreatic cancer and AML, and holds an exclusive license from MIT for cyclodextrin use in ApoE4 Alzheimer's Disease.

Product overview

Rafael Holdings is a clinical-stage biotechnology company that operates as a holding company with a focused portfolio of pharmaceutical investments. Following its 2025 merger with Cyclo Therapeutics and its earlier merger with Rafael Pharmaceuticals, the company concentrates on developing novel drug therapies for difficult-to-treat diseases. The primary product is Trappsol® Cyclo™ for Niemann-Pick Disease Type C1, which completed its pivotal Phase 3 TransportNPC trial in 2026. The company also maintains legacy assets including CPI-613® for oncology indications (pancreatic cancer and AML).

Differentiator

Problem solved

Functional benefit

Products and services

• Trappsol Cyclo Investigational hydroxypropyl-beta-cyclodextrin (HPBCD) drug candidate for treating Niemann-Pick Disease Type C1, a rare, fatal genetic neurodegenerative disease. Completed pivotal Phase 3 TransportNPC study enrollment of 94 patients across 27 sites in 13 countries, with NDA submission planned for H2 2026. Also being developed for Alzheimer's Disease in ApoE4 carriers.
• CPI-613 Legacy oncology compound previously in Phase 3 clinical trials for pancreatic cancer and acute myeloid leukemia (AML), acquired through the 2021 merger with Rafael Pharmaceuticals.

Quantifiable outcome

• 80% of patients ages 0-3 showed CGI scores that improved or remained stable with no serious adverse events considered related to study drug

Segments2 records

Ideal customer profiles1 record

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Two partnerships are on record, tiered core.

• MIT (Massachusetts Institute of Technology)coreOEM/ Whitelabel/ Licensing Partner · 22 April 2026Exclusive licensing agreement 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 license strengthens Rafael Holdings' intellectual property portfolio around Trappsol Cyclo. ApoE4 is estimated to be present in 50-70% of Alzheimer's patients and is a key risk factor in early-onset sporadic AD.
• Cyclo TherapeuticscoreStrategic or Co-development Partner · 22 August 2024Definitive merger agreement combining Rafael Holdings and Cyclo Therapeutics to focus on developing Trappsol Cyclo for Niemann-Pick Disease Type C1. Rafael agreed to fund the ongoing TransportNPC Phase 3 clinical trial. The merger closed in 2025 following shareholder and regulatory approvals. Combined company brings together two innovative companies with mission to deliver breakthrough treatment for NPC patients.

Scale indicators8 records

Recent moves5 records

Expansion highlights5 records

Direct peers

• Ultragenyx Pharmaceutical: Clinical-stage rare disease biotech with multiple late-stage assets targeting ultra-rare genetic and metabolic disorders. Comparable in business model as a focused rare-disease developer building toward commercial launch, though larger and more diversified across several approved and pipeline products.
• BioMarin Pharmaceutical: Specialty biotech focused on rare genetic diseases, including CNS and metabolic disorders. Comparable commercial-stage rare-disease model with multiple approved therapies, offering a benchmark for the trajectory Rafael Holdings is pursuing as it transitions from clinical-stage to commercial.
• Amicus Therapeutics: Late-stage rare disease company focused on lysosomal storage disorders and genetically defined diseases, including Pompe disease and Fabry disease. Closely aligned therapeutic focus with Rafael's NPC program and similar pre-commercial clinical-to-commercial trajectory.
• Travere Therapeutics: Rare disease company developing therapies for serious genetic and metabolic conditions. Similar small-cap rare-disease business model with a focus on indications with high unmet need and orphan-drug pricing dynamics paralleling Rafael's NPC strategy.
• Mallinckrodt Pharmaceuticals: Specialty pharmaceutical company whose product Zavesca (miglustat) is currently the only approved therapy for NPC and represents direct competitive context for Trappsol Cyclo. Relevant peer because it sets the existing standard of care in NPC and benchmarks pricing and market dynamics.

Emerging players

• IntraBio: Clinical-stage rare disease company developing therapies for lysosomal storage disorders and neurodegenerative diseases. Comparable in therapeutic focus on rare neurological indications and similar clinical-stage profile, though smaller and earlier-stage than Rafael.

Broad incumbents

• Alexion (AstraZeneca Rare Disease): Established rare-disease franchise within AstraZeneca with multiple approved therapies for rare and ultra-rare conditions, including neurological disorders. Relevant as a benchmark for rare-disease commercial scaling, though much larger and operating at a different scale.
• Sarepta Therapeutics: Specialty biotech focused on rare neuromuscular diseases with multiple approved therapies. Relevant comparable for commercial-stage rare-disease operations in CNS-adjacent indications, though at a significantly larger scale than Rafael Holdings.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat3 records

Key risks6 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles8 records

Subsidiaries1 record

Funding overview

Funding rounds2 records

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A2 records

Investments3 records

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

Frequently asked questions about Rafael Holdings