Cyprium Therapeutics

What Cyprium Therapeutics does

Cyprium Therapeutics, Inc. is a private clinical-stage biopharmaceutical company founded in 2017 and headquartered in Bay Harbor Islands, Florida, operating as a majority-owned subsidiary of Fortress Biotech (NASDAQ: FBIO). The company focuses exclusively on developing novel therapies for Menkes disease and related copper metabolism disorders, an ultra-rare X-linked recessive pediatric genetic condition caused by mutations in the ATP7A copper transporter gene. Its core product ZYCUBO (copper histidinate, formerly CUTX-101) is a subcutaneous injectable copper replacement therapy that received FDA approval in January 2026 as the first and only approved treatment for Menkes disease in the United States. The company is also developing AAV-ATP7A, an adeno-associated virus-based gene therapy in preclinical stages intended to deliver functional copies of the ATP7A gene as a complementary treatment modality.

Cyprium does not commercialize products directly; commercialization of ZYCUBO is handled by Sentynl Therapeutics, a wholly-owned subsidiary of Zydus Lifesciences, pursuant to a 2021 Development and Asset Purchase Agreement and the December 2023 asset transfer. Under this arrangement, Cyprium retains development responsibility through NDA approval and is eligible for tiered royalties on ZYCUBO net sales (3% up to $75M, 8.75% between $75-100M, and 12.5% above $100M) plus up to $129 million in aggregate development and sales milestones. The company also received $4.5 million at closing of the asset transfer. Clinical evidence supporting the asset includes a nearly 80% reduction in mortality risk (HR=0.21, p<0.0001) and median overall survival of 177.1 months versus 17.6 months in untreated historical controls for early-treated patients.

The company's revenue mechanics also include the March 2026 sale of a Rare Pediatric Disease Priority Review Voucher (PRV) issued upon ZYCUBO's FDA approval, generating $205 million in gross proceeds, of which 20% is payable to NICHD/NIH per the underlying license. Cyprium has historically supplemented capital with non-dilutive grant funding, including a $4.1 million NINDS grant awarded in March 2024 to support AAV-ATP7A preclinical development through Nationwide Children's Hospital. The company partners with NICHD/NIH under a Cooperative Research and Development Agreement (CRADA) for AAV-ATP7A and is led by President and CEO Lung S. Yam, M.D., Ph.D., with Lindsay A. Rosenwald, M.D. (also Chairman, President and CEO of Fortress Biotech) serving as Chairman.

Name

Cyprium Therapeutics

Legal name

Cyprium Therapeutics, Inc.

Website

https://cypriumtx.com

Company type

Private

Founded year

2017

Operating status

Operating

Headcount range

11–50 employees

Short description

Cyprium Therapeutics is a private, Fortress Biotech majority-owned biotech developing copper replacement and gene therapies for Menkes disease and related copper metabolism disorders. Its lead product ZYCUBO is the first FDA-approved treatment for Menkes disease, commercialized by Sentynl Therapeutics, with tiered royalties and milestones flowing back to Cyprium.

Ownership category

akta.pro rank

Product category

Rare Pediatric Disease Therapeutics

akta.pro primary industry

Neurology & Psychiatry (CNS) Pharmaceuticals (HLAIAAAF)

Keywords

• Rare disease therapeutics
• Menkes disease treatment
• Copper replacement therapy
• Gene therapy development
• Biopharmaceutical licensing

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Services

Cost components

Technology or R&D, Personnel, Operations, Others

Revenue model

1. Royalties on ZYCUBO net sales: Cyprium receives tiered royalties on net sales of ZYCUBO: 3% of annual net sales up to $75 million; 8.75% of annual net sales between $75 million and $100 million; and 12.5% of annual net sales in excess of $100 million.
2. Development and sales milestones: Cyprium is eligible to receive up to $129 million in aggregate development and sales milestones from Sentynl Therapeutics.
3. Priority Review Voucher sale: Cyprium sold the Rare Pediatric Disease Priority Review Voucher (PRV) issued upon FDA approval of ZYCUBO for $205 million in gross proceeds, with 20% payable to NICHD/NIH.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels3 records

Core offering

Cyprium Therapeutics develops novel therapeutics for Menkes disease and related copper metabolism disorders. Its core offering is ZYCUBO (copper histidinate, formerly CUTX-101), the first and only FDA-approved copper replacement therapy for Menkes disease, developed in collaboration with NICHD/NIH and licensed to Sentynl Therapeutics for commercialization. The company is also advancing AAV-ATP7A gene therapy in preclinical development for use in combination with ZYCUBO.

Product overview

Cyprium Therapeutics is a biopharmaceutical company focused on developing novel therapies for the treatment of Menkes disease and related copper metabolism disorders. The company's core product portfolio consists of two key therapeutics: ZYCUBO (copper histidinate), which received FDA approval in January 2026 as the first and only approved treatment for Menkes disease, and AAV-ATP7A gene therapy, which is currently in preclinical development to be used in combination with ZYCUBO. The company operates as a majority-owned subsidiary of Fortress Biotech and partnered with Sentynl Therapeutics (a Zydus Lifesciences subsidiary) for the development and commercialization of ZYCUBO.

Differentiator

Problem solved

Functional benefit

Brands

• ZYCUBO: FDA-approved copper histidinate treatment for Menkes disease in pediatric patients (formerly known as CUTX-101). The first and only approved treatment for Menkes disease in the United States.

Products and services

• ZYCUBO (copper histidinate) ZYCUBO is the first and only FDA-approved, bioavailable copper replacement therapy for the treatment of Menkes disease, a rare X-linked recessive pediatric disease caused by mutations in the ATP7A copper transporter gene. It is a subcutaneous injectable formulation of copper histidinate that restores copper homeostasis and maintains copper levels in the normal age-appropriate range. Commercialization rights have been transferred to Sentynl Therapeutics, with Cyprium retaining tiered royalties and milestones.
• AAV-ATP7A Gene Therapy AAV-ATP7A is an adeno-associated virus (AAV)-based gene therapy in preclinical development for the treatment of Menkes disease. It is designed to deliver working copies of the defective copper transporter ATP7A gene and is intended to be used in combination with ZYCUBO copper histidinate therapy to enhance clinical outcomes. The program received FDA Orphan Drug Designation.

Quantifiable outcome

• Nearly 80% reduction in risk of death (HR=0.21, p<0.0001) for early-treated patients vs untreated historical control
• +1 more outcomes

Segments1 record

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

Five partnerships are on record, tiered core and minor.

• National Institute of Neurological Disorders and Stroke (NINDS)coreStrategic or Co-development Partner · 4 March 2024NINDS awarded a three-year grant totaling approximately $4.1 million to the Research Institute at Nationwide Children's Hospital and Principal Investigator Dr. Stephen G. Kaler for preclinical studies, manufacturing, and IND preparation for AAV-ATP7A gene therapy for Menkes disease.
• Sentynl TherapeuticscoreStrategic or Co-development Partner · 6 December 2023Sentynl Therapeutics (wholly-owned subsidiary of Zydus Lifesciences) acquired proprietary rights to CUTX-101/ZYCUBO from Cyprium. Sentynl assumed full development and commercialization responsibility in December 2023. Cyprium received $4.5 million at closing and is eligible for royalties (3-12.5% tiered) and up to $129 million in aggregate milestones. The PRV was transferred to Cyprium upon FDA approval.
• The Menkes FoundationminorStrategic or Co-development Partner · 29 July 2020The Menkes Foundation supported research on targeted next generation sequencing for newborn screening of Menkes disease, published in Molecular Genetics and Metabolism Reports.
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)coreStrategic or Co-development Partner · 1 March 2017Cyprium entered into a Cooperative Research and Development Agreement (CRADA) with NICHD/NIH in March 2017 to advance clinical development of CUTX-101. Also entered into a worldwide exclusive license agreement for AAV-based gene therapy (AAV-ATP7A). Cyprium must pay 20% of PRV sale proceeds to NICHD.
• Nationwide Children's HospitalcoreStrategic or Co-development Partner · 1 March 2017Dr. Stephen G. Kaler, a physician-scientist at the Center for Gene Therapy in the Abigail Wexner Research Institute at Nationwide Children's Hospital, serves as Principal Investigator for CUTX-101 and AAV-ATP7A clinical studies. The hospital received the NINDS grant for AAV-ATP7A preclinical development.

Scale indicators4 records

Recent moves9 records

Expansion highlights5 records

Direct peers

• Ultragenyx Pharmaceutical: Commercial-stage rare disease biotech with multiple approved products for genetic/metabolic disorders. Comparable as a small-cap rare disease company pursuing first-in-class therapies for ultra-rare pediatric conditions with similar regulatory exclusivity dynamics.
• Sentynl Therapeutics: Sentynl is Cyprium's commercialization partner for ZYCUBO and a wholly-owned subsidiary of Zydus Lifesciences focused on rare disease therapeutics. Directly comparable as a rare disease specialty commercializer with single-asset focus and similar regulatory/exclusivity dynamics.
• Sarepta Therapeutics: Gene therapy-focused biotech with approved AAV-based therapies for Duchenne muscular dystrophy. Comparable as a small rare disease company developing AAV gene therapy for pediatric genetic disorders, with similar regulatory pathway and orphan drug dynamics.
• BridgeBio Pharma: Clinical-to-commercial stage rare disease biotech focused on genetic diseases. Comparable as a development-stage company pursuing first-in-class therapies for rare disorders, often using partnership/out-licensing models similar to Cyprium's Sentynl deal.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biotech with a portfolio of approved enzyme replacement and gene therapies for genetic disorders. Comparable business model targeting ultra-rare genetic diseases with high regulatory barriers and orphan drug exclusivity.
• Alexion (AstraZeneca Rare Disease): Large rare disease franchise acquired by AstraZeneca, with multiple approved therapies for ultra-rare genetic and metabolic disorders. Comparable in target market (rare genetic disease patients) and use of orphan drug pathways.

Emerging players

• Ionis Pharmaceuticals: Antisense oligonucleotide platform biotech with multiple approved rare disease therapies including neurological indications. Comparable in targeting rare neurological/genetic disorders with novel modalities.
• uniQure: Gene therapy company with approved AAV-based therapy for hemophilia and pipeline in rare neurological disorders. Comparable as a small AAV gene therapy platform targeting rare diseases with high unmet need.
• Solid Biosciences: Clinical-stage gene therapy company developing AAV-based treatments for Duchenne muscular dystrophy and other rare genetic disorders. Comparable as a small AAV gene therapy developer in the rare disease space.

Others

• Fortress Biotech: Cyprium's parent company and majority shareholder, Fortress operates as a biopharmaceutical holding company with multiple subsidiary biotechs. Comparable operating model of multiple small rare disease programs housed under a holding structure.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat3 records

Key risks6 records

Key highlights6 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles1 record

Funding overview

Funding rounds2 records

Investors1 record

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M&A

Investments

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

Frequently asked questions about Cyprium Therapeutics