Beren Therapeutics

What Beren Therapeutics does

Beren Therapeutics P.B.C. is a clinical-stage biotechnology company organized as a Public Benefit Corporation under Delaware law, headquartered in Thousand Oaks, California, with additional offices in Boston, New Jersey, Budapest, and Tokyo. Founded in 2019 (per the company FAQ; CEO bio on the About page references 2020), the company is among the first biotechnology firms established as a PBC. Beren's core technology is a proprietary cyclodextrin-based therapeutics platform designed to restore intracellular cholesterol trafficking in diseases of defective cholesterol homeostasis, with its lead asset adrabetadex (VTS-270) being a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers administered intrathecally and currently under U.S. FDA Priority Review for infantile-onset Niemann-Pick disease type C (PDUFA target action date November 17, 2026).

Adrabetadex is the company's primary product and serves a single ultra-rare patient population: infants and children with infantile-onset NPC, a severe autosomal-recessive pediatric neurodegenerative disorder caused by pathogenic variants in NPC1 or NPC2 genes. Supporting customer segments include families and caregivers of patients, specialty healthcare providers (pediatric neurologists, metabolic disease specialists, and clinical geneticists), and the global NPC patient advocacy community. Beren is currently pre-revenue; adrabetadex is investigational and is provided to more than 60 patients through an Expanded Access Program across 13-16 sites in the U.S. and Canada at no charge. No commercial pricing has been disclosed.

The business model centers on a future specialty rare disease commercial launch in the U.S. via direct field sales to specialty treatment centers, supported by dedicated rare disease case management, a network of care sites, real-world evidence generation, and peer-to-peer community connectivity. A $55 million royalty financing arrangement with Hercules Capital carries a 7.5% royalty on U.S. net sales and 5% royalty on net sales outside the U.S. (up to a 1.75x cap through 2031), constituting a financing-cost obligation rather than inbound revenue. Long-term operations are supported by a $300 million combined financing closed in June 2026 (comprising $135 million in equity from Wellington Partners, JIC VGI, Founders Fund, Narya Capital, Eisai, and other institutional investors, plus up to $165 million in non-dilutive facility from Hercules Capital), and an innovation agreement with Roquette to expand the cyclodextrin platform beyond NPC.

Name

Beren Therapeutics

Legal name

Beren Therapeutics P.B.C.

Website

https://berentx.com

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

101–250 employees

Short description

Beren Therapeutics is a clinical-stage biotechnology company developing proprietary cyclodextrin-based therapeutics for ultra-rare pediatric neurodegenerative disease. Its lead asset adrabetadex is under FDA Priority Review for infantile-onset Niemann-Pick disease type C, targeting a small patient population with no approved disease-modifying therapy.

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Cardiometabolic Pharmaceuticals (Cardiovascular/Diabetes/Obesity) (HLAIAAAE)

akta.pro secondary industries

Regulatory Strategy & IND/NDA/BLA Submissions Support (HLAIALAH), Small-Molecule CMC Documentation & Regulatory Support (DMF/CMC) (HLAGAHAM)

Keywords

• Rare disease therapeutics
• Cyclodextrin-based medicines
• Niemann-pick disease treatment
• CNS drug development
• Pediatric neurology biopharmaceuticals

Headquarters

HQ city

Thousand Oaks

HQ country

United States

HQ region

North America

Offices5 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Future U.S. and global product sales of adrabetadex: Once approved by FDA (PDUFA target action date November 17, 2026), adrabetadex is expected to be commercialized in the U.S. as a first-in-class disease-modifying treatment for infantile-onset Niemann-Pick disease type C. Beren is preparing a U.S. specialty rare disease launch with global commercialization plans. Adrabetadex is currently provided to over 60 patients through an Expanded Access Program at no commercial revenue.
2. Royalty financing obligation on adrabetadex net sales: Per the $55 million royalty financing arrangement with Hercules Capital (funded upon FDA approval), Beren will pay a 7.5% royalty on U.S. net sales and a 5% royalty on net sales outside the U.S. of adrabetadex, up to a 1.75x cap through 2031, with the ability to redeem at a lower multiple in the first two years. This is a financing-cost obligation, not an inbound revenue stream.

Go-to-market motion3 records

Distribution channels2 records

Marketing channels9 records

Core offering

Beren Therapeutics is a clinical-stage biotechnology company developing adrabetadex (VTS-270), a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers administered intrathecally as a potential first-in-class disease-modifying treatment for infantile-onset Niemann-Pick disease type C. The company operates an Expanded Access Program for adrabetadex through its wholly-owned subsidiary Mandos LLC, currently supporting more than 60 patients across 13–16 specialty sites in the U.S. and Canada, while preparing for U.S. commercial launch pending FDA approval (PDUFA target action date November 17, 2026).

Product overview

Beren Therapeutics P.B.C. operates as a single integrated biopharmaceutical company built around one core product platform — its derivatized cyclodextrin chemistry — and one lead investigational asset, adrabetadex (VTS-270). The company is structured as a Public Benefit Corporation and pursues a fully integrated discovery-through-commercialization model for cyclodextrin-based therapeutics targeting diseases of defective intracellular cholesterol trafficking. Its commercial-stage product offering centers on adrabetadex, an intrathecal 2-hydroxypropyl-β-cyclodextrin mixture in FDA Priority Review for infantile-onset Niemann-Pick disease type C (PDUFA November 17, 2026). The adrabetadex program is administered through Beren's wholly-owned subsidiary Mandos Health (Mandos LLC), which serves as the externally-facing sponsor of the adrabetadex Expanded Access Program and the regulatory sponsor of record, while a growing pipeline of additional derivatized cyclodextrins is being advanced via Beren's internal drug platform. Complementary offerings include long-term patient access and family support programs (diagnosis acceleration, rare-disease case management, network of care sites, real-world evidence generation, and community connectivity) and a dedicated Medical Affairs portal at medical.berentx.com.

Differentiator

Problem solved

Functional benefit

Brands

• BEREN: Trademark of Beren Therapeutics P.B.C. and/or its subsidiary Mandos LLC.
• MANDOS
• MANDOS HEALTH

Products and services

• Adrabetadex (VTS-270) Adrabetadex is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers delivered intrathecally and in development as a potential first-in-class disease-modifying treatment for infantile-onset Niemann-Pick disease type C (I-NPC). It is designed to re-establish intracellular cholesterol trafficking by mobilizing cholesterol accumulated in the endolysosomal compartment. NDA accepted for Priority Review in February 2026 with PDUFA target action date of November 17, 2026; granted FDA Breakthrough Therapy Designation (December 2025), Orphan Drug Designation, and Rare Pediatric Disease Designation.
• Adrabetadex Expanded Access Program (via Mandos Health / Mandos LLC) The adrabetadex Expanded Access Program provides investigational intrathecal adrabetadex to eligible patients with infantile-onset Niemann-Pick disease type C at no charge through specialty treatment centers in the U.S. and Canada, with oversight by principal investigator Dr. Elizabeth Berry-Kravis at Rush University Medical Center. Operated by Beren's wholly-owned subsidiary Mandos Health (Mandos LLC), the EAP serves as the pre-commercial distribution channel for adrabetadex while the NDA is under FDA Priority Review.
• Long-Term Patient Access and Family Support Program for NPC Integrated patient access and family support offering for the global NPC community that combines accelerated genetic diagnostic testing, dedicated rare-disease case management, a network of specialty care sites close to patients' homes, real-world evidence generation, and peer-to-peer community connectivity. Designed to compress the multi-year NPC diagnostic odyssey and reduce family burden across the treatment journey in support of the planned adrabetadex U.S. specialty rare disease commercial launch.

Quantifiable outcome

• 71% reduction in mortality risk vs matched external controls (HR 0.289; 95% CI 0.141–0.593; P<0.0001) in infantile-onset NPC
• +4 more outcomes

Segments4 records

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature5 records

Partnerships

Seven partnerships are on record, tiered core, flagship and minor.

• Wilson Sonsini Goodrich & RosaticoreImplementation/ SI/ Consulting Partner · 10 June 2026Legal advisor to Beren Therapeutics on the combined $300 million financing transactions in June 2026.
• Latham & WatkinscoreImplementation/ SI/ Consulting Partner · 10 June 2026Legal advisor to Hercules Capital on the combined $300 million financing transactions in June 2026.
• Ara Parseghian Medical Research Fund (APMRF)coreOthers · 29 May 2026Annual scientific conference partnership at the Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research at the University of Notre Dame; Beren presents clinical and preclinical data on adrabetadex; CEO Jason Camm participates in fireside chats with APMRF director Sean Kassen.
• National Niemann-Pick Disease Foundation (NNPDF)coreOthers · 8 January 2026Co-hosted community webinars (e.g., January 8, 2026 NPC webinar), shared community communications, partnered on Rare Disease Day activations and NPC Awareness Month, sponsored NNPDF Family Support & Medical Conferences, and coordinated EAP communications.
• INPDR Gateway Ltd (International Niemann-Pick Disease Registry)coreStrategic or Co-development Partner · 1 June 2023Partnership granting access to INPDR's NPC patient registry data covering more than 300 NPC patients. The real-world data provides Beren/Mandos with evidence to accelerate research and analyses on adrabetadex, supporting regulatory submissions.
• RoquetteflagshipStrategic or Co-development Partner · 23 May 2023Strategic investment combined with an innovation agreement to expand the full potential of Beren's cyclodextrin technologies and their medicinal applications. Roquette is a leading provider of pharmaceutical excipients and global leader in plant-based ingredients, operating in more than 100 countries with turnover of ~€5 billion and 8,000+ employees. The collaboration covers chemistry, manufacturing, and controls (CMC) management, safety protocols, and regulatory registration.
• Janssen Pharmaceutical Companies of Johnson & JohnsonminorOthers · 30 November 2021Supported access to unused vials of adrabetadex to help Beren/Mandos build additional drug supply for the Expanded Access Program.

Scale indicators10 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• IntraBio: Clinical-stage rare disease company that has developed NPC therapies (including arimoclomol), with potential combination-therapy overlap with Beren. Direct competitor for the NPC patient population and scientific community.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease biopharma developing therapies for rare metabolic and neurologic diseases, including adjacent lysosomal storage disorders. Comparable specialty rare disease launch model and integrated access infrastructure approach.
• Orchard Therapeutics: Ultra-rare gene therapy company that launched LENMELDY for MLD. Comparable ultra-rare pediatric neurodegenerative launch model and Beren commercial leadership background.

Broad incumbents

• BioMarin Pharmaceutical: Established rare disease biopharma with multiple approved specialty therapies across metabolic and CNS indications. Comparable commercial model for ultra-rare pediatric diseases and pricing dynamics.
• Alexion (AstraZeneca Rare Disease): Global leader in rare disease commercial launches (SOLIRIS, ULTOMIRIS, STRENSIQ). Comparable ultra-rare specialty launch playbook and access infrastructure; key source of Beren's commercial leadership talent.
• Jazz Pharmaceuticals: Specialty biopharma with deep rare neurology franchise. Comparable specialty rare disease commercial footprint and patient access infrastructure investments.
• Recordati Rare Diseases: Specialty rare disease unit of Recordati focused on metabolic, endocrine, and CNS rare diseases. Comparable ultra-rare disease distribution and case-management model.
• Takeda Pharmaceutical (Rare Diseases): Global biopharma with a substantial rare disease and lysosomal storage disorder franchise (e.g., Replagal, Adynovate). Comparable footprint in NPC-adjacent rare metabolic disease markets.

Others

• Mallinckrodt Pharmaceuticals: Original developer of adrabetadex (VTS-270); Beren acquired the asset from Mallinckrodt following its bankruptcy. Historically comparable specialty rare disease portfolio though now in restructuring.
• Roquette: Global leader in pharmaceutical excipients and cyclodextrin chemistry; strategic investor and innovation partner of Beren. Comparable cyclodextrin technology expertise and CMC infrastructure.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat6 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/berentx

Compliance2 records

Revenue estimate

Valuation estimate

Number of profiles

Profiles11 records

Subsidiaries1 record

Funding overview

Funding rounds3 records

Investors8 records

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

M&A

Investments

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

Frequently asked questions about Beren Therapeutics