What IntraBio does

IntraBio is a biopharmaceutical company with a late-stage drug pipeline that includes novel treatments for genetic and neurodegenerative diseases. Their clinical programs leverage the expertise of their scientific founders from the University of Oxford and University of Munich, the preeminent experts and pioneers in discovering and developing small molecule drugs that modulate lysosomal function and intracellular calcium signaling. Their management team and business consultants have vast commercial and regulatory experience in drug product development, including all stages from small molecule manufacturing, clinical studies, and regulatory approvals in the USA and Europe. With their successful track record of drug development and commercialization, IntraBio’s team translates research in the fields of lysosomal biology, autophagy, and neurology into orphan drugs and treatments that will significantly improve the lives of patients, their caregivers, and families.

Name

IntraBio

Legal name

IntraBio Inc

Website

http://intrabio.com/

Company type

Private

Founded year

2015

Operating status

Operating

Headcount range

1–10 employees

Short description

IntraBio is a biopharmaceutical company.

Ownership category

akta.pro rank

Product category

Rare Disease Therapeutics

NAICS

Biological Product (except Diagnostic) Manufacturing (325414)

SIC

Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Rare Neurology & Neurodegenerative Disorder Therapies (HLAIAIAD)

akta.pro secondary industries

Rare Pediatric & Congenital Disorder Therapies (HLAIAIAN), Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

Keywords

• Rare disease therapeutics
• Lysosomal storage disorders
• Neurodegenerative disease treatments
• Orphan drug development
• Specialty pharmaceuticals

Headquarters

HQ city

Austin

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, Infrastructure

Revenue model

1. AQNEURSA Product Sales: IntraBio generates revenue through the sale of AQNEURSA (levacetylleucine), an FDA-approved prescription drug for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15 kg. The company also received European Commission approval for AQNEURSA in the EU for adults and children aged 6 years and older weighing at least 20 kg.

Pricing tiers

Go-to-market motion1 record

Distribution channels3 records

Marketing channels4 records

Core offering

IntraBio develops and commercializes AQNEURSA (levacetylleucine/IB1001), an FDA-approved orally administered modified amino acid therapy for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients. The company also pursues clinical development of the same active compound for Ataxia-Telangiectasia, GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease), and CACNA1A-related disorders, supported by AQNEURSA Cares patient financial assistance and an Expanded Access Program.

Product overview

IntraBio is a biopharmaceutical company focused on discovering, developing, and commercializing therapies for rare neurodegenerative diseases. The company's lead product is AQNEURSA (levacetylleucine), which received FDA approval in September 2024 and European Commission approval in January 2026 for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC). The investigational drug candidate IB1001 (N-acetyl-L-leucine/levacetylleucine) is the active ingredient in AQNEURSA and is also being developed for additional indications including Ataxia-Telangiectasia, GM2 Gangliosidosis, and CACNA1A-related disorders. The company operates an Expanded Access Program and AQNEURSA Cares patient support services.

Differentiator

Problem solved

Functional benefit

Products and services

• AQNEURSA (levacetylleucine) AQNEURSA is an FDA-approved and European Commission-approved orally administered, modified amino acid (N-Acetyl-L-Leucine) prescription therapy for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC). It is approved for adults and pediatric patients weighing ≥15 kg in the US and adults and children aged 6 and older weighing at least 20 kg in the EU, with use as monotherapy or in combination with miglustat. The drug modulates lysosomal function and intracellular calcium signaling through bidirectional TFEB modulation and provides significant improvement in neurological symptoms and functioning within 12 weeks.

Quantifiable outcome

• 118% reduction in annual disease progression after 1 year of treatment as measured by 5-domain NPC Clinical Severity Scale
• +2 more outcomes

Named customers1 record

Segments2 records

Ideal customer profiles1 record

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Three partnerships are on record, tiered core.

• EYcoreImplementation/ SI/ Consulting Partner · 16 September 2024IntraBio engaged EY to implement integrated finance managed services including SAP S/4HANA Cloud Private Edition platform, centralizing treasury, tax planning, procurement, and payments systems. EY estimates its solution reduced IntraBio's projected operating costs by nearly a third while positioning the company for efficient international growth across Europe, the Middle East, and Asia, and accelerating its IPO readiness.
• University of OxfordcoreStrategic or Co-development PartnerIntraBio's clinical programs leverage the expertise in lysosomal function and intracellular signaling of its scientific founders from the University of Oxford, including Professor Frances Platt and Professor Antony Galione. The company's platform technology results from decades of research at Oxford.
• University of Munich (Ludwig Maximilians University)coreStrategic or Co-development PartnerIntraBio's clinical programs leverage the expertise of its scientific founder Professor Michael Strupp from the University of Munich's Department of Neurology. Multiple clinical trials have been conducted with principal investigators from LMU Munich.

Scale indicators7 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• BioMarin Pharmaceutical: BioMarin is a global rare disease biopharmaceutical company with multiple approved enzyme replacement and gene therapies for ultra-rare conditions. It is directly comparable as a commercial-stage rare disease specialist with FDA-approved products and orphan drug designations across multiple indications.
• Ultragenyx Pharmaceutical: Ultragenyx is a commercial-stage rare disease company developing and commercializing therapies for rare and ultra-rare genetic diseases, including metabolic and neurological disorders. Comparable to IntraBio in business model (single platform applied across multiple rare indications) and orphan drug strategy.
• Catalyst Pharmaceuticals: Catalyst Pharmaceuticals is a commercial-stage specialty pharma focused on rare neurological diseases, with FDA-approved therapies and a similar business model of focusing on ultra-rare conditions with high unmet medical need. Directly comparable in business model and patient population profile.
• Travere Therapeutics: Travere Therapeutics is a commercial-stage rare disease company developing therapies for rare neurological and metabolic disorders. Comparable as a similarly sized rare disease biopharma with FDA-approved products and a pipeline of orphan drug candidates.
• Cyclo Therapeutics: Cyclo Therapeutics is a clinical-stage biopharmaceutical company developing treatments for Niemann-Pick disease type C and other rare diseases. Directly comparable as a competitor specifically focused on NPC and adjacent rare neurodegenerative indications.

Broad incumbents

• Alexion (AstraZeneca Rare Disease): Alexion is a major rare disease franchise within AstraZeneca with multiple approved therapies for rare neurological and metabolic conditions. Comparable as a broader incumbent in the rare disease specialty pharma space with established commercial infrastructure for ultra-rare diseases.
• Recordati Rare Diseases: Recordati operates a dedicated rare diseases business unit focused on metabolic and neurological rare conditions, with established US and EU commercial infrastructure. Comparable as a broader incumbent rare disease specialty pharma with multi-region commercialization capabilities.
• Swedish Orphan Biovitrum (Sobi): Sobi is a European-based specialty biopharma focused on rare diseases with a portfolio spanning immunology, genetics, and specialty care. Comparable as a broader incumbent with established rare disease commercialization infrastructure in both US and EU markets.

Emerging players

• Denali Therapeutics: Denali Therapeutics is developing therapies for neurodegenerative diseases including lysosomal storage disorders, with a focus on crossing the blood-brain barrier. Comparable as an emerging player with overlapping therapeutic focus on neurodegenerative diseases and lysosomal biology.
• Homology Medicines: Homology Medicines is a genetic medicines company developing gene therapies for rare diseases including neurological and metabolic disorders. Comparable as an emerging player targeting the same lysosomal storage disorder space with a different therapeutic modality.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Funding overview

Funding rounds2 records

Investors

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 IntraBio