Avenue Therapeutics

What Avenue Therapeutics does

Avenue Therapeutics, Inc. (NASDAQ: ATXI) is a specialty pharmaceutical company founded in 2015 by Fortress Biotech and headquartered in Bay Harbor Islands, Florida. The company is focused on acquiring, in-licensing, developing, and commercializing therapies for rare and neurologic diseases with high unmet medical need. Its pipeline consists of four clinical-stage drug candidates: ATX-04 (clenbuterol), a selective β2-adrenergic agonist licensed from Duke University in February 2026 for Pompe disease with inherited FDA orphan drug designation; IV Tramadol, an intravenous opioid alternative in development for acute pain; AJ201, a Phase 1b/2a asset for spinal and bulbar muscular atrophy (Kennedy's disease); and BAER-101 (now AXS-17), a GABA A α2,3 subtype-selective positive allosteric modulator originally licensed from AstraZeneca and divested to Axsome Therapeutics in November 2025 for up to approximately $82 million in milestones and royalties.

The underlying technology is small-molecule pharmacology rather than a computational or AI-driven platform. Avenue acquires or licenses compounds with prior human proof-of-concept data to reduce development risk — for example, ATX-04 has clinical evidence of improved six-minute walk distance, respiratory muscle strength, and reduced glycogen burden in Pompe disease patients on enzyme replacement therapy, and BAER-101 was originally advanced by AstraZeneca with established safety and efficacy data in CNS indications.

The business model is asset-flip and royalty-oriented rather than vertically integrated commercialization. Avenue has no commercial products, no sales force, and no distribution channels; revenue is expected to come from future product sales, potential milestone payments and royalties (notably the up-to-$82M Axsome arrangement), and continued capital raises through small public offerings. The company operates with 1–10 employees and outsources much of its execution via parent Fortress Biotech, lead underwriter Maxim Group, and academic licensors such as Duke University.

Name

Avenue Therapeutics

Legal name

Avenue Therapeutics, Inc.

Website

https://avenuetx.com

Company type

Public

Founded year

2015

Operating status

Operating

Headcount range

1–10 employees

Short description

Avenue Therapeutics is a pre-revenue specialty pharmaceutical company developing small-molecule therapies for rare and neurologic diseases, including ATX-04 for Pompe disease, IV Tramadol for acute pain, and AJ201 for Kennedy's disease, operating under Fortress Biotech with assets in-licensed from Duke University and AstraZeneca.

Ownership category

akta.pro rank

Product category

Specialty Pharmaceuticals

NAICS

Pharmaceutical and Medicine Manufacturing (3254), Research and Development in the Physical, Engineering, and Life Sciences (54171)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Rare Endocrine & Growth Disorder Therapies (HLAIAIAJ)

akta.pro secondary industry

Pain Management & Anesthesiology Pharmaceuticals (HLAIAAAN)

Keywords

• Specialty pharmaceuticals
• Rare disease therapeutics
• Neurologic disease drugs
• Clinical-stage drug development
• Orphan drug development

Headquarters

HQ city

New York

HQ country

United States

HQ region

North America

Offices2 records

Markets served

GTM type

B2C

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations

Revenue model

1. Pharmaceutical Product Sales: The company is pre-revenue with no commercialized products. Revenue will be generated from sales of approved drug candidates upon successful FDA approval and commercialization.

Go-to-market motion1 record

Marketing channels3 records

Core offering

Avenue Therapeutics is a specialty pharmaceutical company that acquires/in-licenses, develops, and plans to commercialize therapies for rare and neurologic diseases. Its pipeline includes ATX-04 (clenbuterol, a selective β2-adrenergic agonist for Pompe disease), IV Tramadol (intravenous opioid alternative for acute pain), and AJ201 (for spinal and bulbar muscular atrophy). The company operates in clinical development with no commercialized products yet.

Product overview

Avenue Therapeutics is a specialty pharmaceutical company developing a portfolio of therapies for rare and neurologic diseases. The company's pipeline consists of four drug candidates: ATX-04 (a selective β2-adrenergic agonist for Pompe disease, acquired via exclusive license from Duke University in 2026), IV Tramadol (an intravenous opioid alternative for acute pain), AJ201 (in Phase 1b/2a for spinal and bulbar muscular atrophy), and BAER-101/AXS-17 (a GABA receptor modulator for epilepsy, which was divested to Axsome Therapeutics in 2025). The company was founded by Fortress Biotech and operates as a OTC-listed entity (ATXI).

Differentiator

Problem solved

Functional benefit

Products and services

• ATX-04 (clenbuterol) A selective β2-adrenergic agonist in clinical development for the treatment of Pompe disease. It is an orally administered small-molecule drug with established anabolic effects on skeletal muscle and the ability to enhance lysosomal biogenesis and intracellular trafficking of GAA enzyme, reducing glycogen accumulation in muscle tissue. Licensed exclusively worldwide from Duke University.
• IV Tramadol Intravenous tramadol being developed as an alternative to opioids for acute pain management in the United States.
• AJ201 Drug candidate in Phase 1b/2a clinical trial for the treatment of spinal and bulbar muscular atrophy (Kennedy's disease).

Quantifiable outcome

• Improvements in six-minute walk distance, increased respiratory muscle strength, and reduced muscle glycogen burden in Pompe disease patients treated with ATX-04

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

Two partnerships are on record, tiered core and major.

• Duke UniversitycoreTechnology or Integration · 23 February 2026Avenue Therapeutics entered into an exclusive worldwide license agreement with Duke University for patents and know-how pertaining to ATX-04 (clenbuterol) for the treatment of Pompe disease. Duke granted exclusive rights to develop and commercialize products covered by their patents, and Avenue assumed control of Duke's existing clinical and regulatory assets including the IND application and FDA orphan drug designation.
• Axsome TherapeuticsmajorStrategic or Co-development Partner · 6 November 2025Avenue's subsidiary Baergic Bio was acquired by Axsome Therapeutics for up to approximately $82 million in milestone payments and royalties. The acquisition included worldwide rights to BAER-101 (renamed AXS-17), a potential treatment for epilepsy.

Scale indicators5 records

Recent moves6 records

Expansion highlights5 records

Direct peers

• Amicus Therapeutics: Late-stage rare disease biotech with Pompe disease franchise (AT-GAA in pivotal trials). Directly comparable as a small-cap rare disease company pursuing Pompe disease therapies with novel mechanisms, overlapping clinical and commercial pathway with Avenue's ATX-04.
• Larimar Therapeutics: Clinical-stage rare disease biotech developing a treatment for Friedreich's ataxia, another rare neuromuscular disorder. Comparable to Avenue as a small-cap clinical-stage company focused on rare neurological diseases with high unmet need and orphan drug pathway.
• Marinus Pharmaceuticals: Small-cap neurology biotech developing CNS therapies including for epilepsy and rare seizure disorders. Comparable to Avenue as a similarly-sized, clinical-stage CNS drug developer pursuing orphan and specialty neurology indications.

Broad incumbents

• Axsome Therapeutics: CNS-focused commercial-stage biopharma that acquired Avenue's BAER-101 (now AXS-17) for up to $82M. Comparable as a neurology/CNS drug developer, validating that Avenue's CNS assets can attract established specialty pharma acquirers.
• Ultragenyx Pharmaceutical: Commercial-stage rare disease biopharma with multiple approved orphan drugs across metabolic and neuromuscular conditions. Comparable as an established rare disease player that has successfully scaled from clinical-stage to commercial, with Pompe-adjacent metabolic disease experience.
• BioMarin Pharmaceutical: Established rare disease biopharma with multiple commercialized orphan drugs across neuromuscular and metabolic conditions. Comparable as a broader incumbent in the rare and orphan disease space Avenue targets with Pompe and Kennedy's disease programs.
• Acadia Pharmaceuticals: Commercial-stage CNS-focused biopharma with rare disease neurology products. Comparable as a neurology specialty pharma that has scaled from clinical-stage to commercial-stage, relevant to Avenue's CNS pipeline trajectory.
• Sage Therapeutics: CNS-focused biopharma developing neurology and psychiatry therapies including GABA receptor modulators. Comparable to Avenue given the shared mechanism interest (BAER-101 is also a GABA modulator) and overlapping CNS therapeutic focus.
• Catalyst Pharmaceuticals: Commercial-stage rare disease specialty pharma focused on neurology indications including Lambert-Eaton myasthenic syndrome. Comparable as a small-cap rare disease company that has successfully commercialized orphan neurology drugs, representing a potential maturity path for Avenue.

Others

• Fortress Biotech: Avenue's parent company and incubator, which acquires and develops multiple specialty pharma subsidiaries. Comparable as an operating partner that provides shared infrastructure and capital access, and as a precedent for the in-licensing business model Avenue employs.

Market position

Strengths4 records

Weaknesses4 records

Competitive moat2 records

Key risks5 records

Key highlights5 records

Customer concentration

• Xx.com/avenuethera
• LinkedInlinkedin.com/company/avenue-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles4 records

Subsidiaries1 record

Funding overview

Funding rounds3 records

Investors

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

M&A1 record

Investments

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

Frequently asked questions about Avenue Therapeutics