What Augustine Therapeutics does

Augustine Therapeutics is a clinical-stage biopharmaceutical company founded in 2019 as a spin-off from VIB and KU Leuven, headquartered in Leuven, Belgium with a wholly-owned Copenhagen research subsidiary (Augustine Therapeutics Denmark ApS) established in May 2025. The company develops a portfolio of next-generation selective HDAC6 inhibitors built on a proprietary non-hydroxamate, non-hydrazide producing chemotype designed to overcome the specificity limitations, off-target toxicity, and poor drug-like properties of first-generation hydroxamate- and oxadiazole-based HDAC6 inhibitors. Its pipeline targets three therapeutic verticals with high unmet need: neuromuscular diseases led by AGT-100216 in Phase I clinical trials for Charcot-Marie-Tooth disease (a hereditary neuropathy affecting more than 2.5 million people worldwide with no approved disease-modifying treatments); neurodegenerative diseases via brain-penetrant HDAC6 candidates in lead optimization for ALS and other CNS indications; and cardio-metabolic diseases via a second-generation peripherally-restricted program in advanced lead optimization for conditions including heart failure with preserved ejection fraction and atrial fibrillation.

The company's technology platform is grounded in research from Prof. Ludo Van Den Bosch at the VIB-KU Leuven Center for Brain & Disease Research and includes both peripherally-restricted and brain-penetrant selective HDAC6 chemotypes protected by pending patent filings on thiomethyl carbonyl HDAC6/HDAC1/HDAC3 inhibitors. Augustine has built a senior leadership team with extensive big-pharma and biotech experience across Sanofi, Pfizer, Lundbeck, Genzyme, AbbVie, AstraZeneca, Zealand Pharma, and Arkuda Therapeutics, and has constructed a multi-asset pipeline spanning IND-enabling, lead optimization, and clinical stages.

The business model is a traditional pharmaceutical development and commercialization path: Augustine is pre-revenue with no approved products and no disclosed pricing, plans to commercialize through standard pharmaceutical distribution (prescribing physicians, hospital and specialty pharmacies) upon regulatory approval, and acknowledges potential future partnerships with larger pharmaceutical companies for global commercialization. Current funding is sourced from venture capital (cumulative Series A funding of approximately EUR 94.7M led by Novo Holdings, Jeito Capital, Asabys Partners, and Eli Lilly) plus non-dilutive grants from the US ALS Association and the Flanders Agency for Innovation and Entrepreneurship (VLAIO).

Name

Augustine Therapeutics

Legal name

Augustine Therapeutics NV

Website

https://augustinetx.com

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

11–50 employees

Short description

Augustine Therapeutics is a clinical-stage Belgian biopharmaceutical company developing a proprietary portfolio of next-generation selective HDAC6 inhibitors for neuromuscular, neurodegenerative, and cardio-metabolic diseases with high unmet need. The company is currently advancing lead candidate AGT-100216 in Phase I trials for Charcot-Marie-Tooth disease.

Ownership category

akta.pro rank

Product category

Biopharmaceuticals / HDAC6 Inhibitor Therapeutics

NAICS

Research and Development in Biotechnology (except Nanobiotechnology) (541714), Scientific Research and Development Services (5417)

SIC

Pharmaceutical Preparations (2834)

akta.pro primary industry

Genetic & Genomic Rare Disease Therapeutics (HLAIAIAA)

akta.pro secondary industries

Cardiometabolic Pharmaceuticals (Cardiovascular/Diabetes/Obesity) (HLAIAAAE), Rare Cardiovascular & Vascular Disorder Therapies (HLAIAIAH), Target Identification & Validation Platforms (omics-driven, chemoproteomics, genetic validation) (HLAAAIAI), Gene Therapy CMC/Process Development Platforms (capsid engineering, producer cell lines, plasmids, upstream/downstream) (HLAAACAN)

Keywords

• HDAC6 inhibitors
• Small molecule therapeutics
• Neuromuscular disease drugs
• Neurodegenerative disease therapeutics
• Cardiometabolic drug development

Headquarters

HQ city

Leuven

HQ country

Belgium

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Operations

Revenue model

1. Pharmaceutical drug development and commercialization: Biopharmaceutical company developing small molecule HDAC6 inhibitors through clinical development toward regulatory approval and commercialization. Revenue will be generated through product sales upon regulatory approval. The company is pre-revenue as a clinical-stage biotech, with no approved products yet.

Go-to-market motion1 record

Distribution channels2 records

Marketing channels5 records

Core offering

Augustine Therapeutics discovers and develops a portfolio of small-molecule selective HDAC6 inhibitors for chronic diseases. Its lead clinical asset AGT-100216 is a peripherally-restricted selective HDAC6 inhibitor currently in Phase I trials for Charcot-Marie-Tooth disease, with additional pipeline programs in IND-enabling studies (AGT-100XXX), advanced lead optimization (peripheral 2nd-generation HDAC6i for cardiometabolic indications), and lead optimization (brain-penetrant HDAC6i for ALS and other neurodegenerative diseases). All candidates are based on the company's proprietary non-hydroxamate, non-hydrazide chemotype designed to avoid the specificity and toxicity issues of earlier HDAC6 inhibitor generations.

Product overview

Augustine Therapeutics is a biotechnology company pioneering a new generation of HDAC6 (Histone DeACetylase 6) inhibitors for the treatment of neuromuscular, neurodegenerative, and cardiometabolic diseases. The company's pipeline includes multiple programs: lead candidate AGT-100216 (Phase I clinical stage for Charcot-Marie-Tooth disease), AGT-100XXX (IND-enabling stage), second-generation peripherally-restricted HDAC6 inhibitors for cardiometabolic diseases (advanced lead optimization), and central acting/brain-penetrant HDAC6 inhibitors for neurodegenerative diseases like ALS (lead optimization). All candidates are based on Augustine's novel non-hydroxamate, non-hydrazide producing chemotype designed to achieve selective HDAC6 inhibition while preserving beneficial non-catalytic functions.

Differentiator

Problem solved

Functional benefit

Products and services

• AGT-100216 Peripherally-restricted, selective HDAC6 inhibitor lead candidate in Phase I clinical development for Charcot-Marie-Tooth disease; first selective HDAC6 inhibitor from Augustine's pipeline to enter clinical trials.
• AGT-100XXX Peripherally-restricted, selective HDAC6 inhibitor in IND-enabling studies under evaluation for potential clinical studies in several neuromuscular and cardiometabolic indications.
• Peripheral HDAC6i (2nd Generation) Second-generation peripherally-restricted HDAC6 inhibitor in advanced lead optimization for cardiometabolic diseases, designed using Augustine's non-hydroxamate, non-hydrazide chemotype.
• Central Acting HDAC6i Brain-penetrant HDAC6 inhibitor in lead optimization for neurodegenerative diseases such as ALS, designed using Augustine's non-hydroxamate, non-hydrazide chemotype to cross the blood-brain barrier and act on CNS targets.

Quantifiable outcome

• AGT-100216 demonstrated impressive preclinical efficacy in CMT studies, showing ability to halt disease progression, significantly reverse disease phenotype, and rescue axonal integrity in a dose-dependent manner
• +3 more outcomes

Named customers3 records

Segments3 records

Ideal customer profiles3 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature4 records

Partnerships

Four partnerships are on record, tiered core partner.

• Leading European Academic Partners (via VLAIO grant)core partnerStrategic or Co-development Partner · 12 May 2026VLAIO grant project features collaboration with leading European partners (specific partners not named) to broaden insights into HDAC6 biology across different organs and tissues, guiding future clinical development of Augustine's portfolio of next-generation selective HDAC6i molecules.
• US ALS Associationcore partnerOthers · 27 February 2024The US ALS Association awarded a USD 300K Lawrence and Isabel Barnett Drug Development Program grant to support preclinical development of brain-penetrant HDAC6 inhibitors for ALS. The association also provides strategic guidance and patient advocacy support. Augustine collaborates with the VIB-KU Leuven Center for Brain & Disease Research for ALS studies supported by this grant.
• VIB-KU Leuven Center for Brain & Disease Researchcore partnerStrategic or Co-development Partner · 27 February 2024Augustine collaborates with the VIB-KU Leuven Center for Brain & Disease Research on ALS research, using human induced pluripotent stem cells (hiPSCs) and mouse models. The collaboration involves Prof. Ludo Van Den Bosch (founding scientist) and Prof. Philip Van Damme (University Hospital Leuven, LEUCALS). The scientific foundation of Augustine originates from this research center.
• University Hospital Leuvencore partnerStrategic or Co-development PartnerCollaboration with Professor Philip Van Damme from the University Hospital in Leuven (Leuven Center for ALS - LEUCALS) for ALS research using in vitro and in vivo models as part of the ALS Association-funded project.

Scale indicators9 records

Recent moves8 records

Expansion highlights6 records

Direct peers

• Edgewise Therapeutics: Clinical-stage biotech developing therapies for muscle disorders including CMT (EDG-5506 in Phase II) and Duchenne muscular dystrophy. Edgewise is a direct neuromuscular disease peer with overlapping patient populations to Augustine's peripheral programs.
• Pharnext: Late-stage French biotech developing PXT-3003, the most advanced therapeutic candidate in clinical development for Charcot-Marie-Tooth disease. Pharnext is the closest direct competitor to Augustine in the CMT indication, although it uses a different mechanism (combination of approved drugs).
• Regenacy Pharmaceuticals: Clinical-stage biotech developing selective HDAC6 inhibitors for Charcot-Marie-Tooth disease and other peripheral neuropathies. Regenacy's lead program (ricolinostat) is the most direct comparator to Augustine's AGT-100216, targeting the same HDAC6 mechanism for CMT.

Broad incumbents

• Cytokinetics: Late-stage cardiovascular and neuromuscular specialist with cardiac myosin modulator (omecamtiv mecarbil) in development for heart failure and neuromuscular programs in ALS and SMA. Cytokinetics is a broad incumbent directly comparable to Augustine's dual neuromuscular/cardiometabolic positioning.
• Biogen: Major neurodegenerative disease player with established ALS franchise (Qalsody for SOD1-ALS) and ongoing CMT programs. Biogen is a broad incumbent overlapping Augustine's CNS (ALS, AD, PD) and rare neuromuscular disease programs.
• PTC Therapeutics: Established commercial-stage rare disease company with multiple neuromuscular franchises including Duchenne muscular dystrophy (Translarna, Emflaza) and gene therapy programs for CNS disorders. PTC is a broad incumbent in neuromuscular disease that may compete with or partner for Augustine's indications.
• Ionis Pharmaceuticals: Large antisense oligonucleotide platform company with multiple approved and clinical-stage programs for neuromuscular and neurodegenerative diseases (e.g., Spinraza for SMA, tofersen for SOD1-ALS). Ionis is a broad incumbent in the antisense/RNA space overlapping Augustine's ALS and CMT indications.

Emerging players

• Tenaya Therapeutics: Clinical-stage genetic medicines company focused on heart failure and cardiomyopathies with gene therapy and small molecule programs. Tenaya is an emerging player in cardiovascular disease that overlaps with Augustine's HFpEF and atrial fibrillation programs.
• Helixmith: South Korean clinical-stage biotech developing Engensis (VM202), a gene therapy for diabetic peripheral neuropathy and ALS. Helixmith is an emerging peer in the peripheral neuropathy and motor neuron disease space where Augustine operates.
• Acetylon Pharmaceuticals: Early-stage HDAC6-focused biotech acquired by Celgene in 2013 and now operating within Bristol Myers Squibb's pipeline. Acetylon represents historical HDAC6 drug development efforts and a relevant comparator for Augustine's selective HDAC6 inhibitor approach.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/augustine-therapeutics

Revenue estimate

Valuation estimate

Number of profiles

Profiles7 records

Subsidiaries1 record

Funding overview

Funding rounds3 records

Investors11 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 Augustine Therapeutics