What Axonis Therapeutics does

Axonis Therapeutics is a Boston-based, clinical-stage biotechnology company developing first- and best-in-class oral small-molecule medicines that target KCC2 (potassium-chloride co-transporter 2) to restore physiological GABA-mediated inhibition in the central nervous system. Its lead asset, AXN-027, is a first-in-class oral KCC2 potentiator currently in a Phase 1 clinical trial for refractory epilepsy, with planned expansion to chronic pain and spinal cord injury, and a next-generation KCC2 potentiator portfolio targeting psychiatric and neurodevelopmental disorders (including SYNGAP1). The platform is anchored in IP spun out of Boston Children's Hospital, Harvard Medical School, and Laval University, and is built around a proprietary KCC2 Discovery Engine that integrates unbiased in vivo CNS phenotypic screening — including a genome-wide AAV-CRISPR screen co-developed by co-founder Shane Hegarty in Prof. Zhigang He's lab — with customized KCC2-targeted assays built alongside world-leading KCC2 biology experts.

The business is pre-revenue and capital-intensive. Axonis has raised $34M+ in dilutive Seed capital and an oversubscribed $115M Series A in October 2024 (co-led by Cormorant Asset Management and venBio Partners, with a 15-investor syndicate including Sofinnova Investments, MRL Ventures Fund, Perceptive Advisors, Lumira Ventures, Solasta Ventures, SCI Ventures, and Alexandria Venture Investments), bringing cumulative funding to $157M+ since 2019 under CEO Joanna Stanicka. Non-dilutive support has come from NIH, DoD SCIRP, CURE Epilepsy, Wings for Life, SynGAP Research Fund, Praxis Spinal Cord Institute, and others. The intended future commercial model is a specialty CNS pharma field-sales motion targeting epileptologists, neurologists, pain specialists, and major academic medical centers, with anticipated revenue from product sales of AXN-027 and follow-on KCC2 potentiators (and potentially licensing/royalties from partnerships).

The company is led by a mix of scientific co-founders (Joanna Stanicka, Ph.D., CEO/President; Shane V. Hegarty, Ph.D., CSO; Lisa McKerracher, Ph.D., Scientific Advisor) and biotech operator/board leadership (Corey Goodman, Ph.D., Executive Chairman and venBio Managing Partner; Bob Yant, Co-Founder and Board Observer; Marcelo Bigal, M.D., Ph.D., Chief Medical Advisor), with a world-class Scientific Advisory Board chaired by Yves De Koninck (Laval) and including Zhigang He, Clifford Woolf, Jacqueline French, Roger Porter, James Guest, and Wolfgang Liedtke. In October 2024, Axonis added Donald Manning, MD, Ph.D., as Chief Medical Officer and Jeff Imbaro, M.B.A., as Chief Operating Officer to build the clinical and operating muscle required to advance AXN-027 through clinical proof-of-concept and beyond.

Name

Axonis Therapeutics

Legal name

Axonis Therapeutics, Inc.

Website

https://axonis.us

Company type

Private

Founded year

2019

Operating status

Operating

Headcount range

11–50 employees

Short description

Axonis Therapeutics is a Boston-based, clinical-stage biotechnology company developing first-in-class oral small-molecule KCC2 potentiators for refractory epilepsy, chronic pain, and spinal cord injury, with AXN-027 in Phase 1 trials and $157M+ in cumulative funding.

Ownership category

akta.pro rank

Product category

CNS Therapeutics

NAICS

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

SIC

Pharmaceutical Preparations (2834), Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Neurology & CNS Specialty Pharmaceuticals (HLAIACAC)

akta.pro secondary industry

Neural & Spinal Cord Regeneration (HLAAAGAI)

Keywords

• Small molecule therapeutics
• CNS drug discovery
• KCC2 potentiator
• Neurology drug development
• Clinical-stage biotechnology

Headquarters

HQ city

Boston

HQ country

United States

HQ region

North America

Offices3 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pre-revenue / clinical-stage R&D funding: Axonis is a clinical-stage biotechnology company that has not yet commercialized a product and currently generates no product revenue. Capital to date has come from dilutive private financing (Seed and Series A) and non-dilutive grants/awards; future revenue is expected to come from commercialization of AXN-027 and follow-on KCC2 potentiators, either independently or via partnerships/out-licensing.
2. Future drug product sales (planned): Upon regulatory approval, Axonis intends to generate revenue from sales of first-in-class oral KCC2 potentiator medicines (starting with AXN-027) for refractory epilepsy, chronic pain, spinal cord injury and additional neurological indications.

Go-to-market motion2 records

Distribution channels1 record

Marketing channels5 records

Core offering

Axonis Therapeutics is a clinical-stage biotechnology company developing first-in-class oral small-molecule KCC2 potentiator drugs that restore functional inhibition within the brain to treat refractory epilepsy, chronic pain, and spinal cord injury. The company operates a proprietary KCC2 Discovery Engine based on in vivo phenotypic screening and KCC2-targeted assays, with lead candidate AXN-027 currently in a Phase 1 clinical trial.

Product overview

Axonis Therapeutics is a Boston-based, clinical-stage neuro-focused biotechnology company that combines a single proprietary discovery platform — the KCC2 Discovery Engine — with a modular pipeline of oral small molecule KCC2-potentiating drug candidates. The KCC2 Discovery Engine powers the company's product portfolio by leveraging in vivo phenotypic screening and KCC2-targeted assays to identify and advance compounds. Its lead product, AXN-027, is a first-in-class oral KCC2 potentiator currently in a Phase 1 clinical trial for refractory epilepsy, chronic pain, and spinal cord injury. A broader Next-Generation KCC2 Potentiator Portfolio sits in earlier discovery for additional neurological, psychiatric, and neurodevelopmental indications, and the company also conducts collaborative research (e.g., KCC2-Enhancing Compounds for SYNGAP1 Haploinsufficiency Syndrome with the Tang Lab at Boston Children's Hospital) to extend the reach of its KCC2-targeting approach.

Differentiator

Problem solved

Functional benefit

Products and services

• AXN-027 First-in-class oral small molecule KCC2 potentiator currently in a Phase 1 clinical trial. Designed to restore functional inhibition within the brain by enhancing chloride extrusion via KCC2, and is being developed for refractory epilepsy, chronic pain, spinal cord injury, and other CNS pathologies driven by excitation/inhibition imbalance.
• KCC2 Discovery Engine Proprietary, specialized discovery engine built with world-leading KCC2 biology experts inside and outside Axonis that integrates customized in vivo phenotypic screening and KCC2-targeted assays to identify and advance oral KCC2-potentiating small molecule candidates for neurological disorders.
• Next-Generation KCC2 Potentiator Portfolio Pipeline of additional oral KCC2-potentiating small molecules under development for multiple neurological disorders, including epilepsy, pain, psychiatric disorders, and neurodevelopmental disorders associated with KCC2 hypofunction.

Quantifiable outcome

• $157M+ in cumulative funding raised since 2019 under CEO Joanna Stanicka
• +3 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

Twelve partnerships are on record, tiered major, core and supporting.

• SynGAP Research FundmajorStrategic or Co-development PartnerAwarded a $130,000 grant (April 2023) to the lab of Xin Tang at Boston Children's Hospital and Axonis Therapeutics to research KCC2-enhancing compounds for SYNGAP1 haploinsufficiency syndrome. Aligns Axonis' KCC2 platform with a rare pediatric neurodevelopmental disorder; no current KCC2 drugs on market.
• Boston Children's Hospital (Tang Lab, Xin Tang)coreStrategic or Co-development PartnerResearch collaboration with the Tang Lab (Xin Tang) and Axonis Therapeutics to investigate KCC2-enhancing compounds for SYNGAP1, funded by a $130,000 SynGAP Research Fund grant (April 2023). Boston Children's Hospital is also the academic origin of Axonis' KCC2 IP, alongside Harvard and Laval University.
• Creative Destruction LabsupportingStrategic or Co-development PartnerBiotech entrepreneurial program that co-founder/CSO Shane Hegarty participated in; also acknowledged as an Axonis program partner/funder.
• Springboard EnterprisessupportingStrategic or Co-development PartnerRecognized as one of Axonis' supporting accelerator / entrepreneurial programs alongside MassChallenge and Creative Destruction Lab.
• ISS National Lab (with Boeing)supportingStrategic or Co-development PartnerPartnered with ISS U.S. National Laboratory and Boeing via the 2019 MassChallenge 'Technology in Space' Prize, providing Axonis access to microgravity environment for R&D.
• LabCentralmajorStrategic or Co-development PartnerCoworking biotech laboratory operator; awarded Axonis a one-year prepaid bench space Golden Ticket via Boehringer Ingelheim (2019) and via Astellas Pharma (2020), supporting its early growth.
• Boehringer IngelheimsupportingStrategic or Co-development PartnerRecognized Axonis with the 2019 Innovation Prize in Boston for neuro-restorative spinal cord injury research; provided one year of prepaid bench space at LabCentral.
• Astellas PharmasupportingStrategic or Co-development PartnerSponsored the Astellas/LabCentral "Golden Ticket Competition" (2020) — Axonis won one year of prepaid lab space at LabCentral to accelerate growth of innovative science.
• Lundbeck PharmasupportingStrategic or Co-development PartnerAwarded a Lundbeck Pharma-sponsored Golden Ticket in 2024 to Axonis / CEO Joanna Stanicka, providing LabCentral bench space.
• MassChallengesupportingStrategic or Co-development PartnerBoston-based startup accelerator; Axonis was a 2019 MassChallenge winner and recipient of the 2019 'Technology in Space' Prize with ISS National Lab and Boeing.
• Université Laval (Laval University)coreStrategic or Co-development PartnerAcademic origin of Axonis' KCC2 IP — co-founded with Harvard and Boston Children's Hospital. SAB chaired by Laval Professor Yves De Koninck (Canada Research Chair in Chronic Pain; Quebec Mental Health Institute Scientific Director).
• Harvard Medical School / Boston Children's Hospital (Zhigang He Lab)coreStrategic or Co-development PartnerAcademic origin of Axonis' KCC2 IP. CEO Joanna Stanicka and CSO Shane Hegarty conducted postdoctoral research in Prof. Zhigang He's lab at Harvard Medical School/Boston Children's Hospital, where they developed novel approaches to neuroregeneration/neuroprotection (in vivo phenotypic screens including a genome-wide AAV-CRISPR screen). He also sits on Axonis' Scientific Advisory Board.

Scale indicators8 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• BioAxone BioSciences: Clinical-stage biotech developing KCC2-related therapeutics for spinal cord injury and other CNS injuries; founded by Lisa McKerracher, who is also a co-founder and Scientific Advisor of Axonis. Direct peer in the KCC2 target space with overlapping SCI focus.
• Marinus Pharmaceuticals: Clinical/commercial CNS company developing antiseizure medicines (ganaxolone) for refractory epilepsy and rare seizure disorders. Directly comparable clinical-stage-to-commercial neurology business with overlapping refractory epilepsy focus.
• Xenon Pharmaceuticals: Clinical-stage CNS biotech focused on epilepsy and other neurological disorders, including late-stage antiseizure programs. Comparable neurology-focused pipeline and clinical development model targeting refractory epilepsy patients.
• Sage Therapeutics: Neurology-focused biotech developing CNS medicines including antiseizure therapies and other GABA-related mechanism candidates. Comparable clinical development and commercialization model in neurology, with overlapping CNS mechanism focus.
• Ovid Therapeutics: Clinical-stage biotech developing medicines for epilepsy and rare neurodevelopmental disorders (including rare syndromes such as Angelman, SYNGAP1). Directly comparable business focused on refractory epilepsy and rare KCC2-relevant neurodevelopmental indications.
• Praxis Precision Medicines: Clinical-stage CNS biotech developing therapies for epilepsy and other CNS disorders, including rare pediatric epilepsies. Comparable neurology pipeline, clinical-stage profile and indication overlap (refractory/rare epilepsy).
• Engrail Therapeutics: Clinical-stage neuroscience company developing therapies for epilepsy, depression and other CNS disorders. Comparable clinical development profile and CNS mechanism focus in neurology.
• Longboard Pharmaceuticals: Clinical-stage CNS company focused on neurological diseases including rare epilepsy syndromes (e.g., DEE). Directly comparable clinical-stage neurology peer with overlapping pediatric/refractory epilepsy focus.
• Neumora Therapeutics: Clinical-stage neuroscience company developing precision medicines for brain diseases including epilepsy and other CNS indications. Comparable clinical development profile and CNS focus, supported by top-tier biotech investors.

Broad incumbents

• Zogenix / UCB (Fintepla): UCB acquired Zogenix to gain Fintepla (fenfluramine) for refractory epilepsy (Dravet/Lennox-Gastaut). Represents a large incumbent commercializing approved refractory epilepsy therapies, a comparator for downstream commercialization and competitive dynamics for AXN-027.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat5 records

Key risks6 records

Key highlights6 records

Customer concentration

• Xx.com/axonis_us
• LinkedInlinkedin.com/company/axonis-us

Compliance1 record

Revenue estimate

Valuation estimate

Number of profiles

Profiles9 records

Funding overview

Funding rounds12 records

Investors24 records

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M&A

Investments

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Frequently asked questions about Axonis Therapeutics