Audion Therapeutics

What Audion Therapeutics does

Audion Therapeutics is a clinical-stage, venture-backed biopharmaceutical company developing disease-modifying drug therapies for acquired sensorineural hearing loss (SNHL). Founded in Amsterdam, the Netherlands with a laboratory facility established in Cambridge, Massachusetts following its 2014 Eli Lilly investment, the company focuses exclusively on inner-ear hair cell and synapse regeneration as a treatment paradigm that goes beyond the symptomatic prostheses (hearing aids and cochlear implants) that dominate current standards of care. Its core technology platform centers on a small-molecule gamma secretase inhibitor, AUD1001 (also known as LY3056480), originally discovered at Massachusetts Eye and Ear's Eaton-Peabody Laboratory by co-founder Prof. Albert Edge and exclusively licensed to Audion from both Mass Eye and Ear and Eli Lilly. The drug is delivered via intratympanic injection to enable targeted inner-ear exposure while limiting systemic exposure, and is intended to induce supporting-cell transdifferentiation into hair cells as well as synapse formation.

The company's pipeline is single-asset, with AUD1001 representing the entirety of disclosed product development. As of the most recent disclosures, Audion has completed a Phase I multiple ascending dose safety study in 15 patients and a Phase I/IIa trial across UK, German, and Greek sites under the EU Horizon 2020-funded REGAIN consortium, with combined results published in Nature Communications in March 2024. The Phase IIa pre-defined primary endpoints were not met, but post-hoc analyses indicated positive changes in pure-tone audiometry and speech-perception-in-noise measures in 20-25% of patients, with signals persisting up to 12 months post-treatment. Audion does not currently have any ongoing clinical studies and is not generating commercial product revenue; its operating model is to develop evidence to a stage attractive to a pharmaceutical partner or acquirer, with Eli Lilly retaining an option to reacquire technology rights upon successful clinical Proof of Concept.

Cumulatively, Audion has raised approximately €2.5 million in Series B equity from INKEF Capital (September 2015), €5.8 million in non-dilutive Horizon 2020 grant funding through the REGAIN consortium (2015), and an undisclosed Series A from Eli Lilly (February 2014) that accompanied the licensing agreement for LY3056480. The company is led by CEO and co-founder Rolf Jan Rutten, with a small management team (1-10 employees) supplemented by an academic founder (Prof. Albert Edge) and a senior CMO (Prof. Peter Weber) with prior experience at Decibel Therapeutics and Frequency Therapeutics. Audion is currently dependent on raising additional capital or securing a development partner to resume clinical advancement of its lead program.

Name

Audion Therapeutics

Legal name

Audion Therapeutics BV

Website

https://audiontherapeutics.com

Company type

Private

Founded year

2011

Operating status

Operating

Headcount range

1–10 employees

Short description

Audion Therapeutics is a clinical-stage, privately held biopharmaceutical company developing AUD1001/LY3056480, a small-molecule gamma secretase inhibitor delivered intratympanically to regenerate inner-ear hair cells and synapses in patients with acquired sensorineural hearing loss.

Ownership category

akta.pro rank

Product category

Hearing Loss Therapeutics

NAICS

Scientific Research and Development Services (5417)

SIC

Services-Commercial Physical & Biological Research (8731)

akta.pro primary industry

Otology & Neurotology (Ear/Hearing/Balance) (HLAKAMAC)

Keywords

• Sensorineural hearing loss
• Gamma secretase inhibitors
• Hair cell regeneration
• Intratympanic drug delivery
• Otology drug development

Headquarters

HQ city

Amsterdam

HQ country

Netherlands

HQ region

Europe

Offices2 records

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

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

Revenue model

1. Pharmaceutical Product Sales: As a clinical-stage biopharmaceutical company, Audion Therapeutics is developing AUD1001 (LY3056480) for the treatment of sensorineural hearing loss. The company does not currently have commercialized products and has no ongoing clinical studies pending additional funding.

Go-to-market motion1 record

Distribution channels1 record

Marketing channels4 records

Core offering

Audion Therapeutics develops small-molecule gamma secretase inhibitor drug candidates for the treatment of acquired sensorineural hearing loss (SNHL). Its lead product AUD1001 (LY3056480) is a clinical-stage therapeutic designed to regenerate lost inner ear hair cells and synapses through transdifferentiation of supporting cells, delivered locally via intratympanic injection. The company is currently seeking additional funding or a pharmaceutical partner to advance its lead program, with future commercialization expected through licensing to large pharma companies.

Product overview

Audion Therapeutics is a clinical-stage biopharmaceutical company developing a single lead product candidate, AUD1001 (also known as LY3056480), which is a gamma secretase inhibitor for the treatment of acquired sensorineural hearing loss. The company operates with a pipeline-focused approach where this small molecule drug candidate represents the core product. The drug works by inducing transdifferentiation of supporting cells into hair cells and forming synapses to enable sound signal transmission to the auditory nerve.

Differentiator

Problem solved

Functional benefit

Products and services

• AUD1001 (LY3056480) AUD1001 (also known as LY3056480) is a small-molecule gamma secretase inhibitor in clinical development for the treatment of acquired sensorineural hearing loss (SNHL). The drug is delivered locally via intratympanic injection directly to the inner ear, where it induces transdifferentiation of supporting cells into new hair cells and the formation of new synapses, restoring auditory function lost due to aging, noise exposure, or other environmental factors.

Quantifiable outcome

• Phase I confirmed safety and tolerability of 250 microgram dose of LY3056480 in 15 patients
• +3 more outcomes

Segments1 record

Ideal customer profiles2 records

Technology focussed Yes

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature2 records

Partnerships

Eight partnerships are on record, tiered core and minor.

• REGAIN ConsortiumcoreStrategic or Co-development PartnerInternational consortium of 7 partners coordinated by Audion with unique expertise in hearing loss biology, drug development, and clinical expertise. Consortium includes Eli Lilly, Clinquest Services, Ttopstart, University College London, UCLH, University of Tubingen, and National and Kapodistrian University of Athens. Phase I/IIa clinical trial conducted across UK, Germany, and Greece sites.
• SanofiminorStrategic or Co-development PartnerTwo-year research collaboration announced June 2011 for development of potential treatments for hearing loss through optimization of small molecules using regenerative medicine approach. Sanofi has option to license technology rights from Audion related to research conducted under collaboration. Collaboration utilized technology developed at Mass Eye and Ear by Dr. Albert Edge.
• Massachusetts Eye and Ear InfirmarycoreTechnology or IntegrationTechnology and molecules originated from research by co-founder Dr. Albert Edge at the Eaton-Peabody Laboratory at Mass Eye and Ear, one of the world's largest basic research facilities dedicated to the study of hearing and deafness. Technology exclusively licensed to Audion.
• University College London (UCL)coreStrategic or Co-development PartnerClinical study partner in the UK for the REGAIN Phase I/IIa trial. Part of Horizon 2020 REGAIN consortium. UCL Ear Institute provided international network collaboration.
• University of TubingencoreStrategic or Co-development PartnerAcademic partner in the REGAIN consortium conducting Phase I/IIa clinical trials in Germany.
• National and Kapodistrian University of AthenscoreStrategic or Co-development PartnerAcademic partner in the REGAIN consortium conducting Phase I/IIa clinical trials in Greece.
• CilcareminorStrategic or Co-development PartnerPreclinical CRO (Contract Research Organization) partner in the REGAIN consortium supporting preclinical studies.
• Nordic BioscienceminorStrategic or Co-development PartnerIndustrial partner in the REGAIN consortium bringing drug development expertise.

Scale indicators7 records

Recent moves5 records

Expansion highlights4 records

Direct peers

• Frequency Therapeutics: Closest clinical-stage competitor to Audion, also developing a regenerative small-molecule therapy (FX-322) for sensorineural hearing loss via hair cell regeneration. Wound down most operations in 2024 after Phase II setbacks, providing a directly comparable read-through for Audion's gamma secretase approach.
• Decibel Therapeutics: Clinical-stage hearing-loss biotech developing DB-OTO gene therapy and small-molecule programs for SNHL. Acquired by Regeneron in 2023 for approximately $213M, making it the most relevant recent comparable for valuation and exit dynamics in the hearing-loss pharma space.
• Otonomy: Clinical-stage biopharmaceutical company focused on ear therapeutics (Meniere's disease, otitis media, SNHL). Direct competitor in developing locally administered drugs for inner-ear conditions, sharing Audion's intratympanic delivery orientation.
• Sensorion: French clinical-stage biotech developing novel therapies for hearing loss disorders including SENS-401 for sudden SNHL. Directly comparable as a Europe-based, clinical-stage hearing-loss drug developer with similar regulatory and funding dynamics.
• Sound Pharmaceuticals: Clinical-stage biopharma developing novel small molecules (SPI-5557, SPI-3005) for hearing loss including noise-induced and ototoxic SNHL. Comparable as a dedicated hearing-loss drug developer with clinical-stage assets in the same disease area.
• Acousia Therapeutics: German clinical-stage biotech developing small-molecule drugs (e.g., ACOU085) for inner-ear sensorineural hearing loss. Closely comparable as a European small-molecule hearing-loss developer with similar regulatory and reimbursement profile.

Emerging players

• Pipeline Therapeutics: Clinical-stage biotech developing treatments for hearing loss, including otoprotection and regeneration programs. Comparable as an emerging player in the SNHL drug-development space with complementary mechanistic approaches to Audion's gamma secretase inhibition.

Broad incumbents

• Cochlear Limited: Global leader in cochlear implant systems and the dominant incumbent alternative treatment for severe SNHL. Indirect competitor addressing the same patient population with a device rather than a regenerative drug; relevant for understanding the standard-of-care Audion's drug would displace.
• MED-EL: Leading global manufacturer of cochlear implant systems and middle-ear prosthetics, addressing the same severe SNHL patient population as Audion's lead program. Comparable as an incumbent alternative therapy in the hearing-loss treatment landscape.

Others

• Eli Lilly and Company: Series A investor (2014) and licensor of the LY3056480 gamma secretase inhibitor program to Audion, with an option to reacquire rights at clinical POC. Functions as a strategic ecosystem participant and most likely large-pharma exit partner rather than a direct competitor.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• LinkedInlinkedin.com/company/audion-therapeutics-bv

Revenue estimate

Valuation estimate

Number of profiles

Profiles5 records

Funding overview

Funding rounds1 record

Investors2 records

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M&A

Investments

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

Frequently asked questions about Audion Therapeutics