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Details checkedStatus needs checkingHistorical/discontinuedCheckedpublication

LY3056480 by Audion Therapeutics

Also known as: LY3056480, REGAIN, REGAIN trial gamma-secretase inhibitor, VESTA, AUT-003, ISRCTN59733689, NCT05061758

Audion Therapeutics BV

Primary source
Company
Audion Therapeutics BV
Program Type
Therapeutic
Modality
Small Molecule
Stage
Phase 2
Status
Discontinued
Target Gene
—
Route / Mode
Intratympanic injection
Confidence
88%
Status Confidence
Status needs checking
Evidence Strength
Needs more support
Last Reviewed
2026-06-09
Geography
United Kingdom, Germany, Greece, Netherlands, United States
Population
adult, older-adult, mild-to-moderate, sensorineural-hearing-loss
Trial IDs
2
Mechanism

Intratympanic gamma-secretase inhibitor intended to promote cochlear hair-cell regeneration through Notch-pathway inhibition in sensorineural hearing loss.

Indications
Hearing LossSensorineural Hearing Loss
Notes

Verified as a real Audion Therapeutics LY3056480 regenerative-hearing program. The REGAIN Phase I/IIa study of intratympanic gamma-secretase inhibitor completed and was reported in Nature Communications; it was safe and well tolerated but did not meet the primary efficacy endpoint. A later Audion-sponsored Phase 2 VESTA study in stable sensorineural hearing loss was posted on ClinicalTrials.gov as NCT05061758 but was withdrawn before enrollment with the registry reason "Study put on hold." No current active follow-on development was confirmed in public sources in this pass, so HearScape treats the program as discontinued/withdrawn rather than active.

Evidence & Sources
4 structured sources
clinicaltrials
Supports: canonicalName, aliases, company, allCompanies, isIndustrySponsored, programType, modality, mechanism...
IDs: NCT05061758, AUT-003, VESTA, LY3056480
Open
publication
Supports: canonicalName, aliases, company, allCompanies, modality, mechanism, indications, stage...
IDs: LY3056480, ISRCTN59733689, REGAIN, DOI 10.1038/s41467-024-45784-0, PMID 38429256
Open
publication
Supports: trialIds, stage, status, notes, keyEvents
IDs: PMID 38429256, LY3056480, ISRCTN59733689
Open
institution
Supports: status, notes, keyEvents
IDs: LY3056480, REGAIN, Audion Therapeutics
Open
Key Events
2021-09-30
ClinicalTrials.gov first posted Audion VESTA Phase 2 study NCT05061758
2022-08-25
VESTA Phase 2 study was updated as withdrawn before enrollment because the study was put on hold
2024-03-01
Nature Communications published REGAIN Phase I/IIa results for LY3056480
2024-03-05
UCL summarized REGAIN as completed, with safety/tolerability and exploratory activity signals but no primary endpoint success
Clinical Trials (2)
ISRCTN59733689NCT05061758
Data Checks
replaced-unclear-current-status-with-withdrawn-follow-on-trialphase-2-withdrawn-before-enrollmentprimary-endpoint-not-met-in-regainno-confirmed-active-follow-on-program-foundcurrent-ownership-not-clearly-disclosed
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