Recent Updates
Recently added Catalysts

DB-OTO

Phase 1

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF) | Gene therapy | Rare Disease |Regeneron Pharmaceuticals, Inc.|Last Updated: May 5, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment30
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05788536A Study of DB-OTO, an Adeno-Associated Virus (AAV) Based Gene Therapy, in Children/Infants With Hearing Loss Due to Otoferlin MutationsPHASE1 RECRUITING 30 — — Jun 27, 2023Feb 28, 2032May 5, 202616 United States, Germany +3
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Incidence and severity of Treatment-Emergent Adverse Events (TEAEs)
Up to week 104
Achievement of a hearing sensitivity threshold of ≤70 dB assessed by average Pure Tone Audiometry (PTA)
Up to week 104
Secondary Endpoints
Auditory Brainstem Response (ABR) to click
Up to week 48
Achievement of a score ≥3 on the Early Speech Perception (ESP) test
At week 104
Achievement of hearing sensitivity threshold of ≤45 dB assessed by average PTA
Up to week 104
Unlock Study Endpoints
Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
DB-OTO - Unilateral Dose EscalationEXPERIMENTALPart A: Unilateral intracochlear dosing
DB-OTO - Bilateral Dose ExpansionEXPERIMENTALPart B: Bilateral intracochlear dosing using the dose selected based on safety and efficacy data from Part A.
Interventions
NameTypeDescription
DB-OTOGENETICDB-OTO will be administered as a single intracochlear injection into one ear (Part A). * LD Cohort (lower dose) * HD Cohort (high dose) - not implemented
Unlock Study Design Details
Eligibility Criteria
Age RangeN/A — 17 Years
SexALL
Healthy VolunteersNo
Study Sites16

Key Inclusion Criteria: 1. Willingness of at least 1 parent/legal guardian to provide written informed consent (and patient to provide assent, when applicable) and willingness to comply with trial protocol; to consent to genetic testing for the patient (and patient to provide assent, when applicabl...

Countries:United StatesGermanyJapanSpainUnited Kingdom
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
MEDIUMMay 26, 2026NCT05788536primaryCompletionDate: changed
LOWMay 24, 2026NCT05788536studyFirstPostDate: changed