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lufepirsen high dose

Phase 2

Persistent Corneal Epithelial Defect | Small molecule | Ophthalmology |Glaukos Corporation|Last Updated: Jul 24, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment84
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05966493A Study to Evaluate the Safety and Efficacy of NEXAGON® (Lufepirsen Ophthalmic Gel) in Subjects With PCEDPHASE2 RECRUITING 84Aug 17, 2023Dec 1, 2025Jul 24, 202528 United States, Germany +2
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Study Endpoints
Primary Endpoints
Achieve Corneal Re-epithelialization Including Durability (CRC)
End of Study: 28 Days after achieving re-epithelialization

The proportion of subjects achieving corneal re-epithelialization that is maintained for a minimum of 28 days, based on assessment of corneal fluorescein staining images of the PCED by a Central Reading Center (CRC).

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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
NEXAGON® (lufepirsen ophthalmic gel) High Dose ConcentrationEXPERIMENTALLufepirsen (High dose concentration) applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks.
NEXAGON Vehicle (ophthalmic gel)PLACEBO_COMPARATORVehicle applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks.
NEXAGON® (lufepirsen ophthalmic gel) Low Dose Concentration (EU sites only)EXPERIMENTALLufepirsen (Low dose concentration) applied topically to the eye once a week for a minimum of 4 weeks to a maximum of 8 weeks. (EU sites only).
Interventions
NameTypeDescription
lufepirsen high doseDRUGLufepirsen is an unmodified connexin43 antisense oligonucleotide.
VehicleDRUGMatching vehicle without lufepirsen.
lufepirsen low doseDRUGLufepirsen is an unmodified connexin43 antisense oligonucleotide.
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Eligibility Criteria
Age Range2 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites28

Inclusion Criteria: 1. Have a PCED that is at least 2 weeks in duration and refractory to one or more conventional non-surgical standard of care (SOC) treatments 2. Have no clinical evidence of improvement in the PCED within 2 weeks prior to randomization despite the use of non-surgical SOC treatme...

Countries:United StatesGermanyItalySpain
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05966493primaryCompletionDate: changed
LOWMay 24, 2026NCT05966493studyFirstPostDate: changed