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Patidegib

Phase 3

Gorlin Syndrome | Small molecule | Other |Sol-Gel Technologies Ltd.|Last Updated: Oct 14, 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 Enrollment140
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06050122Efficacy and Safety of Patidegib Gel 2% for Preventing Basal Cell Carcinomas on the Face of Adults With Gorlin SyndromePHASE3 ACTIVE NOT_RECRUITING 140Mar 17, 2024Aug 1, 2026Oct 14, 202540 United States, France +5
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Study Endpoints
Primary Endpoints
Number of new BCCs on the face at Month 12
Month 12

New BCCs at Month 12 after twice-daily treatment with either Patidegib Gel 2% or Vehicle Gel for 12 months, as assessed by the CPRB

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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposePREVENTION
Treatment Arms
ArmTypeDescription
Patidegib Gel 2%EXPERIMENTALPatidegib Gel 2% (w/w), applied topically to the face twice daily for 12 months
Vehicle GelPLACEBO_COMPARATORVehicle Gel, applied topically to the face twice daily for 12 months
Interventions
NameTypeDescription
Patidegib Topical GelDRUGPatidegib Gel 2% is a smooth, clear, colorless-to-yellow, viscous, hydro-alcoholic gel for topical administration.
Patidegib Topical Gel with no active patidegibDRUGVehicle Gel consists of the excipients of Patidegib Gel with no active patidegib. It is a smooth viscous gel for topical administration.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites40

Key Inclusion Criteria: 1. The subject must be at least 18 years old at the Screening Visit. 2. The subject must be confirmed to have a PTCH1 mutation. 3. The subject must have at least 10 BCCs on the face at Randomization (Baseline/Day 1). 4. The subject must be willing to abstain from application...

Countries:United StatesFranceGermanyItalyNetherlandsSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06050122primaryCompletionDate: changed
LOWMay 24, 2026NCT06050122studyFirstPostDate: changed