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PLX-200

Phase 3

Juvenile Neuronal Ceroid Lipofuscinosis | Small molecule | Other |Polaryx Therapeutics, Inc. Common Stock|Last Updated: May 29, 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
UNCONTROLLED
Total Trials1
Total Enrollment39
FDA Designations
FAST_TRACK
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04637282Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3PHASE3 NOT YET_RECRUITING 39Feb 1, 2026Mar 1, 2026May 29, 2025 -
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Study Design & Arms
Interventions
NameTypeDescription
PLX-200DRUG15 mg/mL oral solution of experimental drug
PlaceboDRUGTaste and color-matched drug-free solution
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Recent Changes (Last 90 Days)
LOWMay 24, 2026NCT04637282studyFirstPostDate: changed