Juvenile Neuronal Ceroid Lipofuscinosis
1
Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2026
2027
2028
2029
Polaryx TherapeuticsPLX-200
Polaryx TherapeuticsPLX-200
Clinical Trials (2)
Total enrollment: 63 patients across 2 trials
Safety, Tolerability, and Efficacy of PLX-200 in Patients With CLN3
Start: Nov 2026Est. completion: Dec 202639 patients
Phase 3Not Yet Recruiting
Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)
Start: Dec 2026Est. completion: Feb 202924 patients
Phase 2Not Yet Recruiting
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
1 late-stage (Phase 3) programs, potential near-term approvals
2 companies competing in this space
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