Phelan-McDermid Syndrome
2
Pipeline Programs
2
Companies
3
Clinical Trials
1 recruiting
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
1
0
1
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Neuren PharmaceuticalsAustralia - Camberwell
2 programs1
1
NNZ-2591Phase 31 trial
NNZ-2591Phase 21 trial
Active Trials
Human BioSciencesWV - Martinsburg
1 programMapping the Genotype, Phenotype, and Natural History of Phelan-McDermid SyndromeN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
Neuren PharmaceuticalsNNZ-2591
Neuren PharmaceuticalsNNZ-2591
Human BioSciencesMapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
Clinical Trials (3)
Total enrollment: 383 patients across 3 trials
A Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Start: Nov 2025Est. completion: Nov 2027160 patients
Phase 3Recruiting
An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)
Start: Aug 2022Est. completion: Nov 202318 patients
Phase 2Completed
NCT02461420Human BioSciencesMapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
Mapping the Genotype, Phenotype, and Natural History of Phelan-McDermid Syndrome
Start: May 2015Est. completion: Dec 2026205 patients
N/AActive Not 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
1 actively recruiting trials targeting 383 patients
2 companies competing in this space
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.