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NNZ-2591

Phase 3Small molecule

NNeuren Pharmaceuticals Limited

1

Indications

7

Total Trials

2

Active Trials

0

Competitors

Pipeline

Status by Indication[3][1]

Indication

Phelan-Mcdermid SyndromeRecruiting

Phelan-Mcdermid SyndromePhase 3

Recruiting

Timeline

Expected Milestones

Q4 2027

Phase 3Recruiting

Phelan-Mcdermid Syndrome

NCT07281079

Q4 2028

Phase 3Recruiting

Phelan-Mcdermid Syndrome

NCT07593391

Clinical

Trials by Indication[1]

Phelan-McDermid syndrome3 trials

An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

Completed·NNeuren Pharmaceuticals Limited·Phase 2·NCT05025241·18 enrolled

A Phase 3 Randomized, Double-blind, Placebo-controlled Study to Investigate the Efficacy and Safety of Orally Administered NNZ-2591 Compared With Placebo in Pediatric Participants With Phelan-McDermid Syndrome

Recruiting·NNeuren Pharmaceuticals Limited·Phase 3·NCT07281079·160 enrolled

A Phase 3 Open-label Extension Study to Investigate the Long-term Safety and Efficacy of Orally Administered NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome

Recruiting·NNeuren Pharmaceuticals Limited·Phase 3·NCT07593391·180 enrolled

Oral2 trials

An Open-Label Study of Oral NNZ-2591 in Pitt Hopkins Syndrome (PTHS-001)

Completed·NNeuren Pharmaceuticals Limited·Phase 2·NCT05025332·28 enrolled

To Evaluate the Safety, Tolerability and Pharmacokinetics of Oral NNZ-2591 in Healthy Volunteers

Completed·NNeuren Pharmaceuticals Limited·Phase 1·NCT04379869·28 enrolled

Angelman syndrome1 trial

An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome

Completed·NNeuren Pharmaceuticals Limited·Phase 2·NCT05011851·17 enrolled

Prader-Willi syndrome1 trial

An Open-Label Study of Oral NNZ-2591 in Prader-Willi Syndrome (PWS-001)

Withdrawn·NNeuren Pharmaceuticals Limited·Phase 2·NCT05879614

Sources

References

[1]
ClinicalTrials.gov — 7 trial records for NNZ-2591. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — NNZ-2591 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for NNZ-2591. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Neuren Pharmaceuticals Limited company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 3[1]
Company: NNeuren Pharmaceuticals Limited

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