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Pitt Hopkins Syndrome

3
Pipeline Programs
3
Companies
3
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
1
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 2 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Neuren Pharmaceuticals
Neuren PharmaceuticalsAustralia - Camberwell
1 program
1
NNZ-2591Phase 21 trial
Active Trials
NCT05025332Completed28Est. May 2024
Mahzi Therapeutics
Mahzi TherapeuticsCA - San Mateo
1 program
1
MZ-1866Phase 1/21 trial
Active Trials
NCT07135050Recruiting12Est. Jul 2029
Unravel Biosciences
1 program
1
VorinostatPhase 1Small Molecule1 trial
Active Trials
NCT07150026Recruiting5Est. Mar 2027

Trial Timeline

Clinical trial activity over time

2022
2023
2024
2025
2026
2027
2028
2029
Neuren PharmaceuticalsNNZ-2591
Mahzi TherapeuticsMZ-1866
Unravel BiosciencesVorinostat

Clinical Trials (3)

Total enrollment: 45 patients across 3 trials

NCT05025332Neuren PharmaceuticalsNNZ-2591

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

Start: Oct 2022Est. completion: May 202428 patients
Phase 2Completed
NCT07135050Mahzi TherapeuticsMZ-1866

Phase 1/2 Study of MZ-1866, an AAV-9 Gene Therapy Delivered by Intracerebroventricular Injection to Participants With Pitt Hopkins Syndrome

Start: Dec 2025Est. completion: Jul 202912 patients
Phase 1/2Recruiting
NCT07150026Unravel BiosciencesVorinostat

An Exploratory Evaluation of the Safety and Efficacy of Vorinostat in Pitt Hopkins Syndrome

Start: Mar 2026Est. completion: Mar 20275 patients
Phase 1Recruiting

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

2 actively recruiting trials targeting 45 patients
3 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.