An Open-label Study of NNZ-2591 in Pediatric Participants With Phelan-McDermid Syndrome
Neuren Pharmaceuticals Limited180 participants8 Jun 2026 to est. 12 Nov 2028
Phelan-McDermid Syndrome
In PepBank now:147 peptides, 6 blends and 13 other compounds97,577 study results from 17,383 studies8,473 clinical trialsUpdated
Comparing 2 peptides · Data version 308560
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| Peptide | Synthetic analog NNZ-2591 | Synthetic analog Trofinetide | |
|---|---|---|---|
| Identity | |||
| Kind | Synthetic analog | Synthetic analog | |
| Area | Brain and mood | Brain and mood | |
| Evidence | |||
| Amount of evidence | 23 | 204 | |
| Evidence level | Results from animal studies onlyRegistered trials, no results from human studies | Has results from studies in people | |
| Safety | |||
| Safety results | No safety results | 62 (61 in people) | |
| Status | |||
| Approval | Not recorded | ||
| Sport (WADA) | |||
| Registered trials | 7 · 2 active | 13 · 1 active1 more mentions it but does not test it | |
Shared outcomes
From the regulatory data of each peptide.
Outcome groups with study results for at least two of these peptides. Counts include only results from studies that gave the peptide. Bar length is the number of results, on one scale. The bars show how much was studied, not how large an effect was. A peptide with more studies can look stronger here without being stronger. For results, see the randomized results above.
| Outcome group | NNZ-2591 | Trofinetide |
|---|---|---|
| PharmacokineticsPharmacology | 33 | 4242 |
| NeuroprotectionNervous system and cognition |
PepBank is a research reference. Talk to a doctor before you use any peptide.
The FDA label for Daybue says: “Rett syndrome in adults and pediatric patients 2 years of age and older”
88
862 |
| Receptor activity and mechanismNervous system and cognition | 66 | 99 |
|---|
| Exercise and physical functionNervous system and cognition | 33 | 422 |
|---|
| Inflammation markersNervous system and cognition | 11 | 88 |
|---|
| Cognition and memoryNervous system and cognition | 22 | 11 |
|---|
Each analog against its native parent, residue by residue. Substitutions, non-standard residues and modifications are marked. Calculated from the two sequences.
Trofinetide differs from Insulin-like growth factor 1 at 1 position.
What is different
For each outcome that two or more peptides have results for: the best result of a single randomized trial, on the same measure (the same kind of number, the same unit). When a trial has several dose arms, the cell shows the highest dose arm. The trials differ in people, length and dose, so a gap between two cells is not proof that one peptide works better.
No outcome has a randomized-trial result on the same measure for two or more of these peptides yet.
Registered trials, ranked by phase (late phases first), then posted results and enrollment. Status is the registry status. Study results come from published studies. Results posted at ClinicalTrials.gov are shown on each trial page.
Top 3 of 7 trials, ranked by phase, then posted results and enrollment: 2 ongoing · 1 completed · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)
Neuren Pharmaceuticals Limited180 participants8 Jun 2026 to est. 12 Nov 2028
Phelan-McDermid Syndrome
Neuren Pharmaceuticals Limited160 participants12 Nov 2025 to est. 15 Nov 2027
Phelan-McDermid Syndrome
Neuren Pharmaceuticals Limited28 participants14 Oct 2022 to 3 May 2024
Pitt Hopkins Syndrome
Top 3 of 13 trials, ranked by phase, then posted results and enrollment: 2 completed · 1 stopped early · 3 with posted results · source ClinicalTrials.gov (opens in a new tab)
ACADIA Pharmaceuticals Inc.187 participants6 Nov 2019 to 28 Oct 2021
Rett Syndrome
ACADIA Pharmaceuticals Inc.154 participants29 Jan 2020 to 19 Aug 2022
Rett Syndrome
ACADIA Pharmaceuticals Inc.77 participants8 Nov 2020 to 30 Jun 2023
Rett Syndrome
1 more mentions it but does not test it. They only measure it or name it in their text.