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Peptide library / Brain & sleep / NNZ-2591

In clinical trialsBrain & sleep

NNZ-2591

An investigational synthetic analog of a natural brain peptide, in development for rare neurodevelopmental disorders.

Body system
Brain & sleep
Status
In clinical trials
Strongest evidence
Small human studies
Also known as
–

Effects studied or claimed

What it is used or promoted for, and where each effect has actually been seen

  • Phelan-McDermid syndromeSmall human studies
  • Angelman and Pitt-Hopkins syndromesSmall human studies

How it is thought to work

NNZ-2591 is a modified version of a small cyclic peptide made when IGF-1 is broken down in the brain, and is thought to restore synapse function and reduce inflammation.

What the evidence shows

Open-label phase 2 trials by Neuren reported improvements on clinician and caregiver scales. Placebo-controlled confirmation is still needed.

Safety and unknowns

Generally well tolerated in phase 2. Long-term data are limited.

Regulatory status

Not approved. Neuren is moving toward phase 3.

Regulatory status changes quickly. This entry was last reviewed on 27 October 2026. Confirm current status with the FDA before relying on it.

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General information, not medical advice. Do not buy or inject unregulated peptides.