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Current Pipeline
Small Molecule

Neuren • NNZ-2591

Neuren Pharmaceuticals is exploring an investigational drug called NNZ-2591, a synthetic analog related to Insulin-Like Growth Factor-1 (IGF-1), as a potential treatment for Angelman syndrome.

Phase 2

Discovery & Dev

Pre-clinical

Phase 1

Phase 2

Phase 3

To patients

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Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.

Therapeutic Approach

NNZ-2591 is a synthetic analog of a peptide that is related to IGF-1, a growth factor that is stimulated by growth hormone. IGF-1 in the brain is thought to be critical for normal brain development and to maintain biological balance by reducing inflammation, restoring normal cell function of the cells that surround neurons of the brain, and improve the function at the junction of the neurons.

Investigational Drug Name

NNZ-2591

Recent Updates

  • August 2024

    Neuren Pharmaceuticals announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS).

    Learn More
  • August 2024

    COMPLETED: An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome (AS-001)

    Read More

Media

  • Clinical Development Update: NNZ-2591 as a Treatment for Angelman Syndrome

Disclaimer

This website contains information for a broad audience and may include information on current and upcoming programs that are not yet approved or accessible The information provided is for general informational purposes only and is not intended as medical advice, diagnosis, or treatment. While FAST strives to provide accurate and up-to-date information, the content on this site may not always reflect the most current research or clinical guidelines. The inclusion of clinical trial information, treatments or specific healthcare providers does not imply endorsement, recommendation or guarantee of safety, efficacy, or availability. Reliance on any information provided by this website is solely at your own risk. FAST disclaims any liability for any errors or omissions in the information provided or for any decisions made based on this information. For personalized medical advice or specific health concerns including participation in any clinical trial, please consult a qualified healthcare professional.