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Neuren Pharmaceuticals Adds Prader-Willi Syndrome to Development Pipeline

Neuren Pharmaceuticals has added PWS to their development pipeline for compound NNZ-2591 following a successful study in a pre-clinical model.

Neuren Pharmaceuticals has added Prader-Willi syndrome to their development pipeline for compound NNZ-2591 following a successful study in a pre-clinical model. The study showed compelling benefits for treating key symptoms of PWS. 

Key symptoms were eliminated after treatment with NNZ-2591 in Magel2-null mouse model of Prader-Willi syndrome including:

  • Both dose levels normalized all behavioral deficits.
  • Obesity was eliminated, abnormally high insulin levels were reduced to normal, and abnormally low levels of circulating IGF-1 was increased to normal by the high dose.
  • The low dose partially improved obesity, insulin levels and circulating IGF-1 levels.

Neuren CEO Jon Pilcher commented: “These excellent results have once again reinforced the potential for NNZ-2591 to make a difference across multiple neurodevelopmental disorders in which signaling between brain cells and IGF-1 metabolism are impaired. We look forward to working with Prader-Willi families and clinicians, as we are already doing with PhelanMcDermid, Angelman and Pitt Hopkins, to bring this potential treatment to fruition.”

Neuren intends to submit applications for Orphan drug designation in the US and Europe. NNZ2591 has already received Orphan drug designation for Phelan-McDermid, Angelman and Pitt Hopkins syndromes following similarly compelling pre-clinical model results.

Additional study details are available in the Neuren Pharmaceuticals announcement.

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Topics: Research

Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.