Neuren Pharmaceuticals is Happy to Announce the First Site Participating in Their Phase II, Open Label, PWS Study (Neu-2591-PWS-001) is Now Open for Screening!

Important information regarding this exciting milestone:

  • Rare Disease Research (RDR), located in Atlanta, GA, is now welcoming children with PWS and their families to their clinicfor screening into this trial.
  • The duration of active treatment in this study is 13 weeks. In a preclinical study in animals, physiological and behavioral symptoms were normalized within six weeks of dosing.
  • Enrollment will begin in children diagnosed with PWS who are in the older age group (ages 8 through 12).
  • After safety and tolerability data in the older group is independently reviewed, it will be announced when children in the younger group (ages 4 through 7) can start to be screened.
  • As this is an open-label study, all children who participate on this trial will receive study drug.
  • All travel and lodging for in-clinic visits (5 in total) can be reimbursed.
  • If the study drug (NNZ-2591) development moves into Phase 3, all children who participate in this Phase II study may be eligible for an Open-label Extension study that would be opened in parallel to the NNZ-2591 Phase III trial in PWS.
  • Study goals:
    • The overall goal of this first NNZ-2591 clinical trial in children with PWS is to assess safety and inform the design of subsequent registration trials.
    • The primary outcome measures in the study are safety and tolerability as well as measuring how the drug is used and distributed in the body.
    • The secondary goal is to assess the potential benefit of NNZ-2591. The study looks at a range of outcomes that are functionally important to children with PWS: behavioral issues, anxiousness, social interaction, language, cognition/learning, hyperphagia/food behaviors, sleep, and activities of daily living.

For any questions in relation to study participation at Rare Disease Research, please reach out directly to the site at (please note that his email address is actively monitored for incoming emails in need of response). For any general questions, please reach out to Dorothea Lantz (


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