Neuren Announces Results of the NNZ-2566 Clinical Trial for Rett Syndrome

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With Neuren's Phase 2 trial a success, Rettsyndrome.org comments on this advance in Rett treatment drug development.

This is what we, as the Rett community, have been hoping for. Rettsyndrome.org will continue to support Neuren in their goal to develop NNZ-2566 as a drug designated to treat Rett syndrome.

Neuren Pharmaceuticals’ human clinical trial using NNZ-2566 as a potential treatment for Rett syndrome (a trial supported by Rettsyndrome.org) was completed in September of this year. On November 11, Neuren announced the results of this phase 2 placebo-controlled study, which investigated the dosing of NNZ-2566 for safety and tolerability with efficacy being studied as well. The study was carried out at Baylor College of Medicine (Drs. Daniel Glaze and Jeffery Neul), University of Alabama Birmingham (Dr. Alan Percy) and Gillette Children's Specialty Healthcare (Drs. Tim Feyma and Art Beisang).

NNZ-2566 was shown to be well tolerated at the dose levels tested after 28 days of treatment, and no significant safety concerns were identified. The data indicated dose-response and improvement over time in the study. The high dose used in this study showed benefit vs placebo both in the group-level analysis as well as in the individual subject analysis. For the details of the results please refer to Neuren’s announcement.

This is the first step needed to develop a drug for treatment of Rett syndrome. The next steps for Neuren are to meet with the FDA and to apply for Orphan Drug Status and Breakthrough Therapy Status.

Steven Kaminsky PhD, Chief Science Officer of Rettsyndrome.org, commented on the results announced by Neuren: “These are exciting times for Rett syndrome and this trial firmly sets our rudder in the water for the near future. The results will enable engagement with the FDA on the further development of NNZ-2566. This is what we, as the Rett community, have been hoping for. Rettsyndrome.org will continue to support Neuren in their goal to develop NNZ-2566 as a drug designated to treat Rett syndrome."

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ABOUT RETT SYNDROME
Rett syndrome is a rare non-inherited genetic postnatal neurological disorder that occurs almost exclusively in girls and leads to lifelong impairments, affecting nearly every aspect of child’s life: their ability to speak, walk, eat is effected, seizures develop and scoliosis occurs, and many develop irregular breathing patterns. Those diagnosed with Rett syndrome require maximum assistance with basic daily activities. The hallmark sign of Rett syndrome is near constant repetitive hand movements. Cognitive assessment in children with Rett syndrome is complicated, but they understand far more than they can communicate to us, evidenced by their bright and attentive eyes, and their ability to express a wide spectrum of moods and emotions.

ABOUT RETTSYNDROME.ORG
Rettsyndrome.org is the most comprehensive nonprofit organization dedicated to accelerating research of treatments and a cure for Rett syndrome and related disorders while providing information and family empowerment. As the world’s leading private funder of Rett syndrome research, Rettsyndrome.org has funded over $34M in high-quality, peer-reviewed research grants and programs to date. The organization hosts the largest global gathering of Rett researchers and clinicians to establish research direction for the future. Rettsyndrome.org, a 501(c)3 organization, has earned Charity Navigator’s most prestigious 4 star rating year after year. To learn more about our work and Rett syndrome, visit http://www.rettsyndrome.org or call (800) 818-7388 (RETT).

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Melissa Florio
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