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Neuren Pharmaceuticals has opened the first site in the United States for its Phase 2 clinical trial of NNZ- 2591 in Prader-Willi syndrome, aiming to generate necessary data for designing a subsequent registration trial.
Alongside this, Phase 2 trials of NNZ-2591 are also undergoing for three other neurodevelopmental disorders – Phelan-McDermid, Pitt Hopkins and Angelman syndromes, each of which has received orphan drug designation from the FDA.
The open-label Phase 2 trial will examine safety, tolerability, pharmacokinetics and efficacy with NNZ-2591 in up to 20 children aged 4 to 12 years over 13 weeks, with the dosage escalated up to the target dose subject to safety review, and a final follow-up assessment conducted two weeks after the end of treatment.
DAYBUE™ (trofinetide) is now available in the US for the treatment of Rett syndrome in adult and pediatric patients aged two years and older, with Neuren earning US$40m from the launch.
Neuren is eligible to receive ongoing royalties on net sales of trofinetide in North America, plus milestone payments of up to US$350m and one-third of the market value of the Rare Pediatric Disease Priority Review Voucher.
Neuren is also conducting Phase 2 trials for NNZ-2591, targeting Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome, and Prader-Willi syndrome, with all programs granted "orphan drug" designation in the US.