Neuren Pharmaceuticals has initiated the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome (PMS), marking a significant milestone as the first-ever Phase 3 trial for this severe neurodevelopmental disorder. The company received Institutional Review Board approval for the trial, with other sites in the US at various stages of the initiation process.
Trial Design and Patient Population
The randomized, double-blind, placebo-controlled trial will assess 13 weeks of therapy in approximately 160 children aged 3 to 12 with PMS. All participants will be eligible to continue treatment with NNZ-2591 for 12 months in an open-label extension trial, providing long-term safety and efficacy data for this investigational therapy.
Understanding Phelan-McDermid Syndrome
Phelan-McDermid syndrome is caused by a deletion or other change in the 22q13 region of chromosome 22, which includes the SHANK3 gene, or a mutation of the gene. The condition is also known as 22q13 deletion syndrome. The SHANK3 gene codes for the SHANK3 protein, which supports the structure of synapses between neurons in the brain.
The disorder affects an estimated 1 in 8,000 to 1 in 15,000 people and currently has no approved treatments available, representing a significant unmet medical need in the neurodevelopmental disorder space.
Regulatory Alignment and Funding
Neuren conducted an End of Phase 2 Type B meeting and a subsequent Type C meeting with the US FDA, at which alignment was reached on the design of the Phase 3 trial, including the primary efficacy endpoints. The trial program is fully funded by the company's existing cash reserves.
Company Background
Neuren Pharmaceuticals Limited focuses on developing new drug therapies for serious neurological disorders that emerge in early childhood, which currently have no or limited approved treatment options. The company has received 'orphan drug' designation in the United States, which provides incentives for developing therapies for rare and serious diseases.
Neuren's product DAYBUE™ (trofinetide) is approved for the treatment of Rett syndrome, and the company has licensed its development and commercialization to Acadia Pharmaceuticals Inc. NNZ-2591 represents the company's second drug candidate in development for multiple neurodevelopmental disorders.
