Neuren Pharmaceuticals (ASX:NEU) has announced the initiation of the first investigational site in the US for its Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome (PMS), after receiving Institutional Review Board approval.
The company said other trial sites in the US are at various stages of the initiation process.
This is the first-ever Phase 3 trial in PMS, which is a severe neurodevelopmental disorder with no approved treatments.
PMS 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. PMS 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. It is estimated that between 1 in 8,000 and 1 in 15,000 people have PMS.
The randomised, double-blind, placebo-controlled trial will assess therapy for 13 weeks in approximately 160 children aged 3 to 12 with PMS. All participants may be eligible to continue treatment with NNZ-2591 for 12 months in an open-label extension trial.
Neuren said the trial program is fully funded by its existing cash reserves. 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.