Neuren Pharmaceuticals completed all study visits as part of its Phase 2 clinical trial for Phelan-McDermid syndrome, with top-line results expected in December.
The trial targeted PMS, which results from a mutation or change in the 22q13 region of chromosome 22 and affects between 1 in 8,000 and 1 in 15,000 people.
The company is working on potential treatments for PMS using its second drug candidate, NNZ-2591.
Neuren is also carrying out Phase 2 trials of NNZ-2591 in children with three other neurodevelopmental disorders.
The programs have all been granted Orphan Drug designation by the US Food and Drug Administration.