Posted: 14 November 2023
Neuren Pharmaceuticals (ASX: NEU) today announced that all study visits are complete in its Phase 2 clinical trial of NNZ-2591 in Phelan-McDermid syndrome (PMS). Top-line results from the trial are expected to be available in December 2023.
Neuren CEO Jon Pilcher commented “We are grateful to all the people at the trial sites in the United States and in the PMS community who have enabled this groundbreaking trial to be completed as we strive to develop a potential first therapy for PMS. We look forward to releasing next month the first results of treatment with NNZ-2591 in children with PMS.”
Neuren is also conducting Phase 2 clinical trials of NNZ-2591 in children with three other neurodevelopmental disorders – Pitt Hopkins syndrome, Angelman syndrome and Prader-Willi syndrome. All four programs have been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and are being developed under Investigational New Drug (IND) applications. Each syndrome is a seriously debilitating neurological disorder that emerges in early childhood and has no or limited approved treatment options.
About 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. PMS is also known as 22q13 deletion syndrome. The SHANK3 gene codes for the shank3 protein, which supports the structure of synapses between nerve cells in the brain. It is estimated that between 1 in 8,000 and 1 in 15,000 people have PMS. There are no medications, drugs, or therapies specifically for PMS, which has an overwhelming unmet medical need.
PMS has severe quality of life impacts on those living with it, as well as on parents and siblings. The most common characteristics are moderate to severe developmental and intellectual impairment and developmental delay, delayed or absent speech, symptoms of autism, low muscle tone, motor delays, mild to severe epilepsy, difficulties with toilet training and problems with eating.
Further information about PMS is available at: www.pmsf.org
