Neuren Media and Analyst Coverage, page-554

Even made it to the ABC…

2m ago

By Michael Jan

ASX-listed Neuren Pharmaceuticals is the leader among the top 200 companies, surging almost 25% to $21.38, after releasing an update on clinical trials for its catchily named drug NNZ-2591.

This drug is in phase 2 development with hopes of treating some rare but very serious neurodevelopmental disorders.

The latest results come from a trial of NNZ-2591 among 18 children in the US with Phelan-McDermid syndrome (PMS), where a deletion or other change in a part of chromosome 22 affects the SHANK3 genethat codes for a protein that supports the structure of brain synapses.

The company says people with the condition typically exhibit severe developmental and intellectual impairment, including delayed or absent speech, symptoms of autism, low muscle tone, motor delays, epilepsy, difficulties with toilet training and problems with eating.

Neuren has reported that the drug was "well tolerated and demonstrated a good safety profile", while both caregivers and clinicians reported "clinically meaningful" improvements across a range of measures in the vast majority of trial patients.

From a business perspective, the company said approximately 1% of people with autism have a SHANK3 mutation, meaning there are likely to be around 160,000 potential patients across the US, Europe, Japan, urban China, Brazil, Israel, South Korea, New Zealand and Australia.

The company hopes the same drug will also prove useful in the treatment of three other even rarer conditions — Pitt Hopkins, Angelman and Prader-Willi.