Neuren Pharmaceuticals (ASX: NEU) is getting interesting results from a mouse trial of a treatment for Phelan-McDermid syndrome (PMS), a rare genetic illness which exhibits symptoms of conditions including autism and epilepsy.

The study found that a 4x mg/kg dose, the third highest of four levels, was the optimum level in the mouse model, but researchers witnessed a clear response from four escalating dose levels.

The mouse trial was conducted in the shank3 knockout mouse model, a method in which the shank3 gene is deleted to mimic Phelan-McDermid syndrome in humans. As well as causing PMS, disruption of the shank3 gene is thought to be associated with a large number of cases of autism spectrum disorder.

Wild type mice and knockout mice were treated with either a placebo or one of four escalating doses of NNZ-2591 for six weeks.

The study will form the backbone of planned human clinical trials.


Neuren achieved consistent results across all eight behavioural tests of anxiety, repetitive behaviours, memory, learning, sensory motor function, sociability and daily living skills, and seizure incidence.

If you’re wondering how a mouse’s daily living skills are measured, Neuren outlined how it was measuring each behavioural tic.

Anxiety was tested via distance travelled, repetitive behaviour was around self-grooming, learning was time spent with a new object, sociability was time spent with a new mouse, and living skills was around marble burying.

There is currently no treatment for PMS and Neuren has Orphan Drug designation from the US Food and Drug Administration for its drug candidate, as it does for Angelman syndrome and Pitt Hopkins syndrome as well.

Neuren is developing new therapies for neurodevelopment disorders with a high unmet need, using synthetic analogs of neurotrophic peptides that occur naturally in the brain.

Neuren’s lead drug candidate trofinetide is currently in a phase three clinical trial for Rett syndrome, for which it received an extra designation of Rare Pediatric Disease this week, and has completed a phase two clinical trial in Fragile X syndrome.


In other ASX health news:

Dimerix (ASX:DXB) has appointed a new medical advisory board ahead of the read out of two phase two clinical studies in its DMX-200 renal program, due to be completed in mid-2020. The new board will assist with the analysis and interpretation of data and with the design of future studies, including the pivotal phase three study for Focal Segmental Glomerulosclerosis (FSGS), for which Dimerix received guidance from the FDA in November 2019.

Yesterday John Sharman resigned as CEO of Medical Developments International and today has resurfaced as CEO of Universal Biosensors (ASX:UBI), where he will be paid $480,000 a year.

Oncology company Kazia Therapeutics (ASX:KZA) has finished recruiting patients for its phase II study in glioblastoma, using its lead candidate paxalisib, and says new interim data will be presented at American Association for Cancer Research in April.