Neuren Pharmaceuticals (ASX:NEU) - CEO, Jon Pilcher
CEO, Jon Pilcher
Source: Neuren Pharmaceuticals
The Market Online - At The Bell

Join our daily newsletter At The Bell to receive exclusive market insights

  • Neuren Pharmaceuticals (NEU) submits an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for NNZ-2591
  • This application is being made to treat Phelan-McDermid syndrome which is a neurodevelopmental disorder associated with a deletion of chromosome 22
  • Following approval of the application, Neuren will undertake a phase two trial in children with Phelan-McDermid syndrome in the US
  • Neuren has filed an IND application for NNZ-2591 to treat Angelman syndrome and is in the process of finalising an application for Pitt Hopkins syndrome
  • Shares in Neuren are down a slight 0.98 per cent on the market and are trading at $2.03 at 12:57 pm AEST

Neuren Pharmaceuticals (NEU) has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for NNZ-2591.

This application is being made to treat Phelan-McDermid syndrome (PMS) which is a neurodevelopmental disorder associated with a deletion of chromosome 22, resulting in the loss of function of the SHANK3 gene.

The most common characteristics include intellectual disability, epilepsy, delayed or absent speech, motor delays, low muscle tone and autism with roughly 75 per cent of people with PMS diagnosed with autism.

Following approval of the application, Neuren will undertake a phase two trial in children with PMS in the US.

CEO John Pilcher is pleased with the milestone reached.

“This is another important milestone achieved in our plan to develop NNZ-2591 for multiple serious neurological conditions,” Mr Pilcher said.

“We are eager to start the phase two trial in children with Phelan-McDermid syndrome, which we hope will demonstrate the potential for NNZ-2591 to provide an urgently needed treatment option.”

Neuren has already filed an IND application for NNZ-2591 to treat Angelman syndrome and is in the process of finalising an application for Pitt Hopkins syndrome.

The aim of NNZ-2591 is to mimic the normal function of the brain and help control and normalise the biological process.

Shares in Neuren are down a slight 0.98 per cent on the market and are trading at $2.03 at 12:57 pm AEST.

NEU by the numbers
More From The Market Online

Market shrugs as Fisher & Paykel announces mass recall

Fisher & Paykel has initiated a voluntary limited recall of batches of Airvo 1 and my…
The Market Online Video

Brian Leedman’s BlinkLab to join ASX: AI-driven smartphone app screens for autism, ADHD

BlinkLab is a world-first, AI-driven digital healthcare venture, that uses a smartphone and facial recognition to…

Osteopore stock spikes 900% on Singapore and Vietnam approvals

Osteopore's (ASX:OSX) share price was up 38% after the regenerative medicine company received approval for its…

Amplia Therapeutics takes step up in pancreatic cancer treatment

Amplia Therapeutics is on to next step in its pancreatic cancer treatment research after a successful…