New molecule is one step closer to becoming a treatment for Phelan-McDermid syndrome

NewsGuard 100/100 Score

A molecule created at the University of Auckland is one step closer to becoming a treatment for an extremely rare and severely debilitating neurological disorder called Phelan-McDermid syndrome. Children with the disorder showed significant improvements in a phase two clinical trial in the US, Neuren Pharmaceuticals, which is listed on the Australian Securities Exchange, said in December.

Next steps would be a phase three trial and seeking approval from the US Food & Drug Administration. The molecule, NNZ-2591, comes from work years ago by Dr Jian Guan and Professor Sir Peter Gluckman at the Liggins Institute and Distinguished Professor Dame Margaret Brimble in her chemistry laboratory.

That's the same group whose work led to the breakthrough drug Trofinetide, approved last year for treating Rett syndrome, another rare genetic neurological disorder. The treatment is marketed under the name Daybue. 

Neuroprotective qualities of a peptide called cyclic glycine-proline (cGP) were investigated by Guan and Gluckman. In Brimble's lab, Dr Paul Harris produced NNZ-2591, a synthetic analog of cGP, which Neuren Pharmaceuticals hopes will become a Phelan-McDermid treatment. The University spun out a company to commercialize research, ultimately selling out.

Intellectual disability, low muscle tone, developmental delays and symptoms of autism can be among the features of Phelan-McDermid.

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. Likewise, NNZ-2566, the Trofinetide/Daybue molecule, may eventually be used for disorders beyond Rett syndrome.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment

While we only use edited and approved content for Azthena answers, it may on occasions provide incorrect responses. Please confirm any data provided with the related suppliers or authors. We do not provide medical advice, if you search for medical information you must always consult a medical professional before acting on any information provided.

Your questions, but not your email details will be shared with OpenAI and retained for 30 days in accordance with their privacy principles.

Please do not ask questions that use sensitive or confidential information.

Read the full Terms & Conditions.

You might also like...
Unveiling the key role of RNA modification in HIV-1 survival and replication