Neuren Pharmaceuticals triggers up Australian biotech investment

By Leon Gettler, Talking Business >>

THE TREMENDOUS SUCCESS of Neuren Pharmaceuticals (ASX:NEU), the only biotech worldwide that specialises in neurodevelopmental disorders – and best-known for developing Daybue, a world-first treatment for Rett syndrome – is sending a signal to venture capital in Australia to start investing in biotech.

Daybue was launched in the United States in April by Neuren’s licensee, NASDAQ-listed Acadia Pharmaceuticals. The partnership with Acadia was recently expanded from North America to global, with Neuren receiving an upfront payment of US$100m. Neuren receives milestone payments and quarterly royalties on all commercial sales. 

Neuren is now focused on developing a second drug, NNZ-2591, for an additional four rare neurological disorders. Neuren is conducting Phase 2 trials of its second drug candidate, NNZ-2591, for each of Phelan-McDermid syndrome, Angelman syndrome, Pitt Hopkins syndrome and Prader-Willi syndrome.

Neuren granted ‘orphan drug’ status in US

Topline results for Phelan-McDermid are expected December this year.  Recognising the urgent unmet need, all programs have been granted ‘orphan drug’ designation in the US. Orphan drug designation provides incentives to encourage development of therapies for rare and serious diseases.

Generally, venture capital has been reluctant to invest in biotech companies in Australia as it has been seen as too risky.

Neuren Pharmaceuticals CEO Joh Pilcher said that could change with success stories like Neuren. 

“People are going to invest if they see examples of success and we’ve had a few of those now,” Mr Pilcher told Talking Business.

“That shows people what the end value can be and that incentivises them to invest.

“Technology is moving forward the whole time. You might argue there’s less risk than there used to be because we know so much more about biology.”

World first treatment for Rett syndrome

Mr Pilcher said the major challenge for his company was being the first in the world to develop a drug to treat Rett syndrome, a serious neurological disorder that emerges in early childhood.

It affects every aspect of life including walking, talking, breathing, sleeping, hand use and cognitive function. It mainly affects girls and there’s never been any treatment for it.

The drug itself doesn’t fix the mutation. It is not designed to treat one particular symptom. Instead, it focuses on trying to improve the connectivity between the brain cells.

“That’s what’s wrong in Rett syndrome,” Mr Pilcher said. “The connection between the brain cells – and  the signalling that’s happening between them – is not happening properly.

“That’s what our drug is trying to improve and you see impacts across a number of different things. 

“We’re getting stories coming out of the community. Things like improved communication, improved ability to use the hands for purposeful things, even improvements in walking.

“It’s a very exciting time after a long journey.”

Neuren has been developing the drug to treat Rett syndrome for the last 10 years and it has been a huge but vital challenge for the $1.5 billon biotech company.

Mr Pilcher been with the company right from the start and he said he and his team had learned so much. 

“We’ve had to show motivation and determination to get through,” he said. “In some of those periods, we weren’t in the great position we’re in now – not much cash, not great market support – we had to make sure we got through despite that.

“And the other thing is nobody had done anything in Rett syndrome before. We weren’t following what anyone else did. We had to create the path for ourselves.”


Hear the complete interview and catch up with other topical business news on Leon Gettler’s Talking Business podcast, released every Friday at


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