https://www.theaustralian.com.au/business/companies/neurens-lead-work-earns-us-deal/news-story/73f9b702327651847626fca8f058c6b1?login=1

Neuren’s lead work earns US deal

Neuren executive chairman Richard Treagus. Picture: Stuart McEvoy

• EXCLUSIVE

  SARAH-JANE TASKER

  
  

  
• 11:00PM DECEMBER 12, 2018

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Melbourne-based drug developer Neuren Pharmaceuticals has led the world with its work on a rare genetic condition, which has earned it a $US465 million ($645m) deal, with further financial rewards tipped.

The Australian-listed company, which lists billionaire Lang Walker as a major shareholder, has flown under the radar of investors despite recently revealing that it licensed the North American rights of its lead drug, Trofinetide, to US company Acadia Pharmaceuticals.

The drug is being developed for two rare neurological genetic conditions, Rett syndrome and Fragile X syndrome.

“We have really been leading the way as a small, Australian-New Zealand company, in terms of developing a treatment for Rett syndrome,” Neuren executive chairman Richard Treagus said.

“We were the first company in the world to conduct a multi-centre randomised clinical trial in Rett syndrome in 2013 in the US.”

Neuren is focused on the development of orphan drugs — which treat rare conditions — for neurological conditions.

Rett syndrome, which affects about one in 10,000 girls, is a genetic defect on the X chromosome. The girls affected by the condition are wheelchair-bound, have intellectual disabilities and often have seizures.

Neuren completed a phase-two clinical trial for Rett syndrome in March last year and said its drug improved key aspects of the condition.

“We saw a general improvement in the general mood of the girls, their anxiety levels, their communication ability and their interaction,” Dr Treagus said.

“It really got the experts and ourselves very excited that in fact Trofinetide was potentially altering the course of this condition.”

The deal with Acadia will see the US company develop, manufacture and commercialise the drug for Rett and Fragile X, while Neuren will be paid milestones and royalties. Acadia gave Neuren $US10m on signing the deal and it also agreed to pay $US105m in developmental milestones, plus a further $US350m for sales milestones.

“One thing that people didn’t pick up on with this deal is the double-digit royalties we receive, which step up and deliver potentially immense value back to Neuren,” Dr Treagus said.

He said the key to the negotiation with Acadia was that his company had free and full access to all the clinical and regulatory data that Acadia generated.

Acadia also had a right to exclusive talks for a licence for the rest of the world, which it exercised. That exclusivity ends next month.

Dr Treagus said the development of the drug for Fragile X syndrome, which was about 18 months behind the development for Rett, could signal significant potential for Trofinetide.

He highlighted that Acadia recently said sales for Trofinetide in Rett syndrome could be more than $US500m a year.

He said there were about four times more patients with Fragile X than Rett.

“This (drug) has the potential to be a blockbuster in the US alone. The rest of world would be another half of the US sales again,” Dr Treagus said.

Positive news flow is a win for Neuren’s patient investors, which include property developer Mr Lang, who has been a cornerstone investor since 2011 with his 18 per cent stake.

“He has anchored the company for a number of years. He has been very supportive of the company and he understands that good things take time,” Dr Treagus said.

SARAH-JANE TASKER

REPORTER

Sarah-Jane Tasker has been a journalist for more than 20 years, having started her career on The Sunday Times in Perth. Her previous roles include deputy editor of the Kalgoorlie Miner in regional Western Austr... Read more