One of the highest growth ASX 200 healthcare stocks on the market went into a trading halt on Thursday.
Neuren Pharmaceuticals Ltd (ASX: NEU) requested the trading halt before the market open today.
The company said it wanted time to analyse data from its Phase 2 clinical trial of its second drug candidate, NNZ-2591, in the treatment of Pitt Hopkins syndrome.
Neuren intends to prepare a statement announcing the top-line results shortly.
The ASX 200 healthcare stock will remain in a trading halt until either the announcement is released or trading commences on Monday.
What is Pitt Hopkins syndrome?
Neuren develops drugs for serious childhood neurological disorders that have no or limited approved treatments.
All of its drugs have 'orphan drug' designation in the United States, which gives Neuren access to incentives to support its work. It also has an orphan drug designation for NNZ-2591 in Europe.
Pitt Hopkins syndrome (PTHS) is a neurodevelopmental condition that causes developmental delays.
It causes moderate to severe intellectual disability, hyperventilation and/or breath-holding while awake, seizures, gastrointestinal issues, speech difficulties, and sleep disturbances.
Sufferers often have distinctive facial features and are sometimes misdiagnosed as suffering from autism.
Neuren says PTHS is caused by the loss of one copy, or a mutation, of the TCF4 gene on the 18th human chromosome. The incidence of PTHS is estimated at between 1 in 11,000 people and 1 in 41,000 people.
Previously, Neuren tested NNZ-2591 on mice with the tcf4 mutation for six weeks. The company said its drug "normalized the deficits in all the tests for hyperactivity, daily living, learning and memory, sociability, motor performance and stereotype".
ASX 200 healthcare stock up 40% in 6 months
Neuren Pharmaceuticals has been a barnstorming stock of the ASX 200 over the past two years.
It was the ASX 200 healthcare stock of the year in 2023, with its share price skyrocketing 214%.
The road has been a little more challenging for the Neuren Pharmaceuticals share price over the past six months. However, it is still up by 39.56% to $20.71 today.
The latest news from Neuren Pharmaceuticals
Prior to today's trading halt announcement, the last piece of price-sensitive news we received from Neuren Pharmaceuticals was on 9 May.
That's when the company released its Q1 FY24 update on sales of its maiden drug, Daybue.
Daybue is a world-first drug treatment for Rett syndrome. It's approved in the US for adults and pediatric patients aged two years and up.
ASX investors appeared underwhelmed by the progress of Daybue sales in the US. As a result, the ASX 200 healthcare stock lost 3.93% on the day of the release.
Q1 net sales of Daybue in the US totalled US$75.9 million, just missing the guidance range of US$76 million to US$82 million. It was also lower than the previous quarter's net sales of US$87.1 million.
Neuren blamed the fallen sales on seasonal factors, including refills that were due in January and actioned in December before the holidays, and reduced Rett clinic days in January.
The full-year 2024 guidance for net sales is between US$370 million and US$420 million.
Another challenge for this ASX 200 healthcare stock over the past six months was a short seller's report in February, which described Daybue as a "flop" amid "horror stories" of side effects.
Neuren's response failed to stop the ASX 200 healthcare stock from losing its upward trajectory from there.
