Health Check: Neuren says the US Rett syndrome market is a case of glass half full
Look out for a slew of advanced clinical trial results in early 2026
IDT Australia shares plunge up to 39% after CEO departs 'with immediate effect'
Despite firming US sales of its Rett syndrome treatment Daybue, Neuren Pharmaceuticals (ASX:NEU) says two-thirds of eligible patients are yet to try the drug.
Neuren's US partner Acadia Pharmaceuticals overnight reported quarterly net sales of US$96.1 million, up 14% year on year.
The Nasdaq-listed Acadia says a record number of patients received a shipment, growing for the third consecutive quarter.
Acadia holds the global rights to Daybue and pays royalties to Neuren, which initially developed the drug.
Affecting boys, Rett syndrome is a rare neurodevelopmental disorder resulting in slow development after the ages of six to 18 months.
Acadia has guided to net sales of US$380-405 million, which at the current exchange rate will generate $62-67 million of royalties for Neuren in calendar 2025.
Neuren earned $14.7 million in the June quarter, up 16% year on year. By their nature, royalties are pretty much pure profit.
More patients – and they're more persistent
Acadia says a record 987 patients received Daybue in the quarter, up from 954 in the March quarter.
Despite concerns about side effects, 50% of patients persisted after 12 months' treatment.
Neuren adds that 70% of active patients have now been on therapy for 12 months or longer. This was up from 65% previously.
The company says two-thirds of the 5500 to 5800 diagnosed US patients are yet to try Daybue.
The FDA approved the therapy in March 2023 and Acadia expects European assent in the March 2026 quarter.
Neuren shares this morning spurted as much as 8%. They've more than doubled since their mid-April trough of $8.60.
Investors spoil IDT's golden jubilee
Veteran contract drug maker IDT Australia's (ASX:IDT) golden jubilee has been spoiled by a savage share rout, resulting from the news that CEO Paul McDonald would depart 'with immediate effect'.
The company also issued a lacklustre trading update, flagging a boost in revenue for the year to June 30, 2025, but a widening loss.
A former Pfizer exec, McDonald had been in the job for almost three years. Chairman Mark Simari becomes executive chair while the company searches for a new CEO.
IDT says its full-year revenue should come in at $19.9 million, a 40% increase.
The company cites an increase in disbursement revenue. This derived from raw material costs and equipment charged to customers at a 'modest' margin.
IDT says mainly applying to new contracts, disbursements are a 'positive lead indicator'.
IDT also expects a net loss of $7.5 million, compared with a $5.4 million deficit last year. This reflects $1.2 million of bad debts from two customers defaulting on payments.
Founded in 1975, IDT has had a convoluted history, including missing out on a government funded Covid vaccine plant at the last minute.
These days IDT pursues gene technology, antibody drug conjugates, medical marijuana and psychedelic treatments for mental disorders.
The company is in the third year of its five-year strategy, spurred by a board 'refresh' in September 2022.
IDT's $30 million worth is backed by the value of the company's Boronia premises in eastern Melbourne.
This hard-to-replicate is in the books at a conservative $21 million.
We're on track, say drug developers
Investors can look forward to a slew of company-making trial results in 2026, according to presenters at this year's Bioshares summit in Hobart.
Alzheimer's disease drug developer Actinogen Medical (ASX:ACW) says it's on track to report interim results from its advanced phase IIb/III trial, by January next year.
The company is enrolling 220 patients for its Xanamia trial for mild to moderate sufferers, across 15 Australian and 20 US sites.
Actinogen's candidate Xanamem targets excess levels of cortisol in the brain – a novel mechanism of action.
The company expects to unveil data from the first 100 patients, after 24 weeks' treatment. Final results are expected in late 2026.
Stem cell developer Cynata Therapeutics (ASX:CYP) expects to release results of its phase III osteoarthritis (OA) trial between February and April next year.
Cynata's mesenchymal stromal cells modulate the immune system and enable tissue repair.
The OA program is the most advanced of Cynata's four trials that also cover graft-versus-host disease, diabetic foot ulcers and kidney transplants.
Vitiligo is a circa US$500 million market, says Clinuvel
The developer of an approved treatment for a rare sun intolerance disorder abbreviated as EPP, Clinuvel Pharmaceuticals (ASX:CUV) is advancing its proposed treatment for the more common vitiligo.
The company has fully enrolled its 210 patient phase III trial, with a readout due in the June half.
Affecting about 1% of the population, vitiligo is skin discolouration that results from the destruction of pigment-producing cells called melanocytes.
The late Michael Jackson was a celebrity sufferer.
If the drug gets US Food and Drug Administration (FDA) approval, the company estimates US$490-570 million of revenue in years one and two.
Put in context, Clinuvel reported revenue of $35.6 million in the 2024 December half, up 10%. These sales derived from the company's EPP treatment, Scenesse.
Doing the numbers on Imricor
Broker Canaccord reckons Imricor Medical Systems (ASX:IMR) could be worth more than twice its current valuation – but faces constrained hospital budgets for the time being.
In a 60-page appraisal of the cardiac ablation catheter developer, the firm estimates 120 US hospitals could be using the device within five years.
Assuming 35% penetration of the ablation procedures, this equates to a $2.50 per share valuation compared with around $1.40 now.
'The big question ... is how quickly can these installs happen,' the firm says.
Uniquely, Imricor's catheters are guided in real-time with magnetic resonance imaging – MRI – rather than via x-ray.
The procedure is called interventional cardiac magnetic resonance cardiac ablation (ICMR CA).
The company initially is focused on the US$12b arrhythmia market.
Canaccord believes Imricor is still 12-18 months away from a 'material revenue ramp'.
But after that it's off to the races.
Imricor has an initial US target market as 120 large academic hospitals, 20 of them high volume.
The firm estimates that facilities will install 190 ICMR US labs globally five years post launch.
Of these, 100 will be in the US with total revenue of US$250 million.
Imricor currently is undertaking a trial aimed at FDA approval for atrial flutter procedures, with a decision by the end of 2026.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
SBS Australia
a minute ago
- SBS Australia
No new taxes before the next election, declares PM
No new taxes before the next election, declares PM Published 7 August 2025, 9:03 am The Prime Minister has all but ruled out any tax changes before the next election. There have been competing pitches for reforms ahead of the government's upcoming economic roundtable. One proposal is increase to the GST, but with every Australian to receive a cashback compensation.
SBS Australia
5 minutes ago
- SBS Australia
The effects of scrolling social media on your brain
Scrolling social media is a guilty pleasure ... that can quickly spiral into distraction and people are certainly becoming more aware of it. "Definitely scroll on Instagram way too frequently, way more than I need to, yeah it's in the back of my mind but I'm not super conscious about it." "You just lose focus for even a second - you just go in there - it's like a doom scroll, you start doom scrolling for an hour or something, so you're just unproductive after that, that's it." It's a new age challenge - with an impact that's yet to be fully understood . Dr Alexandra Gaillard of Swinburne University says her new research sought to explore the effects of screen time on the brain. "The main takeaways that our research really showed that time spent on social media may be a waste of resources for the brain can lead to decreased focus and increase in stress." The research has found 18 to 25 year olds exposed to phone screens for just three minutes, experienced changes in mood, energy, tension, focus and happiness. The pilot study of 27 people compared responses to gaming, television and social media on these devices. Dr Gaillard said this tracked brain activity by using small electrodes that track oxygen levels. "With social media there was the biggest increase in blood flow to those regions, but it doesn't actually get used - compared to something like gaming where a lot of blood goes to the region but it's actually used up, people engaging there's problem solving, they're doing things with that oxygen and that brain area." The findings indicate even short periods of screen time can have measurable effects. But Professor Michael Dezuanni of Queensland University of Technology says the full impact on young people is a nuanced and evolving field of research. "We need to think about how social media can have negative impacts on young people, but when it comes to learning - we can't just have a kind of generalised view." From December the 10th, a federal government ban on under 16s accessing social media takes Professor Suzanne Schweizer of UNSW Sydney says it's important efforts continue to better understand the impact of social media. "Because young people spend so much time interacting online - we need to know does it actually influence their brain development and if so again how so those are reasons why it's important that we invest more in experimental research."
News.com.au
12 minutes ago
- News.com.au
How big government is making life more expensive
We're in trouble … Right now in Australia, more than half of working-age adults get their main source of income from the government. That includes from JobSeeker, family payments, disability support, and more, as well as from income in public sector jobs. According to The Australian Financial Review, we're at the highest level of dependency since World War II. Meanwhile, the shape of the job market is shifting dramatically. According to The Australian, 82 per cent of all jobs created over the past two years were government-funded positions. In 2024, the private sector added just 53,000 jobs, while public employment surged at five times the normal rate. Economists are warning this is creating serious distortions in the labour market and driving Australia's ongoing productivity slump. But here's the worst part. To sustain this level of income support and public hiring, the government has to dramatically increase both taxes and spending. And that's what they've done. Total tax revenue hit a record $801.7 billion in 2023–24, or nearly 30 per cent of GDP – the highest in modern history. Income tax alone is expected to rise above $343 billion by 2025–26. That's a bigger tax burden on individuals and businesses than we've seen in decades. But it hasn't just been funded through tax. Over the past decade, the Reserve Bank and the government have massively expanded the money supply. Australia's M3 broad money measure – essentially the total pool of cash in the system – has grown to more than $3.15 trillion, up 80 per cent since 2015. That's more money in the economy, chasing the same supply of goods, housing, and services. And that's exactly what causes inflation. We're seeing that play out in everyday prices – at the supermarket, in energy bills, and most of all, in housing. Government overspending has helped weaken the Australian dollar. That makes imported goods and building materials more expensive – driving up construction costs. Meanwhile, inflation has pushed investors toward property as a store of value, sending demand – and prices – even higher. So, while the government talks about 'affordability,' its own policies are fuelling the very forces that make housing less affordable. And the pressure isn't just on prices. It's on productivity. To keep the machine running, the government is taxing more – especially on income, savings, and investment. But high taxes make it harder for people to start a business, grow one, or take financial risks. The result? Investment slows. Innovation stalls. Businesses hesitate. And Australia's ability to actually produce more – to meet all this demand – keeps falling behind. That's when inflation really sticks: when demand is pumped up by public spending, but supply can't keep up. Prices rise faster, and stay higher. According to former Treasury Secretary Ken Henry, Australia's poor productivity performance over the past 25 years has cost the average worker around $500,000 in lost income. And the Centre for Independent Studies puts it plainly: 'Australia's prosperity depends on private sector-led growth – not government spending propping up demand.' But right now, that's exactly what we're doing. We're building an economy on dependence – not on productivity. We're growing jobs in government – not in business. We're taxing more – but getting less. And we're printing money – instead of creating value. This isn't just about budgets or ideology. It's about whether we build an economy that rewards effort, innovation, and work – or one that makes us more dependent, more taxed, and more stuck. And right now, we're heading down the wrong path.
