This $1.3b biotech is still run out of a spare bedroom in Melbourne
The chief executive of Neuren Pharmaceuticals has overseen two capital raisings and the rapid growth of the company, which has developed the world's first approved drug to treat Rett syndrome, a neurological disorder disrupting signalling between brain cells mainly in young girls.
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News.com.au
07-08-2025
- News.com.au
Health Check: Neuren says the US Rett syndrome market is a case of glass half full
Neuren's US partner Acadia reports a 14% sales boost 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.
News.com.au
03-06-2025
- News.com.au
Biocurious: Inspired by Neuren, Nyrada aims to change the treatment landscape for brain and heart disorders
Nyrada is focused on a particular ion pathway to tackle disorders including stroke and traumatic brain injury Preclinical work has shown a significant reduction in secondary brain injury and cardiac damage CEO James Bonnar spent 15 years at Neuren Pharmaceuticals in the company's formative stage Nyrada (ASX:NYR) CEO James Bonnar is too modest to admit it, but he played a key role in developing and commercialising Neuren Pharmaceuticals' (ASX:NEU) neurological drug Daybue. Bonnar was one of the then New Zealand-based Neuren's first employees and spent a decade at the company, which now bears a $1.7 billion market cap. Bonnar seeks to channel Neuren's virtues as Nyrada pursues small molecule treatments for both brain and heart protection. Before Neuren, Bonnar was with Protemix, which worked on a drug for diabetes-related cardiomyopathy. 'This is not my first rodeo." Nyrada's indications of interest include traumatic brain injury (TBI), which Neuren pursued before switching development to the childhood disorder Rett syndrome. 'You learn a lot of lessons along the way, competing in both successful and unsuccessful programs,' Bonnar says. Have a nice TRPC Nyrada's lead drug was called NYR-BI03, but last week the company bestowed the 'proper' name of Xolatryp to reflect the drug's 'advancing development'. The science is based on bodily transient receptor potential canonical (TRPC) ion channels (often pronounced 'tripsy'). These conduits regulate calcium influx into cells. 'Following ischemia or injury, these ion channels are activated and let in calcium into the cell,' Bonnar says. 'That calcium builds up to the extent where they're toxic.' As with Neuren, Nyrada had a spare clinical program in its back pocket. The Delaware-incorporated Nyrada listed in 2020, having spun off from immunology play Noxopharm (ASX:NOX). Nyrada initially focused on oral inhibitors which failed preclinical toxicology studies. 'Fortunately, we had a promising secondary program, which is now the lead program," Bonnar says. Xolatryp blocks TRPC channels to protect key cells in vital organs when under stress. In the case of brain neurons, that could be after ischaemia (a blood clot) and reperfusion (the sudden flow of blood returning to an organ or tissue). Ischemic-reperfusion injury is also a key cause of tissue damage following the restoration of blood flow to the heart post-injury. It's a knock-out! Using a 'knock out' mice model, pioneering work at the University of NSW (Uni NSW) showed the rodents were protected from stroke. The 'knock out' doesn't refer to concussing the critters with a mallet, but genetically modifying and cross breeding them so they no longer express the TRPC channels. 'This confirmed that completely knocking out the channel is safe and for all intents and purposes these mice lived a normal life,' Bonnar says. Over the last 12 months, Nyrada has focused on expanding efficacy data that builds on this 'knock out' mouse work. 'We went through a few iterations of molecules that showed promise, but they weren't target specific,' Bonnar says. Let's get clinical Preclinical work suggests Xolatryp is delivering significant reduction in secondary brain injury and cardiac damage For cardiac reperfusion, rat models showed an 86% reduction in injury (there are no other approved treatments). With ischemic strokes, a mouse study showed 42% protection. The company also ran a neuroprotection study with the Walter Reed Army Institute Research and Uni NSW, deploying a TBI model. Based on follow-up magnetic resonance imaging, this showed a 'statistically significant' neuroprotective benefit. Now for a human study As they say, everything works in mice … or rats. Nyrada's attention now has turned to a phase I healthy volunteer study to appraise dosing safety. The randomised, double blinded effort involves three-hour infusions, compared with 20 hours for the first preclinical study. The study consists of three eight-patient cohorts, six on the active drug and two on the placebo in each group. Bonnar says the trial will guide the company on what indication to pursue in a phase II study. 'In each one, there is first-in-class potential with large markets and unmet need.' The company is hoping to complete the study in September. Later, the company also would like to run a TBI study in the US, given the large addressable population. 'You would struggle to do that in Australia,' Bonnar says. Tackling a barren treatment landscape Bonnar describes Xolatryp as a 'Swiss Army knife' drug candidate, in that it could have broad uses. He says the current TBI treatments only alleviate the symptoms, rather than the condition. 'It's a similar situation with ischaemic stroke, but not quite so bad.' While clot-busting drugs work effectively, they need to be administered in a narrow time window (the 60 minutes after a stroke is called the Golden Hour). Given that, they only apply to 10-15% of patients. As for cardio protection, existing drugs can reduce the ticker's workload after a heart attack, but none of them protects the heart tissue. Can Nyrada 'do a Neuren?' Despite Xolatryp's snazzy name, Nyrada faces many years and development dollars before it 'does a Neuren'. Of course, most drug developers tripsy-up before reaching the end game. Bonnar dubs TRPC as a 'hot' research area reflected in Nyrada's share price: up 68% over the last month and 160% over the last year, ascribing a $38 million market cap. 'Big Pharma is looking at cardio protection in particular and we think our novel molecule … should attract interest,' Bonnar says. At the end of March Nyrada had $4.7 million of cash, enough to fund the phase I study. 'We are pretty lean; we work as a virtual company with a low head count.' Bonnar notes Boehringer Ingelheim has developed a TRPC inhibitors for renal fibrosis, while elsewhere work is afoot to develop one for the chronic condition of cardiac hypertrophy. 'But as far as I'm aware, we are the first company to be looking at a TRPC channel blocker in the clinic in our chosen indications.'
News.com.au
08-05-2025
- News.com.au
Health Check: Neuren says more patients are staying the course as US Daybue sales lift
Neuren says the number of patients discontinuing its Rett syndrome drug has fallen by two-thirds over the last year LTR Pharma launches a secondary program for swallowing disorders Telix targets throat – and other - cancers Neuren Pharmaceuticals' (ASX:NEU) global marketing partner Acadia has reported strong March quarter sales of the duo's treatment Daybue in the US, for the rare neurological disorder Rett syndrome. More heartening is a drop-off in patient discontinuation rates – a sensitive topic given the drug's known side effects (diarrhoea, vomiting, possible weight loss). A rare genetic disorder evident mainly in girls, Rett syndrome affects brain development, resulting in slower development and often a shorter lifespan. The Nasdaq-listed Acadia disclosed net sales for the quarter of US$84.6 million ($130 million), 11% higher than a year previously. Sales were 15% lower than the December quarter, owing to seasonal variations and a small cut in the per-bottle price, as determined by the US Medicare system. Acadia has the US rights to Daybue, on which Neuren generates royalty income. Neuren pocketed royalties of $13.5 million, up 17%. Acadia has stuck with its calendar 2025 sales guidance of US$380-405 million, implying $62-67 million of royalty income for Neuren. Acadia says a record 954 patients received Daybue, with discontinuations down 35% on the December quarter and 66% year on year. Put another way, more than 50% of patients have continued the therapy at 12 months, while 65% of 'active patients' have been on the drug for 12 months or longer. Neuren says there's still plenty of untapped potential, given two-thirds of the 5500 to 5800 diagnosed Rett patients are yet to try the drug. Meanwhile, Neuren expects European approval in the March 2026 quarter, with pending distribution agreements to sell in certain Latin America, Middle East and Asia Pacific geographies. Neuren was due to present at Macquarie's corporate jamboree today. The developer of a nasally delivered spray treatment for erectile dysfunction, LTR Pharma (ASX:LTP) has also turned its mind to a secondary program. LTR has signed a development agreement to develop Oroflow, a spray for oesophageal motility disorders (OMD) – a group of conditions that cause impaired swallowing (dysphagia). The compact is with Strategic Drug Solutions of the US. As well as not being able to down solids and liquids, OMD patients suffer regurgitation, chest pain and sometimes weight loss. Substandard current treatments include dilation, surgery, or Botox injections. Oroflow aims to deliver relief in 10 minutes. 'There is a clear need for effective, non-invasive treatment options that can provide rapid relief for patients with swallowing difficulties,' executive chairman Lee Rodne says. The company values the OMD market globally at US$4.49 billion, with the US accounting for US$1.94 billion. The condition affects about 10.8 in every 100,000 people, but for unexplained reasons the incidence is higher in Australia (16.9). We blame all the flies. Telix's side program could be FAP-ulous Nuclear medicine house Telix Pharmaceuticals (ASX:TLX) also reports promising results from an acquired secondary program – with a throaty theme as well. The program pertains to the fibroblast activation protein – FAP – as expressed by several cancers. As reported in the American Thyroid Association's official journal, unsurprisingly monikered Thyroid, FAP showed 'encouraging efficacy' against aggressive thyroid cancer in a 73-patient study. The heavily pre-treated patients showed median progression-free survival of 29 months, which meant their tumours didn't get bigger. Overall survival was 32 months, with a 'manageable' safety profile. Telix last year acquired the FAP program, dubbed TLX-400, from its inventors at Germany's Johannes-Gutenberg University. The terms were €10 million ($17.6 million) cash and €132 million of milestones if the program progresses as either a diagnostic tool or a therapy. Telix chief medical Dr David Cade says the results compare positively to the current treatment – tyrosine kinase inhibitor drugs – which can have nasty side effects. The company is eyeing multiple solid tumour types, including bladder cancer. Telix has two approved prostate cancer diagnostics on market and is angling for US Food & Drug Administration (FDA) consent for a kidney cancer diagnostic. A Roxsta moment for Memphasys Last week we mentioned Memphasys (ASX:MEM) and its successful trial of its sperm separation device for assisted reproduction purposes, Felix. There's another leg to the Memphasys tale: its Roxsta device that measures antioxidant levels in human and animal biological samples. Memphasys today said various pilot studies had shown Roxsta's ability to perform mass testing in various contexts, including oxidative stress in cattle and antioxidant levels in professional footballers. In the case of the latter, the technology 'demonstrated the capacity to process 96 samples in less than an hour, delivering highly accurate data in a fraction of the time compared to traditional methods.' Currently, commercially available methods can take up to 16 hours to deliver a single result, while Roxsta can do in around three minutes. 'To date, much of the focus has been on smaller-scale assessments of Roxsta, ensuring the results are accurate and the device is capable of being deployed in a commercial setting,' CEO Dr David Ali says. Memphasys shares gained 40% – or two-tenths of a cent – this morning. Robot says 'yes' on the best drug combos Why carry out expensive drug trials if a robot can do the job instead? We're being a tad facetious, but minnow Algorae Pharmaceuticals (ASX:1AI) highlights the potential for artificial intelligence to short-cut the initial stages of drug development. Algorae has partnered with the Victorian Centre for Functional Genomics, an arm of Melbourne's Peter MacCallum Cancer Centre, to validate AI-predicted drug interactions in oncology. Alogorae has developed AlgoraeOS, a machine learning tool to identify the best drug combinations. The study will focus on 24 AI-generated drug candidates, which are tested for synergies across four cancer cell lines (glioblastoma, pancreatic, breast and prostate). The company expects to release preclinical data within six months.
