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.
Hashtags
Try Our AI Features
Explore what Daily8 AI can do for you:
Comments
No comments yet...
Related Articles
ABC News
2 hours ago
- ABC News
Female tennis players undergoing fertility procedures to get protected ranking
Female players who choose to undergo a fertility protection procedure will be allowed to take time away from the sport and return to competitive action with a protected ranking, the governing body of women's tennis has said. The new rule aims to support women athletes to balance their family goals and career ambitions, and comes three months after the WTA offered players up to 12 months of paid maternity leave for the first time. "The new rule means that players can now take time away from professional tennis for a fertility protection procedure, such as egg or embryo freezing, and safely return to competition with a protected ranking," the WTA said in a statement on Wednesday. "Eligible players will receive a Special Entry Ranking (SER), which can be used to enter up to three tournaments, based on the 12-week average of their WTA Ranking from eight weeks prior to the start of their out-of-competition period." Sloane Stephens, the 2017 US Open champion, has previously called for egg freezing to be recognised as a protected ranking activity, and called Wednesday's announcement a "ground-breaking" move. "I'm incredibly proud of our sport in recognising the importance of fertility treatments for female athletes. For any woman, the conversation of family life versus a career is nuanced and complex," the former world number three said. "The WTA has now created a safe space for players to explore options and to make the best decisions for themselves." The WTA also said players will benefit from paid maternity leave and grants for fertility protection through the WTA Maternity Fund sponsored by the Saudi Public Investment Fund.
SBS Australia
20 hours ago
- SBS Australia
Mass arrests in LA as an emergency curfew comes into effect
The Los Angeles Police Department says officers have conducted mass arrests, after a curfew was implemented in the city. In a statement, the LAPD says several groups of protesters are still gathered in a designated curfew zone, prompting them to conduct mass arrests. The city's mayor, Karen Bass, who issued the curfew, has urged the Trump administration to end the immigration raids. Speaking earlier at the airport, US President Donald Trump insists that it's necessary to keep National Guard troops inside the city. "A very simple rule of engagement. If they're dangerous, if they're throwing concrete or bricks, if they're spitting in the face of the police or whoever's in front of them, if they're punching people, if they're doing all of the things that you see done for the last three nights, that I would say is engagement. We need to keep our city... I want to save Los Angeles. And (Governor Gavin) Newsom is totally incompetent. He's not going to... Look at the fires he had." The regulation of Australia's fertility sector will be discussed at a meeting of state and territory health ministers on Friday, after a second instance of an error during an embryo transfer done by Australian fertility group Monash IVF. In a statement to the stock exchange, the company says it transferred the wrong embryo to a patient on June 5 at the company's Clayton laboratory in Melbourne. Monash IVF has issued an apology to the impacted couple - and is also conducting an internal investigation. The Victorian Health Regulator says it has also begun an external investigation into how the error occurred. Assistant Minister for Social Services Ged Kearney says the Federal Health Minister Mark Butler is closely watching the case. "Well this is something that simply shouldn't happen, and my heart goes out to the people affected by the situation. Really it's a tragedy in some ways. It's unacceptable, and it shouldn't have happened, let's just put that on the table. And I know that Minister Butler is very interested in this, and has called for this to be on the agenda of the health minister meeting, which is actually happening on Friday, very timely. So it would be an important issue to be discussed there." Negotiations for a Treaty between Victoria's Indigenous peoples and the state government resumes today. Victoria became the first jurisdiction to begin work on Treaty with its Indigenous peoples more than eight years ago, with official treaty negotiations began last year in November. First Peoples' Assembly Co-Chair, Ngarra Murra says she is looking forward to building on the progress made so far on issues including: the ongoing representation of First Peoples in Victoria; an accountability mechanism to ensure policies and programs are achieving positive outcomes for First Peoples; and continuing Truth-Telling beyond the conclusion of the Yoorrook Justice Commission's work. Australia is one of the only Commonwealth countries that has not signed a federal treaty with First Nations peoples. Business and industry groups have welcomed the prime minister's announcement that the government will hold a productivity forum in August this year. Anthony Albanese announced the round-table late on Tuesday, saying it will bring together unions, civil society and business groups to exchange ideas. Australian Industry Group Chief Executive Innes Willox says the sector hopes the meeting will help spur private investment. Australian Chamber of Commerce and Industry CEO Andrew McKellar says an ambitious productivity agenda is vital for the nation's future. "Without productivity growth, you can't have sustainable increases in living standards. That's the only way that you're going to get real wages increasing without it being chewed up by inflation. It's about taking the pressure off small business, more time for business, reducing red tape, the impact of regulation, ensuring that they've got technologies that make it easier to work. So it's of benefit to employees and to businesses." In sports, Spanish cyclist Iván Romeo has taken the overall lead at the Criterium du Dauphiné with a stunning solo victory on Stage 3. In the second win of his career, Romeo completed the hilly run across the southeast of France in 4 hours 34 minutes 10 seconds. The 21-year-old says he will be looking to maintain his advantage in the upcoming 17.4-kilometre time trial. "I'm going go for it for sure. I hope yellow will give me wings because I will need them. We will see. I am here for Enric (Mas). We are going to do everything for him to put him on the podium. I think this is good for the team to boost their confidence, so I hope the week keeps this way."
News.com.au
a day ago
- News.com.au
Doctors' warning about ‘insidious' social media trend
It's no secret that young girls are obsessed with beauty — just look at the popularity of 'get ready with me' videos on social media. In a survey last year, more than three in four parents reported that their seven to 17-year-old girls have a 'skin care routine'. A groundbreaking new study out of Northwestern University warned that these girls are shelling out a lot of money for skin care that may cause redness and not protect them from the sun's harmful rays, New York Post reported. 'It's problematic to show girls devoting this much time and attention to their skin,' said corresponding study author Dr. Molly Hales, a postdoctoral research fellow and a board-certified dermatologist at Northwestern University's Feinberg School of Medicine. For this study, Hales and another researcher created their own TikTok accounts under the guise that they were 13. They collected 100 unique videos suggested in the 'For You' tab. They analysed the demographics of content creators, the products used and the total cost of routines, finding that girls 7 to 18 are applying an average of six facial products at once. Some girls use more than a dozen. Researchers estimated that these kids fork out an average of $A257 for about a month's worth of products. In the jaw-dropping cases, they are spending more than $A765. The products in the top viewed videos contained 11 active ingredients on average. In one clip, a creator rubbed 10 products on her face in six minutes. 'As she's applying the products, she begins to express discomfort and burning, and in the final few minutes, she develops a visible skin reaction,' said senior study author Dr. Tara Lagu, an adjunct lecturer of medicine and medical social sciences at Feinberg. Hales noted that the irritation stems from using products with clashing active ingredients, as well as applying the same active ingredient over and over again, not knowing it's in several products. Girls also face the risk of sun sensitivity and a skin allergy known as allergic contact dermatitis, which causes a rash. Only 26 per cent of daytime skin care regimens included sunscreen, the Northwestern study found, even though it's key to preventing skin cancer. The research — billed as the first peer-reviewed study to explore the pros and cons of teen skin care routines shared on social media — was published Monday in the journal Pediatrics. Beyond the health risks, mental distress is also a concern. Beauty videos on social media can contribute to lower self-esteem and pressure to conform to unrealistic beauty standards. Lagu noted that many videos 'emphasised lighter, brighter skin.' 'We're setting a very high standard for these girls,' Hales said. 'The pursuit of health has become a kind of virtue in our society, but the ideal of 'health' is also very wrapped up in ideals of beauty, thinness and whiteness,' she added. 'The insidious thing about 'skincare' is that it claims to be about health.' TikTok is only for users 13 and older, a rep reminded CNN, and creators who are too young are removed from the platform. The company also works with third-party adolescent development experts and doctors to establish safeguarding policies, the spokesperson added.
