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.

LTR Pharma’s latest news is easy to swallow

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.