Latest news with #Amplia
News.com.au
23-07-2025
- Business
- News.com.au
Health Check: Aussie biotechs are navigating a US regulatory minefield
Telix faces unwanted US regulatory attention – but it's not alone Amplia raises $25 million, with another $2.5 million to go A home-grown uni biotech innovation shines again Telix Pharmaceuticals (ASX:TLX) shares this morning slumped up to 16% on news that the radiopharmacy play has attracted unwanted attention from the Securities and Exchange Commission (SEC). But the company is no Robinson Crusoe, in that US regulatory and legal glitches pose a regular minefield for ASX biotechs doing business there. The companies watchdog has subpoenaed 'various documents and information'. These mainly relate to the company's disclosures about its prostate cancer therapeutic program. 'The company is fully cooperating with the SEC and is in the process of responding to the information request,' Telix says. 'At this stage, this matter is a fact-finding request'. Telix adds the SEC's entreaty does not mean that Telix has violated US security laws 'or that the SEC has a negative opinion of any person, entity or security'. Barring more detail, the episode sounds like a case of 'probably nothing or very much something'. Overnight, Nasdaq investors were more chill about the affair, marking Telix shares only marginally lower. At the very least, the SEC's nosing around is likely to distract management from the company's busy agenda. 'In our experience of similar matters, these investigations tend to drag on for years without resolution,' broker Jefferies says. Trouble and strife Other ASX biotechs in the US have run into the odd bit of strife over patent disputes and regulatory interventions. ResMed (ASX:RMD) regularly parries with rivals Fisher & Paykel Healthcare (ASX:FPH) and Philips Respironics over sleep apnoea patents. In 2018 a jury ordered Cochlear (ASX:COH) to pay US$268 million in damages for a patent infringement. The charitable Alfred E. Mann Foundation lodged the action. In 2020 Cochlear lost an appeal. In 2013 CSL (ASX:CSL) paid US$64 million to settle an antitrust class action lodged by hospital groups. 'Business as usual' Telix says it will continue with the prostate cancer program. The SEC action also will not affect its nearer-stage imaging programs for kidney and brain cancers. Telix also reported June quarter revenue of US$204 million, 63% higher year on year, whilst maintaining calendar 2025 revenue guidance of US$770-800 million. This revenue derived mainly from Telix's prostate cancer imaging agent, Illucix. 'Dose volumes for Illuccix rose 7% quarter-on-quarter in the US, reinforcing the strength of our market position and continued customer demand,' Telix co-founder and CEO Dr Chris Behrenbruch says. He adds that 'despite emerging competitive pricing pressure', Telix has 'effective strategies' to maintain average selling prices. Next month, Telix lifts the kimono on June (first) half earnings. Jefferies expects a $50.6 million net profit, with a calendar 2025 tally of $88.3 million. Interestingly, this is a decline on the previous year's US$105.4 million, but the firm plugs in US$183 million for calendar 2026. Perhaps they lost our phone number? Speaking of US regulatory glitches, stroke drug developer Argenica Therapeutics (ASX:AGN) is yet to hear back from the US Food and Drug Administration (FDA) as to why the agency plonked a 'clinical hold' on its US trial plans. On June 10 Argenica said the FDA had deemed the company's supportive material as not being adequate to support its Investigational New Drug application. The trial was aimed at approval under the FDA's fast-track route. Argenica expected to hear back from the FDA within 30 days, but the agency's 'resourcing challenges' have blown out this timeline. In the meantime, Argenica is on track to report topline results from its local, proof-of-concept trial in the current quarter. The 92-patient phase II study tests Argenica's candidate ARG-007, in view of safety and preliminary efficacy in ischaemic (blockage) stroke patients. Courtesy of $4 million of government and private grants, Argenica is also investigating the 'potential utility' of ARG-0007 for other neurological conditions, including traumatic brain injury. Argenica disclosed June quarter cash burn of $2.36 million and a closing cash balance of $10.5 million. Nyrada advances heart protection trial In other trial news, Nyrada (ASX:NYR) is advancing its locally developed drug Xolatryp into phase IIa stage. Xolatryp targets the unmet need of protecting the heart following cardiac injury where patients are at high risk of tissue damage. Nyrada notes there's been no significant new cardiac drug developed for more than two decades. Xolatryp could become the first drug of its kind to protect the heart actively from ischemia-reperfusion injury. Nyrada anticipates a randomised, double-blind, placebo-controlled study, enrolling 150 subjects. These patients have acute myocardial infarction undergoing percutaneous coronary intervention (stenting). The company hopes to kick off the study in the March quarter of 2026. We now have Ample-ia funds, says cancer drug developer Prostate cancer drug hopeful Amplia (ASX:ATX) has raised $25 million in an institutional placement and is eyeing a further $2.5 million in a share purchase plan. The deed was done at 23 cents, a 19% discount to last Friday's 'frozen' price ahead of a trading halt. Amplia shares have been on a tear on the back of remarkable phase II results, covering patients with advanced metastatic forms of the deadly disease. Last month Amplia reported 17 partial responses, which meant the tumour size shrunk by at least 30%. Two of the 55 patients reported a complete response, which pretty much means a – can we say it? – cure. Amplia now plans a US trial combining Amplia's candidate AMP-945 with a different chemotherapy. The raising takes Amplia's cash to $42.7 million, including $8.2 million of expected R&D tax refunds. Don't forget who invented it Sanofi's $2.5 billion purchase of vaccine tech outfit Vicebio again highlights the role of Australian universities as a springboard for money-making life sciences ideas. The Paris-based Big Pharma is paying up to US$1.6 billion for the UK-based Vicebio and its so-called molecular clamp technology. The deal involves an upfront payment of US$1.15 billion, with regulatory milestones of US$450 million. Discovered at the University of Queensland (UQ), the clamp stabilises viral proteins enabling the immune system to respond to them more effectively. The upshot is the quicker development of fully liquid combination vaccines that can be stored at fridge temperatures. Vicebio was formed in 2018 to develop the clamp to make vaccines against life-threatening respiratory viral infections. During the pandemic, Vicebio had a stab at a Covid-19 vaccine. Courtesy of a US$100 million Series B financing last year, Vicebio's investors include Goldman Sachs Alternatives, Avoro Ventures and Venbio Partners. Uniquest, UQ's commercialisation arm has an unquantified direct investment and benefits from a licensing arrangement. Uniquest formed more than 130 start-ups, which went on to raise more than $1 billion and glean $86 million in product sales. This included licensing the UQ-invented cervical cancer vaccine Gardasil. The UQ bright sparks responsible for the molecular clamp are professors Paul Young, Daniel Watterson and Keith Chappell. UQ describes the deal as 'the largest involving a company that is commercialising intellectual property from an Australian university.'
Herald Sun
21-07-2025
- Business
- Herald Sun
Health Check: On Lamington Day, biotechs serve up their chocolate-dipped quarterly morsels
Today's news is all sweet on trial progress, revenue and cash Amplia to raise capital after runaway share performance Tryptamine starts binge eating 'magic mushie' trial Today is National Lamington Day and biotechs are serving up a solid sponge-like base of news encased in chocolate and coconut – occasionally interspersed with a jam layer. Hold the cream and pink jelly though – it just doesn't work. Like so many drug discoveries, the lamington had serendipitous origins. The story goes that one of Queensland governor Lord Lamington's maids was serving up a yellow sponge, but accidentally dunked it into molten chocolate. Lord Lammo recommended the squares to be rolled into coconut shavings for ease of eating – and the rest is history. As is this non-sequitur. Now onto the biotech news. Lumos glows after last week's mega deal Point-of-care diagnostics house Lumos Diagnostics (ASX:LDX) reports revenue of $12.4 million for the year to June 30, up 12%. June quarter revenue declined 26% to $2.6 million, owing to the end of the US flu season. Lumos reported cash outflows of US$1.7 million for the quarter, taking June 30 cash to US$2 million. Investor interest has focused on last week's mega US distribution deal with Phase Scientific, which could deliver Lumos up to US$317 million ( $487 million) over six years. This pertains to the company's bacterial-versus-viral rapid lateral flow test, Febridx. Lumos has also signed a term sheet for a $5 million loan facility, proffered by shareholders Tenmile Ventures (Andrew Forrest) and Ryder Capital. At its discretion, Lumos can draw down the facility over the next 12 months. READ: Why $487 million US distribution deal is a 'watershed moment' for skyrocketing biotech Lumos Cleo eyes FDA approval for ovarian cancer assay Still on diagnostics, Cleo Diagnostics (ASX:COV) says it aims to submit a US Food & Drug Administration (FDA) marketing approval application for its ovarian cancer assay next year. The company is on track to complete a supportive US trial in the December quarter. Thanks to government grants and tax incentives, Cleo reported cash inflows of $38,000, taking end of quarter cash to $6.46 million. Turning to drug development, genetic disease specialist PYC Therapeutics (ASX:PYC) reports cash outflows of $17.6 million. At quarter's end the company still had cash of $153 million. The company has dosed the first patient in a combined phase 1a/2b trial for polycystic kidney disease. PYC has achieved 'alignment' with the FDA on the structure of a registrational trial for its lead program, the blinding eye disease retinitis pigmentosa type 11. The company believes the regulator will require only a phase II trial. Tryptamine BEDS down eating disorder trial Psychedelic medicines house Tryptamine Therapeutics (ASX:TYP) has started recruiting patients for a world-first binge eating disorder (BED) study. In the open-label trial, 12 patients will be administered intravenously infused psilocybin, combined with psychotherapy. Melbourne's Swinburne University is undertaking the study, with first dosing this quarter and top line results due by the end of the year. BED is the most common eating disorder in the US and second most prevalent in Australia here. The condition can result in depression, anxiety, post-traumatic stress disorder and compulsive behaviour. Amplia passes the hat Meanwhile Amplia (ASX:ATX) shares this morning entered trading halt, ahead of a share placement and share purchase plan. Amplia thus continues the rich tradition of companies leveraging clinical trial results, in this case its stunning data for hard-to-treat pancreatic disease. Amplia has reported 17 'partial response' rates in it Accent trial, in 17 out of 55 advanced disease patients. A confirmed partial response is tumour shrinkage of more than 30%, sustained for two or more months with no new cancerous lesions detected. Amplia is testing AMP-945 (narmafotinib). AMP-945 appears to inhibit the protein FAK, which is overexpressed in pancreatic cancers. Amplia shares have surged 376% in the past 12 months, but investors still value the company at a modest $110 million. As of the end of March, Amplia had cash of $10.8 million. 'Perplexed' Imugene laments soft-as-a-sponge share price Imugene (ASX:IMU) chairman Paul Hopper hopes the company's lamington-soft share price will 'do an Amplia' (our words) and reflect the company's progress with its multiple cancer trials. The company last Monday announced its phase 1b study for an aggressive blood cancer had resulted in two additional 'complete responses' (that is, the tumours disappeared). Imugene is trialing Azer-cel, its allogeneic Car-T drug made from healthy donor T-cells rather than the patient's. Naturally, Imugene announced a $22.5 million and share purchase plan for up to $15 million. On reinstatement, the shares lost 4.5 cents, or 10%, taking the loss over the past year to around 80%. This allows for a one for 34 share consolidation. 'We are very disappointed with the share price performance,' Hopper says. 'We are perplexed why we had such a lukewarm reception to the earlier data at the start of year.' Cashed up for pivotal trial But with the share raising in train, Imugene investors should no longer fear that such a dilutionary event is around the corner. Post raising Imugene should have cash of $64 million, with management costing a 60-80 patient pivotal trial at $30-40 million. If approved, Azer-cel would be the first commercial allogenic Car-T treatment, enabling mass produced, off-the-shelf therapies. 'We are in active discussions with partners on the strategy for developing the drug and getting it approved,' Hopper says. Shares settle in orbit after last week's Meso-blast off Mesoblast (ASX:MSB) shares have taken a breather after Friday's 35% surge on the back of initial US sales of its first US-approved stem cell product. The company reported unaudited June quarter revenue of US$13.2 million ($20.3 million) for Ryoncil, its treatment for childhood graft-versus-host disease. The FDA approved Ryoncil in December last year. Mesoblast founder and CEO Prof Silviu Itescu points to higher sales in the current quarter, given US Centres for Medicare and Medicaid Services coverage became effective on July 1. The company has signed up more than 25 transplant centres and hopes to enlist all 45 priority centres by October. Bell Potter analyst John Hester says the sales were in line with the firm's expectations. 'The figure is inclusive of sales to the distributor, nevertheless it represents a reasonable proxy for hospital demand.' The firm values Mesoblast at $3.50 a share, implying 55% of upside. At Stockhead, we tell it as it is. While Lumos and Tryptamine are Stockhead advertisers, the companies did not sponsor this article. Originally published as Health Check: On Lamington Day, biotechs serve up their chocolate-dipped quarterly morsels
News.com.au
07-07-2025
- Business
- News.com.au
Health Check: Amid headwinds, Nanosonics goes bells and whistles on core device
Nanosonics has launched tweaked versions of its Trophon medical probe steriliser Amplia shares soar a further 36% on pancreatic cancer promise Dimerix looks to broaden its repertoire Nanosonics (ASX:NAN) has launched updated iterations of its medical probe sterilising device Trophon, pending rollout of its all-new tool called Coris. The 'next generation' Trophon 3 has 40% faster cleaning cycles, with 'greater digital integration and the broadest traceability capabilities". The company has also launched Trophon 2 Plus, a tweaked version of an existing software upgrade for users of earlier Trophons. The company first commercialised the Trophons in 2009. Trophons clean ultrasound probes much more safely and reliably than traditional manual techniques. In mid-March the US Food & Drug Administration (FDA) approved the company's an all-new product Coris, for more fiddly flexible probes (such as for colonoscopies). The company expects to launch Coris in the US later this year. Nanosonics initially is launching the Trophon upgrades here and in the UK and Europe, with the FDA reviewing an approval submission. What the analysts think RBC Capital Markets analyst Craig Wong-Pan estimates only about 35% of the company's current US users have upgraded to a Trophon 2. "Therefore we believe there is a reasonable runway for further upgrade sales.' He's not sure whether the Trophon 3 features will convince enough hospitals to upgrade, given their budgetary pressures. 'Nonetheless, we believe the launch of these offerings is positive for Nanosonics, given the company has seen new competing products enter the ultrasound probe high level disinfection market.' RBC rates the company a 'hold' with a target price of $5. Meanwhile, broker Canaccord retains a 'buy' call, but has revised its target price from $5.74 to $5.15. The firm cites the impact of a competitor, Lumicare, which is not FDA approved but looks to be winning market share here. Canaccord expects Nanosonics to report 2024-25 revenue at the bottom of its cited $188.7 million to $193.8 million range. Amplia continues its winning ways One of the deadliest of tumours, pancreatic cancer is having its day in the sun courtesy of progress reports from Amplia (ASX:ATX) and OncoSil Medical (ASX:OSL). Amplia shares today vaulted a further 36%, having soared 25%, on Friday. That was after the company confirmed a 16 th 'partial response' in its phase 1b/2a advanced pancreatic cancer trial, called Accent. A confirmed partial response is when a tumour shrinks by more than 30% and the effect is sustained for two months or more. More to the point, Amplia earlier recorded two 'pathological complete responses' (PCRs). Rare in advanced pancreatic cancers, a PCR means there are no signs of cancer in tissue examined by a pathologist following surgical removal. Accent is evaluating Amplia's drug candidate narmafotinib, in combination with standard-of-care chemotherapies. Of the 55 patients enrolled, 20 of them remain on the trial so the company hopes for more positive responses. The trial is being conducted at seven sites in Australia and five sites in South Korea. Amplia shares have gained 330% since the start of the year. Oncosil completes trial enrolment Also on Friday, Oncosil said it had enrolled all 20 advanced pancreatic cancer patients in a phase I/II study of its eponymous targeted device. A novel brachytherapy for pancreatic and liver cancers, the treatment involves irradiating tumours via a direct injection, using endoscopic ultrasound guidance. Dubbed Pancosil, the study is investigating a novel percutaneous (through the skin) delivery for the spherical devices, guided by computed tomography. As investigator, The Amsterdam University Medical Center is conducting the study. Oncosil CEO Nigel Lange says the percutaneous approach could 'simplify administration and lower barriers to adoption, supporting wider market penetration and real-world clinical use'. The idea is not to cure the cancer, but reduce the tumors to the point where they are operable. Currently 30 countries have approved the device, including in the European Union, Britain, Turkey and Israel. The company should release preliminary Pancosil data in late 2025. Dimerix eyes other indications While the cashed-up Dimerix (ASX:DXB) remains focused on its phase III kidney disease program, it's eyeing secondary activities to broaden its portfolio. The company has raked in $65 million in upfront payments from four separate partnering deals for its lead indication, the regressive focal segmental glomerulosclerosis (FSGS). But not all the cash is needed to support the 286-patient phase III trial. 'Now that the funds have come in, we are in a very strong position and can turn attention to our pipeline,' CEO Dr Nina Webster says. She is working with the board on what program to pursue, but it will be 'in an area of high unmet need' and not too far from the company's expertise. 'Generally speaking, our competency is inflammatory disease and we understand rare diseases and kidneys well,' Webster says. We know for sure it won't be something like oncology injectables. Nor will it be diabetic kidney disease, which is much more prevalent than FSGS but a competitive field. In the meantime, Dimerix hopes it might be able to win accelerated FDA approval for FSGS, which the agency classes as an 'orphan' disease. … while PYC expands kidney disease trial Still on the topic of spuds. Following ethics approval, PYC Therapeutics (ASX:PYC) will advance to the second part of its safety and dosage study for polycystic kidney disease (PKD). PYC hopes its drug candidate, PYC-003 will address the underlying cause of PYC, the most prevalent monogenic disease in humans marked by extreme swelling of the organ. PYC will launch part B of the single ascending dose study of PKD patients, as well as escalating dosing to healthy volunteers. By combining existing ribonucleic acid (RNA) drug design with its proprietary delivery platform, PYC is developing precision therapies for patients with genetic diseases that have no treatment options. PYC's lead program is for the rare eye disease retinitis pigmentosa, but the company believes the kidney program shows much promise.
News.com.au
01-07-2025
- Business
- News.com.au
Health Check: And the EOFY biotech winner is … gasp … a pot stock
Bioxyne stars with a 720% gain in the 2024-25 year Paradigm shares soar 34% after $41 million convertible note deal Biotechs turn to debt funding The ASX biotech sector's best EOFY performer has come from left field: the local and European focused medicinal cannabis supplier Bioxyne (ASX:BXN). According to the Health Check Biotech Pulse – trademark pending – Bioxyne shares soared 720% in 2024-25, leaving its largely poorly performing pot peers in the dust. Bioxyne's fortunes have been driven by its Breathe Life Sciences arm, which purveys cannabis products including not just flowers and oils but pastilles, vapes, pessaries and suppositories (we kid you not). The company recently upgraded full-year revenue guidance from $25 to $28 million and promised positive cash flow and – gasp! – profitability. Amplia (ASX:ATX) took second place, with a 233% gain (300% over the last month). The stock soared after Amplia unveiled clinical trial results showing two 'complete responses' among a cohort of advanced pancreatic cancer patients. Orthocell (ASX:OCC) shares vaulted 220% on the back of US Food & Drug (FDA) approval of its novel nerve repair tool Remplir. Imricor Medical Systems (ASX:IMR) shares ended the year 189% to the good, as the company eyes FDA approval of its world's first MRI-guided ablation catheter. Other triple digit dazzlers were myelofibrosis drug developer Syntara (ASX:SNT) (up 130%), autism testing device play Blinklab (ASX:BB1) (up 116%) and the beloved radiology imaging tearaway ProMedicus (ASX:PME) (up 110%). Sorry – there's no prize for trying Sadly, there's still too much red ink in the rankings. In your columnist's opinion, investors have marked down many worthy stocks unfairly. But we're not in primary school and everyone doesn't get a ribbon for trying, so the record books will show that medication compliance group MedAdvisor (ASX:MDR) led the falls with an 82% decline. Other laggards are the multi-pronged Universal Biosensors (ASX:UBI) (down 76%), Proteomics International Laboratories (ASX:PIQ) (down 62%, see below), lung imager 4D Medical (ASX:4DX) (down 56%) and Clarity Pharmaceuticals (ASX:CU6) (down 51%). Shares in the busy Clarity shares peaked at $8.74 last October – a 70% gain for the year – but even after the subsequent sell off the company still bears an $800 million market cap. Strictly speaking, Opthea shares fared the worst after the company's infamous eye disease trail failure in March. Opthea shares never resumed trading, so the official records show a 73% gain when in fact the stock is worth next to nothing. Genetic Technologies and Nuheara are also missing from the laggards list, only because they went into administration and subsequently de-listed. Paradigm shifts to convertible notes With the equity capital raising outlook still looking shaky, Paradigm Biopharmaceuticals (ASX:PAR) has become the latest in a string of biotechs to tap alternative funding sources. The developer of a knee osteoarthritis (OA) drug candidate has tapped US$27 million ($41.2 million), by way of convertible notes. These have been issued to New York based alternative funder Obsidian Global Partners. Under the terms, Paradigm will draw an initial US$7 million to fund patient recruitment, trial operations and regulatory milestones. The balance of the facility is available at Paradigm's discretion, 'offering operational flexibility and strategic control over future funding needs'. Barring default, the notes are interest free. The cash will help to fund Paradigm's pivotal phase III trial of its repurposed drug candidate pentosan polysulphate sodium (PPS, or Zilosul). Investors have keenly awaited this trial initiation – and confirmation of how it will be funded. Reflecting this, Paradigm shares this morning surged 34%. The 466-patient study in underway across up to 15 Australian and 50 US sites. The company says it is now fully funded up to the interim analysis of the first 50% of patients, due in mid 2026. Last week Paradigm hedged its bets by paying as much as $16.5 million for Proteobioactives Pty Ltd ($500,000 upfront) This company owns an early-stage oral candidate for minor to mild OA, which combines PPS with a COX-2 inhibitor (Coxib). When debt is not a dirty word The term 'debt' can have unfortunate connotations – especially in the context of your columnist's household budget. But it can be cheaper than equity and has the benefit of being non-dilutive and more flexible with the timing of drawdowns. Often, it's simply more accessible than equity. The developer of better working anti-infectives, Recce Pharmaceuticals (ASX:RCE) last month availed of a US$20 million ($30 million) draw-down facility from the New York based Avenue Capital Group. Recce pockets an initial US$7.5 million and a further US$5 million between April and September this year. The remainder is available in calendar 2027, with all the amounts subject to 12.75% interest. The funds will support Recce's two phase III registrational studies on diabetic foot infections and acute bacterial skin and skin structure infections. Recce also recently raised $15.8 million of equity. Last month, dermatology group Botanix Pharmaceuticals (ASX:BOT) unveiled a circa US$30 million ($48 million) debt facility with Kreos Capital. Kreos is an arm of the world's biggest investment manager Blackrock. The facility provides for circa $US20 million to be drawn now, with the remainder to be tapped by October 2026 at the company's option. Under certain conditions, Kreos Capital can convert 20 percent of the loan into Botanix shares, at 33 cents apiece. Botanix developed Sofdra, a treatment for excessive underarm sweating and has started selling the product in the US. In mid-April Botanix also raised $40 million of equity, via an institutional placement. Proteomics secures non-dilutive $6 million Then there's non-dilutive grant funding. The Perth-based Proteomics today said it had secured a $6 million investment from the federally funded Bioplatforms Australia and the WA government. The funding will support developing an accredited protein biomarker analysis platform, in partnership with the University of Western Australia. Proteomics chips in $1 million over the three-year funding period. Proteomics has commercialised a predictive test for diabetic kidney disease and is developing assays for endometriosis, esophageal cancer and oxidative stress. Meso-blast off for FDA application? Stem-cell drug developer Mesoblast (ASX:MSB) says the company and the FDA are 'aligned' on what the company needs to do before it lodges a US marketing application for its heart disease candidate, Revascor. In late December Mesoblast won FDA approval for its childhood graft-versus-host disease (GvHD) candidate. This followed years of the company and the agency being decidedly 'unaligned'. Given the heart disease heart indication is much bigger than GvHD, today's news is more significant than it may appear at first blush. Mesoblast intends to file for accelerated approval by the end of the year. This is to treat ischemic heart failure patients with reduced ejection fraction and inflammation.
News.com.au
24-06-2025
- Business
- News.com.au
Health Check: Inoviq cops ASX ‘speeding ticket' query over cancer study headline claims
The bourse's 'please explain' to the cancer drug developer has elicited a lengthy response Last week's tearaway performer, Amplia will launch a second pancreatic cancer trial The private Myro to launch neuroblastoma trial As any wizened sub editor will impart to an impressionable cub reporter – or what's left of either – a headline needs to be short and punchy or else readers will go no further. In adopting a similar approach, cancer drug developer Inoviq (ASX:IIQ) has attracted the gaze of the ASX compliance cops. The bourse took issue with last Wednesday's spiky disclosure headlined: 'New treatment kills 88% of breast and lung cancer cells'. Investors certainly read on, with the shares surging from 38.75 cents to a high of 47 cents (21%). The ASX asked the company whether the announcement conveyed a "fair and balanced' impression of the contents, 'given the early stage of the research'. The bourse asked how Inoviq justified the use of the term "treatment", given the disclosure lacked the 'usual detail' to justify the claims. In response, Inoviq furnished the compliance cops with a response that ran to nine pages, including 12 tables and 27 references. Inoviq said the release made it clear the claims were based on in vitro studies, using cancer cell lines in a laboratory. 'Therefore, the header of the announcement was both fair and balanced in the context used.' 'Best practice' The company said the in vitro work is recognised as standard best practice. This work is 'often mandated by regulatory authorities and scientific guidelines for generating credible, reliable and interpretable results in both laboratory and clinical research settings.' Inoviq also said the potential advantages of its CAR-exosomes over autologous CAR-T therapies in solid tumours were 'generally well known and accepted in the scientific literature based on previous researchers' in vitro and in vivo studies.' CAR-Ts are T-cells that have been tricked up to improve their cancer-busting abilities. CAR-T therapies to date have used patients' own cells. Inoviq strives for an 'off the shelf' treatment availing of donor material. Inoviq noted its headline was limited by the ASX's proforma requiring a 60-word limit. As any grizzled sub will tell you, the headline needs to match the allocated space (now, AI will do the job in a flash). Not the first time In late May, the ASX suspended Inoviq shares and lodged a query after a 25% share romp. Inoviq pointed to the online publication of an abstract poster to American Society of Clinical Oncology (ASCO) conference. This outlined an independent patient validation study of the company's ovarian cancer test. The 'outstanding' blood test results showed more than 94% accuracy. The issue centred on what data was 'new new', as opposed to being previously released data validated by Inoviq's learned peers. Amplia to launch second pancreatic cancer trial Tearaway pancreatic cancer drug developer Amplia (ASX:ATX) has won US ethics clearance to launch a second phase II trial, combining its candidate with standard chemotherapy. Amplia shares last week surged as much as 400% after the company announced separately two 'complete responses' in a local trial. A complete response is defined as the complete disappearance of a tumour lesion, maintained for more than two months. That sounds awfully close to a cure but – corporate cops note – the company did not use the 'c' word. The trial combined Amplia's protein inhibitor narmafotinib (AMP-945) with the chemotherapies gemcitabine and Abraxane (nab-paclitaxel). The proposed second trial will combine AMP-945 with Folfirinox. Folfirinox is a cocktail of four chemo agents, more commony used in the US for advanced pancreatic cancers. Having won central US Institutional Review Board (IRB) ethics assent, Amplia is selecting up to six US sites and two Australian venues. The company expects to recruit 60 to 70 patients, which has a single arm (no placebo group). The study is also open label (the researchers and patients know what treatment is being administered). Take it as Red, Myrio is tackling neuroblastoma Myrio Therapeutics is not listed – or not yet anyway – but keep an eye on this immunotherapy play because of its sole focus on neuroblastoma. Neuroblastoma is a rare but aggressive childhood cancer. Myrio recently won FDA approval to carry out a phase I road test of its CAR-T therapy. This is inelegantly named PHOX2B PC-CAR T and is based on Myrio's tech dubbed Red. Developed with the Children's Hospital of Philadelphia, the proposed therapy targets a specific protein. You guessed it, that's PHOX2B – as expressed in neuroblastoma cells. Red can also treat a much broader range of patients, via a mechanism best described by Google rather than us. Myrio plans to enrol the first patient in mid-2025. The most common tumour of the sympathetic nervous system, neuroblastomas account for 15% of paediatric cancer-related deaths. So, it's rare, but not so rare. Current treatment options result in low response rates and toxicity issues. Clarity abandons program There's a dearth of neuroblastoma work in the ASX-listed sphere. In April Clarity Pharmaceuticals (ASX:CU6) abandoned its program called CL04, which was in phase I/II trial stage across five US sites. The study road-tested Clarity's copper isotope-based agent, 64Cu/67Cu Sartate. While the safety profile was OK, Clarity decided to focus its Sartate program on neuroendocrine tumours, a 'a faster and larger market opportunity'. Clarity has two Rare Paediatric Disease designations from the FDA, thus opening the prospect of winning valuable Priority Review Vouchers (PRVs). What's not to like? The answer is the 'risk to the continuation of the PRV program under the current US administration, the changes to the FDA and the very small patient population in neuroblastoma, despite the high unmet need.' ... but Arovella remains in the game In early May Arovella Therapeutics (ASX:ALA) announced an exclusive option with the Baylor College of Medicine, to license two novel CAR-T receptors. These receptors target solid tumours, including neuroblastoma (highly prevalent in children) and hepatocellular carcinoma (liver cancer). Both CARs have been studied in human clinical trials, reducing the need for extensive preclinical testing. Elsewhere, neuroblastoma progress has been snail-paced. In 2020 the FDA approved Y-mAbs Therapeutics naxitamab (Danyelza). This is a monoclonal antibody for relapsed or refractory high-risk neuroblastoma in the bone or bone marrow. But in late 2022 the agency's oncologic drug advisory committee voted 16 to nil to reject Y-mAbs' omburtamab (Omblastys). The European Medicines Agency swiftly followed suit.
