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Health & Biotech
Several ASX-listed companies are making significant strides in their phase III clinical trials, the critical final stage before potential regulatory approval.
Phase III trials aim to confirm the safety and effectiveness of groundbreaking treatments, bringing hope for new therapies to tackle often challenging medical conditions with limited therapeutic options available.
It’s a pivotal moment for listed companies, which have often spent millions of dollars in the clinical development of their treatments.
Success – and anticipated success – can drive share prices higher, while failure often causes sharp declines.
For many ASX biotechs, the outcome can make or break their future, highlighting the high-stakes nature of this critical stage.
Let’s dive into some ASX companies undertaking phase III trials.
Dimerix is forecasting the second interim analysis of its ACTION3 global phase III trial of lead drug DMX-200 to treat focal segmental glomerulosclerosis (FSGS) in around mid-CY25.
The phase III study is titled Angiotensin II Type 1 Receptor (AT1R) & Chemokine Receptor 2 (CCR2) Targets for Inflammatory Nephrosis – or the far-more catchy ACTION3 for short.
FSGS is a rare kidney condition that damages filtering units, leading to permanent damage and eventual organ failure, often requiring dialysis or a transplant. With no approved treatments worldwide, options are limited.
DMX-200 has been granted Orphan Drug Designation in the US and Europe, as well as the equivalent Innovative Licensing and Access Pathway (ILAP) designation in the UK.
The retinal-diseases-focused biotech is awaiting two phase III trial results in CY25. Opthea said its drug candidate sozinibercept, known as OPT-302, blocks the activity of two vascular endothelial growth factor (VEGF) proteins – VEGF-C and VEGF-D – known for contributing to wet age-related macular degeneration (wet-AMD).
Opthea said topline data from its COAST phase III trial investigating OPT-302, in combination with aflibercept for the treatment of wet-AMD, was due early in Q2 CY25.
Results for its phase III ShORe trial investigating OPT-302 in combination with Ranibizumab in wet AMD are due in mid CY25.
If successful OPT-302 would be the first superior treatment in more than 15 years for wet-AMD, which affects around 3.5 million people in the US and Europe alone and has a multi-billion dollar market.
READ: End in sight as Opthea looks to be key player in US$10 billion wet AMD market
Neuren is actively preparing to kick off a phase III trial for its second drug candidate NNZ-2591 in Phelan-McDermid syndrome.
The company recently announced alignment with the US Food and Drug Administration had been reached regarding a single phase III trial, with the same dose, population and duration as the phase II trial
Neuren said negotiations with the FDA were continuing on the primary efficacy endpoints to be used in the trial. Neuren said it also planned to advance Pitt Hopkins syndrome and Angelman syndrome to phase III trials, subject to FDA consultation, following positive phase II trial results.
The company’s first drug candidate trofinetide – marketed as DAYBUE – is approved by the FDA and Health Canada for
the treatment of Rett syndrome.
Neuren has granted an exclusive worldwide licence to Nasdaq-listed Acadia Pharmaceuticals Inc for the development and commercialisation of trofinetide.
Recce is preparing to start its registrational phase III clinical trial assessing RECCE 327 as a topical gel for the treatment of diabetic foot infections in Indonesia in Q1 CY25.
The company is focused on developing a new class of synthetic anti-infectives designed to address the urgent global health problems of antibiotic-resistant superbugs.
Recce announced in December it had been was granted approval to conduct the trial in Indonesia, which has one of the world’s largest diabetes patient populations.
Recce said the phase III trial was one of the largest DFI studies in the world and the first of its kind across Indonesia.
2025 is shaping up to be a big year for Imricor, which is developing MRI-compatible consumable devices, such as single-use ablation catheters, required to perform cardiac ablations in an iCMR lab.
In the pharmaceutical sector, phase III clinical trials signal a company’s progression towards regulatory approval and market entry for a new drug.
The medical device industry also faces rigorous testing and regulatory scrutiny. Companies developing Class III devices—those that sustain or support life, are implanted, or present potential high risk to patients—must conduct Premarket Approval (PMA) clinical trials.
These trials, similar in their thoroughness to phase III drug trials, assess the safety and effectiveness of these devices. A successful PMA submission to the FDA not only demonstrates compliance but also opens the door to market entry.
Imricor has started its pivotal VISABL-AFL clinical trial supporting US FDA approval of its products for use in atrial flutter (AFL) ablations.
These products are a mix of capital and consumables that will follow a 510k or a PMA pathway depending on their classification.
The company is expecting FDA approval inH2 CY25 for AFL and said it had begun building out its US sales team in anticipation of the commercial launch later this year.
Imricor is also about to kick off a pivotal European trial for ventricular tachycardia (VISABL-VT) which is the next indication the technology is focused on and where the company believes there will be the largest unmet need.
Having finally obtained breakthrough FDA approval for its childhood graft-versus-host disease treatment Ryoncil after three attempts, stem cell developer Mesoblast has supersized a phase III study for the bigger indication of chronic lower back pain.
Dual Nasdaq-listed Mesoblast has started enrolling the first of 300 patients in the placebo-controlled study, across 40 US sites (expanded from 15).
Mesoblast already had conducted a 404-patient phase III trial in 2021. While the study showed pain relief, the data wasn’t convincing enough and the FDA demanded another study.
The primary endpoint – pain reduction over 12 months from a single injection – is the same as for the first trial.
In 2020, Mesoblast also carried out a 537-patient heart trial that missed the primary endpoint of reducing heart failure in chronic patients.
But the company is vying for FDA accelerated approval for a needy sub group: end-stage heart failure patients dependent on left ventricle assist devices (LVADs).
Mesoblast chief Prof Silviu Itescu told Stockhead Ryoncil’s approval supported the company’s extended pipeline, which also covers diseases such as inflammatory bowel disease.
“We understand how to bring an allogeneic [off-the-shelf] cell therapy product to market, based on meeting the FDA’s stringent criteria,” he said.
“We have created a benchmark for the whole industry.”
Paradigm is scheduled to start enrolment of patients in Q1 CY25 for a phase III trial of PARA_OA_012, its repurposed anti-inflammatory drug pentosan polysulfate sodium (aka PPS, aka Zilosul), to treat knee osteoarthritis (OA).
In 2024, Paradigm determined the optimal dose of PAR_OA_012 (iPPS) for the phase III trial and had its trial protocol approved by the FDA.
The trial aims to demonstrate improvement in pain and function with subcutaneous injections of iPPS compared with subcutaneous injections of placebo in participants with knee OA pain.
Paradigm said duration of the study will be up to 64 weeks with treatment duration six weeks.
The phase III primary endpoint is the change in knee pain from baseline at day 112, measured by the weekly average daily pain score (0-10 scale).
Key secondary endpoints include knee pain, function, stiffness, quality of life and rescue medication use through to day 365.
Based on positive phase II trial results and FDA guidance Paradigm said it had elevated structural improvements —measured via MRI and X-ray — from exploratory to secondary endpoints in the phase III trial.
Phase II data showed reductions in bone marrow lesion size, favourable biomarker changes, and increases in cartilage thickness, suggesting iPPS’s potential impact on structural aspects of OA.
At Stockhead, we tell it like it is. While Dimerix, Imricor Medical Systems, Recce Pharmaceuticals and Paradigm Biopharmaceuticals are Stockhead advertisers, they did not sponsor this article.
Health & Biotech
Health & Biotech
Health & Biotech
