The ASX stocks leading clinical trials in Australia

The clinical trials sector is boosting our economy - and the hopes of those battling conditions from motor neurone disease to osteoarthritis and erectile dysfunction.

Pic: Getty Images

• Australia's clinical trials sector generated $1.6bn for the nation's economy in 2022 according to MTPConnect

• PharmAust is currently undertaking a Phase 1 open label extension study at two sites in Australia

• Dimerix has seven Australian sites as part of its ACTION3 trial of of DMX-200 to treat focal segmental glomerulosclerosis

Australia has a hard-earned global reputation as a ‘go to’ destination to conduct clinical trials, which is continuing to grow, according to MTPConnect’s latest Clinical Trials Sector Report.

MTPConnect CEO Stuart Dignam said clinical trial growth contributed $1.6bn to the economy in 2022 – a 4% increase from 2019.

Dignam said growth was achieved despite the 2020 Covid-19 pandemic downturn with patient-centric outcomes on the rise.

So who are some of the ASX companies undertaking clinical trials in Australia and what is the attraction of running a trial Down Under?

Earlier this week, Stockhead took a look at Argenica Therapeutics (ASX:AGN), a brain injury-focused clinical-stage biotech developing its lead drug candidate ARG-007 – a cationic, arginine-rich peptide with neuroprotective properties designed to protect brain cells from injury after a stroke and other neurological conditions.

And we also learnt more about Neurotech (ASX:NTI), a clinical-stage biopharmaceutical development company focused predominantly on paediatric neurological disorders, where there is persistent neuroinflammation.

If you didn't catch it, you can read that article here > Australia proves its reputation as ‘go-to’ destination for clinical trials

Here, though, we follow up further with other stand-out innovators in the biotech, healthcare and life sciences sectors of the ASX…

PharmAust (ASX:PAA)

Chief operating officer John Clark told Stockhead that PharmAust was undertaking a Phase 1, open label extension study (OLE) at two trial sites in Australia – Calvary Health Care Bethlehem in Melbourne, led by Associate Professor Susan Mathers, and Macquarie University in Sydney, led by Professor Dominic Rowe.

The OLE study is investigating the long-term safety, tolerability, and efficacy of its lead drug monepantel (MPL) in patients with motor neurone disease -also known as amyotrophic lateral sclerosis (ALS) - who had previously completed its Phase 1 MEND Study.

Enrolled patients will receive a daily dose of 10 mg/kg body weight of (MPL) for an additional 12 months.

The company recently provided a four-month progress update demonstrating that treatment with MPL continues to be well-tolerated, slowed disease progression and improved survival when compared to untreated matched controls from the PRO-ACT Historical Database, the largest publicly available repository of merged MND/ALS clinical study data.

"The OLE study is planned to conclude in mid 2025, and next month will be the two-year anniversary since the first patients started treatment with MPL," said Clark.

Dimerix (ASX:DXB)

This clinical-stage biotech has seven Australian sites as part of its ACTION3 global Phase 3 trial of of lead drug, DMX-200, to treat focal segmental glomerulosclerosis (FSGS).

The Phase 3 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.

DXB CEO and managing director Dr Nina Webster told Stockhead clinical sites in the Australian arm of the trial included:

• Sunshine Hospital, Royal North Shore Hospital, Liverpool Hospital, Prince of Wales Hospital in Sydney
• St. Vincent’s Hospital and Nepean Hospital in Melbourne
• Princess Alexandra Hospital in Brisbane

Webster said that in general, Australia was a great place for recruiting clinical trials, with an excellent health system and a great source of innovation.

"The challenge for Dimerix is that we are recruiting in a rare disease population," she said.

"Australia has a relatively small population, and thus we need to also recruit overseas.

"It is also a requirement that, for regulatory agencies such as the FDA in the US and EMA in Europe, we recruit populations that meet the demographic, and thus we are also required to recruit overseas."

Neuren Pharmaceuticals (ASX:NEU)

Neuren is also focused on rare diseases.

CEO Jon Pilcher told Stockhead  the company had done Phase 1 trials in healthy volunteers in Australia for both of its drugs - trofinetide and NNZ-2591.

One of these drugs already has a stellar record.

NEU became the market darling of the biotech sector in 2023 after their Nasdaq-listed partner Acadia Pharmaceuticals received FDA approval for trofinetide (marketed as DAYBUE) in Rett Syndrome.

"There is a well-established capability and multiple Phase 1 units available," Pilcher said.

"Phase 1 units are specialised facilities designed specifically to conduct these trials, in which participants usually have to be confined to the unit for a long period and closely monitored, so very different to later patient trials."

Pilcher said operating in rare diseases meant that it was more challenging to do patient trials in Australia and get sufficient participants in a reasonable timeframe.

"However, we did recently complete our Phase 2 trial in Angelman syndrome at three Australian hospitals," he said.

NEU in August reported promising results from the Phase 2 trial of NNZ-2591 for Angelman syndrome, a rare genetic disorder that mainly affects the nervous system and severely disrupts development.

Percheron Therapeutics (ASX:PER)

Formerly named Antisense Therapeutics, PER is focused on treatments for rare diseases and is undertaking a Phase 2B trial of its lead program avicursen (ATL1102) into non-ambulant boys with Duchenne muscular dystrophy (DMD).

Avicursen is an antisense oligonucleotide targeting CD49d, a receptor involved in white blood cell activity and has shown effectiveness in multiple inflammatory conditions, including multiple sclerosis and DMD.

CEO and MD Dr James Garner told Stockhead the study was being undertaken at three sites in Australia: The Royal Children's Hospital in Melbourne, Westmead Hospital in Sydney and The Queensland Children's Hospital in Brisbane.

The Phase 2B sites also has sites in Turkey, Serbia, Bulgaria and the UK with initial results from the study expected in December and further readouts in mid-CY25.

"On the whole Australia is a good place to do clinical trials essentially because of quality," he said.

"Many of the hospitals we are working with here have extensive clinical research experience.

"The clinicians do a good job with these trials which can be complex and demanding and they're ready for the challenges."

However, like DXB Garner said Australia's smaller population of ~27 million meant rare diseases trials mostly also needed to be undertaken overseas to adequately recruit patient numbers.

He said furthermore, there could also be differences in standards of care between countries.

"That obliges us to look more broadly than Australia for patients because they may have had different prior treatments in other countries we want to be able to capture in our trial," he said.

PER previously did the Phase 2a trial just in Australia at the Royal Children's Hospital with earlier studies also undertaken here.

"In a way what we are doing is quite common for Australian companies where earlier studies are purely in Australia and then become international as they get more late stage," he said.

Paradigm Biopharmaceuticals (ASX:PAR)

Late-stage drug development company PAR has also conducted multiple clinical trials both in Australia and internationally.

Managing Director Paul Rennie told Stockhead the company's Phase 2b trial PARA_005 and Phase 2 trial PARA_OA_008 of its lead asset injectable pentosan polysulfate sodium (iPPS) for knee osteoarthritis (OA) had been run at various sites across Queensland, Victoria, Western Australia, New South Wales, and South Australia.

"In addition, we have completed other Phase 2 studies like the MPS-1 (Mucopolysaccharidosis Type I) trial in Adelaide, and the Ross River study in Queensland," he said.

The company's first phase of its Phase 3 program of iPPS for knee OA PARA_OA_002 involved sites not only across Australia but also in Europe, the UK, Canada, and the US.

The PARA_OA_002 study has completed with the optimal dose identified to continue into the next stage of the phase 3 program, which will be PARA_OA_012.

"This international and domestic approach ensures comprehensive data and robust outcomes for our clinical programs," he said.

PAR plans to run PARA_OA_012 trial at multiple sites across Australia, including Queensland, New South Wales, Victoria, South Australia, and Western Australia, alongside international sites.

The company this week announced it had received clarity from the FDA on the design of the proposed Phase 3 study.

"Australia is an excellent environment for clinical trials due to its strong regulatory framework, access to world-class medical facilities, and highly skilled investigators," Rennie said.

"Additionally, the federal government's R&D tax rebate program provides significant incentives, allowing companies like ours to reinvest in innovation and further development.

"This, combined with the ability to recruit diverse patient populations across metropolitan and regional areas, makes Australia a highly advantageous location for running clinical trials."

Optiscan (ASX:OIL)

OIL announced in July it had received ethical clearance from the Royal Melbourne Hospital Human Research Ethics Committee to undertake an in vivo clinical study for assessment of cancer margins in patients presenting for surgical treatment of breast cancer.

The company is developing, manufacturing, and commercialising confocal endomicroscopy imaging technologies for medical, translational, and pre-clinical applications.

The study will assess the clinical workflow and real-time imaging capability of OIL’s InVue precision surgery imaging platform and used during surgery to collect in vivo imaging data of the surgical cavity intraoperatively after tumour removal to determine clearance of the tumour in real-time.

Fifty patients undergoing breast conserving cancer surgery lumpectomy procedures at the Royal Melbourne Hospital, Frances Perry House and Epworth Hospital will be recruited for the study.

CEO and managing director Dr Camile Farah told Stockhead the company was also planning a clinical study in Perth to assess the utility of its InVivage device in oral cancer surgery.

The study is planned to be undertaken by head and neck cancer surgeons to collect feasibility data on surgical margin assessment and workflow requirements.

"OIL is keen to undertake clinical feasibility studies in Australia because of the relatively straightforward nature in initiating these in a local environment and the ability to provide local support for collaborating surgeons," he said.

"Larger scale clinical trials are planned for the US given the FDA’s preference to have data collected in populations where OIL’s devices will be marketed."

LTR Pharma (ASX:LTP)

LTP recently completed a pivotal bioequivalence clinical study in Sydney of its lead asset Spontan nasal spray treatment for erectile
dysfunction (ED). 

Initial results of the study showed Spontan was able to achieve rapid absorption and faster onset of action than traditional oral PDE5 inhibitors, encompassing household names like Viagra.

LTP announced it had achieved a significant milestone in August with the first patients having begun using Spontan under the Therapeutic Goods Administration (TGA) Authorised Prescriber Scheme (APS).

"Spontan is a truly innovative, fast-acting treatment, which is on track to disrupt the established blockbuster oral PDE5 market. Current oral tablets can take up to one hour to take effect, and many men are dissatisfied with the results as they don’t always work or in the time desired," said chair Lee Rodne.

"We were thrilled to welcome men in Australia to participate in our pivotal study recently – a crucial step in clinical validation and our expedited regulatory journey for Spontan, as we prepare to bring this product to the world.

The speed of recruitment and preliminary results we shared recently, are truly encouraging."

At Stockhead, we tell it like it is. While PharmAust, Dimerix, Paradigm Pharmaceuticals, Optiscan and LTR Pharma are Stockhead advertisers, the companies did not sponsor this article.

Originally published as The ASX stocks leading clinical trials in Australia