Cancer remains one of the most formidable challenges in modern medicine — but it is also one of the most dynamic frontiers of pharmaceutical innovation.
Over the past decade, oncology has become the centre of gravity for the global biotech and pharmaceutical industries. Drugmakers are investing billions of dollars in therapies aimed at harnessing the immune system, targeting tumour genetics or delivering highly precise treatments directly to cancer cells. At the same time, clinical trial design and research technologies are evolving rapidly, creating new ways to identify promising drugs and bring them to patients faster.
The scale of activity is striking. Hundreds of oncology clinical trials are under way globally, and regulators have approved a steady stream of therapies across cancers from melanoma and lung cancer to leukaemia, lymphoma and other rare tumours. Breakthrough immunotherapies such as Merck & Co Inc (NYSE:MRK, XETRA:6MK)’s blockbuster checkpoint inhibitor Keytruda have transformed treatment for many patients and reshaped expectations across the industry.
Yet cancer treatment remains far from solved. Many patients still fail to respond to the most advanced medicines, while others develop resistance after initially responding to therapy. Even with widely used drugs like Keytruda, durable responses are far from universal — a reality that continues to drive the search for new treatments capable of improving survival and extending the benefits of existing therapies.
Across the industry, the next phase of innovation is emerging at the intersection of new drug technologies, smarter trial designs and a deeper understanding of tumour biology.
The immunotherapy revolution
Much of oncology’s recent progress has been driven by immunotherapy — treatments designed to stimulate the body’s immune system to recognise and destroy cancer cells.
The most successful of these drugs are immune checkpoint inhibitors, which block molecular signals tumours use to evade immune attack. Medicines targeting the PD-1 and CTLA-4 pathways have become a cornerstone of cancer care over the past decade.
Merck’s Keytruda is the best-known example, now approved across dozens of cancer types and widely used as a first-line therapy in many settings. Similar drugs from companies including Bristol-Myers Squibb Co (NYSE:BMY, XETRA:RM, OTC:BMYMP), Roche and AstraZeneca PLC (LSE:AZN, NASDAQ:AZN) have also delivered durable responses for patients who previously had few options.
But even these therapies have limits, prompting researchers and biotech companies to look beyond the first generation of checkpoint inhibitors in search of additional immune targets and complementary approaches that could broaden the reach of immunotherapy.
The next wave of oncology technologies
As scientists explore new ways to treat cancer, several emerging technologies are drawing particular attention.
Cell therapies such as CAR-T treatments genetically engineer a patient’s immune cells so they can recognise and destroy cancer cells. These therapies have produced remarkable outcomes in certain blood cancers and are now being explored for solid tumours.
Antibody-drug conjugates (ADCs) are another rapidly expanding field. These medicines combine a tumour-targeting antibody with a potent chemotherapy payload, allowing drugs to deliver toxic agents directly to cancer cells while limiting damage to healthy tissue.
Radiopharmaceuticals are also gaining traction, delivering radioactive material directly to tumours through molecular targeting mechanisms that allow radiation to be applied far more precisely than traditional radiotherapy.
Alongside these approaches, researchers are increasingly exploring immune checkpoints beyond the PD-1 and CTLA-4 pathways in hopes of overcoming resistance to existing therapies.
Targeting the next generation of immune checkpoints
ASX-listed Percheron Therapeutics Ltd (ASX:PER, OTC:PERCF) is one company pursuing such targets through its VISTA-focused immunotherapy candidate HMBD-002.
Percheron chief executive James Garner said interest in alternative immune checkpoints has grown as researchers look for ways to extend the benefits of existing immunotherapies.
“Existing immune checkpoint inhibitors, which almost all target PD-1/PD-L1 or CTLA-4, have made an enormous impact in the treatment of a wide range of cancers,” Garner said. “However, many patients do not respond to these therapies and, of those that do, their effect often wanes over time.”
Researchers have increasingly focused on identifying additional checkpoints that may help tumours evade immune responses.
“VISTA has emerged as one of the most promising such targets,” Garner said.
“It is widely expressed on a broad range of cancers. High expression generally correlates to a worse prognosis, which is the hallmark of an important target. And, most interestingly, VISTA seems to be implicated in resistance to existing therapies such as Keytruda which target PD-1.”
Rather than replacing existing treatments, Garner believes the next generation of immunotherapies will likely be used alongside them.
“In Percheron’s view, the long-term future of immune checkpoint inhibitors will likely consist substantially in combinations of drugs, administered in a tailored regimen for each patient, providing greater efficacy and longevity than any individual therapy,” he said.
“To this end, the very favourable safety profile of Percheron’s HMBD-002 is critical — the drug already appears to be very well tolerated in combination with Keytruda, for example.”
Precision oncology and the tumour microenvironment
Another major area of innovation is precision oncology — the effort to match treatments more closely to the biological drivers of an individual patient’s cancer.
Researchers are increasingly focusing on therapies built around specific biological pathways that can be combined depending on the characteristics of a patient’s tumour. Scientists are also paying growing attention to the tumour microenvironment — the network of immune cells, blood vessels and signalling molecules surrounding a tumour that can influence how cancers grow and respond to treatment.
ASX-listed Prescient Therapeutics Ltd (ASX:PTX) is among the companies pursuing this strategy through its targeted oncology program, including its lead drug candidate PTX-100.
Prescient chief executive James McDonnell said the industry is increasingly shifting toward therapies designed around distinct biological mechanisms that clinicians can combine to treat individual patients.
“We are focused with PTX-100, which has a unique mode of action, and that's really what clinicians are looking at now — specific modes of action that they combine to treat individual patients depending on what their tumours are,” McDonnell said.
“So it’s quite an interesting phase we're going through with combination approaches, but also single-agent activity.”
PTX-100 works by inhibiting geranylgeranyl transferase-1, an enzyme involved in activating Ras proteins that play a key role in cancer cell growth.
McDonnell said the industry is increasingly moving away from traditional chemotherapy toward more targeted biological therapies designed to attack specific cancer pathways.
“With PTX-100 we have a particular pathway and a particular focus, and biologics have their particular targets as well,” he said.
“Clinicians are now looking at what they can combine, because the tumour microenvironment is becoming quite a popular topic, and they realise that to have just one active working on the tumour may not be the ideal approach.”
Interest in combination therapies is also accelerating as researchers explore ways to improve response rates and overcome resistance to existing drugs.
“Those therapies will be quite diverse in terms of biologics therapies and small molecule targeting enzymes and other pathways,” McDonnell said. “So, it is quite exciting.”
Australia’s emerging oncology biotech sector
Prescient and Percheron are part of a broader wave of Australian biotechnology companies developing novel cancer therapies.
Imugene Ltd (ASX:IMU, OTC:IUGNF, FRA:ILA), for example, is advancing its allogeneic CAR-T therapy azer-cel, an off-the-shelf immunotherapy designed to target CD19-positive blood cancers, and has reported encouraging early clinical results.
Other ASX-listed companies are pursuing different immuno-oncology strategies. Immutep Ltd (ASX:IMM) is developing therapies targeting the LAG-3 immune checkpoint, while Chimeric Therapeutics Ltd (ASX:CHM, OTC:CHMMF) and Racura Oncology Ltd (ASX:RAC, FRA:FN3) are exploring cell therapy and targeted chemotherapy approaches.
Designing trials for a complex treatment landscape
As cancer therapies become more specialised, clinical trial strategies are evolving as well.
Traditional studies often test a single drug in a single tumour type and can take years to produce results. Increasingly, however, researchers are adopting adaptive designs that allow multiple tumour types or treatment approaches to be evaluated within a single trial.
Garner said Percheron’s upcoming Phase II trial for HMBD-002 reflects this shift, with the company aiming to test the drug in several tumour types in parallel.
“Partly, this mitigates the binary risk that can be so challenging for biotech investors,” he said. “Commercially, it helps to broaden both the validation and the addressable market for the drug.”
The study will also use an adaptive structure designed to evaluate results at several points during the trial.
“This means that, instead of recruiting a large number of patients and waiting until the end of the study to see results, the Percheron Phase II study will recruit patients in several blocks and check the efficacy after each block to make sure that the study is progressing as hoped,” Garner said.
Prescient is adopting a similar approach as it advances PTX-100 through clinical development, with its Phase II trial evaluating multiple dosing levels while working closely with regulators on the path toward later-stage studies.
The realities of biotech drug development
For emerging biotech companies, scientific innovation is only part of the challenge.
Developing new cancer medicines requires years of clinical testing and significant financial resources — a process that can be particularly demanding for smaller public companies.
“For an ASX-listed biotech, it’s about the funding of it, but also the pathways and the uncertainty moving forward,” McDonnell said, noting that clinical trials can take years to complete, especially when studying rare cancers where patient recruitment is more difficult.
“We have a rare disease focus, so patients are not popping up everywhere, and that creates its own challenges.”
Data milestones drive the sector
For investors watching the oncology sector, clinical data remains the most important catalyst.
McDonnell said upcoming trial readouts often represent critical inflection points for biotech companies, as will be the case with PTX-100.
“As we get halfway through the Phase IIa study or reach any data readout, that will be an inflection point,” he said.
“The data provides us an opportunity to talk to relevant partners around the area and look to how we might commercialise and go forward, provided the data is strong.”
A rapidly evolving oncology ecosystem
Across the industry, oncology is undergoing rapid transformation. New technologies are expanding treatment options, while advances in data science and clinical trial design are helping researchers test therapies more efficiently.
For investors, the opportunity is significant — but so is the uncertainty. Oncology drug development remains one of the riskiest areas of biotech, where promising scientific ideas must still survive the long and expensive process of clinical testing.
If the trajectory of the past decade is any guide, the next wave of cancer treatments may emerge not from a single breakthrough drug but from increasingly sophisticated combinations of therapies, data-driven trial strategies and precision targeting of tumour biology.
For patients, that shift could mean more personalised treatment plans and longer-lasting responses. For drug developers and investors, it underscores how the future of oncology will likely be shaped not by one dominant technology, but by how multiple innovations are brought together in the clinic.