One of the most exciting areas in medicine is the emerging field of CAR-T cell therapy, a potentially revolutionary treatment in cancer and a range of other serious diseases. CAR-T therapy is an innovative form of immunotherapy that involves modifying a patient’s own T-cells (natural disease-fighting cells in the human immune system, that develop from stem cells in the bone marrow) to express a chimeric antigen receptor (CAR) on their surface, with the CAR designed to recognise specific proteins on the cancer cells and target and destroy these cells, effectively enhancing the immune system’s ability to combat the disease.
In CAR-T cell therapy, T-cells are extracted from a patient’s blood, and genetically re-engineered into a “one-off” treatment that attacks cancer cells, and reinfused back into the patient, programmed into a treatment that can potentially provide a functional, personalised cure for cancer. CAR-T cells have had considerable success against blood cancers, such as leukaemia and lymphoma, but applying CAR-T cell therapy to solid tumours remains very challenging.
As the Peter MacCallum Cancer Centre in Melbourne – Australia’s only public hospital dedicated to cancer care and research – puts it:
“The immune system consists of many cells and organs. They almost always protect the body from infection and cancer. An important part of the immune system is T cells. T cells can hunt down and destroy abnormal cells, including some cancer cells. Sometimes cancer cells find ways to evade the immune system. When this happens, we need to retrain the immune system to recognise and attack cancer cells. CAR T-cell therapy is one way that we can train and strengthen the immune system to attack some forms of cancer.”
The market for CAR-T cell therapies is growing very significantly; it is expected to reach about $30 billion a year by the end of this decade, as it moves out of its origins in oncology and into other disease markets. One of the limiting factors is the manufacturing – the re-engineering of harvested stem cells into cancer-fighting CAR-T cells – itself.
As a world-class centre of medical research, Australia is at the forefront of this area. Here are four ASX-listed companies involved in this fascinating area.
Readers will notice the bugbear of ASX life sciences: tiny market valuations, despite strong science. This area has to be considered speculative: however, if you do your research, and if you get satisfied that any of these stocks are on the right track, a punt could be rewarding – and you may end-up supporting life-changing research for severely ill patients.
- Allovera Therapeutics (ALA, 10 cents)
Market capitalisation: $119 million
1-year total return: –31%
3-year total return: 54.7% a year
Analysts’ consensus price target: n/a
Perth-based Allovera is developing its novel invariant natural killer T (iNKT) cell immunotherapy platform, for which it holds an exclusive global licence from the platform’s developer, Imperial College London. iNKT cells, which are one of the body’s rarest kinds of immune cells, work alongside antibodies and T cells as part of the body’s natural immune defences. iNKT cells are distinct from the more well-known natural killer, or ‘NK’ white blood cells, but unlike NK cells they are capable of influencing a longer-term immune response and activating other components of the immune system.
The therapy will use iNKT cells that have been combined with lab-made CARs, which cause the immune cells to recognise and activate against target cancer cells, beginning with blood cancers such as leukaemia, lymphoma, and multiple myeloma. The active cells are called CAR-iNKT cells.
The approach resembles CAR-T therapy, but the major difference is that the CAR-iNKT cells can be produced as an ‘off-the-shelf’ treatment – that is, the CAR-iNKT cells used in the new therapy are derived from a donor (they are ‘allogeneic’) rather than the patient, making them an “off-the-shelf” therapy option. CAR-iNKT cells offer potential advantages over traditional CAR-T cells, including a reduced risk of graft-versus-host disease (GvHD) – conventional T cells cannot be transplanted from a donor to a recipient because they would attack the recipient’s cells, which is why they are removed and re-engineered. Thus, CAR-iNKT cells can be used in a much broader patient population and can be mass-produced at much lower cost using iNKT cells provided by healthy donors. (CAR-T cells derived from the patient’s own immune cells, in other therapies, are called ‘autologous.’)
Arovella plans to seek US Food & Drug Administration (FDA) approval for its first-in-human trial of blood cancer patients, under the US investigational new drug (IND) pathway. The fully funded Phase 1 trial, set for 2026, will be conducted in non-Hodgkin’s lymphoma and leukaemia patients exhibiting the CD-19 biomarker, the target the company’s CAR-iNKT cells recognise. The company has also licensed two novel CAR receptors targeting solid tumours, including neuroblastoma (highly prevalent in children) and a form of liver cancer; and has also signed a deal with US-based biotech company SparX Group, to develop a world-first CAR-iNKT cell therapy targeting Claudin 18.2, a validated target that is expressed in gastric cancers, gastro-oesophageal junction cancers and pancreatic cancer.
- Imugene (IMU, 27 cents)
Market capitalisation: $59 million
1-year total return: –81%
3-year total return: –68.7% a year
Analysts’ consensus price target: n/a
Imugene works in immuno-oncology, the anti-cancer field in which the body’s immune system is artificially stimulated to boost its natural ability to fight the disease (sometimes called “cancer immuno-therapy”). Imugene is developing four new treatments that activate the immune system of cancer patients to identify and eradicate tumours.
Imugene’s intellectual property (IP) is mainly based on its platform technologies for cancer immunotherapy, including oncolytic virotherapy (the term ‘oncolytic’ refers to a virus that selectively infects and destroys cancer cells while leaving healthy cells unharmed), and its CAR T-cell therapies.
Imugene’s CAR T-cell work came out of a collaboration with California-based cancer research centre, City of Hope, on a novel CAR T-cell therapy approach called OnCARlytics, which combines oncolytic viruses with CAR T-cells that target a protein called CD19, a protein found on the surface of some cancer cells, to treat solid tumours. This strategy involves using an oncolytic virus (CF33) engineered to express CD19 on cancer cells; it enters solid tumour cells and forces them to express the CD19 protein on the cell surface, presenting a target for the modified CAR-T cells that are infused back into the patient – they are primed to seek out and attack CD19-positive cancer cells. This approach is being explored in a Phase 1 clinical trial.
Imugene’s other CAR T approach is its Allo CAR T Cell Therapy, azer-cel, which is an allogeneic CAR-T cell drug that targets CD19 to treat blood cancers. Azer-cel is currently in a Phase 1b trial, under the FDA IND pathway, being tested on patients with diffuse large B-cell lymphoma (DLBCL) who have relapsed off autologous CAR-T therapy. DLBCL is a highly aggressive blood cancer, and these patients have failed multiple therapies. Azer-cel aims to address a large unmet need in rare lymphomas and the relapsed/failed autologous CAR T setting.
Imugene also has its oncolytic virus VAXINIA in the Phase I MAST trial in patients with late-stage metastatic advanced solid tumours, under the FDA IND pathway. VAXINIA (or CF33-hNIS) was also developed at City of Hope and licensed by Imugene. It is designed to selectively kill cancer cells and activate the immune system against tumours. The virus has been shown to shrink colon, lung, breast, ovarian and pancreatic cancer tumours in pre-clinical laboratory and animal models; in the MAST trial it has shown good early results in bile tract cancer. The virus is being tested both as a monotherapy and in combination with Merck’s immunotherapy drug Keytruda, which is a PD-1 inhibitor, meaning it works by protecting PD1 receptors on T-cells, allowing them to activate the cell death pathway and destroy cancer cells. VAXINIA has been granted ‘fast track’ designation by the FDA: the ‘fast track’ status is designed to speed-up the development and regulatory review of novel drugs that address serious conditions with significant unmet medical needs. In granting an FTD, the FDA assesses preliminary results to date.
The company also has its PD1-Vaxx vaccine immunotherapy targeting the PD-1 protein currently in a Phase II Neo-POLEM trial in operable mismatch repair-deficient/microsatellite instability (dMMR/MSI-high) colorectal cancer (approximately 15% of patients with colorectal cancer have the dMMR/MSI-high subtype.) Neo-POLEM is a neo-adjuvant study, meaning PD1-Vaxx is administered before surgery, aiming to enhance treatment outcomes. The trial is recruiting across Australia and the United Kingdom.
- AdAlta (1AD, 0.3 cents)
Market capitalisation: $3 million
1-year total return: –83.8%
3-year total return: –59% a year
Analysts’ consensus price target: n/a
Melbourne-based drug developer AdAlta has taken an antigen-binding domain found in shark blood and transformed it into a humanised single domain antibody that can potentially change treatments for diseases such as fibrosis and cancer. Called i-bodies, the size and structure of the next-generation antibodies mean they can interact with difficult-to-access targets that are implicated in many serious diseases. The i-bodies are smaller, and their binding region is different from traditional antibodies, giving them the ability to bind to therapeutic targets that traditional antibodies cannot.
The platform has generated a lead product candidate, called AD-214, which targets a particular receptor known as CXCR4 and is being developed as a ‘first-in-class’ therapeutic for a type of lung fibrosis known as idiopathic pulmonary fibrosis (IPF), a rare and highly lethal disease in which the current treatment options only slow the disease’s progression. AD-214 only targets diseased human tissue – there are no effects on normal lung tissue. In laboratory tests, AD-214 appears to be more effective than the two existing drugs approved for IPF. AD-214 has been shown to be safe in Phase I clinical studies, and the company is progressing it toward Phase II clinical trials. As a large unmet medical need, the global IPF market size is US4.54 billion ($7 billion) a year, and it is projected to reach as high as US$9.23 billion ($14.2 billion) by 2033.
Where AdAlta comes into the CAR-T space is that the company is collaborating with Carina Biotech, an Australian pre-clinical immunotherapy company that researches and develops chimeric antigen receptor T cell (CAR-T) therapies to treat solid cancers, to discover and optimise panels of i-bodies that bind to five solid tumour antigen targets. Carina will then incorporate the i-body sequences into CARs which it then engineers into the T cells. The i-CAR-T cells will be mixed with cancer cells that express the antigen the i-bodies target, to see if the T cells can kill the cancer cells and then used to treat mice growing these cancers to identify CAR-T product candidates. The collaboration leverages the small size of i-bodies, which mean that they can access unique targets that traditional antibodies cannot; and it is also easier to put more i-bodies into the cells, allowing the researchers to target multiple antigens at the same time.
- Chimeric Therapeutics (CHM, 0.3 cents)
Market capitalisation: $9 million
1-year total return: –84.2%
3-year total return: –71.6% a year
Analysts’ consensus price target: n/a
Melbourne-based Chimeric Therapeutics is a cancer cell therapy company with a diversified portfolio that includes autologous CAR T cell therapies and allogeneic NK cell therapies. Chimeric’s assets are being developed across multiple different disease areas in oncology with four programs, all in Phase 1/1b clinical trials. Chimeric raised $6.6 million May 2025 and also received $2.5 million in R&D tax incentive to support trials.
The lead candidate, CHM CDH17, is in a phase 1/2 clinical trial assessing its effect on advanced gastrointestinal and neuro-endocrine cancers and colorectal cancers. The CDH17 novel CAR-T cell therapy was invented at the world-renowned cell therapy centre at the University of Pennsylvania, in the laboratory of Dr. Xianxin Hua, professor of cancer biology in the Abramson Family Cancer Research Institute at Penn. CDH 17 targets the cancer biomarker associated with poor prognosis and metastases in common gastrointestinal tumours.
Preclinical evidence in a CHM CDH17 CAR-T mice study published in 2022 demonstrated complete eradication of tumours in seven types of cancer. Chimeric launched the Phase 1/trial last year to determine a recommended dose of CHM CDH17 and evaluate its safety and objective response rate in patients with advanced colorectal cancer, gastric cancer and intestinal neuroendocrine tumours. The trial is being conducted in four US sites, under the IND pathway. In June, the CHM CDH17 program received FDA Fast Track designation for treatment of gastroentero/pancreatic/neuroendocrine tumours (GEP-NETs). This week, the CHM CDH17 trial advanced to Dose Level 2, following no safety concerns or off-target effects being observed at dose level 1.
The second asset, CHM CLTX (also known as CHM 1101), is being aimed at the brain cancer glioblastoma. CLTX is a novel and promising CAR T therapy that is unique in its use of chlorotoxin, a peptide derived from deathstalker scorpion venom, as its tumour targeting domain. (A ‘tumour targeting domain’ refers to a specific region or component of a therapeutic molecule, like a CAR T cell, that is designed to recognise and bind to cancer cells, enabling targeted delivery of the therapy. This domain essentially acts as a homing device, ensuring the therapeutic agent focuses its action on the tumour, while minimising harm to healthy tissues.)
Preclinical studies identified the potential of CHM 1101 to bind in other solid tumours such as melanoma, colorectal cancer and prostate cancer. The therapy is now in a Phase 1A clinical trial testing its safety and efficacy on patients with recurrent and/or glioblastoma multiforme (GBM) – a fast-growing and aggressive brain tumour. According to the US-based National Brain Tumour Society, the five-year survival rate for glioblastoma patients is only 6.8%, and the average length of survival for glioblastoma patients is estimated to be only eight months; survival rates and mortality statistics have been virtually unchanged for decades.
Chimeric also has its CHM CORE-NK cell therapy in trials. CHM CORE-NK is a potentially best-in-class, clinically validated NK cell platform; it is an allogeneic (universal donor) natural killer (NK) cell therapy that can be administered ‘off-the-shelf.’ The company is running the ADVENT-AML Phase 1B clinical trial, evaluating CORE-NK cell therapy in conjunction with standard-of-care treatments of acute myeloid leukaemia (AML). This is the first frontline AML study globally to incorporate a cell therapy. So far, CHM says the results support the potential of CHM CORE-NK to enhance responses in elderly or unfit patients with newly diagnosed AML, who are not candidates for intensive chemotherapy or allogeneic stem cell transplant. Another Phase 1b trial is testing the combination of CHM CORE-NK and the anti-cancer drug Vactosertib in patients with advanced colorectal and blood cancers. In October 2024, Chimeric announced that a patient with AML had achieved a complete response in the CORE-NK-Vactosertib trial – a “complete response” means the disappearance of all detectable signs of cancer in the patient: scans and other tests no longer show any evidence of cancer, but it does not necessarily mean the cancer has been cured.
Across the applications it is working on, Chimeric is targeting a global oncology market projected to grow from US$39 billion ($60 billion) in2025) to more than US$80 billion ($123 billion) by 2034, spanning gastro-intestinal (GI), neuro-endocrine, AML and glioblastoma.
