Cartherics is a biotechnology company focused on developing innovative cell therapies for cancer and other diseases. The company works to create precisely-defined immunotherapy treatments to combat a variety of cancers and other difficult diseases. Pulse 2.0 interviewed Cartherics CEO Professor Alan Trounson to learn more about the company.
Alan Trounson’s Background
What is Alan Trounson’s background? Alan Trounson is a cell biologist who pioneered human in vitro fertilization (1980s and 1990s); developed human embryonic stem cells and pluripotent stem cells for regenerative medicine (1990s and early 2000s), led the Australian Stem Cell Centre funded by the Australian Federal Government (AU$110 million grant) in 2003 and was appointed President of the Californian Institute for Regenerative Medicine (2007-2014) – the US$3 billion granting agency for driving stem cells and regenerative medicine. In 2014, he returned to Australia and formed Cartherics Pty Ltd to develop chimeric antigen receptor (CAR) technology in combination with pluripotent stem cells as a new approach to the control of cancer.
Formation Of Cartherics
How did the idea for Cartherics come together? Trounson brought together – Professor Richard Boyd (an internationally recognised immunologist from Monash University); Dr. Ian Nisbet, a scientist and executive with deep experience in the biotechnology and pharmaceutical industries in Australia and the US; Dr. Peter Hudson from CSIRO with deep experience in antibody design and function; and Robert (Bob) Moses, a well-credentialed businessman who has provided leadership to many successful major and smaller companies in biotechnology. These were the founders of Cartherics. The company started operations in facilities at the Monash Medical Centre in 2016, supported by a seed investment from Shunxi Holdings of China. Further investment was provided by individuals in Malaysia and Australia. Cartherics was awarded a number of competitive state and federal grants for its research.
Favorite Memory
What has been Trounson’s favorite memory working for the company so far? Trounson said:
“These would be the demonstration of very efficient killing of cancer in the lab and in animal models; the efficient manufacture of our CAR-iNK cells from gene-edited stem cells (iPSCs) in bioreactor systems; building a platform technology that enables many different products that may control cancer but also may have other potential applications such as autoimmunity, fibrotic diseases, endometriosis, and degenerative brain diseases.”
Core Products
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What are the company’s core products and features? Trounson explained:
“The lead product for the company is a CAR-iNK cell with two gene knock-outs of immune inhibitory genes, termed CTH-401, targeting ovarian cancer. These cells have most of the known natural killer cell activators present and are armed with a CAR (targeting the tumor antigen TAG-72). These cells will be delivered through intra-peritoneal (i.p.) administration, so they have direct access to ovarian cancer lesions in the pelvic cavity. The clinical studies will involve a dose escalation phase, beginning at a low dose, followed by treatment of an expanded cohort of patients at the highest dose level shown to be safe. We hope to begin seeing anti-cancer effects at the higher dose levels. NK cells have been shown to be very safe in other clinical trials.”
“We also have an autologous CAR-T cell product (CTH-004) that we expect will enter phase I clinical trials in 2025 in Melbourne and in China. This has the same CAR and gene knock outs as CTH-401. It is also aimed at treating ovarian cancer.”
“We are also developing CAR-iNK products targeting triple-negative breast cancer, brain cancer and endometriosis, and expect these to begin entering the clinic in the next few years.”
Challenges Faced
What challenges have Trounson and the team faced in building the company? Trounson acknowledged:
“Financing is always a challenge for early-stage biotechnology companies and often the major issue to address. Manufacturing to GMP standards and regulatory demands is also a major challenge, including the need to find GMP grade reagents and GMP-certified facilities. We have been fortunate to have very supportive investors wishing us to succeed and have invested in creating our own clean rooms for early clinical-stage cell manufacturing.”
Evolution Of Cartherics’ Technology
How has the company’s technology evolved since launching? Trounson noted:
“The company was established to develop allogeneic cell therapy products based on iPSCs derived from triple HLA homozygous umbilical cord blood, with an initial focus on iT cells. In validating the gene modifications to be incorporated into its iT cells, the company saw the opportunity to develop an autologous CAR-T therapy, which is the product CTH-004 that we expect will be trialed in Melbourne in 2025 for ovarian cancer and in greater China. The company switched its focus from iT to iNK cells platform to yield the product CTH-401 and now has a platform for producing iNK cells, iT cells, and iMacrophages. These cell types will be armed with CARs and genetically modified for increased immune cell function, and for targeting different cancers and other disease conditions. The lead product is the first of these products to be tested in patients.”
Significant Milestones
What have been some of the company’s most significant milestones? Trounson cited:
– Multiple successful rounds of funding from private investors.
– Development of a CAR-T autologous product to target ovarian cancer.
– Development of an off-the-shelf, allogeneic stem cell-derived CAR-NK cell product for ovarian cancer.
– Establishment of a platform technology for addressing immune cell therapy for cancer and other intractable diseases.
Challenges Faced
What challenges have Trounson and the team faced in building the company? Trounson acknowledged:
“Funding is a constant challenge for a research-based biotechnology company. The funding of research, as distinct from licensing discoveries from other institutions or companies, is a heavy financial burden for biotech companies like Cartherics to conduct their own discovery operations. Preclinical studies and early-stage clinical trials are also extremely expensive. While Cartherics has been able to access some government support through competitive grants, its funding is fundamentally based on investor support, complemented by the R&D tax incentives available in Australia. Cartherics will need continued investor support and will be seeking future private and public mezzanine funding. An IPO will be considered at the appropriate time.”
Differentiation From The Competition
What differentiates the company from its competition? Trounson affirmed:
“Cartherics can utilize its stem cell platform to mix and match various gene edits (gene sequence knock-ins and knock-outs), producing a variety of immune cell products (T cells, NK cells, macrophages etc.) that can be used to target cancer, endometriosis, autoimmune disease, fibrosis, inflammatory disorders and potentially neural disorders. The potential is substantial. Unlike most of its competitors, Cartherics has focused its activities to date on solid tumors, which represent the majority of cancers affecting patients and the greatest unmet need. We are also developing CAR-NK cells targeting endometriosis and actively exploring triple-negative breast cancer, another condition with little therapy available. These targets, therapeutic cell products, and target expansion capacity differentiate us from other companies.”
Total Addressable Market
What is the company’s total addressable market (TAM) size? Trounson assessed:
– Spending on cancer medicines is expected to reach US$375 billion globally by 2027, up from US$196 billion in 2022 (IQVIA Report on Global Oncology Trends 2023).
– Ovarian cancer TAM in 2022 was US$3.3 billion (Global Data); Gastric cancer is at US$5.32 billion in 2024 (Mordor Intelligence); Pancreatic cancer is at US$3.83 billion in 2023 (Research and Markets). Global brain cancer market is forecast to grow from US $2.51 billion in 2023 to US$5.39 billion in 2031 (Markets & Data).
Triple-negative breast cancer had a TAM of US$614.8 million in 2022 (Persistence Market Research).
The global endometriosis treatment market size is expected to reach US$3.2 billion by 2030 (Grand View Research).
Future Company Goals
What are some of the company’s future company goals? Trounson concluded:
“Future goals are:
- Establish CAR-iNK therapies for adenocarcinomas (pancreatic, gastric, liver, kidney, prostate etc.), triple negative breast cancer.
- Demonstrate effective immune control of endometriosis using CAR-iNK cells, CAR-macrophages.
- Develop CAR-iT cells for brain cancer.
- Explore the role of NK cell products in the control of neurological diseases.”
