Cartherics is a biotechnology company focused on developing innovative cancer therapies. Pulse 2.0 interviewed Cartherics CEO Prof. Alan Trounson to learn more about the company.
Dr. Trounson’s Background
What is Prof. Trounson’s background? Prof. Trounson said:
“Graduated as a wool technologist (BSc in Wool & Pastoral Sciences, University New South Wales, Sydney). Working in sheep genetics and physiology (MSc UNSW) and then PhD in sheep embryology and reproduction – Sydney University).”
“Postdoctoral Research Fellow at ARC Unit Biochemistry and Reproductive Physiology, Cambridge UK (1974-77). Joined Professor Carl Wood in 1978 (Dept Obstetrics & Gynaecology, Monash University) to develop assisted reproductive technologies, including IVF. Pioneered the use of fertility drugs for successful IVF, embryo freezing, egg and embryo donation, sperm injection into human oocytes (subzonal injection) for male infertility, vitrification of oocytes and embryos, and freezing of ovarian tissues (see review attached). Appointed Professor at Monash University 1991.”
“Pioneered the development of human embryonic stem cells (1998-2000). Awarded the Australian Biotechnology of Excellence – Australian Stem Cell Centre by Australian Government ($110 million) 2002. Returned to Monash University to direct Monash Immunology & Stem Cell Labs.”
“Appointed President of the Californian Institute for Regenerative Medicine (CIRM) (2007-2014) – $3 billion initiative to drive stem cell research and clinical applications.”
“Returned to Australia – formed Cartherics Pty Ltd to develop stem cell immune CAR (chimeric antigen receptor) therapies for cancer.
Formation Of Cartherics
How did the idea for the company come together? Prof. Trounson shared:
“I thought chimeric antigen receptor (CAR) research was the most promising work in cancer we were supporting at CIRM.”
“I wanted to combine stem cells with CARs for off-the-shelf therapies for human cancer and was convinced this was the best way forward. We needed expertise in immunology (Professor Richard Boyd from Monash Uni, Chief Scientific Officer), international biotechnology expertise (Dr. Ian Nisbet, Chief Operating Officer), antibody design expertise (Dr. Peter Hudson) and business (Robert Moses) – together, we founded the company in 2015 and began operations with private investment support in 2016.”
Favorite Memory
What has been your favorite memory working for the company so far? Prof. Trounson reflected:
“Seeing the incredible efficiency of our CAR-T cells killing human ovarian cancer in immune suppressed mice and then the stem cell-derived natural killer (NK) cells likewise, or even more efficiently, killing and controlling human ovarian cancer in immune suppressed mice. Then to be able to manufacture tens of billions of these CAR-NK cells in bioreactors that may be used to control ovarian cancer in patients.”
Core Products
What are the company’s core products and features? Prof. Trounson explained:
“The lead product (CTH-401) is a CAR-NK cell that targets ovarian cancer. The CAR target is TAG-72, a marker that appears on >95% of tumors in patients with ovarian cancer. This marker is not expressed by normal tissues but is commonly expressed on all adenocarcinomas of the major organs of the body. CTH-401 also has two genes knocked out which are involved in pathways that tumors use to suppress the killing capacity and longevity of immune cells. These knockouts prevent the natural tumor suppression of CAR-NK and other immune cells.”
“We will explore the use of CTH-401 for control of other cancers in basket studies of patients who have TAG-72 expressing cancers other than ovarian cancer (gastric, colorectal, uterine, pancreatic, prostate, kidney, etc.).”
“We also have a new CAR target – Tissue Factor (TF) – that targets triple negative breast cancer, currently in development. This CAR target was accessed under an agreement with Ohio State University.”
“We have additional products in the pipeline that include dual CARs for “hold and punch” killing, as well as additional gene knockouts that may further improve control of cancers.”
Challenges Faced
What challenges has Prof. Trounson faced in building the company? Prof. Trounson acknowledged:
“Our most difficult challenge has been to obtain funding to advance our lead product into initial clinical trials and expand our pipeline. We have been well funded to date, but our IND-enabling studies require substantial funding for expensive activities, such as outsourced non-clinical toxicology and safety studies and GMP manufacture of our CAR-NK cells from our master cell bank of gene-edited stem cells.”
“Our pre-IND meeting with the US FDA was very positive and we know what needs to be completed for our IND submission. Since we are still a private, pre-clinical-stage company, finding funding is challenging. But we are determined to bring CTH-401 into clinical trials and progress our pipeline products to demonstrate the power of the stem cell immune therapy approach for cancer management.”
Evolution Of Cartherics’ Technology
How has the company’s technology evolved since launching? Prof. Trounson noted:
“The company initially developed an autologous CAR-T cell product but passed the clinical development to others, to concentrate on the allogeneic (off-the-shelf) version based on stem cells. For this we have partnered with ToolGen (a Korean company) for gene-editing tools and rights to specific gene knockouts. We have since increased our partnerships with other companies and universities to enable the development of a full pipeline of products that enable mix and match of CARs, gene knockouts and other enabling technologies. These can be developed using the manufacturing blueprint of CTH-401. This is a platform technology that enables rapid preparation of new and enhanced products targeting specific cancers and other medical applications.”
Significant Milestones
What have been some of the company’s most significant milestones? Prof. Trounson cited:
“The most significant milestones are pre-clinical and include positive discussions with the FDA (pre-IND) on CTH401; efficacy studies on immune-suppressed mice for the CAR-T and CAR-NK products; the establishment of GMP master cell bank; selection of critical clones with no detected mutations after gene editing the stem cells.”
Customer Success Stories
After asking Prof. Trounson about the company’s customer success stories, he highlighted:
“Since we are still at the pre-clinical stage of development, we don’t have customers per se at this stage. However, we do engage closely with clinicians who may ultimately use our products and with potential pharmaceutical company partners. We also interact with patient advocates and patients, who are extremely supportive. Some individuals with ovarian cancer want to be included in the clinical trials and one has joined the company Board as an advisor.”
Funding
After asking Prof. Trounson about the company’s funding information, he revealed:
“To date, the company has raised about $30 million in private capital, which it has supplemented with grant income and the Australian government’s R&D tax incentive. The company has its own purpose-built R&D facility in Melbourne, Australia. We are capital raising at present to ensure our lead product enters clinical trials in mid-2025 and to bring the next CAR-NK product though preclinical development. We are seeking lead investors to commit around AUD$5 million to underpin an AUD$10 million to AUD$20 million capital raise prior to a public offering expected in 2026.”
Total Addressable Market
What total addressable market (TAM) size is the company pursuing? Prof. Trounson assessed:
“Spending on cancer medicines is expected to reach $375 billion globally by 2027, up from $196 billion in 2022 (IQVIA Report on Global Oncology Trends 20923)
Ovarian cancer TAM in 2022 was $3.3 billion (Global Data); Gastric cancer is at $5.32 billion in 2024 (Mordor Intelligence); Pancreatic is at $3.83 billion in 2023 (Research and Markets). Our new CAR-TF targets Triple Negative Breast Cancer with a TAM of $614.8 million in 2022 (Persistence Market Research).
Differentiation From The Competition
What differentiates Cartherics from its competition? Prof. Trounson affirmed:
“Cartherics has a platform stem cell technology that allows mix and match of CARs created in iPSCs, with enhancers and gene knockouts that can be readily optimized for cancer therapeutics. Few competitors are focused solely on solid tumors where the largest unmet need exists (e.g., ovarian, gastric, pancreatic, and triple-negative breast cancers).”
“Our lead product is a stem cell-derived NK cell, clonally derived and DNA deep sequenced to ensure safety from secondary cancers and other undesirable mutations. These features do not appear in competitor products. We have the capacity to also introduce stem cell-derived T cells or macrophages for use in cancer treatments or other disease conditions.”
Future Company Goals
What are some of the company’s future company goals? Prof. Trounson concluded:
“We aim to bring our lead product CTH-401 to clinical trials in ovarian cancer; undertake a basket study of adenocarcinomas expressing TAG-72 to determine the effectiveness of CTH-401 in cancers of the internal organs; complete our dual CAR-NK cell with novel gene knockouts through preclinical development for ovarian and gastric cancers; bring our new CAR-TF through preclinical development for targeting triple negative breast cancer; we intend to explore the use of CAR-technology in other disease conditions with appropriate partners.”
“For more information on the TF CAR please see our recent announcements detailing collaborations with Ohio State University and TiCARos.”
