Four Doctors for One, or What's Going on at Captor Therapeutics? [INTERVIEW]

In May, Captor initiated administration of CT-01 at a clinic in Barcelona, ​​a leading trend in liver cancer treatment. This is the first phase of clinical trials, which has already demonstrated good tolerability of the molecule. A partnering agreement for this oncology drug candidate could be announced next year. The company plans to launch clinical trials for CT-03 in the third quarter of 2026. The stakes are high, as the company's management estimates that the market potential for CT-01 significantly exceeds $1 billion annually, and for CT-03, we're talking about several billion at peak sales. Investors are focusing on the development progress of CT-01, perhaps losing sight of the potential of CT-03. And the results of the CT-03 project are so good that some potential partners are looking at them with disbelief.

The process of discovering drug candidates is complex, especially when it comes to the incredibly demanding field of oncology. More than one daredevil has failed miserably, ending their business careers in disgrace. Cancer therapies are both a lucrative slice of the biopharmaceutical pie and address vital patient needs. Therefore, it's no surprise that many healthcare companies, undeterred by the failures of some, are participating in the quest for Jason's golden fleece. Among them is the Wrocław-Basel-based Captor Therapeutics (CTX), whose drug candidate, CT-01, was recently nominated by industry magazine Drug Hunter as the molecule of the month for June.

Especially for the readers of politykazdrowia.com, four leading CTX managers shared their observations on what is happening in the business and how research and development work is progressing.

"Four doctors per patient" – we haven't played this one out before. Each of my interviewees holds a doctorate in their field. It was worth taking such an unconventional approach to the interview, as it revealed a range of views on the Captor Therapeutics business from different perspectives. Who are they?

Michał Walczak, President of the Management Board and Chief Scientific Officer (CSO) , Responsible for research and strategy implementation. A biophysicist and structural biologist with a doctorate from the Swiss Federal Institute of Technology in Zurich (ETH Zurich). He also graduated from the University of Wrocław and completed his master's thesis at the University of Virginia in Charlottesville. He completed a postdoctoral fellowship at the Friedrich Miescher Institute for Biomedical Research in Basel (FMI Basel), part of the Novartis Institutes for Biomedical Research.

Sylvain Cottens, co-founder and SVP Chemistry , is responsible for executing chemistry activities and strategy. Previously, he served as Global Head of the Center for Proteomic Chemistry at Novartis Institutes for Biomedical Research, where he played key roles in numerous successful R&D programs (e.g., Afinitor™ and the in-licensing Gilenya™). He holds a PhD in organic chemistry from EPFL in Lausanne.

Robert Dyjas, head of Medical Affairs and Clinical Development , responsible for the clinical development processes of drug candidates. A medical doctor with a specialization and doctorate in internal medicine with over twenty years of experience in the clinical development of innovative drugs, both in global pharmaceutical companies, a global CRO and a biotechnology company.

Anna Pawluk, Operations Director , oversees the implementation of optimal processes, standards, and solutions to support the smooth operation of the company. She holds a PhD in biological sciences in biotechnology from the University of Wrocław and is a graduate of the Executive MBA program at the WSB University in Wrocław.

Marcin Kuchciak (MK): Do you know what I pay attention to first when it comes to any listed company, and especially in the healthcare business?

Michał Walczak (MW): No.

MK: On what it knows about its competitive environment. Because you can navigate the bumpy road of the operational process, equipped with superhuman confidence in your development concept, but if you have no idea what the competition is doing, you're essentially walking around like a child in a fog. Unless you're so unique that you're developing a product that the Russians say "niet analogue v mirie," and even that's a bit risky, because someone could come up with something new any minute now.

To me, it's madness to select a molecule for development without a thorough analysis of patient needs and what business rivals are doing or want to do. So let's start with what's happening in the competitive landscape surrounding Captor Therapeutics.

MW: I think it's a great approach, and one we use ourselves. I'll even go a step further, because we have to try to predict what the competition and patient needs will look like in 5-6 years , when we're administering drugs to patients.

The very concept of competition is multifaceted, as we can speak of technological, design, and disease-related competition. The former, of course, refers to competitors pursuing the same technology and pose a threat to us from the perspective of partnering in the discovery of new drugs for Big Pharma. Here, we have competitors in the form of companies like Kymera, Arvinas, Nurix, C4 Therapeutics, and MonteRosa. Each company has its own advantages in technological nuances and limited capacity in terms of partnering opportunities. We're also competing technologically with Asian companies, but these are service-oriented companies.

Captor stands out in that it has a proposition for a large group of smaller pharmaceutical companies that cannot pay the upfront fee of USD 50 million and that would also like to give themselves a chance to develop in targeted protein degradation - this is how we started cooperation with Ono Pharmaceutical, and previously with Sosei Heptares.

Compared to service companies, our heritage is rooted in drug development, i.e., developing molecules from early discovery to clinical phases. This means we work in an interdisciplinary manner, with close collaboration between disciplines and an understanding of the trade-offs that must be made. In service companies, each element of the drug discovery chain is practically a company within a company.

The second type of competition is design competition, where other companies are developing molecules targeting the same protein targets as us. Here, the Swiss-American MonteRosa, which has a GSPT1 protein degrader in Phase I/II clinical trials for the treatment of prostate cancer, as well as NEK7 protein degraders, is on the horizon .

Regarding GSPT1, our molecule is primarily a prodrug that is selectively activated in diseased liver and possibly in diseased lungs. Additionally, our molecule removes a second disease-causing protein, NEK7. Due to the highly complex mechanism of action of this drug candidate, I like to think it is potentially a "first in class" drug—similar to Revlimid in the treatment of myelodysplastic syndromes.

Our dark horse project is a molecule targeting the MCL-1 protein, one of the horsemen of the cancer apocalypse. We don't see any competition here, which stems from the fact that there have been so many failures in the development of drugs targeting this protein. However, we have something different, but Sylvain will discuss that.

The third type of competition is disease competition, and it's powerful, if only because we have relatively few diseases and many more companies, most of which have multiple projects . Why this happens is a topic for a separate discussion.

MK: The R&D process in biopharma is truly about people. Please tell us about the specific qualities of the team you lead, as this increases the likelihood of success. And how you manage it, as the process seems incredibly important in this area.

MW: Teamwork is definitely key in R&D, and in biotechnology, experience is more important than IQ, which can't be gained alone, as in IT or academia . Furthermore, the drug discovery process is very time-consuming and changes significantly as the project progresses. We're fortunate to have a significant amount of key experience at Captor.

Let me start by saying that to be able to talk about science and R&D, you need to have facilities and support. By facilities, I mean laboratories, offices, internal services (e.g., internal order logistics), external services, administration, IT, grant writing, and tenders.

We're very fortunate to have Ania Pawluk (a board member since June of this year) overseeing everything. She's been with the company from the very beginning and has built it. She also has a deep understanding of biotechnology, which allows her to efficiently manage all work to ensure everything runs "agile," using IT terminology.

The second board member is Tomáš Drmota , who worked for over 15 years at the outstanding pharmaceutical company Astra Zeneca, where he rose through the scientific ranks from laboratory scientist to executive director. Tomáš, an excellent drug hunter, has introduced many of the processes he learned and implemented at Big Pharma.

Sylvain Cottens has also been with us since the beginning of Captor – a veteran of another Big Pharma, Novartis, where in his last role he managed a group of 300 people in Basel and Boston at the elite R&D institute, NIBR (short for Novartis Institutes for Biomedical Research). To learn how fantastic NIBR was, I recommend reading this text: https://www.alexkesin.com/p/the-day-novartis-chose-discovery .

I'll add that Sylvain discovered the drug everolimus and licensed the molecule fingolimod to Novartis . Both have become huge sales hits.

The next person I'll mention is Robert Dyjas , Director of Medical Affairs and Clinical Development. Robert has unprecedented experience in this area, having previously worked at Big Pharma, a mid-sized biotech company, a startup, and companies providing clinical development services to small entities. This experience allows us to design clinical trials that are cost-effective yet deliver maximum results. Furthermore, Robert's knowledge, experience, and contacts enable us to rapidly recruit patients and access the world's best medical specialists.

We also have Adam Łukojć , a well-known figure on the capital market, who oversees finances and, although he has only been with us for a short time, has found his place in the technology start-up very well.

Finally, I'd like to mention Anthony Rowena-Brown , a business development director who was a lecturer at Oxford, knows the drug development process, has worked at several biotech companies, and his group developed a molecule that recently became an approved drug for the treatment of breast cancer - inavolisib .

There are many other equally talented and committed people working at Captor, but it is difficult to list them all.

When it comes to management, I prefer to think of it in terms of collaboration—we have regular one-on-one meetings, group meetings, and reviews of project progress and budget strategies. We create business and scientific plans and goals together, and we constantly revise them.

MK: How did it all actually begin? Capital market investors closely monitor progress in subsequent phases of clinical trials, and ultimately, once a drug is approved by global regulators, they observe how the molecule is adopted by patients. With this perspective, few people understand the conceptual phase of developing a new drug and what preclinical testing entails.

What did these processes look like for you? Let's jokingly assume that one day someone wakes up from a prophetic dream and declares: I have an idea for how to obtain a molecule essential for the treatment of hepatocellular carcinoma.

Sylvain Cottens (SC): For us, it all started with the desire to discover the degraders of a transcription factor (SALL4) believed to play a key role in hepatocellular carcinoma, the most common form of liver cancer. This project was co-financed by a grant from the National Centre for Research and Development.

A key element of our strategy was to test a library of potential degraders for their ability to kill liver cancer cells without affecting other cells. As a result, we identified a lead compound with these properties.

To our surprise, however, we discovered that the identified compound did not bind to SALL4 on its own but was converted by the enzyme VAP-1 into an active metabolite. Because this enzyme is abundant in the human liver, this provided an opportunity to identify a drug candidate that would be produced locally in the liver. We found that the resulting metabolite was a potent degrader of GSPT1 and NEK7, both important targets in hepatocellular carcinoma, but only a weak degrader of SALL4. We demonstrated that GSPT1 degradation was the primary mechanism of direct action against liver tumors.

We then optimized the compound for pharmacokinetic properties and tested the drug candidate in vivo in a range of human tumor models in mice, where the compound demonstrated remarkable potency. The next step was to assess the compound's safety in animal studies before initiating clinical trials.

Source: Captor Therapeutics

MK: Now that we've reached this point, it's impossible to ignore the role of artificial intelligence in the invention process. Some say that if you don't use AI, you're a dinosaur who doesn't know how models can do good. What's it like for you? Do you use AI in research and identifying promising drug candidates? If so, what exactly does it involve?

MW: We do use it, though not as extensively as in other disciplines. When it comes to AI, for it to work well, it must first learn its domain on good data, and statistics show that in biology, scientists fail to replicate even 70-80% of the results published in top journals.

We use AI techniques to predict certain properties of chemical molecules and how they interact with proteins. We also have our own unique biophysical and structural datasets that could be used to develop new AI methods. But for this, we'll need a partner specialized in AI and with an understanding of drug development.

To sum up, we use artificial intelligence tools and create simpler solutions ourselves.

MK: Captor Therapeutics specializes in Targeted Protein Degradation (TPD) technology. What does it involve?

Robert Dyjas (RD): Targeted protein degradation is an innovative therapeutic method that utilizes the cell's natural protein degradation mechanism—the ubiquitin-proteasome system—to selectively target proteins involved in the pathogenesis of disease and eliminate them through a cellular system that ensures homeostasis. Unnecessary proteins are eliminated by being marked for degradation by the ubiquitin system, and the protein structures thus marked for degradation are destroyed by the proteasome, a cellular structure that acts as a "grinder" for their destruction.

It has been shown that TPD technology, compared to commonly used drugs , which are most often inhibitors with a chemical structure of a small molecule, in the treatment of many diseases, including cancer , has the ability to target pathogenic proteins, including non-enzymatic ones, which were previously considered impossible to eliminate , as factors causing the development of diseases.

The first protein degraders entered human clinical trials in 2019, a new field of experimental treatment. The results of studies on the first TPD molecules in clinical development indicate a satisfactory safety profile and favorable pharmacokinetic (how the body responds to the drug candidate) and pharmacodynamic (how the drug candidate affects the body) parameters, justifying their use in the treatment of diseases, primarily cancer.

MK: Looking at the stage of the discovery cycle, Captor Therapeutics is clearly a "young" biopharmaceutical company. It doesn't have any approved drugs. In May of this year, you entered Phase I clinical trials with one molecule (CT-01), targeting hepatocellular carcinoma. So let's take a look at your current state of innovation, considering the company's multi-product pipeline.

RD: Captor Therapeutics is in clinical development with its first program , CT-01, for the treatment of hepatocellular carcinoma. Following European regulatory approvals , the first patient received CTX's drug candidate on May 19, 2025, in a Phase 1 (First in Human) study at a renowned liver cancer clinic in Barcelona, ​​led by Dr. Maria Reig, who also serves as Principal Investigator . This center sets global standards for the diagnosis, staging, and treatment of liver cancer.

The study drug is being administered to the next patient at a higher dose and is well tolerated. The study aims to assess safety and tolerability, evaluate pharmacokinetic and pharmacodynamic parameters, and determine the dose for subsequent phases of clinical trials following the use of CT-01 . The study design is adaptive, meaning that subsequent stages of the study can be implemented in full or in limited doses, and their initiation will depend on the analysis of the obtained results.

Another project, CT-03 , using an innovative MCL-1 protein degrader, the removal of which may contribute to obtaining a positive therapeutic response in patients diagnosed with acute myeloid leukemia, is currently in the last stages of preclinical development, and the start of the clinical part, i.e. treatment of patients, is planned for the third quarter of next year .

In the CT-02B and CT-02S projects, we have molecules that could be clinical candidates. However, we will conduct the required preclinical studies either with a partner or only after securing funding.

Source: Captor Therapeutics

MK: The CT-01 project is the most advanced. What about the other leading drug candidates, CT-03, CT-02B, and CT-02S?

SC: In the CT-03 project, we discovered a highly potent and selective degrader of MCL-1, a protein that plays a key role in many blood cancers and solid tumors such as lung and breast cancer . MCL-1 inhibitors were the Holy Grail for treating these cancers, but unfortunately, they failed to demonstrate activity in clinical trials. This is because these inhibitors increase the amount of MCL-1 in the body, counteracting the inhibition caused by the drugs and leading to adverse effects on patients' hearts.

Our MCL-1 degrader works completely differently and doesn't have the drawbacks of inhibitors . We are now confirming the safety of our unique MCL-1 degrader in a four-week, GLP-compliant animal study.

Our CT-02S and CT-02B molecules degrade NEK7, an enzyme that plays a key role in inflammatory diseases . NEK-7 activates the NLRP3 pathway, which is attracting significant interest from pharmaceutical companies. These degraders potentially address a wide range of indications, including osteoarthritis, gout, high-risk cardiometabolic syndrome, fatty liver disease, Parkinson's disease, and amyotrophic lateral sclerosis , to name a few. Our NEK-7 degraders are highly selective and allow for once-daily treatment , a key advantage over current NLRP3 inhibitors.

MK: Let's now talk about TAMs, or target market values ​​for individual discovered drugs. Investors are probably most curious about the situation with CT-01 and CT-03, as they are the most advanced in the process. Sometimes analysts also calculate peak sales. What do you estimate this to be? What competitors will we face if the entire process is successful? Would it be fair to say that any other biotech company is a direct competitor to Captor, and that the company's achievements should be best compared to them?

SC: Our CT-01 degrader addresses a significant unmet medical need. The current standard of care for hepatocellular carcinoma only extends patients' lives by less than a year. The number of patients with liver cancer is growing rapidly.

Historically, accurately predicting sales of any drug has been a challenge. Nevertheless, the market potential for CT-01 is significant, well over $1 billion annually. In the case of our CT-03 degrader, to the best of our knowledge, no other MCL-1 degrader is currently in preclinical development. The patient populations that could benefit from our drug candidate are large, and the medical need is high, as in most cases, patients have no other treatment options. Therefore, the market potential at peak sales is several billion dollars annually.

It's not as if any biotech company could create similar drug candidates. First, our molecules work by degrading proteins, and few biotech companies are capable of utilizing this approach. Furthermore, our capabilities in protein engineering, biophysics, X-ray crystallography, and medicinal chemistry are unique among biotech companies working in this field.

MK: Companies in the sector that rely solely on discovery tend to burn through cash, and future cash flows are usually delayed. Unless, of course, we share the discovery risk through various partnering agreements. What is the company's management's approach in this regard? The burn rate shows that CTX has the cash to conduct research and maintain the business until early 2026. Let's assume we break even in Q1 2026. What next steps does the company plan to take to continue operations?

Anna Pawluk (AP): Burning through cash is typical for biotech companies developing their projects. Dutch-based Argenx, the largest biotech company in Europe, reported an operating loss as recently as 2024 – and by the end of 2024, it was valued by investors at over €30 billion, more than the largest Polish company listed on the Warsaw Stock Exchange.

But we know we won't get as much patience from investors as Argenx did. Therefore, our strategy involves entering into partnering agreements at a stage where we don't yet have to incur the large costs associated with a phase II or III clinical trial, but can already receive decent royalties from the sale of the drug registered by our partner. The optimal time for this seems to be the first phase of a clinical trial.

The cash we currently have will last us until approximately mid-2026. We have submitted four grant applications and are also in talks about partnering agreements and research collaborations. We are also considering a share issue, although at the current share price, we would prefer not to dilute existing shareholders. We believe our projects are worth many times more than the stock market currently values ​​them at.

MK: I assume that at least preliminary partnering efforts for CT-01 are underway. If I'm not mistaken, who are you talking to? Who is within the company's sphere of interest? What level of potential revenue can we expect from this? And how are these tranches distributed over the timeline?

There is a huge movement in the partnering business at TPD, which can be seen from the slides on pages 32 and 33 of this presentation (the Captor Therapeutics business is described in detail on almost 70 pages) – link: https://captortherapeutics.pl/relacje-inwestorskie/materialy-inwestorskie/prezentacje

AP: US-Swiss Monte Rosa Therapeutics has entered into a partnering agreement with Novartis. The agreement covered a project with significant overlap with CT-01. Monte Rosa Therapeutics received $150 million in upfront payments from Novartis and could receive up to $2.1 billion in milestone payments and undisclosed royalties. The agreement was reached after the drug had been administered to only a few patients.

This is a major advantage of targeted protein degradation: quite early in a clinical trial, a significant amount can be said about the drug's chances of effectiveness. Degradation, or removal, of the target protein can be observed shortly after drug administration. Similar conclusions can be reached later with many other types of drugs.

We don't expect our initial payment to be as high as the one Monte Rosa received. However, we hope to conclude a satisfactory partnering agreement in 2026.

Clinical trials for liver cancer should move forward quickly because there are many patients and a shortage of good drugs, so we should probably start receiving milestone payments and royalties relatively soon after concluding the partnering agreement .

But we're not just talking about CT-01. Our results in the CT-03 project are so good that some potential partners are skeptical. This project has the potential to be a major breakthrough in oncology, so we'd like to conclude a partnering agreement on truly favorable terms.

We're also seeing interest in our autoimmune drug projects: CT-02(B), CT-02(S), and CT-05. For these projects, a preclinical partnering agreement would yield less revenue than we'd like, but it would help us achieve success in other projects.

MK: Until recently, some people were excited about the millennium bug, due to the change in date from the 20th to the 21st century. No catastrophe occurred, although some experts, feeding humanity with fear, predicted apocalyptic scenarios. A quarter of a century has flown by like a whip. The world is developing, thanks to scientific discoveries. If you had to name the world's five greatest discoveries in healthcare that have made it from the clinical to the commercial phase, what would they be and why?

SC: First, I'd like to mention lenalidomide, the first drug to act by degrading proteins . It has helped tens of thousands of cancer patients, generating peak sales of over $10 billion annually and cumulative sales of over $100 billion.

Then there are kinase inhibitors, such as imatinib , which work by blocking mutant kinases in tumors. These are important to me because they illustrate the concept of personalized medicine and were made possible only by advances in basic science, in this case DNA sequencing, whose discovery was awarded the Nobel Prize in 1980, and by advances in automation and nanotechnology in the early 21st century.

Treatments for HIV-infected patients are also crucial . These drugs became commercially available shortly after the virus was discovered and have been continuously improved over the years. They have transformed a rapidly progressive, fatal disease into a chronic, remitting one. Recently, therapies have emerged that can prevent transmission of the virus . It's been an incredible odyssey in a short time.

Among the newer drugs, weight-loss medications are of interest not only because of their enormous commercial success but also because they illustrate advances in drug-injection devices . Remarkably, these drugs have already demonstrated significant benefits in reducing cardiometabolic risk, and one has already been approved for the treatment of fatty liver disease. The benefits of these drugs clearly extend beyond weight loss.

MK: Thank you for the interview.