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Partnership for Innovation: The Strategic Role of Investigator-Initiated Trial (IIT) in Drug Development In the dynamic and rapidly evolving pharmaceutical industry, particularly in the area of modern technologies such as Targeted Protein Degradation (TPD), the key to success is not only innovation in the lab but also strategic clinical partnership. Biotechnology companies developing first-in-class drugs, such as Captor Therapeutics, reach beyond their own research resources and join forces with leading medical centers. An excellent example of this strategic approach is the collaboration between Captor Therapeutics and MD Anderson Cancer Center - a global leader in cancer treatment. The synergy between Captor's innovative technology and MD Anderson's clinical experience creates a new value in drug development. We invite you to explore the mechanism of this partnership, understand the fundamentals of clinical research, and discover how this synergy of science and strategic risk management builds not only knowledge but also higher value for companies, patients, and investors. The Foundations of the Clinical Research Ecosystem To better grasp the significance of Investigator-Initiated Trials (IITs), it's worth reviewing the basics of the clinical trial process and the roles of the individual participants. Two key roles are distinguished in the process of developing new drugs: the Investigator and the Sponsor. Investigator: This is typically a specialist physician working at a clinical center who conducts the trial, has direct contact with patients, administers the investigational drugs, and monitors the safety of the therapy. Sponsor: This is the entity (e.g., a biopharmaceutical company) that takes full legal, financial, and ethical responsibility for the trial before regulatory authorities (FDA, EMA). The development of new drugs requires a precise plan, which is the clinical development strategy. This strategy defines in which diseases (indications) the drug has the greatest potential, what studies must be conducted to register the drug, and the order in which to execute subsequent trial phases, minimizing risk and maximizing the chances of success. The process of bringing new drugs to market is divided into four main phases: Phase I: The first administration of the drug to humans, focusing on safety assessment and establishing the appropriate dose. Phase II: Assessment of the drug's efficacy in treating a specific disease and further safety monitoring. Phase III: Confirmation of efficacy and safety in a large group of patients (often thousands of people), comparing the new drug with currently used therapies. The results of this phase are the basis for drug registration. Phase IV: Studies conducted after drug registration, aimed at monitoring long-term safety and efficacy in a broad patient population. The Mechanism of the Investigator-Initiated Trial (IIT) In the context of collaboration with academic centers, the Investigator-Initiated Trial (IIT) plays an increasing role. An IIT constitutes a key stage of advanced clinical research in the development of innovative therapies. Participation in such trials provides patients with access to the latest, advanced therapies and specialist medical care, which is a significant benefit for participants. An Investigator-Initiated Trial is a specific model where a beneficial role reversal occurs for innovative companies: Initiator and (Legal) Sponsor: The independent investigator or scientific institution becomes the originator of the study and takes on the regulatory and operational responsibility, and crucially, covers the lion's share of the costs. They are responsible for patient safety and data quality before regulatory bodies, such as the FDA or EMA. Supporter (Technological Partner): A company like Captor Therapeutics supplies the innovative drug, a small portion of the funding (grant), and/or technical expertise. Depending on the agreement, the Technological Partner may participate operationally in the trial. Thanks to this model, the company can leverage the enormous intellectual and infrastructural resources of the partner without fully burdening its own operational resources with the entire trial management process. Strategic Value and Benefits of IITs for the Company and Patients Collaboration in the IIT model brings many benefits to both companies like Captor Therapeutics and to researchers and patients. Faster Generation of Scientific Data: IITs allow for the quick testing of new hypotheses and drug applications that may not be included in the company's main clinical programs. This allows for an expansion of knowledge about the drugs and their mechanisms of action at a relatively low cost. Resource and Cost Optimization: The company incurs significantly lower costs and operational responsibility associated with conducting the trial, enabling more efficient capital management and risk minimization in early development phases. Independent Validation: Results from independent, academic sources have high credibility and can contribute to a better understanding of the therapy's safety and efficacy. Strengthening Relationships with Opinion Leaders: Collaborating with key researchers and clinical centers builds strong partnerships that are valuable in the further development and commercialization of drugs. Exploration of New Indications: The trial's objective often involves exploring new indications, studying the drug's mechanisms of action, testing therapies in rare or difficult-to-treat patient groups, and checking safety and efficacy in specific clinical contexts. The Perspective of Collaboration for Investors For investors, information about a company's collaboration with researchers within an IIT is an important signal. The interest of independent scientists in the product being studied, such as protein degraders, demonstrates its value and therapeutic potential. Data regarding the efficacy and safety of the therapy, which can be decisive for the project's further development, is crucial here. Moreover, this collaboration indicates the efficient use of company resources, investing in development and research while benefiting from academic expertise and infrastructure. IITs often test drugs in new diseases or unique patient subgroups, which can lead to market expansion and an increase in the company's value. Investors also pay attention to the challenges associated with drug resistance and the potential of new therapies, such as those being developed by Captor Therapeutics, in overcoming these mechanisms. Captor Therapeutics and MD Anderson Partnership Captor Therapeutics is strategically utilizing the Investigator-Initiated Trial model to accelerate the development of its leading molecule - the MCL-1 protein degrader (CT-03p), primarily intended for the treatment of hematological cancers. The company has entered into a collaboration agreement with the renowned MD Anderson Cancer Center (MDACC). The clinical trial, which will be led by Dr. Abhishek Maiti from MD Anderson, is a continuation of promising preclinical studies that showed CT-03p effectively degrades MCL-1 and acts synergistically with other therapies, even in resistant cells. Such collaboration with a center possessing a unique patient cohort, like MDACC, allows Captor Therapeutics to reduce risk and more efficiently manage clinical progress, increasing the chances for drug registration. This combination of an innovative molecule with prestigious clinical validation is a key component in increasing the company's value and lowering investment risk. Furthermore, MDACC is the center that conducted key studies for venetoclax - the most important drug for combination therapy with MCL-1 degraders. From Clinical Results to a Billion-Dollar Valuation: The Story of ImCheck Therapeutics The history of the French company ImCheck Therapeutics is an excellent testament to how the synergy of science and business translates into financial success. This company, developing the drug ICT01 (γ9δ2 T-cell activator), based its success on close integration with leading clinical centers within the EVICTION trial. Independent researchers played a key role here, such as Dr. Abhishek Maiti from MD Anderson Cancer Center, who presented promising data on the treatment of acute myeloid leukemia (AML) at the AACR 2025 conference. The credibility of the results, confirmed by medical authorities, became the foundation for a business transaction, and the market predicts further effects of the synergy between science and business in the form of subsequent breakthroughs. The result? The pharmaceutical giant Ipsen announced the acquisition of ImCheck for up to 1 billion euros (approx. 1.16 billion USD) to include the clinically verified drug in its portfolio. The groundbreaking effects of this clinical collaboration confirm the importance of innovative mechanisms of action and the impact of implementing new therapies. The course of ImCheck Therapeutics' development clearly demonstrates that strategic cooperation is the main financial lever in biotechnology, due to clinical validation by renowned centers: Risk Reduction: Partnering with clinical practitioners reduces risk at an early stage, which directly translates into a leap in company valuation. This allows for the quicker identification of optimal patients. Collaboration is the Path to Success: Strategic cooperation is the key to rapid growth in enterprise value and effective entry into advanced registrational study phases. Validation by researchers is the main financial lever, not just additional data. Summary: The Value of Investigator-Initiated Trials Investigator-Initiated Trials are a strategic lever for biopharmaceutical companies, extending beyond merely being an additional source of data. This model, by transferring operational responsibility to independent centers, enables the optimal use of capital and minimization of risk in the early development phases of innovative therapies, such as those based on Targeted Protein Degradation (TPD) technology. IITs accelerate the exploration of new indications and the building of relationships with key opinion leaders. For investors, the crucial point is that IIT results provide independent validation of the technology, confirming its market potential. This strategic synergy of science and industry is the key to faster introduction of breakthrough therapies and increased treatment efficacy. Sources: ICH Official web site : ICH Perspectives in Clinical Research Investigational New Drug (IND) Application | FDA What Are Clinical Trials? - NCI Investigator-initiated studies: Challenges and solutions - PMC ImCheck Ipsen Captor Therapeutics & MD Anderson Cancer Center