In the second half of September 2022, we announced an optimization of the development plan in the PB004 project, introducing the clinical phase 0 into the plan, during which we plan to obtain the first important information on the effectiveness of the drug candidate at the very beginning of clinical development.
Obtaining the results of phase 0 will allow us to obtain the first information about the effectiveness of the compound in humans faster than by the classic approach, based on phase I and II clinical trials. At the same time, these data will be obtained with significantly lower (compared to the classic approach) financial outlays. Additionally, we decided to improve the construct of the potential drug under development, thanks to which we expect to obtain better clinical results.
The Phase 0 clinical trial will involve administering microdoses of a drug candidate directly to neoplastic tumors, which will allow us to confirm the mechanism of action of the PB004 molecule in the complex tumor microenvironment and to better estimate the potential efficacy of a drug candidate in anti-cancer therapy.
– Data collected under near-real conditions in the context of the intended use of the drug will positively affect the commercial potential of the project. First, a potential partner entering the transation will have access to the first data demonstrating the effectiveness of PB004 in humans. Secondly, the results will help to design the next phases of clinical trials without causing delays in the project, explains Dr. Niina Veitonmäki, Project Manager PB004.
The implementation of the Phase 0 study as the first step in the clinical development of immuno-oncology projects is in line with Pure Biologics’ smart clinical development strategy to obtain valuable pharmacodynamic data at an early stage of molecule development to reduce the risk of failure of costly later clinical stages and significantly increase project valuation at an early-stage clinical development.
– Biological compounds have statistically better safety profile than chemical molecules. At the same time, the statistics in the field of immuno-oncology indicate a higher probability of success in particular stages of clinical development than in the case of traditional drugs. Therefore, there is a high chance that PB004 would have successfully passed the first phase of clinical trials, where safety is primarily studied, and only in the second phase of the study would we obtain the first data on effectiveness. Pharmaceutical companies are also aware of this, therefore, by signing partnering agreements for this type of drug candidates, they have strong arguments in negotiations. Therefore, when thinking about early partnering for PB004, we want to have preliminary data confirming the effectiveness of our molecule in humans, to maximize the value of a potential transaction, explains Dr. Filip Jeleń, co-founder, significant shareholder and President of the Management Board of Pure Biologics.
At the same time, the Company decided to change the molecular format of the drug candidate, which results from Pure Biologics’ own research, as well as observations of market reports. The expected dosing regimen is likely to require a longer drug half-life than is achievable for the originally proposed BIKE (bispecific killer engager) format. This issue is addressed by the improved molecular format introduced as part of the annex.
– The proposed new construct is expected to obtain a longer half-life in the body, due to the molecular properties of the molecule. In the long term, we expect that this will contribute to better parameters of the developed drug, explains Dr. Niina Veitonmäki.
The PB004 project, part of the company’s biological drug development portfolio, aims at the development of a new therapeutic molecule in the format of a non-classical antibody, which is to stimulate the immune response of NK cells to tumor cells expressing the ROR-1 tumor antigen, leading to their direct killing by the ADCC mechanism. The PB004 molecule is being developed for the treatment of solid tumors, including triple negative breast cancer, as well as selected hematological neoplasms, including chronic lymphocytic leukemia.