Pure Biologics has received approval from the U.S. Food and Drug Administration (FDA) to conduct a Phase 0 clinical trial in Project PB004 for the PBA-0405 molecule.
- The study is expected to provide preliminary information on the effectiveness of PBA-0405, including the activation of immune cells and the destruction of cancer cells. The study will involve the direct injection of microdoses of the PBA-0405 molecule into patients’ tumors.
- The study will be multicenter and open. It will be conducted in eight to twelve hospitals and centers in the US, involving 12 to 15 patients with solid tumors, including head and neck cancer, soft tissue sarcoma, and triple negative breast cancer.
- The Company anticipates that the study will span approximately six months and will be finalized by mid-2024. It will be conducted in an open-label format, signifying that the Company will systematically gather information on the study’s progress and outcomes on an ongoing basis.
- The company’s strategic priority remains the signing of a partnership agreement, and the results of Phase 0 could influence the decisions of pharmaceutical companies.
Dr. Pieter Spee, Vice President and Chief Scientific Officer of Pure Biologics:
This is a very important milestone for the company and a tribute to the hard work of the Pure Biologics team in the PB004 project. We see tremendous potential for the PBA-0405 molecule, which combines the unique expression pattern of ROR-1 limiting tumor growth with the validated mode of action of proven anti-cancer therapies such as Rituxan, and which we believe is much more suitable for the treatment of, for example, chronic lymphocytic leukemia (B-CLL) than the ROR1-targeted therapies currently being tested in the clinic. Phase 0 will not only provide us with information on the efficacy profile of our molecule, but will also allow us to more effectively plan its further development in the clinic.
Dr. Filip Jelen, co-founder, major shareholder and CEO of Pure Biologics:
The FDA’s approval to proceed with Phase 0 is a very important formal element of our plan. The regulator’s decision mitigates our risks and brings us closer to unlocking the value of the PB004 project generated over the years. Discussions with potential partners in recent quarters indicate that the results of Phase 0 may be crucial to their decision-making processes.
The company plans to sign a partnering agreement in a co-development model, in which the costs of further development of PB004 will be transferred to the partner. In such a model, it will be possible to use the already-acquired, non-dilutive Medical Research Agency grant worth PLN 32 million in the Phase 1 clinical trial, which is an important argument in partnering talks.
Project PB004 is a potential biologic drug. It involves the development of a novel therapeutic molecule in the format of an affucosylated IgG1 class antibody to stimulate the immune response of NK cells against tumor cells bearing the tumor antigen ROR-1, leading to their direct killing by an antibody-dependent cellular cytotoxicity (ADCC) mechanism. The PB004 molecule is being developed for the treatment of solid tumors, including triple-negative breast cancer, as well as for the treatment of selected hematological malignancies, including chronic lymphocytic leukemia, among others.