multiQure SA

Pure Biologics today announced the successful completion of its Phase 0 clinical study for the novel cancer therapy, PBA-0405. Conducted by Presage Biosciences (Seattle, USA), the multi-center study demonstrated PBA-0405’s potential to activate tumor-infiltrating immune cells and induce cancer cell killing in real cancer patient settings. The open-label study enrolled six patients with head and neck carcinoma and soft tissue sarcoma, administering intra-tumoral microdoses of PBA-0405, dosed in up to six different sites in the tumor, prior to scheduled surgery. Biomarkers indicating drug activity were analyzed in 84 different tumor sites from five evaluable patients, with four patients showing clear signs of immune-cell activation and cancer cell killing. No significant safety concerns were observed. These results underscore the therapeutic promise of PBA-0405. Pieter Spee, Chief Scientific Officer of Pure Biologics, commented: The high failure rate of drug candidates in clinical development often stems from inadequate efficacy and safety profiles. By demonstrating PBA-0405’s intended therapeutic mode of action in actual cancer patients early in development, we have significantly de-risked this program, which can now move forward into further clinical development. We are thrilled by these results and eager to move forward with our oncology pipeline. The high failure rate of drug candidates in clinical development often stems from inadequate efficacy and safety profiles. By demonstrating PBA-0405’s intended therapeutic mode of action in actual cancer patients early in development, we have significantly de-risked this program, which can now move forward into further clinical development. We are thrilled by these results and eager to move forward with our oncology pipeline. Jason Frazier, VP of Technology at Presage Biosciences, said: The success of the PBA-0405 phase 0 CIVO study is highly encouraging and follows on previous successful studies conducted with other leading oncology companies. We are excited to collaborate further on Pure Biologics’ next phase 0 study for PBA-0111, set to begin patient enrolment next month. The success of the PBA-0405 phase 0 CIVO study is highly encouraging and follows on previous successful studies conducted with other leading oncology companies. We are excited to collaborate further on Pure Biologics’ next phase 0 study for PBA-0111, set to begin patient enrolment next month. The next step for Pure Biologics involves launching a phase 0 study for PBA-0111, which has received FDA approval and is poised to commence shortly. These developments reinforce Pure Biologics’ strategic focus on advancing breakthrough therapies that address unmet medical needs in oncology.

Pure Biologics has entered into an agreement to sell global rights to molecules PBA-0405 (project PB004) and PBA-0111 (project PB003G). The other party to the transaction is Promittens Corporation, an American company founded by experienced executives with many years of experience in the capital markets and pharmaceutical and biotechnology industries. Pure Biologics will be entitled to an upfront fee (“upfront”) in the total amount of $8 million to $9 million, depending on the results of the Phase 0 clinical trials conducted in the aforementioned projects. The agreement also provides for potential milestone payments (“milestones”) totaling up to $299 million and royalties (“royalties”). Pure Biologics is preparing to unveil the Company’s development plans for the coming years in the first quarter of 2025. Dr. Filip Jelen, co-founder, major shareholder and CEO of Pure Biologics: The signed agreement represents a very important step in Pure Biologics’ development. Its conclusion signifies the implementation of a key element of the strategy adopted years ago, involving the development of potentially best-in-class drug candidates, from the discovery stage to clinical trials. Proceeds from the sale will allow us to stabilize the Company’s financial position and focus on new projects. We intend to present plans for the Company’s further development later in the first quarter of 2025. Devrim Aran, co-founder and CEO of Promittens Corporation: Our years of experience in the industry, combined with access to the global capital markets, will allow us to advance the antibodies developed by Pure Biologics. They have the potential to become breakthrough drugs for the treatment of various solid tumors and hematological malignancies. PBA-0405 (Project PB004) is a therapeutic antibody of the IgG1 class, which is expected 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. PBA-0405 was developed by Pure Biologics to improve the treatment of B-cell malignancies and solid tumors. The antibody was designed to optimally activate the immune system to eliminate cancer cells, with minimal risk of side effects. In studies to date, it has shown significantly better performance than other antibodies targeting ROR1. PBA-0405 has shown very promising results in the first clinical trials involving patients with head and neck cancers and soft tissue sarcomas, which is currently being conducted in the US. PBA-0111 (PB003G) is a dual-action therapeutic antibody that simultaneously eliminates immunosuppressive regulatory T (Treg) cells in the tumor microenvironment and stimulates cytotoxic NK and T lymphocytes to directly target tumor cells. It targets GARP, a protein expressed on tumor cells, and immunosuppressive regulatory T cells, which inhibit the immune system’s ability to eliminate tumor cells. PBA-0111 was developed by Pure Biologics and has the potential to become a best-in-class drug for the treatment of solid tumors. PBA-0111 is designed to act by a dual mechanism: to overcome immunosuppression by blocking and eliminating regulatory T cells, and to enable the immune system to target and destroy tumor cells targeted by PBA-0111. The FDA has approved a Phase 0 clinical trial protocol for PBA-0111 in patients with head and neck cancers and soft tissue sarcomas. The U.S. trial is scheduled to begin in early 2025.

SEATTLE, July 25, 2024 /PRNewswire/ -- Presage Biosciences, a biotechnology company that directly measures activity of oncology drugs within human tumors, announced the Phase 0 dosing of the first cancer patient with Pure Biologics' PBA-0405, an antibody that targets ROR1. PBA-0405 has been engineered to induce tumor cell killing by cytotoxic immune cells. This exploratory study is designed to study the biological effects of PBA-0405 within the human tumor microenvironment. In January, Presage and Pure Biologics announced the U.S. Food and Drug Administration issued a Study May Proceed notification for Phase 0 testing of a pre-GMP first-in-human drug candidate with Presage's Comparative In Vivo Oncology (CIVO®) platform. CIVO enables microdosing directly in a human tumor that is planned for surgical removal. Extensive molecular profiling technologies determine drug effectiveness in the tumor microenvironment by analysis of the expression of thousands of genes. "With the dosing of the first patient in our exploratory Phase 0 study, PBA-0405 is the first ROR1-targeting compound with a unique mode of action to enter studies in patients. We're encouraged by the preclinical data we've seen so far and are eager to evaluate its pharmacodynamic activity in human studies", said Dr. John Weinberg, Chief Medical Officer of Pure Biologics. ROR1 is a tumor-associated antigen expressed on many solid tumors and B cell malignancies. "We are very excited to partner with Pure Biologics to evaluate their pre-GMP oncology drug in a first-in-human Phase 0 CIVO study" said Dr. Patrick Gray, Presage CEO. "Testing of pre-GMP compounds in human tumors in situ simplifies and speeds up the drug development process. Our Phase 0 studies can determine tumor killing activity, synergism with approved or investigational drugs, and immuno-oncology activity." About CIVO Comparative In Vivo Oncology (CIVO) is Presage's patented platform that enables multiplexed intratumoral microdosing and generation of detailed tumor profiling. The CIVO device can deliver up to eight different drugs or drug combinations simultaneously into trackable drug columns. Presage's CIVO technology and analysis capabilities are unparalleled at providing insight into drug-exposed areas of the intact tumor microenvironment. Presage is pairing the use of CIVO with immunohistochemistry, in situ hybridization, and molecular profiling technologies in both preclinical and Phase 0 trials to inform and de-risk oncology drug development. About Presage Presage Biosciences is a translational oncology company dedicated to understanding the complexity of drug response in the tumor microenvironment. Presage partners with oncology-focused pharmaceutical companies through strategic alliances along with other innovators who are at the forefront of spatial biology and oncology drug development. Presage is privately held and based in Seattle. For more information, visit . For more information: [email protected] SOURCE Presage Biosciences Inc