SBIR Phase I: Novel, Accurate and Reproducible Platform for the Developability Assessment of Protein Therapeutics

Broader impact/commercial potential of this Small Business Innovation Research (SBIR) Phase I project is to (1) improve the decision making towards protein therapeutics development (also known as developability), (2) reduce Research and Development costs for the Biotechnology industry in general, (3) improve timelines of new protein therapeutic candidates, thus proceeding to clinical phase trials sooner, (4) resulting in fewer candidate withdrawals from clinical trials, (5) reduce risk associated with protein aggregation and immunogenicity, a potentially fatal outcome; leading to (6) overall quicker times for approval-to-market, and finally (7) a reduction of product recall risk. The market opportunity for protein characterization and identification market by instruments also known as life sciences tools is estimated to be $ 80-85 billion dollars. Our product is an innovative patented technology platform and related services which will support the developability evaluation of new protein therapeutic candidates in the biologics and biosimilars product portfolio. As an additional funding source, we have adopted the production of a high quality aggregate free potential diagnostic candidate biomarker for prostrate and pancreatic cancer to allow for further research and development in other research institutions. Completion of Phase I in the evaluation of formulation conditions of protein therapeutics including monoclonal antibodies (mAbs) will ensure progress towards the development of a high throughput platform for Phase II evaluation. The proposed project will address the current bottleneck of new protein therapeutics within the biologics and biosimilars industry due to protein aggregation. This is the single most prevalent reason that has hampered the release of biotherapeutics into the market. Protein aggregation leads to loss of efficacy and potentially to immunogenicity risks. Formulation is critical to downstream processing, dosing, storage, and delivery of protein therapeutics. Our goal is to test different formulation conditions through the use of Design of Experiment strategies to identify the formulation conditions under which the monoclonal antibody candidate is stable and aggregation free, even under stress. We have designed an innovative patented platform using two dimensional infrared (2D IR) correlation spectroscopy and perturbation correlation (PC) analysis which is accurate, reproducible and does not use probes to determine the mechanism and extent of aggregation, and stability of a mAb. A potential outcome will be the developability assessment of novel candidates ensuring a pipeline of protein therapeutics early in the research and development. If successful, Phase II will involve a high-throughput platform to address the evaluation of protein aggregation in plasma and final IV delivery conditions, for the design of a predictive model for immunogenicity risk assessment.