Periodic Reporting for period 1 - MABIOS (The first microalgae platform for the production of anticancer biopharmaceuticals) Reporting period: 2019-08-01 to 2020-01-31 Summary of the context and overall objectives of the project Manufacturing costs remain one of the major obstacles to providing effective treatment of patients with oncologic disorders. Anticancer agents are often complex biopharmaceuticals that are expensive to manufacture, requiring use of living animal/plant/microorganism cell lines with difficult-to-control production processes, limited yields and time-consuming purification of the active biomolecules. There is an urgent unmet need for an improved manufacturing process of (anticancer) biopharmaceuticals, to meet the high demand and lower the price of oncologic treatments.We will revolutionize the way biopharmaceuticals are produced by developing a microalgae based biorefinery platform. These biorefineries will utilize genetically engineered microalgae to efficiently produce pharmaceutical agents, such as therapeutic peptides, recombinant proteins or small molecules. The objectives of this feasibility study are to;(1) create a clear overview of the market opportunity of micro-algae based production of paclitaxel, (2) develop a de-risked plan for the technical development of our platform, (3) evaluate the necessary regulatory roadmap to enter the market Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far In this project we have;1. identified our position in the market by analysing our competitors and new players in the market, and current patents by, together with a leading RTO (Research and Technology Organization);SINTEF, reading company reports, analysing press releases and articles and searching through proprietary databases.2. acquired consultation from an expert team of SINTEF and together developed an optimal proof of concept project plan as a development strategy. This maximizes the chance of achieving success in development. In this plan we have considered the techno-economic aspect to assure a commercially feasible result. This setup also matched with the requirements from regulatory experts regarding regulatory approval.3. investigated the necessary regulatory roadmap and build a schedule for when to begin interactions with the regulatory authorities. As the CEO Roy Abraham has over a decade of experience in pharmaceutical process development, and aware of which regulatory bodies to contact in order to develop the correct roadmap to market entry.Results achieved so far: 1. Findings from our market study are very promising as it showed that our technology is not yet on the market for producing paclitaxel (not patented either). There is a shift towards plant cell fermentation (10-20% of the market), but this technique provides low quantity with high associated production costs.2. Our technical analysis concluded the following;(1) After re-evaluating the ideal production platform (plant cell, bacteria, fungi, algae), we concluded to stick to our initial plan by using microalgae as production platform, potentially being the most stable and cost-efficient method.(2) A techno-economic analysis concluded a specific concentration requirement out of kg dry algae mass, which would make the production profitable (confidential)(3) For next technical development steps 0.4 – 0.6 M is needed (2-year framework)3. To set up the quality management system the ICH Q7 document will be followed as well as appropriate Good Manufacturing practices at all stages of the API supply chain, including distribution. Following regulatory procedures to enter the market, we have decided to use the CEP procedure for registration in Europe and we will submit a DMF file to register in the U.S.A. Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far) Current biomanufacturing platforms for producing biopharmaceuticals include mammalian cell lines, plant cells, insect cell lines, yeast, and bacteria, and now microalgae are entering the field. Each of these production platforms have specific strengths and limitations. The production of recombinant proteins using mammalian cells is already a multibillion-dollar business. Biomanufacturing with microalgae is an innovative platform in pharmaceutical industry with great potential that goes beyond the state-of-the-art. Microalgae are eukaryotic cells with fast growth rates, and a large cell mass can be obtained, therefore the potential yield is high.Microalgae have PTM (Post-translational modification) machinery, enabling production of complex proteins such as monoclonal antibodies (mAb), currently produced by mammalian cells (low yields and complex purification steps). Active agents are easily purified from the biomass, reducing production complexity and costs. Microalgae platforms are scalable and flexible enabling to up/downscale the process based on demand. The state of the art solutions within this sector have not changed since the application of the SME instruments report MAW company logo
