Periodic Reporting for period 1 - BloodStemPLUS (Commercial feasibility study of a technology that multiplies blood stem cells to increase the success of transplants for cancer treatment) Reporting period: 2016-06-01 to 2016-09-30 Summary of the context and overall objectives of the project GrowingFormula Lda (trading as StemCell2MAX) has investigated the marketing and commercial feasibility of the clinical application of their proprietary mix of neurotrophic factors. The value proposition of StemCell2MAX's technology relies on the ability to expand hematopoietic stem cells (HSCs) up to 20 times over the current state-of-the-art while maintaining unique stem cell properties, as published in Nature (Fonseca-Pereira et al, Nature 2014). Despite the promise of HSC transplantation for a variety of malignant and non-malignant diseases, its full potential cannot yet be fully exploited due to the limited availability from existing sources (bone marrow, umbilical cord blood, pe-ripheral blood). This drives the global need for reliable techniques to multiply hematopoietic stem cells.StemCell2MAXTM Mix, the technology developed by StemCell2MAX, is currently commercially available for Research Use Only with an international client base. The main goal of this feasibility study was to investigate the commercial feasibility of StemCell2MAXTM Med, focusing on the potential clinical applications of the technology. The project was designed to generate a strategy to address challenges for bringing a safe, reliable, regulatory-compliant and cost-effective product to the market. It includes an assessment of the market and most relevant target areas, an inventory of stakeholders and potential partners for clinical validation, an analysis of Intellectual Property, the design of clinical development strategy, identification of reimbursement issues and the development of a funding strategy. Finally, the results of this feasibility study were consolidated in a sound business plan. Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far This project benefited from work executed in 6 tasks, of which the results were presented in a feasibility report (project deliverable) and consolidated in a sound business plan.Task 1: Market study and risk assessment. The potential of StemCell2MAXTM Med was established by determining the market and most relevant target areas. We have defined unmet needs, developed marketing and pricing strategies, analysed reimbursement policies and generated sales forecasts. We have identified competitive development and trends, opportunities and threats in the market.Task 2: IP management. To maintain the strong position for StemCell2MAXTM Med and protect the extensive know-how present within StemCell2MAX, we have performed a freedom-to-operate analysis.Task 3: Regulatory landscaping and clinical trial design. For this task we have consulted an experienced regulatory expert to identify regulatory demands. The regulatory guidelines for StemCell2MAXTM Med implementation have been described and incorporated in an extensive regulatory strategy, including clinical grade manufacturing and quality requirements. Additionally, the clinical trial design has been explored. Task 4: Financing and deals. In this task we have developed a funding strategy for the development of StemCell2MAXTM Med. Multiple financial scenarios have been taken into account, including the overall investment need for validation. We have identified possible partnerships and other funding opportunities.Task 5: Partner search. This task was dedicated to making an inventory of interested partners for clinical validation, with a main focus on Europe (but also including the USA and Asia). The partners have been investigated for their willingness to support the implementation of StemCell2MAXTM Med, their power to accelerate the implementation of StemCell2MAXTM Med and the means necessary to convince them.Task 6: Business plan consolidation. This task has consolidated the input from tasks 1-5 in a sound business plan focused on the clinical application of StemCell2MAXTM Med. It includes information on goals and ambitions for this application, business and IP strategy, business model, market size and dynamics, logistical analyses, technical and operational planning, SWOT analysis, investment plan and financial forecasts. Based on the results of this feasibility study, we have concluded that the development of the Good Manufacturing Practices (GMP) version of StemCell2MAXTM Mix will enable its use as a manufacturing platform for different clinical applications. Following GMP development, a comprehensive analysis of the different potential clinical markets clearly indicated that the company should focus primarily on the development of StemCell2MAXTM Med for the field of HSC transplantation. The remaining markets should be tackled by licensing deals, either directly with other companies developing their own products or with manufacturing service providers that would use StemCell2MAX’s GMP platform for HSC-based advanced medicinal products. 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) The current feasibility report has prepared StemCell2MAX to undertake the next development steps towards clinical application of their blood stem cell multiplication technology. The project will be further pursued by applying for followup funding in the SME Instrument phase 2 for establishing a GMP manufacturing platform. The socio-economic impact of this platform is enormous, as StemCell2MAX's clinical platform will be further developed as an advanced therapy medicinal product, to improve haematopoietic stem cell transplantation with blood stem cells derived from umbilical cord blood. This product can directly serve the unmet need market (approximately 21.600 patients in the EU/US that are currently not treated) and serve to improve the over 50.000 HSC transplantations that are performed yearly in the EU/US. In addition, the platform will support the clinical development of new HSC-based therapies. The remaining markets could be tackled by licensing deals, either directly with other companies developing their own products or with manufacturing service providers that would use StemCell2MAX’s GMP platform for HSC-based advanced medicinal products. Company logo
