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Report

Teaser, summary, work performed and final results

Periodic Reporting for period 1 - O-POINTED (Osteoarthritis patient cohorts in the dish - personalised cellular drug development tools for osteoarthritis precision medicine)

Teaser

Summary

Pharma companies, biotechs and research institutes invest billions of Euros developing drugs for the growing osteoarthritis patient population. Still, they have failed to bring a drug to the market that would slow disease progression. This is because osteoarthritis is a heterogeneous disease and currently used drug development tools completely fail to represent patient variability. We offer O-POINTED, a tool thatâ€”for the first timeâ€”enables personalised osteoarthritis drug development before expensive clinical trials are started. O-POINTED are cellular test systems build from libraries of expandable osteoarthritis cell lines that mimic the three main osteoarthritis disease phenotypes (bone-driven, cartilage-driven and synovioum/inflammation-driven). The feasibility assessment revolved around the validation of economic and financial, commercial and market, and technical feasibility. The main conclusions of this project are the confirmation of the overall business idea, which was updated to broaden the disease focus beyond osteoarthritis. This evolution was influenced by technical details but mainly by an updated market analysis and feedback from potential customers.

Work performed

The economic and financial viability assessment revolved around the analysis of precision medicine initiatives and their influence on the market for O-POINTED, as well as the validation of the business model. The business model validation was positive and our combined business models received positive feedback from potential customers. Our analysis of precision medicine initiatives showed that a widened disease focus for O-POINTED would better align with the scopes of the initiatives. The analysis of the commercial and market feasibility allowed us to identify key decision-making processes in pharma companies, one of our main target customers. Discussions with a current user of a MVP and a potential user, gave us valuable feedback towards the product design and the market size. This feedback also convinced us to update our business idea to include more diseases in our patient cohorts in the dish. The main two parts of the technical feasibility assessment were the FTO analysis and the expansion of our supplier network for the O-POINTED source material. We were able to confirm our FTO and also obtain a preliminary quote for the O-POINTED source material.

Final results

The original mission of the project was to address the lack of cell-based tool for personalised osteoarthritis drug development and patient stratification. We translated feedback from potential customers and also academic collaborators into requirements that guided the evolution of our product idea. The idea matured to include 4 other priority diseases as well. This significantly enlarges our potential customer base while keeping our development workload similar to the original idea. This also means we will make personalised drug development tools available that have the potential to benefit millions of more patients than anticipated at the start of the project.