REPO4EU News - Jan 2023

NEWSLETTER NO. 1 –JANUARY 2023 Dear colleague, Welcome to REPO4EU’s very first newsletter and thank you for your interest in our work! Below, you will find REPO4EU’s core principles and milestones we have focused on these past 5 months since the project started. Looking forward to a spectacularly successful 2023 letting our paths cross! Harald Schmidt COO REPO4EU Maastricht University About REPO4EU Why drug repurposing? Finding a new application for a drug that is already on the market reduces the time and costs of drug development. It allows us to prioritize patient benefit and safety, as most side effects are already known. Why mechanism-based? Currently, most diseases are defined by symptoms in specific organs. There is no understanding of its molecular causes. This makes current approaches to drug development (and drug repurposing) risky and imprecise. Thus, our platform combines all legal and business requirements to repurpose a drug with world-class research to completely redefine diseases by their molecular causes, implementing precision diagnostics for patient selection and selecting the optimal drugs to treat the underlying cause. REPO4EU is the Euro-Global Platform for Mechanism-Based Drug Repurposing,an ambitious initiative that we believe will usher in a profound change to medicine and patient benefit. Making connections at the start of our journey Our coordination team organised a very successful project kick-off in Maastricht, together with the RExPO22 conference.We also met the RepoOG operating group of European national regulators on drug repurposing. The road so far… Setting the stage for the future of drug repurposing As part of the project’s foundational work, we have started collecting tools,data sources and standards relevant for drug repurposing for later prioritisation and open access. Similarly, and in order to streamline preclinical validation efforts, we have prepared an overview of accessible data,contacts and ethical requirements to get access to samples stored at different European biobanks. This is essential to train our diagnostics platform technology. In parallel, an internal baseline technology readiness assessment has already been initiated to start the build-up of RepoScope, a unique database making repurposing-relevant patents and peer-reviewed papers from 2010 onwards available in a useful linked form. Real-world data-driven Artificial Intelligence Our AI experts have developed a plan for a generative statistical model that can predict mechanistic disease subtype labels. The model can be trained in a semi-supervised way, using both labelled and unlabelled data. This technology will be applied in our upcoming REPO-HYPER clinical trial for resistant hypertension. Unlocking the full potential of drug repurposing clinical trials REPO4EU has produced guidelines for conducting an extended freedom to operate (FtO) analysis and analysed all intended clinical trials. REPO4EU mainly enables academics (the untapped resource) to perform and consider all relevant steps in drug repurposing up to conducting a clinical phase II (proof of concept) trial. One of these trials (REPO-HYPER) will be conducted at the Karolinska Institutet, Stockholm. An inquiry has already been prepared to the Swedish component authority for this purpose. To guarantee an ethics-bydesign process, two surveys have been developed to map ethical and data protection issues. The pilot will be applied to REPO-HYPER, including the data protection challenges in Machine Learningassisted patient screening. Our experts have started brainstorming on a white paper on Health Technology Assessment (HTA) in drug repurposing. Feedback is now being collected to ensure a broader view from all stakeholders is adequately integrated within REPO4EU. Ethics in the spotlight A new outlook on HTA Calling all stakeholders - REPO4EU is an open platform! REPO4EU invites all key actors in drug repurposing to be part of our platform to ensure its inclusiveness and completeness. We are currently developing an open publishing portal, archiving and journal structure for the entire community - our collection on Drug Repurposing Research publications on ScienceOpen is already live! © REPO4EU 2023 This year, we will also organize several national events to get in direct contact with local stakeholders. The first one is already underway: it will take place in sunny Madrid this March. And that’s not all - many more exciting opportunities are on the horizon! National events in Munich and Vienna are in preparation. The upcoming RExPO23 conference will be hosted in Stockholm…and a US edition of our RExPO series is coming in 2024. Stay tuned - 2023 has just begun!