Registration and Welcome Coffee

Plenary One – Innovative changes to drive patient impact

Change is essential to accelerate repurposing success and deliver more medicines to patients. Here, we highlight the need for change, and the impact that innovative thinking from patient groups, funders, and regulators is already having in the drug repurposing space.

Welcome	EATRIS Don Lo EATRIS Anton Ussi Beacon: for rare diseases Mary Rose Roberts Anticancer Fund Gauthier Bouche
Welcome to the EU	Member of the European Parliament Tomislav Sokol
The Power of Living Fully: My Journey with PNH (Paroxysmal Nocturnal Hemoglobinuria)	Suzanne Morris-Wade
Repurposing and reformulation to treat vascular anomalies?	Université catholique de Louvain (UCLouvain) Miikka Vikkula
Not all that glitters is gold: reclaiming & retooling innovation for patient benefit	æqua Els Torreele
Wrap up	Anticancer Fund Gauthier Bouche

Morning Coffee Break

Plenary Two – Creating repurposing ecosystems

While isolated repurposing projects can drive small scale success, synergies are vital to exchange knowledge, scale impact, and generate systemic change. Here, we explore different repurposing ecosystems, both nationaland condition-focussed, and their key drivers for success.

Introduction	EATRIS Don Lo
Strategies to facilitate drug repurposing in Spain	Spanish Ministry of Health César Hernández
Stimulating medicines repurposing in the EU EMA/AEMPS, building on the STAMP initiative	Spanish Agency of Medicines and Medical Devices (AEMPS) Yoana Nuevo-Ordóñez
	Spanish Ministry of Health César Hernández Spanish Agency of Medicines and Medical Devices (AEMPS) Yoana Nuevo-Ordóñez Foundation 29 Julián Isla Spanish Association Against Cancer (AECC) Marta Puyol EATRIS Don Lo
Read more Panel Session: Creating a complete ecosystem to bring more and effective treatments to patients Spanish Ministry of Health César Hernández Spanish Agency of Medicines and Medical Devices (AEMPS) Yoana Nuevo-Ordóñez Foundation 29 Julián Isla Spanish Association Against Cancer (AECC) Marta Puyol EATRIS Don Lo
REMEDi4ALL Funders Network: Connecting funders to maximise the potential of drug repurposing research	ZonMw Heleen van der Meer
Wrap up	Beacon: for rare diseases Mary Rose Roberts

Lunch Break

RARE I - When is repurposing right for rare diseases?

Repurposing is not right for every rare disease. This session will explore why and when repurposing is chosen relative to other therapeutic approaches, circumstances that make it a viable strategy, and how decisions are made in practice. Speakers will set the scene for how repurposing fits into the broader rare disease landscape, highlighting key considerations for deciding which diseases and patient groups are most suited.

Introduction	Beacon: for rare diseases Rick Thompson
To bring new purpose...	Illumina Inc. Shirlene Badger
Learning on the job - insights on repurposing collaborations from the CATS Foundation	Cure & Action for Tay-Sachs (CATS) Foundation Dan Lewi
From Concept to Clinic: Early Lessons from Drug Repurposing Trials in Malaysia and the ASEAN Region	City St George’s, University of London | International Affordable Diagnostics and Therapeutics Alliance (IA-DATA) Nafeesa Mat Ali
Repurposing afatinib to treat head and neck carcinomas in the cancer predisposition syndrome Fanconi anemia	Sant Pau Research Institute | Autonomous University of Barcelona| Biomedical Research Networking Center (CIBER) Jordi Surrallés
Wrap up	Beacon: for rare diseases Rick Thompson

DISCOVERY I - Artificial Intelligence in drug repurposing

With a wealth of new AI companies and platforms appearing every year, the potential of AI to uncover new therapeutic opportunities has never been greater. But what is the best approach to deploy this technology and ensure that this vast computational power can be directed to meet some of the most pressing unmet medical needs worldwide? This session will explore different approaches to AI in the repurposing space and assess their potential for patient impact.

Introduction	University of Helsinki Tero Aittokallio
NetMedGPT: A network medicine foundation model for disease mechanism mining and drug repurposing	Institute for Computational Systems Biology (CoSy.Bio), University of Hamburg Farzaneh Firoozbakht
From Hypothesis to Patient Outcome: An AI-Driven End-to-End Drug Repurposing Platform	Every Cure Robyn Macrae
Mendelian randomization improves machine learning prediction of clinical success in drug development	biotx.ai Roman Fudim
Virtual models, clinical impact. Advancing drug repurposing through in silico new approach methodologies	BioLizard Alexander Koch
Wrap up	University of Helsinki Tero Aittokallio

Afternoon Coffee Break

RARE II - New approaches to drive repurposing research in rare diseases

Novel strategies are needed to accelerate repurposing research in rare diseases. This session will showcase emerging approaches from industry, regulatory, clinical, and patient groups to drive forward repurposing research. Our speakers will explore how collaboration and innovation can make repurposing more effective and patient-relevant, and ensure repurposing research can break through translational barriers and deliver patient access.

Introduction	Beacon: for rare diseases Megan Burley
Drug repositioning in overgrowth syndromes and vascular malformations	Hôpital Necker–Enfants Malades, Université Paris Cité Guillaume Canaud
Building a platform trial for NF1-driven tumors: how patient organisations can drive the infrastructure	Children’s Tumor Foundation Europe Dariusz Adamczewski
From Advocate to Architect: How a Patient Organisation Built an Ecosystem for Rare Disease Drug Repurposing	Citrin Foundation Barbara Yu
Capturing all outcomes that matter thanks to the Net Treatment Benefit	IDDI (International Drug Development Institute), CluePoints and One2Treat Marc Buyse
Wrap up	Beacon: for rare diseases Megan Burley

DISCOVERY II - Building a discovery pipeline – combining methodologies to find the best repurposing routes

While AI has huge potential, it relies on processing good data, based on a diverse range of scientific methods. Furthermore, hypotheses must be tested and validated to ensure that only ideas with a real chance of patient impact move towards the clinic. This session will explore how lab-based science is driving forward repurposing opportunities.

Introduction	EATRIS ERIC Martin de Kort
A Translational Path to Clinical Readiness: Repurposing Strategy for DDX3X Syndrome	Fraunhofer Institute for Translational Medicine and Pharmacology Maria Kuzikov
Not all drugs are created equal	Institute for Molecular Medicine Finland (FIMM) Kristen Nader
TerbinaTwo - The Second Life of an Antifungal	pua venture resources Lukas Kadawy
CURE ID: a drug repurposing treatment registry for hypothesis generation	U.S. FDA Heather Stone
Wrap up	EATRIS ERIC Martin de Kort

Poster Session and Networking

Registration and Welcome Coffee

COMMUNITY I - The community in action

Here we explore the example of the national repurposing network being developed in the Netherlands, before highlighting the work of REMEDi4ALL’s own ecosystem through our delegate-proposed lightning talk session.

Panel Session - The Dutch repurposing landscape: collaborations, support, and how to create a national repurposing network	ZonMw Dunja Huijbers ZonMw Heleen van der Meer RARE-NL Sibren van den Berg Centre for Future Affordable and Sustainable Therapy development (FAST) Saco de Visser Amsterdam University Medical Center and University of Amsterdam Remco Molenaar
Introduction to Lightning Talks	Beacon for rare diseases Hannah Harvey
Lightning Talk: 3 Ways to Maximize Impact with Limited Resources	Cure OTCD Kate Vinokurov
Lightning Talk: Systematic Drug Repurposing Identifies Translational Candidates for Multiple Sulfatase Deficiency	Fraunhofer Institute for Translational Medicine and Pharmacology (ITMP-TNM) Alisa DeGrave
Lightning Talk: An iDR25 Success Story! A Global Registry project for the world-wide EB community	Dendrite Clinical Systems Ltd Martin Twycross
Lightning Talk: Patient-led drug repurposing in PACS2 syndrome: combining cell imaging with proteomics	PACS2 Research Foundation Dominik Cysewski
Lightning Talk: Developing the First-Ever Dedicated Therapy for Cri du Chat / 5p minus Syndrome	Sherwood Pharmaceuticals Yannick Lampo
Lightning Talk: Repurposing shrouded as new drug developments	Ayna Baladi Nejad
Wrap up	ZonMw Heleen van der Meer

INDUSTRY I - Building a repurposing business model to attract investment

Finding an effective business model for a repurposed asset is a persistently cited barrier – but what options are available to a company in this space, and what can be done to increase the appeal of commercial repurposing?

Introduction	EATRIS Don Lo
From shelf to patient: how patient-led research organisations unlock innovation - lessons from the Gomekli story	Children’s Tumor Foundation Europe Dariusz Adamczewski
Barriers and opportunities to unlocking shelved assets	Duke-Margolis Institute for Health Policy Beth Boyer
Precision Medicine Meets Reality: Data Access and Translational Barriers in KCNQ2 Therapy Development	VIB-Uantwerp Sarah Weckhuysen KCNQ2 e.V. Aila Coulmann
Panel session - How can repositioning of clinical-stage drug assets be made more systematic and more impactful?	Children’s Tumor Foundation Europe Dariusz Adamczewski Ignota Labs Kelly Gray EATRIS Don Lo VIB-Uantwerp Sarah Weckhuysen EFPIA Nick Sykes
Wrap up	Institute for the Study of the Human (iHuman) - University of Sheffield Paul Martin University College Dublin Tineke Kleinhout-Vliek

Morning Coffee Break

COMMUNITY II - Improving skills and creating collaborations

Partnerships which bring together diverse skills and perspectives are vital to deliver a repurposed drug from concept to market. In this session we will highlight public private-partnerships as a model in the repurposing ecosystem, before exploring the opportunities, successes and challenges of upskilling people to deliver more successful repurposing studies.

Introduction	Beacon: for rare diseases Rick Thompson
Driving innovation: the example of the Innovative Health Initiative	Innovative Health Initiative (IHI) Nathalie Seigneuret
Open Science & Best Practices on the Drug Repurposing Central Publication Portal	ScienceOpen Nina Tscheke
Panel Discussion: Growing the size and skill base of the repurposing community	University of Birmingham Felix Chan U.S. FDA Heather Stone ZonMw Dunja Huijbers Beacon: for rare diseases Rick Thompson CHAMP1 UK Scott Hutchings UCL Carmel Reilly

INDUSTRY II - Unlocking value: the pathway to commercializing off-patent repurposed medicines

Despite the perception, there are many successful cases of repurposing in the commercial space. Here we will examine both success stories and initiatives designed to make repurposing more attractive and effective for the pharmaceutical industry.

Introduction	Medicines for Europe Raluca Radu
REMEDi4ALL's research into perceived barriers to off-patent drug repurposing	Syreon Research Institute Dalma Hosszu
Gloricard: Drug repurposing in practice?	Tiofarma B.V. Ivo van der Bijl
Derisking Clinical Repurposing Trials for Later-Stage Investment and Commercialization	Cures Within Reach Barbara Goodman
Key questions to help repurposing practice	Medac Thomas Grub EATRIS Don Lo Cures Within Reach Barbara Goodman Tiofarma B.V. Ivo van der Bijl
REMEDi4ALL’s research into selected push and pull mechanisms	Syreon Research Institute Dalma Hosszu
Panel discussion	Medicines for Europe Raluca Radu EATRIS Don Lo Tiofarma B.V. Ivo van der Bijl Cures Within Reach Barbara Goodman Syreon Research Institute Dalma Hosszu

Lunch Break

Patient Engagement Panel – Having a drug isn't enough

Even when repurposed treatments exist, patients often face barriers that prevent access or meaningful benefit. This panel, led by patient advocates and representatives, will showcase how patient communities are actively addressing these inequalities, highlighting their influence in shaping research priorities, driving access initiatives, and ensuring treatments meet real patient needs. It will demonstrate why patient insight and leadership are essential to turning scientific advances into tangible improvements for rare disease communities.

Introduction	Beacon: for rare diseases Rick Thompson
	Malaysian Rare Disorders Society Nadiah Hanim Abdul Latif OIFE (Osteogenesis Imperfecta Federation Europe) Alessandra Tolaccia Hypersomnolence UK Matthew Baker Citrin Foundation Barbara Yu Beacon: for rare diseases Rick Thompson

Afternoon Coffee Break

Closing Plenary – The future of drug repurposing

With legislative changes on the horizon affecting Europe’s approach to repurposing regulation, combined with the growing interest in repurposing science, we can be sure that the repurposing of tomorrow will differ from today. Here we explore the future of repurposing, the projects that bring us hope in the near future, and the challenges that still need to be addressed.

Introduction	Beacon: for rare diseases Rick Thompson
Winning Poster presentation	Beacon: for rare diseases Megan Burley
	DG SANTE, European Commission Olga Solomon Anticancer Fund Patricia Vandamme Anticancer Fund Lydie Meheus Medicines for Europe Adrian van den Hoven EMA EUROPA Christelle Bouygues OIFE (Osteogenesis Imperfecta Federation Europe) Alessandra Tolaccia Medical Products Agency, Swedish Ministry of Health and Social Affairs Charlotte Asker Hagelberg
Looking into the future	EATRIS Don Lo
Closing remarks	Beacon: for rare diseases Rick Thompson