SIMPATHIC – Accelerating drug repurposing for rare neurological, neurometabolic and neuromuscular disorders by exploiting SIMilarities in clinical and molecular PATHology

SIMPATHIC’s main objective is to accelerate drug repurposing for rare neurological, neurometabolic and neuromuscular disorders. The project’s main accelerating innovation is the simultaneous drug development for groups of patients with different genetic diagnoses but overlapping neurological symptoms and molecular pathomechanisms.  

SIMPATHIC’s key outputs accelerating the drug repurposing pathway include:  

• Standard operating procedures (SOPs) for culturing stem cell-derived neuronal cell models with proven relevance for clinical symptoms and amenable to high-throughput drug screens;  
• New drug repurposing candidates with proven efficacy in advanced brain-on-a-chip and 3D brain organoid models, as demonstrated by reversal of molecular biomarker signatures and cellular readouts associated with clinical symptoms;  
• Designs of innovative basket clinical trials to which patients with different disorders are recruited, utilising and aggregating personalised clinical endpoints;  
• A training module for patients and patient organisations to empower them as drivers of the drug repurposing pathway;  
• Blueprints for intellectual property strategies, business models, regulatory dossiers and patient access strategies, developed in co-creation between all relevant stakeholders.  

SIMPATHIC’s ambition is to develop a fast and effective drug repurposing pathway for neuro-RDs in co-creation with all stakeholders, reducing the time from discovery to patient access by up to 4 years. 

SIMPATHIC consortium 

SIMPATHIC brings together an interdisciplinary group of partners that jointly co-create the drug repurposing process, and can cover the different phases of the drug development pathway. Partners Radboudumc, AMC, UT, UKHD and CHEORI combine preclinical and clinical research in the neuro-RD under study. They are also national and/or regional clinical expert centres for these conditions, and developed and/or help to maintain patient registries for the disorders under study. 

EATRIS role in the project 

EATRIS leads WP5 – Exploitation and patient access models for repurposed drugs in which will be developed exploitation pathways for the most promising repurposing candidates pursued in WP2-4. EATRIS will also coordinate the IP management that will consist of a preliminary freedom to operate analysis and monitoring of new (shared) IP generated (e.g. new formulation, use patent, ODD filing in concert with T5.3), in liaison with ELPEN and Tech Transfer Offices of the academic beneficiaries to pursue in/out-licensing opportunities. 

EATRIS will also lead the development of the Sustainability plan (WP6) to ensure exploitation of SIMPATHIC results and achievement of long-term impacts. The sustainability plan will be aligned with the business models and exploitation plan (WP5) supporting the development and market uptake of repurposing candidates identified in WP4. It will list the innovative tools and training programs produced by WP1-4 and describe how these will be offered to the research community as services to REMEDi4ALL (EU-funded project coordinating by EATRIS related to drug repurposing) and REPO4EU as emerging state-of-the-art platforms in the drug repurposing ecosystem.