Repositioning Drugs and Drug Combinations to treat patients with unmet medical need Rare Diseases Program

SlideA disease or disorder is defined as rare when it affects fewer than 1 in 2000 people in Europe, and 1 in 1500 people in the US. There are over 6000 rare diseases affecting more than 400 million people globally, including 40 million in Europe and 30 million in the US. 95% of all rare diseases do not have a single approved drug treatment. 80% of these diseases have a genetic origin, and 50% of them affect children. 20% of all cancers are rare. 3-10% of all hospitalizations, regardless of age, are related to a rare disease. (Ref. Global Genes, Eurordis). About Rare Diseases

SlideBioXplor applies it's data-driven platform to accelerate and derisk drug repositioning including combination drug candidates. Patient data is required to match treatments to responder populations. Drugs are identified and assessed for licensing potential before being prioritized for preclinical validation in cell and animal models. To achieve this, BioXplor partners with rare disease patient groups, academic teams and pharmaceutical companies. BioXplor works with patients to collect data from electronic medical records, genomics sequencing results, patient reported insights and wearables data via a custom designed rare disease-specific patient app. Partnership Program

SlideAs part of an ongoing collaboration with a leading cancer research team studying a rare and particularly aggressive form of Leukemia (ETP-ALL). BioXplor set out to analyse the ZEB2 pathway to identify optimal drug combinations. Our team identified a novel drug combination for ETP-ALL with strong supporting evidence to modulate the known disease causing proteins, and the combination has already shown safety and efficacy in another indication. This drug combination is being validated in vitro and in vivo using patient tissue and transgenic models. Case Study - Rare Leukemia Drug Combinations