On average it takes about 14 years for a new drug to travel from the research lab to market approval at an average cost of ≥€2 billion. Only 10% of compounds that enter preclinical testing ever make it into clinical trials, with only 20% of these achieving marketing approval. It is therefore very important to advance approaches to decrease the time, reduce costs and improve success rates during the development of drugs.
One approach is the repurposing of high-quality pharmaceutical industry compounds that have stalled at some stage during research or development. Many of these compounds have already undergone preliminary testing in humans, but have not been progressed further because they were not found to be sufficiently effective in the indication for which they were originally developed.
These compounds represent valuable tools that researchers can use to test their novel hypotheses for alternative therapeutic indications, with the ultimate aim of identifying alternative uses for these compounds in other indications. Since partial preclinical and clinical documentation packages have been developed for these assets, any positive findings hold the opportunity to progress towards the market more quickly and cost-effectively, with the ultimate goal of benefiting patients in diseases of high unmet need.
This asset-sharing repurposing programme through IMI2 JU aims to provide researchers across the EU to form hypothesis, to engage in collaborative research with industry and to access discontinued compounds that have already passed through several stages of the drug development process.
In this call, compounds, together with key information on their mechanism of action, pharmacology, safety, tolerability and exclusions, are made available for exploration in the area of Rare/orphan diseases (please find more details in the topic text). More precisely, applicants are invited to submit proposals to utilise these assets to test their hypotheses, to generate clinical data and, if needed, prerequisite preclinical data, with the ultimate aim of taking these assets to the market in alternative indications to those that they were originally developed for.
The overall objective of this pilot programme is to take one of the previously deprioritised clinical compounds and investigate their therapeutic potential in new clinical indications in the area of Rare/orphan diseases.
These compounds are listed in the topic text together with key information including mechanism of action, original indication, route of administration pharmacology, safety, tolerability and links to previous clinical studies and publications, to facilitate idea generation by investigators with hypotheses for novel uses. These compounds have all been through clinical phase 1 studies.
This programme intends to support only innovative clinical development for the compounds. This means that proposals for clinical development should not be considered in an indication which has been already tested (i.e. original primary indication or additional studies) or if there are already ongoing or planned clinical studies on identical or related disease indications with the compound or with a compound with overlapping mechanism of action that impacts the novelty of a given proposal.
Information on original primary indications, already tested indications, ongoing and/or planned clinical studies for each of these ten compounds can be found in the topic text.