Off-Label Ltd

Use the Off-Label.com Target Library to translate preclinical hits into potential clinical applications

The Off-Label.com Target Library represents an invaluable resource for supporting key decision-making when effecting the transition from laboratory assay to clinical study. Current data reported by FDA show that attrition for lead candidates in progressing from laboratory to clinical testing can exceed 90%, with devastating effects upon time and resources. The Off-Label.com Target Library fills a void, enabling for the first time an additional screen that could result in significant labour and financial savings.

“Reaching a more systematic and dynamic understanding of human disease will require major additional scientific efforts as well as significant advances in bioinformatics……as candidates emerge, the best tools available should be used for their evaluation……and working to identify ways to bridge between the laboratory and the whole organism and correlate early markers of safety and benefit with actual outcomes in patients.”

Challenge and Opportunity on the Critical Path to New Clinical Products, FDA 2004

Off-Label.com maintains two datasets in constant fidelity

The Core Clinical Database at Off-Label.com catalogs licensed and unlicensed uses for the existing pharmacopoeia, with newly-reported unlicensed uses compiled daily; in addition, a second dataset, the Target Library is likewise maintained in a state of constant fidelity.

Through these datasets, Off-Label.com provides the only resource that enables high-fidelity translational research with, for the first time, the real prospect of zero attrition. Researchers can now screen preclinical hits at any stage in development by selecting targets from the Target Library, and viewing potential clinical applications that converge upon these targets.

Translational research at Off-Label.com…From bench to bedside and back

Given the billions and billions of molecules that have been screened as potential candidates for use in the clinic, it is a sobering thought that there are only about 1,000 regularly-prescribed, small molecules in the US pharmacopoeia. The convergence of such a small number of drugs upon an equally small number of druggable targets (about 500), qualifies this select population of molecules as a collective "memory" or "library" of pharmacophores or templates of druggable targets, upon which tens of thousands of diseases converge. At Off-Label.com we make this wealth of knowledge available for systematic study.

By connecting druggable targets with clinical outcomes, translational research at Off-Label.com means that researchers can reduce the probability of making type I/II errors in critical decision-making, discover new outcomes and search for "off-target" effects and "hidden phenotypes"; all at a clinical level. The Core Clinical Database, that grows by about 200-300 new reports of novel (ie unlicensed) use each month, can be queried through a metadata layer that comprises about 500 human genome-derived druggable targets (with about 30 nonhuman). This metadata layer of druggable targets functionally unites drug and target promiscuity with clinical divergence.

Providing solutions for a changing industry

For meaningful advances in today’s drug discovery, there is much to be learned through systematic study of the convergence of both 1,000 clinically efficacious drugs and some 20-30,000 human disorders upon about 500 human genome-derived druggable targets (and about 30 nonhuman).

SAMPLE SEARCH USING A SINGLE TARGET