While all applications submitted in 2013 were not accepted for filing as of December 31, 2013, CDER forecasts that about 36 total applications were submitted for 2013, meaning nine were left on the table for one reason or another.
This information is of particular value to VistaGen Therapeutics, a small-cap biotech company focused on reviving once-promising drug candidates that were shelved due to toxicity issues. The expense of failure is staggering for the company and investors that have invested considerable time and money in their drug candidates. This high cost of failure associated with unexpected heart and liver toxicity and metabolism issues drives the cost to develop drug candidates higher and results in relatively low annual number of FDA-approved NMEs.
VistaGen believes that the majority of pricey and unexpected heart and liver toxicity and metabolism issues stem from limitations of the major toxicological testing systems currently used in the pharmaceutical industry, namely animal testing and cellular assays based on transformed cell lines and human cadaver cells.
The company’s solution is its Human Clinical Trials in a Test Tube™, which utilizes human pluripotent stem cells (hPSCs), cells that have the ability to be geared and developed into any other kind of cell in the human body, and differentiates them into useable human heart cells and human liver cells. VistaGen’s strategy is to apply this technology combined with medicinal chemistry to generate new, proprietary drug rescue variants of once-promising small molecule drug candidates that were discarded for safety concerns.
Using mature human heart cells produced from its stem cell technology, VistaGen has developed CardioSafe 3D™, a novel, three-dimensional (3D) bioassay system for predicting the in vivo cardiac effects, both toxic and non-toxic, of drug rescue variants and other small molecule drug candidates before they are tested in animals and humans. The company is further expanding its drug rescue capabilities by developing LiverSafe 3D™, a novel bioassay system using hPSC-derived human liver cells to assess potential liver toxicity and adverse drug-drug interactions in drug rescue variants and other new drug candidates early in development, before animal and human testing. Identifying toxicity issues early in the development process can save considerable research, money and other resources.
Of the 27 drugs recently approved by CEDR, at least two pertained to cardiac disease. VistaGen is well-positioned to take advantage of unmet needs in this niche market of the healthcare industry.
For more information on VistaGen visit www.vistagen.com
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