VistaGen Therapeutics, Inc. (VSTA) is biotechnology company applying stem cell technology for drug rescue, predictive toxicology, and drug metabolism screening. Drug rescue combines human stem cell technology with modern medicinal chemistry to generate new chemical variants ("drug rescue variants") of once-promising drug candidates that have been discontinued during late-stage preclinical development due to heart or liver safety concerns.
VistaGen's versatile stem cell technology platform, Human Clinical Trials in a Test Tube™, has been developed to provide clinically relevant predictions of potential heart and liver toxicity of promising new drug candidates long before they are ever tested on humans.
By more closely approximating human biology than conventional animal studies and other nonclinical techniques and technologies currently used in drug development, VistaGen's human stem cell-based bioassay systems can improve the predictability of the drug development cycle and lower the cost of new drug research and development by identifying product failures earlier in the cost curve. According to the Food and Drug Administration even only a ten percent improvement in predicting failure before clinical trials could save $100 million in development costs, which savings ultimately could be passed on to patients.
Using mature human heart cells produced from stem cells, VistaGen has developed and internally validated CardioSafe 3D™, a novel three-dimensional (3D) bioassay system for predicting the in vivo cardiac effects of new drug candidates before they are tested in humans. VistaGen is now focused on using CardioSafe 3D™ to generate up to two new, safer small molecule drug rescue variants every twelve to eighteen months. VistaGen anticipates that these drug rescue variants will be modified versions of once-promising new drug candidates that have been discontinued by pharmaceutical companies and academic research institutions because of heart toxicity concerns, despite substantial prior investment and positive efficacy data demonstrating their potential therapeutic and commercial benefits. In most cases, VistaGen plans to license or sell its new, safer drug rescue variants in strategic partnering arrangements with global pharmaceutical companies, arrangements providing VistaGen with both near term and downstream milestone payments and economic participation rights but without future development cost obligations.
VistaGen's lead drug candidate, AV-101, is in Phase Ib development in the U.S. for treatment of neuropathic pain, a serious and chronic condition causing pain after an injury or disease of the peripheral or central nervous system.
Neuropathic pain affects approximately 1.8 million people in the U.S. alone. Although the current active AV-101 IND is for the treatment of neuropathic pain, VistaGen's development plan and regulatory strategy for AV-101 has been designed to allow its Phase 1 safety studies to support Phase 2 development for depression, epilepsy, Huntington's Disease and Parkinson's disease, indications for which there is now supporting preclinical efficacy data. To date, VistaGen has been awarded over $8.5 million from the U.S. National Institutes of Health (NIH) for development of AV-101.
VistaGen is also developing LiverSafe 3D™, a novel predictive liver toxicity and drug metabolism bioassay system for drug rescue applications. In parallel with drug rescue activities, the Company is funding early-stage nonclinical studies focused on potential cell therapy applications of its Human Clinical Trials in a Test Tube™ platform.