VistaGen Therapeutics stands at the forefront today of the rapidly developing world of stem cell technology, with a tight portfolio of IP that cuts hard and fast across multiple, critical applications in the areas of drug rescue and regenerative medicine. The company’s Human Clinical Trials in a Test Tube™ platform fuses together proprietary technologies for controlled development/differentiation of human pluripotent (having the potential to differentiate into almost any cell) stem cells into many types of mature, functional adult cells and the utilization thereof.
This incredible capacity to produce large quantities of functional adult cells has thus far led to two 3D “micro-organ” bioassay system applications for drug rescue, CardioSafe 3D™ and LiverSafe 3D™. Using stem cell-derived, functional heart and liver cells (respectively) to create mock-up 3D cell networks and tissue structures, which accurately model the actual organs in the body, presents the multibillion dollar pharma development space with a safer small molecule Drug Rescue Variant™ solution pathway. This salvage opportunity technology is able to help drug developers recoup the massive capital outlays associated with creating once-promising new drug candidates that got dropped due to bad drug interactions and/or toxicity concerns, in essence offering drug developers a way to hit the reset switch and come away with a safer, more effective product instead of lost capital. Moreover, CardioSafe 3D and LiverSafe 3D offer pharma developers a drug metabolism and toxicity screening solution that ably surmounts one of the major causes of the high failure rates among otherwise promising drug candidates, the persistent limitations of preclinical drug testing.
VistaGen is also developing a novel, orally available small molecule prodrug candidate known as AV-101, which has shown solid results in Phase 1 clinical development for neuropathic pain in the U.S., under an active Investigational New Drug application with the FDA. With apparent traction in depression and epilepsy, as well as potentially in other neurological conditions, including Parkinson’s, AV-101 is of particular interest in that it acts as a synthetic analog for a naturally-occurring regulatory compound in the central nervous system, kynurenic acid (AV-101 converts into the analog, 7-chlorokynurenic acid, inside the brain). High oral bioavailability, excellent blood-brain barrier transport dynamics and preferential conversion to synthetic kynurenic acid at sites of neural damage, all reinforce the $8.8M in funding VSTA has received from the NIH for AV-101’s development.
Advancements in the broader stem cell space, like the recent announcement that Japanese researchers at the RIKEN Center for Life Science Technologies have identified how CCL2 (a chemokine that plays a role in immune response) functions as a pluripotency enhancer in human induced pluripotent stem cells, makes VSTA’s portfolio start to look more and more interesting. The potential for modeling drug interactions in vitro with high-fidelity in vivo results guidance (long before substantial development costs start to add up), as well as the potential to salvage otherwise deprecated, yet promising drug candidates, combined with an eventual ability to re-grow various tissues and break through to new horizons in regenerative medicine, makes VSTA an exciting company for its size and market cap. Inducing the body to take up cultured stem cells and re-grow blood, bones, cartilage and other tissues, even heart, liver and pancreatic tissue, is serious blue sky potential that may even dwarf the value of chemical variant salvaging on drug candidates for pharmaceutical and biotech customers.
With recent patent expansions in both the U.S. and Canada this year for their stem cell technology platform, VistaGen is feeling supremely confident about the company’s performance. CEO of VSTA, Shawn K. Singh, JD, even noted how the Canadian patent allowance in particular expands the company’s core IP in a key target market the company has been after for years now. This particular patent, under exclusive license from the Icahn School of Medicine, Mount Sinai, New York, covers pluripotent stem cell culture systems which produce endoderm lineage cells, including liver, lung, pancreas, parathyroid, and thyroid cells. The equivalent U.S. patent expansion covers three patents under similar license and roundly reinforces the company’s already strong LiverSafe 3D position, while also opening up collaboration potential in the area of liver biology and drug metabolism assay, as well as regenerative therapy for diabetes using pancreatic beta-islet cells.
The recent (July 2) announcement that University at Buffalo researchers have identified the “master switch” which triggers myelination in the brain, a process where fatty layering (a sign of healthy central nervous system functionality) accumulates on the neuronal axon (or main shaft), allowing the brain cell to transmit data quickly and reliably (enabling more complex brain activity to take place), should give investors some idea of where this sector is heading. The identification of this “master switch,” a transcription factor in human brain cells known as SOX10, puts multiple sclerosis for instance directly in the crosshairs for development of an efficient treatment. An MS treatment based on transplantation of myelin-generating progenitor cells that doesn’t take a year or more, says it all about the potential of the stem cell space really. In this particular case the potential exists for a small molecule drug candidate that could switch on SOX10 as well.
The immense potential of stem cell technology for fundamentally transforming several areas of medicine is fully inherent in companies like VistaGen Therapeutics, whether we are talking small molecule drug development or regenerative stem cell therapies and organ modeling.
More data on VistaGen Therapeutics is available at: www.vistagen.com
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