Vistagen has a versatile human pluripotent stem cell technology platform and proprietary methods for differentiating stem cells into mature cell types for applications in drug rescue, discovery, and cell therapy. They are focused on developing "drug rescue variants" - molecules derived from previously terminated drug candidates that show similar efficacy but less toxicity. Vistagen has multiple preclinical programs in areas like heart and liver repair, and their drug AV-101 is in a Phase 1b trial for neuropathic pain. With over 12 years of research and $40 million invested, Vistagen aims to leverage their stem cell technology and expertise in drug rescue.

1. →
www.vistagen.com

2. Investment highlights
• Versatile human pluripotent stem cell • Focused on building a pipeline of “drug
technology platform rescue variants” – small molecules with
• Proprietary differentiation of human promising efficacy of original new drug
pluripotent stem cells into mature cells candidates but less toxicity
• Mature cells used for drug rescue, drug • Multiple pilot preclinical cell therapy
discovery and cell therapy programs focused on heart, liver and
cartilage repair and autologous bone
• Drug rescue leverages prior investment in marrow transplantation
new drug candidates “put on the shelf”
during preclinical development due to • AV-101 in Phase 1b clinical development for
toxicity concerns neuropathic pain
• Not an early stage concept; 12 years and
$40 million invested to date
→
Monday, April 23, 2012 www.vistagen.com 2

3. FDA’s emphasis on drug safety
is increasing
“FDA Pulls
Darvon
“FDA to Restrict Painkiller
“FDA Revamps Avandia, Due to Safety
Process for Citing Heart Risks”
Safety of Drugs Risk” San Francisco Chronicle
November 2010
News York Times
After Approval” September 2010
Washington Post
January 2007
→
low tolerance for heart and liver toxicity issues
Monday, April 23, 2012 www.vistagen.com 3

4. Once the best-selling antihistamine in the world
Seldane
“FDA Wants
Drug Seldane
”
Off Market
Washington Post January 1997
US Prescriptions in 1996
“ FDA Says
Seldane Should
”
Be Withdrawn
Los Angeles Times January 1997 →
Monday, April 23, 2012 www.vistagen.com 4

5. Drug rescue works
GLOBAL SALES
IN 2004
caused fatal
and serious
cardiac
toxicities
efficacy of
Seldane without
serious cardiac
→
toxicities
Monday, April 23, 2012 www.vistagen.com 5

6. Late-stage safety failures are just the tip of the iceberg
The Safety Iceberg
$1.5B
Marketed and late-stage clinical $1B
$75M
$35M
Deep inventory of promising $25M
drug candidates put on the shelf
due to heart or liver toxicity $15M
$5M
Preclinical and early-stage clinical
Monday, April 23, 2012 www.vistagen.com 6