Our lead drug candidate, AV-101, also known as “L-4-chlorokynurenine” and “4-Cl-KYN”, is a novel, orally available prodrug focused on the treatment of diseases and disorders involving the central nervous system (CNS), including Major Depressive Disorder (also known as MDD or refractory depression), epilepsy, neuropathic pain, and Parkinson’s disease.

AV-101 is converted in the brain into an active metabolite, 7-chlorokynurenic acid (7-Cl-KYNA), which is an antagonist of the N-methyl-D-aspartate (NMDA) receptors. 7-Cl-KYNA is a synthetic analogue of kynurenic acid (KYNA), which is a naturally occurring CNS regulatory compound, and is one of the most potent and selective blockers of the regulatory GlyB-site of the NMDA receptor. However, 7-Cl-KYNA does not cross the blood-brain barrier.

In preclinical studies, AV-101 is rapidly and efficiently transported across the blood-brain barrier, and is converted into 7-Cl-KYNA in the brain and spinal cord, preferentially, at the site of seizures and potential neural damage.

We have successfully completed Phase I development of AV-101, demonstrating that AV-101 is well tolerated without any significant safety issues. AV-101 is a candidate for the treatment of multiple diseases and disorders involving the central nervous system (CNS), including, Major Depressive Disorder (MDD or refractory depression), refractory epilepsy, neuropathic pain, and neurodegenerative diseases such as Parkinson’s disease and Huntington’s disease. We believe our AV-101 IND application on file at the U.S. Food and Drug Administration, and the safety studies we have completed successfully in Phase I development of AV-101, will support our development of AV-101 for the multiple CNS indications noted above.

To date, the U.S. National Institutes of Health (NIH) has awarded us $8.8 million of grant funding for our preclinical and Phase 1 clinical development of AV-101. We are now preparing to begin Phase 2 clinical development of AV-101 with the NIH.

A non-confidential AV-101 program summary is available below. For additional information, please contact our Business Development Department at info@vistagen.com.