Full Press Release Details
VistaGen Therapeutics Receives FDA Clearance of IND for Phase 2
Clinical Study of AV-101 as a Potential Treatment of Dyskinesia in
Parkinson's Disease Patients
Company also Receives a Notice of Allowance from the USPTO for a
New AV-101 U.S. Patent for Treatment of Dyskinesia Induced by
SOUTH SAN FRANCISCO, Calif., January 30, 2020 VistaGen
Therapeutics (NASDAQ: VTGN), a clinical-stage
biopharmaceutical company developing new generation medicines for
central nervous system (CNS) diseases and disorders with high unmet
that its Investigational New Drug (IND) application for AV-101, the
Company's oral NMDAR (N-methyl-D-aspartate receptor) glycine
site antagonist, as a potential treatment of dyskinesia in patients
with Parkinson's disease receiving levodopa-based therapy has
been cleared by the U.S. Food and Drug Administration (FDA). The
FDA's IND clearance permits VistaGen to proceed with Phase 2
clinical development of AV-101 in this indication. The Company also
announced that the U.S. Patent and Trademark Office (USPTO) has
issued a Notice of Allowance for U.S. Patent Application 16/003,816
related to therapeutic use of AV-101 for treatment of dyskinesia
induced by the administration of levodopa. The patent, once issued,
will be in effect until at least 2034.
Current drug treatment options for levodopa-induced
dyskinesia, or LID, may cause serious side effects, including
hallucinations and sedation. In all clinical studies to date,
AV-101 has not been associated with any psychotomimetic side
effects or drug-related serious adverse events. With its
exceptional safety profile, recently successful preclinical studies
in the leading primate model for LID, and the successful Phase 1b
NMDAR target engagement clinical study conducted by Baylor College
of Medicine in healthy volunteer U.S. military Veterans, we are
excited by AV-101's potential as a novel therapy for
Shawn Singh, Chief Executive Officer of VistaGen. These are
important milestones for our AV-101 program, both a key regulatory
advance and expanded commercial protection for AV-101 in the U.S.
AV-101 Preclinical Data in LID
In November 2019, at the 7th International
Conference on Parkinson's and Movement Disorders in
Th r se Di Paolo, Professor in the Faculty of Pharmacy at
Laval University and among the world's leading researchers focused
on Parkinson's disease and LID, presented preclinical data involving
AV-101 in the gold standard MPTP non-human primate
model for reproducing motor complications of Parkinson's disease,
including dyskinesia observed in Parkinson's disease patients
treated with levodopa. In the MPTP primate model, the
antidyskinetic activity of AV-101 compared favorably with prior
observations with amantadine in parkinsonian monkeys. However,
better than amantadine, with its known side effects (in humans with
Parkinson's disease and in parkinsonian monkeys), no adverse
effects with AV-101 were observed. In the study, AV-101's efficacy
against LID was measured through behavioral scores on a dyskinesia
scale, and a parkinsonian disability scale was used to measure
levodopa antiparkinsonian efficacy. Importantly, this study
demonstrated that AV-101 significantly (p = 0.01) reduced LID
without affecting the timing, extent, or duration of the
therapeutic benefits of levodopa.
About Parkinson's Disease and LID
disease is the second most common neurodegenerative disease
worldwide, affecting approximately one million people in the U.S.
and ten million people worldwide, according to the Parkinson's
Foundation. There is no "one-size-fits-all" description of
Parkinson's disease. Rather, it is a complex
neurodegenerative disorder that occurs when brain cells that make
dopamine, a chemical that coordinates movement, stop working or
die, resulting in progressive deterioration of voluntary motor
control. Classic motor symptoms of Parkinson's disease
include muscular rigidity, resting tremor, and postural and gait
impairment. Typically, patients with Parkinson's disease
present with a combination of motor and non-motor symptoms.
Non-motor symptoms may include cognitive impairment, sleep
disorders, pain and fatigue. There is currently no medication to
slow, delay, stop or cure Parkinson's disease, and currently
available treatments are symptomatic. Treatment of motor symptoms
with oral levodopa, introduced about 50 years ago, remains the
gold standard treatment.
in patients with Parkinson's disease receiving levodopa-based
therapy is a disorder that is characterized by unpredictable,
involuntary and purposeless movements after continuous long-term
use (often longer than five years). Although clinical
manifestations of LID are heterogenous, these motor complications
tend to become more severe as a patient's Parkinson's
disease progresses and as the duration of levodopa treatment is
extended, until the impact of LID may compromise the advantage of
treatment with levodopa. Once LID develops, Parkinson's
disease patients treated with levodopa may be faced with a choice
between immobility due to untreated and uncontrolled
Parkinson's disease, or mobility with the associated
AV-101 (4-Cl-KYN) belongs to a new generation of investigational
medicines in neuropsychiatry and neurology known as NMDAR
(N-methyl-D-aspartate receptor) modulators. The NMDAR is a pivotal
receptor in the brain and abnormal NMDAR function is associated
with numerous CNS diseases and disorders. AV-101 is an oral prodrug
of 7-chloro-kynurenic
potent and selective full antagonist of the glycine site of the
NMDAR. With its exceptional side effect and safety profile in all
studies to date (no psychological side effects or safety concerns
similar to those that may be caused by amantadine and ketamine),
AV-101 has potential to be a new oral, at-home, non-sedating
treatment for multiple large market CNS indications where current
treatments are inadequate to meet high unmet patient needs. The FDA
has granted Fast Track designation for development of AV-101 as