Full Press Release Details
VistaGen Announces Positive Results of Newly Published Exploratory
Phase 2a Study of PH10 for Rapid-Onset Treatment of Major
PH10, a Neurosteroid Nasal Spray, Demonstrated Significant
Rapid-Onset Antidepressive Benefit versus Placebo, without
Psychological Side Effects or Safety Concerns Often Associated with
Ketamine-based Therapy
SOUTH SAN FRANCISCO, Calif., February 20, 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
medical need, today announced positive
results from a newly published exploratory Phase 2a clinical study
of PH10, its investigational first-in-class, rapid-onset synthetic
neurosteroid nasal spray, for treatment of major depressive
disorder (MDD). Results of the double-blind, randomized,
placebo-controlled Phase 2a study have been published in the
British Journal of Pharmaceutical and Medical
In the single-site exploratory Phase 2a study (n=30), randomized
participants received placebo or either a 3.2
neuroactive nasal spray twice daily for eight weeks. Change in
total score on the 17-item Hamilton Depression Rating Scale
(HAM-D-17), a multiple-item questionnaire used to provide an
indication of depression as a guide to evaluate recovery, was
measured at the end of each week of treatment. A rapid
antidepressant benefit from the PH10 6.4
g dose was evidenced by
changes in HAM-D-17 scores at the end of the first week of
treatment. After one week of treatment, the mean reduction of
HAM-D-17 scores for the PH10 6.4
points, which was statistically greater (p = 0.03) than the mean
reduction in the placebo group of 4.2 points from baseline. Also,
at the end of the last week of treatment (Week 8), the PH10
g group showed a mean
HAM-D-17 score reduction of 17.8, which was statistically greater
than the mean reduction in the placebo group of 10.9 points from
baseline (p = 0.02). Thus, in the PH10 6.4
g treatment group, the
HAM-D-17 score improved significantly from the baseline within one
week and this effect was sustained until the Week 8 study endpoint.
Notably, both the PH10 3.2
showed strong effect sizes after one week of treatment (0.72 for
g dose and 1.01 for the
g dose) and at the Week
8 study endpoint (0.74 for the 3.2
g dose and 0.95 for the
There were no reports of serious adverse events. At the end of
treatment, all subjects reported to have tolerated twice daily
self-administration. The results of this peer-reviewed published
study suggest PH10's potential as a non-systemic,
rapid-onset, new generation antidepressant for the millions of
people suffering from depression around the world. VistaGen is
currently preparing for Phase 2b clinical development of PH10 in
the U.S. for treatment of MDD.
The results of this exploratory Phase 2a clinical study
demonstrate PH10's exciting potential to treat patients with
MDD with a first-in-class, non-systemic, rapid-onset antidepressant
without the serious psychological side effects and safety concerns
of ketamine-based therapy. The large separation from placebo seen
at one week, the first time point measured, and sustained through
eight-week completion is very encouraging. The significant
reduction in HAM-D-17 with the 6.4
g dose at the end of
the first week of treatment suggests that the antidepressant effect
of PH10 may have started even earlier than 1 week. Our plan for
Phase 2b development of PH10 in MDD includes a next-step study of
four weeks in duration, with an earlier initial measurement of
antidepressant effect, likely within the first 24 to 48 hours of
initial dose administration, stated Mark
Smith, M.D., Ph.D., Chief
Medical Officer of VistaGen.
Depression remains a highly prevalent and difficult to treat
mental illness, but we believe these data suggest a new path
forward in providing treatment for the millions of individuals with
MDD. The significant reduction in HAM-D-17 scores for the PH10 6.4
g group versus the placebo group not only offers evidence of
PH10's potential to be a fast-acting, easily administered MDD
treatment alternative, but also supports the nasal chemosensory
system as a novel way of delivering CNS active medications, which
is also relevant for VistaGen's other neuroactive nasal
spray, PH94B for social anxiety disorder entering Phase 3
development later this year, added Dr. Smith.
Therapeutics is a multi-asset, clinical-stage biopharmaceutical
company developing new generation medicines for CNS diseases and
disorders where current treatments are inadequate, resulting in
high unmet need. VistaGen's pipeline is
focused on clinical-stage CNS drug candidates with a differentiated
mechanism of action, an exceptional safety profile, and therapeutic
potential in multiple large and growing CNS markets. For more
information, please visit www.vistagen.com
and connect with VistaGen on Twitter, LinkedIn and Facebook.
PH10 is an investigational first-in-class, odorless, fast-acting
synthetic neurosteroid with therapeutic potential in a wide range
of neuropsychiatric indications involving depression and suicidal
ideation. VistaGen is initially developing PH10 as a potential
fast-acting, non-sedating, non-addictive new generation treatment
of MDD that can be conveniently self-administered at home. Upon
self-administration, a non-systemic microgram-level dose of PH10
sprayed into the nose binds to nasal chemosensory receptors that,
in turn, activate neural circuits in the brain that lead to
rapid-onset antidepressant effects, without side effects, systemic