Positive results in Phase 3 open label study of fasedienol (PH94B) in social anxiety disorder (SAD)
Positive U.S. Food and Drug Administration (FDA) feedback on the use of the Liebowitz Social Anxiety Scale (LSAS) as the primary efficacy endpoint in future Phase 3 studies of fasedienol in SAD
Successful itruvone (PH10) U.S. Phase 1 study supports previous successful Phase 1 studies and a positive Phase 2A study in major depressive disorder (MDD) conducted outside the U.S. and facilitates next-step U.S. Phase 2B development in MDD
Clinical-stage pipeline expanded to six differentiated product candidates targeting multiple large CNS markets
Pursuing multiple global and regional strategic development and commercialization partnerships to accelerate clinical and regulatory milestones across the CNS pipeline
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Vistagen (Nasdaq: VTGN), a late clinical-stage biopharmaceutical company aiming to transform the treatment landscape for individuals living with anxiety, depression and other central nervous system (CNS) disorders, today provided a corporate update and reported financial results for its fiscal year 2023 ended March 31, 2023.
“We continue to make significant progress across our CNS pipeline, which now includes six innovative clinical-stage product candidates, each with a differentiated mechanism of action, a favorable safety profile and substantial potential to improve millions of lives affected by anxiety, depression, hot flashes due to menopause and several other large market CNS disorders without therapeutic options that adequately satisfy patient needs,” said Shawn Singh, Chief Executive Officer of Vistagen. “Our deep understanding of the science and therapeutic potential underlying each of our product candidates provides a firm foundation for our confidence in our future. Given the collective body of positive safety and efficacy studies supporting our clinical-stage pipeline, now is the opportune time to amplify our efforts with multiple global and regional strategic development and commercialization partnerships across our portfolio to accelerate achieving key clinical and regulatory milestones within each program.”
Corporate Update
Positive results from PALISADE Phase 3 open label study amplify placebo-controlled Phase 2 studies of fasedienol (PH94B) in SAD and inform next steps in Phase 3 development to address a large and growing global unmet need.
Earlier this year, the Company reported that long-term administration of 3.2 µg of fasedienol, taken as-needed up to four times per day in a real-world setting, was well-tolerated with no new safety findings or trends identified, regardless of the number of doses administered by each subject. Overall, patients self-administered over 30,000 doses of fasedienol during the study with a mean duration of 4 months and a maximum study duration of over 10 months. Additionally, exploratory efficacy results from the study demonstrated clinically meaningful reductions in fear, anxiety and avoidance of anxiety-provoking social and performance situations in daily life, as measured by the Liebowitz Social Anxiety Scale (LSAS). We believe the continued improvement in LSAS scores observed in the study indicates the therapeutic potential of multiple, patient-tailored as-needed administrations of fasedienol over time to help patients build confidence and resilience, enabling them to engage in anxiety-provoking social and performance situations in their daily lives more frequently and with less fear and anxiety.
Positive FDA feedback on the path forward for development of fasedienol for SAD – FEARLESS Phase 3 program to center on the three prior FDA approvals in SAD using the LSAS as the primary efficacy endpoint.
In the first quarter of calendar 2023, the Company met with the FDA to discuss the next steps in its FEARLESS Phase 3 development plan for fasedienol in SAD, including conducting a potential New Drug Application (NDA)-enabling Phase 3 study of fasedienol in a real-world setting using the LSAS as the primary efficacy outcome measure in a manner similar to the registration studies for the three FDA-approved treatments for SAD. Support for an LSAS-based study design is demonstrated in the Company’s prior placebo-controlled Phase 2 study of fasedienol in SAD, in which the amount of separation between fasedienol and placebo as measured by the LSAS at the end of the first 2 weeks was comparable to what was observed after 12 weeks in the registration trials for the antidepressants currently approved by the FDA for treatment of SAD. Using the LSAS as the primary efficacy endpoint, all prior registration studies for these approved medications were positive.
Positive feedback from the FDA confirmed the acceptable use by the Company of the LSAS as a primary efficacy endpoint in its Phase 3 study of fasedienol for the treatment of SAD. Previously, the FDA granted Fast Track designation for the development of fasedienol for the treatment of SAD. Accordingly, the Company is now positioned to finalize key components of its NDA-enabling FEARLESS Phase 3 development program for fasedienol in SAD.
In contrast to the Company’s PALISADE Phase 3 single administration public speaking challenge studies in SAD conducted during the acute stage of the COVID-19 pandemic, the Company’s FEARLESS program in SAD will align with the LSAS-based study design supporting the precedent-setting NDA-enabling programs for the three antidepressants currently approved for treatment of SAD. The Company’s FEARLESS Phase 3 studies will be designed to assess multiple administrations of fasedienol, on a patient-tailored, as-needed basis in their daily lives, up to six times per day, in a real-world outpatient setting over a multiple-week period, with the clinician-administered LSAS as the primary efficacy endpoint. Dr. Michael R. Liebowitz, a Columbia University psychiatrist, former director and founder of the Anxiety Disorders Clinic at the New York State Psychiatric Institute and current Managing Director of The Medical Research Network LLC in New York City, is the creator of the LSAS. Dr. Liebowitz is a renowned authority on SAD and will serve as the Principal Investigator for the Company’s FEARLESS Phase 3 program in SAD.
Successful itruvone (PH10) U.S. Phase 1 clinical trial supports previous successful Phase 1 and Phase 2 studies conducted outside the U.S. and enables U.S. Phase 2B development in MDD.
Recently, the Company reported favorable safety and tolerability data from its U.S. Phase 1 clinical trial of itruvone, its innovative stand-alone, rapid-onset Phase 2 product candidate for the treatment of MDD. Itruvone nasal spray was well-tolerated and consistently continued to demonstrate a favorable safety profile. Results from this study build on previous successful Phase 1 studies and a positive placebo-controlled Phase 2A study of itruvone in MDD conducted outside the U.S. The successful U.S. Phase 1 study is expected to facilitate next step Phase 2B clinical development of itruvone in the U.S. During the past year, the FDA granted Fast Track designation for the development of itruvone for the treatment of MDD.
Positive results reported from Phase 2A study of PH80 in women diagnosed with vasomotor symptoms (hot flashes) due to menopause.
The Company recently reported that PH80 nasal spray demonstrated statistically significant efficacy versus placebo in a previously unreported exploratory placebo-controlled Phase 2A study for the acute treatment of vasomotor symptoms (hot flashes) due to menopause. PH80 induced a statistically significant reduction in the daily number of menopausal hot flashes compared to placebo at the end of the first week of treatment (p