Zynerba Pharmaceuticals Presents Positive Data from Phase 2 INSPIRE Trial in 22q at The Society for the Study of Behavioural Phenotypes (SSBP) 24th International Research Symposium
Zynerba Pharmaceuticals (Nasdaq: ZYNE) presented positive results from the Phase 2 INSPIRE trial at the SSBP Symposium in Oslo, which evaluated Zygel for children with 22q11.2 deletion syndrome.
The trial showed statistically significant and clinically meaningful improvements in anxiety and behavioral symptoms, particularly on the Pediatric Anxiety Rating Scale (PARS-R) and other assessments. Zygel demonstrated a favorable tolerability profile with mild adverse events noted in a few patients. CEO Armando Anido expressed optimism about Zygel's potential and the FDA regulatory path.
- Statistically significant improvements in anxiety and behavioral symptoms in the INSPIRE trial.
- Zygel demonstrated excellent tolerability, consistent with previous trials.
- Positive risk-benefit profile suggests Zygel's potential in treating anxiety in 22q patients.
- None.
INSPIRE trial in 22q achieved statistically significant and clinically meaningful improvements from baseline in multiple efficacy assessments, with excellent tolerability profile
DEVON, Pa., Sept. 08, 2022 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, announces an oral presentation at The Society for the Study of Behavioural Phenotypes (SSBP) 24th International Research Symposium, being held in Oslo, Norway, September 8-10, 2022. Helen (Honey) Heussler, MBBS FRACP MRCPCH, DM, Associate Professor, Faculty of Medicine, Child Health Research Centre, University of Queensland, Australia, presented data from the open-label Phase 2 INSPIRE (Assessing the Impact of Zygel™ [Transdermal CBD Gel] on Pediatric Behavioral and Emotional Symptoms of 22q11.2 Deletion Syndrome) trial. A copy of Professor Heussler’s presentation is available on the Zynerba corporate website at www.zynerba.com/publications.
The presentation titled, “An Open-Label, Tolerability and Efficacy Study of ZYN002 (Cannabidiol) Administered as a Transdermal Gel to Children and Adolescents with 22q11.2 Deletion Syndrome (INSPIRE),” includes data that suggests a positive risk-benefit profile for Zygel in improving anxiety-related and behavioral symptoms in children and adolescents with 22q11.2 deletion syndrome (22q). Statistically significant improvements from baseline were seen in the Pediatric Anxiety Rating Scale (PARS-R), the total score and all five subscales of the Anxiety, Depression and Mood Scale (ADAMS) and all five subscales of the Aberrant Behavior Checklist – Community (ABC-C). In addition, the majority of patients showed clinically meaningful improvements as demonstrated by the Clinical Global Impression – Improvement (CGI-I). Zygel was shown to be well tolerated, and the safety profile was consistent with previously released data from other Zygel clinical trials. Three patients reported treatment related adverse events which were all mild application site adverse events. One patient discontinued treatment due to adverse events not related to Zygel.
“The data from the Phase 2 INSPIRE trial are very encouraging, including the significant improvement in anxiety as measured by the clinician-rated PARS-R scale,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We believe the Phase 2 data are very encouraging and suggest the potential of Zygel for the treatment of anxiety and behavioral symptoms in children and adolescents with 22q, and we look forward to discussing the regulatory path forward with the FDA.”
About Zygel
Zygel is the first and only pharmaceutically-manufactured cannabidiol formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally (i.e. through the skin). Recent studies suggest that cannabidiol may modulate the endocannabinoid system and improve certain behavioral symptoms associated with neuropsychiatric conditions. Zygel is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS), 22q11.2 deletion syndrome (22q) and autism spectrum disorder (ASD). The Company has received orphan drug designation for cannabidiol, the active ingredient in Zygel, from the FDA and the European Commission in the treatment of FXS and by the FDA for the treatment of 22q. Additionally, Zygel has been designated a Fast Track development program for treatment of behavioral symptoms of FXS.
About 22q11.2 Deletion Syndrome (22q)
As the second most common chromosomal disorder after Down syndrome, 22q is caused by a small missing piece of the 22nd chromosome. The deletion occurs near the middle of the chromosome at a location designated q11.2. It is considered a mid-line condition, with physical symptoms including characteristic palate abnormalities, heart defects, immune dysfunction, and esophageal / GI issues, as well as debilitating neuropsychiatric and behavioral challenges. Anxiety is among the most common neuropsychiatric symptoms of 22q and researchers have found that for children with 22q, anxiety is linked to poorer adaptive behaviors such as self-care and communication skills that affect daily life. Children with 22q also experience social withdrawal, attention deficit hyperactivity disorder, cognitive impairment, and autism spectrum disorder. Later in life, they are at an increased risk of developing mental illnesses such as schizophrenia. It is estimated that 22q occurs in between one in 3,000 and one in 6,000 live births, suggesting that there are approximately 83,000 people living with 22q in the U.S.
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, 22q11.2 deletion syndrome and autism spectrum disorder. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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