Zynerba Pharmaceuticals Presents Positive Data from Phase 2 INSPIRE Trial in 22q11.2 Deletion Syndrome at the National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit
October 17 2022 - 7:00AM
Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for orphan neuropsychiatric disorders, presented a poster
at the 2022 National Organization for Rare Disorders (NORD) Rare
Diseases and Orphan Products Breakthrough Summit being held October
17-18, 2022, in Washington, D.C. A copy of the poster is available
on the Zynerba corporate website at www.zynerba.com/publications.
The poster, 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),”
reports data from the first 14-week treatment period 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). This study enrolled 20
patients in the U.S. and Australia. 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.
“We are encouraged by the results from our Phase 2 INSPIRE
study, where we saw statistically significant and clinically
meaningful improvements from baseline in multiple efficacy
assessments, including 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 suggests 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 later this half.”
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 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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Zynerba Contact
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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