Zynerba Pharmaceuticals Announces Completion of Enrollment in Phase 2 Trial of Zygel™ in 22q11.2 Deletion Syndrome
February 28 2022 - 7:00AM
Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced the completion of enrollment for the 14-week Phase 2
INSPIRE (Assessing the
Impact of Zygel
[Tra
nsdermal CBD Gel] on
Pediatr
ic
Behavio
ral and
Emotional Symptoms
of 22q11.2 Deletion Syndrome) trial of Zygel in the treatment of
behavioral symptoms of 22q11.2 deletion syndrome (22q) in children
and adolescents. The Company continues to expect topline data from
this trial mid-year 2022. The Company has previously received
orphan drug designation for Zygel in 22q from the FDA.
“The completion of enrollment in our INSPIRE trial is an
important step forward in advancing Zygel as a potential new
treatment option for patients suffering from 22q,” said Armando
Anido, Chairman and Chief Executive Officer of Zynerba. “Pending
results from the INSPIRE trial and subsequent discussion with the
FDA on the regulatory path forward, we plan to advance our clinical
development program in 22q with Zygel. We are thankful for the
combined efforts of our researchers, investigators and the patient
and caregiver community.”
The 14-week INSPIRE trial is an open-label, multi-dose, Phase 2
clinical trial designed to evaluate the efficacy and safety of
Zygel in children and adolescents (ages six through 17) with
genetically-confirmed 22q. Enrolled patients receive weight-based
doses of 250 mg daily or 500 mg daily of Zygel. The efficacy
assessments include the Aberrant Behavior Checklist-Community
(ABC-C), the Anxiety, Depression and Mood Scale (ADAMS), the
Qualitative Caregiver Reported Behavioral Problem Survey, and
Clinical Global Impression – Severity and Improvement. A total of
20 patients have been enrolled in the INSPIRE trial.
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), autism spectrum
disorder (ASD), and 22q11.2 deletion syndrome (22q). Zygel has been
granted orphan drug designation by the FDA in the treatment of FXS
and 22q. Additionally, the FDA has designated Zygel 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 withdrawn behavior, ADHD, cognitive
impairment, and autism spectrum disorder that affect communication
and social interaction. Later in life, they are at an increased
risk of developing mental illnesses such as schizophrenia. It is
estimated that 22q has a prevalence rate of approximately one in
4,000 people in the U.S.1,2, suggesting that there are
approximately 83,000 people living with 22q in the U.S.
1 The 22q Family Foundation2 The International 22q11.2
Foundation
About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative
pharmaceutically-produced transdermal cannabinoid therapies for
rare and near-rare neuropsychiatric disorders. We are committed to
improving the lives of patients and their families living with
severe, chronic health conditions including Fragile X syndrome,
autism spectrum disorder, and 22q11.2 deletion syndrome. Learn more
at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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Statements
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Zynerba Contact
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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