Zynerba Pharmaceuticals Presents Longer Term Tolerability and Efficacy Data of Zygel™ in Children and Adolescents with Autism Spectrum Disorder (ASD) at The Society for the Study of Behavioural Phenotypes (SSBP) Conference 2021
September 09 2021 - 7:00AM
Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
presented data from the Phase 2 BRIGHT trial describing
tolerability and efficacy of Zygel (cannabidiol formulated in a
transdermal gel [ZYN002]) in children and adolescents with autism
spectrum disorder (ASD) over a longer term, 38-week treatment
period. These data were presented as an oral presentation at The
Society for the Study of Behavioural Phenotypes (SSBP) Conference
2021 being held virtually from September 9-10, 2021. A copy of the
presentation is available on the Zynerba corporate website at
http://zynerba.com/publications/.
Helen (Honey) Heussler, MBBS FRACP MRCPCH, DM,
Associate Professor, Faculty of Medicine, Child Health Research
Centre, University of Queensland, Australia, delivered the oral
presentation titled, “Longer Term Tolerability and Efficacy of
ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with
Autism Spectrum Disorder (ASD): An Open-Label Phase 2
Study (BRIGHT [ZYN2-CL-030]).” The presentation shows that
through 38 weeks of treatment, the BRIGHT trial provides initial
evidence suggesting a positive benefit-risk profile for Zygel when
administered in addition to stable standard of care in children and
adolescents with moderate-to-severe ASD. Furthermore, in patients
who completed the 38-week treatment period, statistically
significant improvements compared to baseline were sustained in all
efficacy measures of ASD compared to baseline.
“Current management options for ASD are
restricted to behavioral therapies and a limited number of approved
pharmacologic treatments, highlighting the substantial unmet need
for novel therapies in this difficult-to-treat population,” said
Joseph M. Palumbo, M.D., LFAPA, MACPsych, Chief Medical Officer of
Zynerba. “These data are encouraging, and if confirmed,
suggest meaningful therapeutic utility in ASD."
The open-label Phase 2 study evaluated the
safety, tolerability and efficacy of Zygel in children and
adolescents aged 3-17 years with ASD in addition to stable standard
of care. Eighteen of the 27 patients that completed week 14 (Period
1) demonstrated ≥35% improvement in the Aberrant Behavior
Checklist-Community (ABC-C) irritability subscale at week 14 and
were allowed to continue treatment for an additional 24 weeks
(Period 2). In the 18 patients who completed treatment through
Period 2, Zygel showed improvement in all the Aberrant Behavior
Checklist-Community (ABC-C) subscale scores (51% to 61% across
domains; P<0.0001), as well as improvements in the Parent-Rated
Anxiety Scale-ASD score (42%; P<0.0001), the Autism Parenting
Stress Index (40%; P<0.0001) and the Autism Impact Measure (19%
to 36% across domains; P≤0.0008), relative to baseline.
Zygel was generally well tolerated, and the
safety profile was consistent with data from previous Zygel
clinical trials. Adverse Events (AEs) were mild (80%) or moderate
(20%). Treatment-related AEs were reported in 19% of patients; most
were mild and transient. One patient discontinued due to
application site reaction. No serious or severe AEs or clinically
significant changes in vital signs, laboratory tests or ECG
parameters were reported during the study.
In addition, Randi J. Hagerman, M.D., Medical
Director and Endowed Chair in Fragile X Research at UC Davis MIND
Institute and Distinguished Professor at the Department of
Pediatrics at UC Davis School of Medicine, will present
previously-announced data from the CONNECT-FX trial, a randomized,
double-blind, placebo-controlled trial which assessed the efficacy
and safety of Zygel as a treatment for the behavioral symptoms of
FXS, on September 10, 2021, at 2:15 p.m. at SSBP. A copy of the
presentation, titled “A Pivotal Study of ZYN002 Cannabidiol (CBD)
Transdermal Gel in Children and Adolescents With Fragile X Syndrome
[CONNECT-FX (ZYN2-CL-016)]” will be available on the Zynerba
corporate website following the presentation at
http://zynerba.com/publications/.
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, 22q11.2 deletion syndrome, and
a heterogeneous group of rare and ultra-rare epilepsies known as
developmental and epileptic encephalopathies. Learn more at
www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
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Zynerba Contacts
Peter VozzoWestwicke/ICROffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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