Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, is
presenting a poster describing data from the Phase 2 BRIGHT (An
Open-La
bel
Tole
rab
ility and Efficacy Study
of ZYN002 Administered as a Transdermal
Gel to
C
hildren and Adolescen
ts with
Autism Spectrum Disorder) trial further demonstrating the potential
for Zygel™ (ZYN002) to improve the core behavioral symptoms of
autism when administered in addition to stable standard of care in
children and adolescents with moderate-to-severe ASD. These open
label data are being presented at the virtual Joint 16th
International Child Neurology Congress (ICNC) & 49th Annual
Child Neurology Society (CNS) Meeting. These data will also be
presented as an oral presentation during the “Research Pipeline:
New Findings on Diagnostic and Therapeutics” session of the virtual
American Academy of Child and Adolescent Psychiatry (AACAP) 2020
Annual Meeting on Friday, October 23rd, 2020.
A copy of the poster entitled, “Tolerability and Efficacy of
ZYN002 Cannabidiol (CBD) Transdermal Gel in Children and
Adolescents With Autism Spectrum Disorder: An Open-Label Phase 2
Study [BRIGHT (ZYN2-CL-030)]” is available on the Zynerba corporate
website at http://zynerba.com/publications/.
“The Phase 2 BRIGHT trial provides the first clinical evidence
of the potential for Zygel to improve behavioral symptomology in a
group of highly symptomatic pediatric and adolescent patients with
ASD,” said Helen Heussler, FRACP, Associate Professor at Children’s
Health Queensland, Medical Director Child Development and principal
investigator in the BRIGHT trial. “In these children receiving
Zygel, the observed changes from baseline are promising. In
particular, the improvements seen in core symptoms of autism, as
specifically assessed by the Autism Impact Measure, are of special
interest. Though open label, these results are compelling and we
look forward to continuing the evaluation of Zygel in ASD in future
placebo-controlled clinical trials.”
The BRIGHT Phase 2 trial is an exploratory, single-center,
open-label Phase 2 study evaluating the safety and tolerability and
efficacy of Zygel in children and adolescents with ASD who are 3 to
<18 years old. The study enrolled patients with Clinical Global
Impression (CGI)-Severity score ≥4 (moderate or greater) and
Aberrant Behavior Checklist-Community (ABC-C) Irritability score
≥18. The primary objective of the trial was to evaluate the safety
and tolerability of Zygel for up to 38 weeks (14-week treatment
period and a 6 month extension period). Secondary objectives
comprised evaluation of the efficacy of Zygel in the treatment of
symptoms of ASD, including measuring parental/caregiver stress,
Autism Impact Measure (AIM), and caregiver reported behavioral
problems. The results provided in the poster are after 14 weeks of
treatment with Zygel.
New data presented today include that patients receiving Zygel
in this study achieved statistically significant caregiver-reported
improvements compared to baseline across all subscales of the AIM,
which was designed to track incremental change in frequency and
impact of core ASD symptoms: Atypical behavior (p<0.001),
Communication (p<0.001), Peer Interaction (p<0.001),
Repetitive Behavior (p<0.001), and Social Reciprocity
(p=0.0053).
Figure 1.
Statistically Significant Improvements in Autism Impact Measure
Scores
https://www.globenewswire.com/NewsRoom/AttachmentNg/73c3a0dc-15b2-4959-82f1-cfdaff740f7e
In addition, statistically significant improvements compared to
baseline were observed at week 14 of treatment with Zygel in the
Autism Parenting Stress Index (p<0.0001).
Figure 2. Statistically Significant Improvements in
PRAS-ASD, APSI, and CGI-I
https://www.globenewswire.com/NewsRoom/AttachmentNg/3838f1c3-2cfa-4e27-88c2-1c5153c5a307
Zynerba also measured notable improvements in behaviors
utilizing the Qualitative Caregiver Behavioral Problems Survey
after 14 weeks of study drug. Clinically meaningful improvements
were observed by a majority of surveyed caregivers in behavioral,
social and emotional behavioral problems.
Figure 3. Notable
Improvements in the Qualitative Caregiver Behavioral Problems
Survey at Week 14
https://www.globenewswire.com/NewsRoom/AttachmentNg/6fde1c59-6af5-4ebe-af13-e3c5a89aa379
The authors of the poster concluded that:
- 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;
- Zygel showed improvement in all ASD measures (ABC-C, AIM,
PRAS-ASD, CGI and Qualitative Caregiver Assessments);
- Further controlled studies are warranted in this
difficult-to-treat population.
These results supplement the topline data initially disclosed by
the Company in May 2020 (Press release), including that:
- All five subscales
of the ABC-C showed both statistically significant (p<0.0002)
and clinically meaningful improvements at 14 weeks of treatment
from baseline.
- The results of other
efficacy assessments reinforce the results demonstrated in the
ABC-C, including a mean improvement of 46% at week 14 from baseline
as measured by the Parent Reported Anxiety Scale for ASD (PRAS-ASD;
p<0.0001) and 57% of patients were assessed as “very much
improved” or “much improved” at week 14 as measured by the Clinical
Global Impression - Improvement scale (CGI-I).
- Zygel was very well
tolerated in this trial and the safety profile was consistent with
previously released data from other Zygel clinical trials. No
serious or severe adverse events were reported.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals is the leader in 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.
Cautionary Note on Forward-Looking
Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. We may, in some cases, use terms such as “predicts,”
“believes,” “potential,” “proposed,” “continue,” “estimates,”
“anticipates,” “expects,” “plans,” “intends,” “may,” “could,”
“might,” “will,” “should” or other words that convey uncertainty of
future events or outcomes to identify these forward-looking
statements. Such statements are subject to numerous important
factors, risks and uncertainties that may cause actual events or
results to differ materially from the Company’s current
expectations. Management’s expectations and, therefore, any
forward-looking statements in this press release could also be
affected by risks and uncertainties relating to a number of other
factors, including the following: the Company’s cash and cash
equivalents may not be sufficient to support its operating plan for
as long as anticipated; the Company’s ability to obtain additional
funding to support its clinical development programs; the results,
cost and timing of the Company’s clinical development programs,
including any delays to such clinical trials relating to enrollment
or site initiation; clinical results for the Company’s product
candidates may not be replicated or continue to occur in additional
trials and may not otherwise support further development in a
specified indication or at all; actions or advice of the U.S. Food
and Drug Administration and foreign regulatory agencies may affect
the design, initiation, timing, continuation and/or progress of
clinical trials or result in the need for additional clinical
trials; the Company’s ability to obtain and maintain regulatory
approval for its product candidates, and the labeling under any
such approval; the Company’s reliance on third parties to assist in
conducting pre-clinical and clinical trials for its product
candidates; delays, interruptions or failures in the manufacture
and supply of the Company’s product candidates the Company’s
ability to commercialize its product candidates; the size and
growth potential of the markets for the Company’s product
candidates, and the Company’s ability to service those markets; the
Company’s ability to develop sales and marketing capabilities,
whether alone or with potential future collaborators; the rate and
degree of market acceptance of the Company’s product candidates;
the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates; the timing and outcome of current and
future legal proceedings; and the extent to which health epidemics
and other outbreaks of communicable diseases, including COVID-19,
could disrupt our operations or adversely affect our business and
financial conditions. This list is not exhaustive and these and
other risks are described in the Company’s periodic reports,
including the annual report on Form 10-K, quarterly reports on Form
10-Q and current reports on Form 8-K, filed with or furnished to
the Securities and Exchange Commission and available
at www.sec.gov. Any forward-looking statements that the
Company makes in this press release speak only as of the date of
this press release. The Company assumes no obligation to update
forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press
release.
Zynerba ContactWill Roberts,
VP Investor Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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