Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, will
present an overview of its Zygel™ (ZYN002) development program in
Fragile X syndrome (FXS) and additional caregiver-reported data
from its 14-week pivotal CONNECT-FX (
Clinical
study
of Ca
nnabidiol (CBD) in
Childr
en and
Adoles
cen
ts with
Fragile
X) trial during the 17th
NFXF International Fragile X Conference Research Roundup. The
multi-national, randomized, double-blind, placebo-controlled trial
assessed the efficacy and safety of Zygel™ CBD gel as a treatment
for behavioral symptoms of Fragile X syndrome (FXS) in 212
patients; topline results were announced on June 30, 2020. (Press
release).
The presentation entitled, “Zygel (ZYN002) Development Program
in Fragile X Syndrome” will take place at 3:45PM ET today, July 22,
2020. Additional information on the conference and registration are
available here:
https://fragilex.org/get-involved/international-fragilex-conference/.
A copy of today’s presentation will be made available prior to the
time of presentation on the Zynerba corporate website at
http://zynerba.com/publications/.
“I am very pleased to participate in today’s Fragile X Research
Roundup on Fragile X Awareness Day,” said Joseph M. Palumbo, MD,
FAPA, MACPsych, Chief Medical Officer of Zynerba. “We believe that
the caregiver data that we are presenting today further support the
statistically significant improvement we achieved in the primary
endpoint of social avoidance in patients with full methylation of
their FMR1 gene. We look forward to discussing these and other data
with the U.S. Food and Drug Administration as soon as possible
regarding a potential regulatory path forward.”
Qualitative Caregiver Reported Behavioral
Survey
Consistent with guidance from the FDA on capturing the voice of
the patient in drug development, the Company collected qualitative
data on the importance of various FXS behaviors to caregivers
entering the trial. Utilizing the Qualitative Caregiver Reported
Behavioral Survey, caregivers were asked to describe their most
important behavioral challenges at baseline. The results of the
survey indicate that caregivers found anxiety, socially avoidant
behaviors (including elopement and isolation seeking), and
disruptive behaviors (including aggression and temper tantrums) to
be the most challenging.
Figure 1. Results of Qualitative Caregiver Reported Behavioral
Survey is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/67243db8-e2cb-48f4-9ba9-71f82b6355c8
Caregiver Global Impression - Change from Baseline to
Week 12
Using the Caregiver Global Impression - Change survey,
caregivers were asked to complete a questionnaire rating how their
child’s behavior changed with respect to social avoidance and
isolation, irritable and disruptive behaviors, social interactions,
and the child’s overall behavior at the end of the 12 week
treatment period compared to the beginning of the trial, utilizing
a seven point scale from ‘much worse’ to ‘much better’. The results
of this survey show a broad shift toward global improvement from
baseline to week 12 for patients with full methylation of their
FMR1 gene (FMet patients), with three of the four behavioral
domains showing a statistically significant change in favor of
patients on Zygel and the fourth domain trending toward
significance.
Figure 2. Results of Caregiver Impression - Change: Full
Methylation Group is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/87e1db62-97e8-4dc6-9655-db036a073f20
These statistically significant Caregiver Global Impression -
Change data in socially avoidant behavior support the statistically
significant improvement observed in the CONNECT-FX primary endpoint
of social avoidance in FMet patients who received Zygel compared to
placebo (p=0.020).
About Fragile X Syndrome (FXS)
Fragile X syndrome is a rare genetic developmental disability
that is the leading known cause of both inherited intellectual
disability and autism spectrum disorder, affecting 1 in 3,600 to
4,000 males and 1 in 4,000 to 6,000 females. It is the most common
inherited intellectual disability in males and a significant cause
of intellectual disability in females, and the leading genetic
cause of autism spectrum disorder (ASD). The disorder negatively
affects synaptic function, plasticity and neuronal connections, and
results in a spectrum of intellectual disabilities and behavioral
symptoms, such as social avoidance and irritability. In the
US, there are about 71,000 people suffering with FXS, approximately
60% of whom have full methylation of the FMR1 gene.
FXS is caused by a mutation in FMR1, a gene which modulates a
number of systems, including important effects on the
endocannabinoid system, and most critically, codes for a protein
called FMRP. This protein helps regulate the production of other
proteins and plays a role in the development of synapses,
which are critical for relaying nerve impulses, and in regulating
synaptic plasticity. The FMR1 mutation manifests as multiple
repeats of a DNA segment, known as the CGG triplet repeat. In most
neurotypical people, the FMR1 gene correctly codes for the FMRP
protein. In neurotypical individuals, there are CGG repeats, but
these repeats only occur between 5 and 40 times. As a result, FMRP
is manufactured at levels that enable control over behaviors like
social avoidance and anxiety. In people with full mutation of the
Fragile X gene, the CGG segment is repeated more than 200 times and
in most cases causes the FMR1 gene to not function. However, the
methylation of the FMR1 gene also plays a role in determining
functionality of the gene. At greater than 90% methylation, which
is considered “full methylation”, the FMR1 gene is silenced,
therefore, no FMRP is produced, and the systems and processes that
are expected to be affected by FMRP become dysregulated.
People with genetically confirmed full mutation Fragile X and
full methylation of their FMR1 gene are generally the most severely
impacted by the disorder.
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 ContactWilliam Roberts, Vice President,
Investor Relations and Corporate CommunicationsZynerba
Pharmaceuticals484.581.7489 robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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