Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for orphan neuropsychiatric disorders, today announced
that the Company is revising its target for announcing topline
results from the pivotal Phase 3 RECONNECT trial of Zygel in
Fragile X syndrome (FXS). The Company now expects topline results
in the first half of 2024, rather than the second half of 2023.
The impact of environmental factors including unusually high
rates of RSV (respiratory syncytial virus) and influenza and the
continued impact of COVID-19 among children, adolescents, family
members, and investigational center personnel, has resulted in
higher than expected cancellations of initial screening visits at
the investigational centers which has led to slower than
anticipated enrollment. Despite these issues, clinical
investigators at the 27 sites in the U.S., Australia, the UK and
Ireland remain committed to completing the trial.
“Though we are still striving to report topline results by the
end of 2023, when we look at the current and projected impact of
the Tripledemic, we believe that the first half of 2024 is a more
reasonable timeframe to have topline results from RECONNECT,” said
Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We
have also prioritized our corporate business plans to focus almost
exclusively on completing RECONNECT and now expect we have cash
into mid-year 2024.”
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 the treatment of 22q. Additionally, Zygel
has been designated a Fast Track development program for treatment
of behavioral symptoms of FXS.
About RECONNECT
RECONNECT is a randomized, double-blind, placebo-controlled,
multiple-center efficacy and safety trial of Zygel administered as
a transdermal gel to children and adolescents ages 3 through 17
with Fragile X syndrome. For more information regarding
participation visit www.fragilexhelp.com.
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.
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 4,000 males
and 1 in 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 U.S., there are
about 78,000 people suffering with FXS, approximately 60% of whom
have complete 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. As a
result, FMRP is produced at levels that enable control over
behaviors like social avoidance and anxiety. In people with full
mutation of the FMR1 gene, the CGG segment is repeated more than
200 times, and in most cases causes the gene to not function.
Methylation of the FMR1 gene also plays a role in determining
functionality of the gene. For patients with complete methylation,
no FMRP is produced. With no FMRP, the systems and processes that
are modulated by FMRP become dysregulated.
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 expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
incentive and other tax credit eligibility, collectability and
timing, and availability of and the need for additional financing;
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,
the European Medicines Agency and other 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 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; and the extent to which inflation or global
instability, including political instability, may disrupt our
business operations or our financial condition. 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 Contact
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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