Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announces the initiation of a pivotal, multinational randomized,
double-blind, placebo-controlled, multiple-center, efficacy and
safety (RECONNECT) Phase 3 trial. The RECONNECT trial is designed
to evaluate the efficacy and safety of Zygel (cannabidiol
formulated in a transdermal gel) in children and adolescents with
Fragile X syndrome (FXS). The trial is planned to confirm the
positive results observed in a population of responders in the
Company’s 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. FXS is a
genetic condition that causes intellectual disability, behavioral
and learning challenges and is the most common known single-gene
cause of autism spectrum disorder.
“The RECONNECT trial provides us with an opportunity to confirm
the positive results observed in a population of responders in our
CONNECT-FX trial and further demonstrate the effect of Zygel on
behaviors associated with FXS,” said Armando Anido, Chairman and
Chief Executive Officer of Zynerba. “A significant unmet medical
need continues to exist for therapeutics to treat patients with
FXS. If successful, we believe the study could serve as the basis
for Zygel approval in the U.S. for patients with FXS.”
“Children with FXS exhibit a number of developmental and
behavioral symptoms including anxiety, social avoidance,
hyperactivity, and socially unresponsive behaviors that
significantly impact the family and the child’s capacity to
interact with them, their peers, and care providers,” said Caroline
Buchanan, M.D., Greenwood Genetic Center, Greenville, S.C., and an
investigator in the RECONNECT trial. “Potentially having an
approved treatment option for these vulnerable patients would be a
significant step forward for FXS patients and their families.”
RECONNECT will be an 18-week trial that is expected to enroll
approximately 200 children and adolescents, aged three through 17
years, at approximately 25 clinical sites in the United States,
Australia, the UK and Ireland. Approximately 160 of the patients
enrolled will have complete (100%) methylation of their FMR1 gene
and approximately 40 patients will have partial methylation of
their FMR1 gene. Patients will be randomized 1:1 to either Zygel or
placebo. Randomization will be stratified by gender, methylation
status and weight.
The primary endpoint for the trial will be the change from
baseline to the end of the treatment period in the Aberrant
Behavior Checklist-Community FXS Specific (ABC-CFXS) Social
Avoidance subscale in patients who have complete methylation of
their FMR1 gene. The ABC-CFXS Social Avoidance subscale is the same
primary endpoint used in the CONNECT-FX trial. Key secondary
efficacy endpoints include:
- The change from baseline to the end
of the treatment period in the ABC-CFXS Irritability subscale in
patients who have complete methylation of their FMR1 gene.
- Percent of patients with any
improvement on the Caregiver Global Impression of Change (CaGI-C)
at the end of the treatment period for Social Interactions among
patients with complete methylation of the FMR1 gene.
- Percent of patients rated as
improved on the Clinical Global Impression- Improvement (CGI-I)
scale among patients with complete methylation (100%) of the FMR1
gene.
- The change from baseline to the end
of the treatment period in the ABC-CFXS Social Avoidance subscale
among all randomized patients (complete and partial methylation of
the FMR1 gene).
Topline results for the RECONNECT trial are expected in the
second half of 2023.
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.
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 U.S.,
there are about 71,000 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
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 Contacts
Peter VozzoWestwicke/ICROffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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