- Company Will Focus on Four Childhood
Neuropsychiatric Disorders: FXS, DEE, Autism Spectrum Disorder and
22q11.2 Deletion Syndrome -
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
provided an update on its clinical progress.
- Zynerba remains on track to report top line results from the
CONNECT-FX (Clinical
study of Cannabidiol (CBD)
in Children and
Adolescents
with Fragile X) study
in Fragile X Syndrome in the second half of 2019;
- The Company has completed enrollment in its Phase 2 BELIEVE 1
(Open Label Study to Assess the Safety and
Efficacy of ZYN002 Administered as a Transdermal
Gel to Children and
Adolescents with Developmental
and Epileptic Encephalopathy) clinical trial in
children and adolescents with developmental and epileptic
encephalopathies (DEE). Top-line results will be reported in the
third quarter of 2019;
- Zynerba has expanded its pipeline with the addition of two new
childhood neuropsychiatric clinical targets for ZYN002: Autism
Spectrum Disorder (ASD) and 22q11.2 Deletion Syndrome (22q Deletion
Syndrome; 22q), a rare genetic syndrome leading to significant
impairments, including neuropsychiatric disorders. The Company
expects to initiate open label Phase 2 studies in these indications
in the first half of 2019 and report top-line results in first half
2020;
- The Company has postponed the initiation of its clinical trial
in adult epilepsy until after the completion of the four childhood
neuropsychiatric studies; and
- As a result, Zynerba expects to extend its cash runway into the
second half of 2020.
“Our aspirations and expectations are clear: To work closely
with the U.S. Food and Drug Administration to expand the
opportunity for pharmaceutically-developed CBD treatments that meet
their rigorous medical and manufacturing standards, and in doing
so, continue toward our goal of addressing significant unmet
medical needs in neuropsychiatric disorders,” said Armando Anido,
Chairman and Chief Executive Officer of Zynerba. “With the new
indications of ASD and 22q, in addition to FXS and DEE, we now have
four shots on goal to show the clinical benefit of ZYN002
transdermal gel in patient populations that have few, if any,
therapeutic options available.”
Fragile X Syndrome Pivotal Data Expected in
2H2019Enrollment is progressing in CONNECT-FX, a pivotal,
multi-national, randomized, double blind,
placebo-controlled study evaluating the efficacy and safety of
ZYN002 in three through 17-year old FXS patients with full mutation
of the FMR1 gene. The primary endpoint is the change from baseline
to the end of the treatment period in the Aberrant Behavior
Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance
subscale. Clinical investigative sites are enrolling patients in
the United States, Australia, and New Zealand. Patients who have
completed the double-blind phase are now enrolling into the
12-month open label phase. The Company is on track to report top
line data in the second half of 2019; there are currently no
approved products indicated for FXS.
Completion of Enrollment in BELIEVE 1 The
Company has completed enrollment in BELIEVE 1, an open label
multi-dose Phase 2 clinical trial evaluating the efficacy and
safety of ZYN002 in children and adolescents (three through 17
years) with developmental and epileptic encephalopathies (DEE), as
classified by the International League Against Epilepsy (ILAE)
(Scheffer et al. 2017). Children and adolescents with a variety of
DEEs have been enrolled in BELIEVE 1 from sites in Australia and
New Zealand. Once completing a four-week baseline period to
determine seizure frequency, patients are treated with ZYN002 for
26 weeks, initially receiving weight-based doses of 250 mg or 500
mg daily of ZYN002 CBD gel during a two-week titration period,
followed by maintenance doses of 250 mg up to 1,000 mg daily for 24
weeks. The primary efficacy assessment is reduction in seizure
frequency at week 26 compared to baseline. Patients successfully
completing the study may elect to enter a 6-month extension study.
The Company expects to announce topline data from this study in the
third quarter of 2019.
DEE is a heterogeneous group of rare and ultra-rare epilepsy
syndromes that manifest with seizures, behavioral disturbances, or
EEG abnormalities that can directly worsen cognition and behavior.
These disorders are often progressive and are highly resistant to
treatment. The syndromes involve significant developmental
impairment (developmental encephalopathies) or regression of
developmental progress (epileptic encephalopathies). DEEs include a
number of epilepsy syndromes, including Doose, Dravet,
Lennox-Gastaut, and Ohtahara (early infantile epileptic
encephalopathy); and early-onset epilepsy syndromes caused by
variants of genes including SYNGAP1 and SCN1A, among others.
Autism Spectrum Disorder (ASD) in Pediatric
PatientsASD is a developmental disability defined by
anxiety, the presence of restricted, repetitive patterns of
behaviors, impairments in social communication and interactions,
deficits in verbal and non-verbal communication, and deficits in
developing, understanding and maintaining relationships. This
near-rare neuropsychiatric disorder affects approximately one
million pediatric and adolescent patients (five to 17 years of age)
in the U.S. Recent studies suggest that ASD may be associated with
a disruption in the endocannabinoid system; clinical and anecdotal
data show that modulation of the endocannabinoid system with CBD
has shown therapeutic potential in ameliorating certain behaviors
associated with ASD, including social avoidance and anxiety. The
Company expects to initiate an open label Phase 2 study in
pediatric patients with ASD in Australia in the first half of
2019.
22q11.2 Deletion Syndrome (22q)22q is the most
common chromosomal known contiguous gene deletion syndrome. This
syndrome is associated with congenital anomalies, cognitive
deficits, and neuropsychiatric symptoms including anxiety
disorders, ADHD, developmental delays, cognitive impairment and
psychoses. Early onset of neuropsychiatric symptoms disrupts
development and quality of life, and is thought to heighten the
risk of development of psychotic disorders later in life. This rare
disorder affects approximately 81,000 patients in the U.S. CBD may
treat the neuropsychiatric symptoms of 22q due its activity as an
agonist at serotonin 1A receptors, an antagonist at GPR55
receptors, and a modulator of the endocannabinoid system. The
Company expects to initiate an open label Phase 2 study in
pediatric patients with 22q in Australia in the first half of
2019.
Adult Refractory Focal EpilepsyThe Company will
postpone the initiation of its planned Phase 2B study of ZYN002 in
adult refractory focal seizures until after reporting data from the
four childhood neuropsychiatric studies.
Financial OutlookManagement believes its
current cash and cash equivalent position is sufficient to fund
operations and capital requirements into the second half of 2020,
past data readouts from the CONNECT-FX pivotal trial in Fragile X
Syndrome, the BELIEVE 1 Phase 2 trial in DEE, and the planned Phase
2 trials in both ASD and 22q.
Conference call
informationZynerba management will host a live
conference call and webcast today at 8:30 am Eastern Standard
Time to provide a corporate update. The call can be accessed
by dialing (866) 573-0180 (U.S. and Canada) or (430) 775-1345
(international) and referencing conference ID 6892856. To access
the live webcast or the replay, visit the investor page of the
Company’s website at http://ir.zynerba.com/. The webcast will
be recorded and available on the Company’s website for 30 days.
About ZYN002 Zynerba’s ZYN002 CBD gel is the
first and only pharmaceutically-manufactured CBD formulated as a
patent-protected permeation-enhanced clear gel, designed to provide
controlled drug delivery into the bloodstream transdermally (i.e.
through the skin).
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 and a heterogeneous group
of rare and ultra-rare epilepsies known as developmental and
epileptic encephalopathies (DEE). 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;
and the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates. 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.
Investor Contact
Will Roberts, VP Investor Relations and Corporate
CommunicationsZynerba
Pharmaceuticals484.581.7489robertsw@zynerba.com
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