Zynerba Pharmaceuticals Announces Achievement of Enrollment Target in Phase 2 Trial of Zygel™ in Autism Spectrum Disorder
January 13 2020 - 6:45AM
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced the achievement of its enrollment target for the 14-week
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 of Zygel™ for the treatment of
pediatric and adolescent patients with autism spectrum disorder
(ASD). The Company expects to announce topline results from this
study in the second quarter of 2020.
“We are committed to developing new treatment
options to improve outcomes in patients suffering from a variety of
neuropsychiatric conditions including autism spectrum disorder, and
achieving our enrollment target in the BRIGHT trial is an
especially meaningful step toward this goal,” said Joseph M.
Palumbo, MD, FAPA, MACPsych, Chief Medical Officer of Zynerba. “We
would like to thank everyone involved in this study to date,
particularly the participating patients and their caregivers, as
well as the investigators. We look forward to announcing topline
results from this study in the second quarter of 2020.”
The 14-week exploratory Phase 2 BRIGHT trial has
enrolled 36 patients with ASD at a single clinical site in
Australia. The trial is designed to evaluate the efficacy and
safety of Zygel in children and adolescents (ages four through 17)
with ASD as confirmed by DSM-5 diagnostic criteria for ASD.
Enrolled patients are receiving weight-based initial doses of 250
mg daily or 500 mg daily of Zygel. The efficacy assessments include
the Aberrant Behavior Checklist (ABC), Parent Rated Anxiety Scale –
Autism Spectrum Disorder (PRAS-ASD), Autism Impact Measure (AIM),
the Children’s Sleep Habit Questionnaire (CSHQ), and Clinical
Global Impression – Severity and Improvement (CGI-S, CGI-I). After
completing dosing in the 14-week period, participants may enroll in
a six-month extension trial.
Using the Autism Diagnostic Observation Schedule
(ADOS-2) which is administered at baseline by a qualified
clinician, 94% of enrolled patients had moderate-to-severe symptoms
of ASD at baseline. The mean baseline ABC-C Irritability
subscale score of 30.0 further supports the severity of the
enrolled patient population. Thirty-three (92%) are male. The mean
age of these patients is 9.3 years.
About Autism Spectrum Disorder
(ASD)Autism Spectrum Disorder is a developmental disorder
that affects communication and behavior in approximately one
million pediatric and adolescent patients between the ages of five
and 17 in the U.S. It refers to a range of conditions characterized
by anxiety, repetitive patterns of behavior, impairments in social
communication including verbal and non-verbal communication, and
deficits in developing and maintaining relationships. Although
autism can be diagnosed at any age, it is said to be a
“developmental disorder” because symptoms generally appear in the
first two years of life. Research suggests that genes can act
together with influences from the environment to affect development
in ways that lead to ASD. Newer studies suggest that ASD is linked
to disruption in the endocannabinoid system.
About Zygel™ Zygel (cannabidiol
[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). Recent studies suggest that
Fragile X syndrome (FXS) and other neuropsychiatric conditions
including ASD may be associated with a disruption in the
endocannabinoid (EC) system. Clinical and anecdotal data suggest
that CBD may modulate the EC system and improve certain core social
and behavioral autism-related symptoms, including social avoidance
and anxiety.
Zygel is being studied in clinical trials in a number of rare
and near-rare neuropsychiatric conditions. Target enrollment has
been achieved in the Phase 2 BRIGHT trial of Zygel in ASD, with
topline data anticipated in the second quarter of 2020. Enrollment
is ongoing in CONNECT-FX, a multi-national, randomized, double
blind, placebo-controlled pivotal clinical trial of Zygel in
Fragile X syndrome (https://www.connectfxtrial.com/) and in the
Phase 2 INSPIRE trial in 22q11.2 deletion syndrome. Zynerba also
expects to meet with the U.S. Food and Drug Administration in the
first half of 2020 to discuss the recently announced positive
topline safety and efficacy data from its Phase 2 BELIEVE 1
clinical trial in patients with developmental and epileptic
encephalopathies, and the clinical path forward.
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. For example, there can be no guarantee that the
Company will obtain approval for Zygel from the U.S. Food and Drug
Administration (FDA) or foreign regulatory authorities; even if
Zygel is approved, the Company may not be able to obtain the label
claims that it is seeking from the FDA. 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 FDA 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.
Zynerba ContactWilliam Roberts, Vice President,
Investor Relations and Corporate CommunicationsZynerba
Pharmaceuticals484.581.7489 robertsw@zynerba.com
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