Zynerba Pharmaceuticals Initiates Phase 2 Trial of Zygel™ in Autism Spectrum Disorder
March 07 2019 - 7:00AM
- Topline Data from BRIGHT Study Expected in the
First Half 2020 -
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced that it has initiated the 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. The trial will assess the safety,
tolerability and efficacy of Zygel (previously referred to as
ZYN002) for the treatment of child and adolescent patients with
Autism Spectrum Disorder (ASD). The Company expects to present
topline data from this study in the first half of 2020.
“Autism spectrum disorder can have a devastating
impact on a child and their family,” said Armando Anido, Chairman
and Chief Executive Officer of Zynerba. “The medical need is
significant and unmet despite high awareness and advocacy efforts.
Though there has been an accelerating rate of diagnosis, to date
there are only two FDA approved products indicated for the
treatment of ASD symptoms. Both have significant side effect
profiles, and neither have been approved to address the key
symptoms of social impairment and anxiety. We are excited to
initiate the BRIGHT trial evaluating the role of Zygel in ASD and
are hopeful that Zygel may improve some of the core social and
behavioral symptoms of ASD. We look forward to presenting topline
data in the first half of 2020.”
The 14-week BRIGHT trial is an open-label
multi-dose Phase 2 clinical trial designed to evaluate the efficacy
and safety of Zygel in approximately 36 children and adolescents
(ages four through 17) with ASD as confirmed by DSM-5 diagnostic
criteria for ASD. Enrolled patients will receive weight-based
initial doses of 250 mg daily or 500 mg daily of Zygel. The
efficacy assessments include the Aberrant Behavior Checklist,
Parent Rated Anxiety Scale – Autism Spectrum Disorder, Autism
Impact Measure, and Clinical Global Impression – Severity and
Improvement.
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.
About Zygel™ Zygel (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 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.
Enrollment is ongoing in a multi-national, randomized, double
blind placebo controlled Clinical study
of Cannabidiol (CBD) in
Children and
Adolescents with
Fragile X (CONNECT-FX), a pivotal
clinical trial of ZYN002 in FXS (https://www.connectfxtrial.com/);
topline data from CONNECT-FX are expected in the second half of
2019. Additionally, Zynerba expects topline data from its Phase 2
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 (BELIEVE 1) clinical
trial in the third quarter of 2019.
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
StatementsThis 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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