Zynerba Pharmaceuticals Announces Initiation of Phase 2 Trial of Zygel™ in 22q11.2 Deletion Syndrome
May 29 2019 - 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 that it has initiated the Phase 2 INSPIRE (Assessing the
Impact of Zygel [Tra
nsdermal CBD
Gel] on
Pediatr
ic
Behavio
ral and
Emotional Symptoms
of 22q11.2 Deletion Syndrome) trial. The INSPIRE trial will assess
the safety, tolerability and efficacy of Zygel (ZYN002 CBD gel) for
the treatment of behavioral symptoms of 22q11.2 Deletion Syndrome
(22q). The Company expects to present topline data from this study
in the first half of 2020.
“Children born with 22q often require surgeries to
rectify acute physical concerns, like anomalies of the heart and
palate; once corrected, there are a myriad of behavioral symptoms
that need to be addressed,” said Armando Anido, Chairman and Chief
Executive Officer of Zynerba. “Parents of children with 22q report
significantly higher rates of withdrawn behavior, affective
disorders, pervasive developmental problems and anxiety in their
children compared to non-affected children. There is also an
increased risk of developing psychoses such as schizophrenia later
in life, compared to the general population. We are very excited to
initiate the INSPIRE trial to assess the potential impact of Zygel
in these children and adolescents, and look forward to presenting
topline data in the first half of 2020.”The 14-week INSPIRE trial
is an open-label multi-dose Phase 2 clinical trial designed to
evaluate the efficacy and safety of Zygel in approximately 20
children and adolescents (ages six through 17) with
genetically-confirmed 22q. Enrolled patients will receive
weight-based doses of 250 mg daily or 500 mg daily of Zygel. The
efficacy assessments include the Aberrant Behavior
Checklist-Community (ABC-C), the Anxiety, Depression and Mood Scale
(ADAMS), the Columbia Suicide Severity Rating scale (C-SSRS), the
Qualitative Caregiver Reported Behavioral Problem Survey, and
Clinical Global Impression – Severity and Improvement.
About 22q11.2 Deletion Syndrome (22q)As the second
most common chromosomal disorder after Down syndrome, 22q is caused
by a small missing piece of the 22nd chromosome. The deletion
occurs near the middle of the chromosome at a location designated
q11.2. It is considered a mid-line condition, with physical
symptoms including characteristic palate abnormalities, heart
defects, immune dysfunction, and esophageal / GI issues, as well as
debilitating neuropsychiatric and behavioral challenges. Anxiety is
among the most common neuropsychiatric symptoms of 22q and
researchers have found that for children with 22q, anxiety is
linked to poorer adaptive behaviors such as self-care and
communication skills that affect daily life. Children with 22q also
experience withdrawn behavior, ADHD, cognitive impairment, and
autism spectrum disorder that affect communication and social
interaction. Later in life, they are at an increased risk of
developing mental illnesses such as schizophrenia. It is estimated
that 22q occurs in between one in 3,000 and one in 6,000 live
births, suggesting that there are approximately 81,000 people
living with 22q in the U.S. 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
Fragile X Syndrome (FXS) and other neuropsychiatric conditions 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 symptoms,
including social avoidance (prefers isolation from others, prefers
solitary activities, avoids new social activities), irritability
(aggressive to others, tantrums/outbursts, and stubbornness),
social unresponsiveness/lethargy (lack of attention/interaction,
inactive/lack of movement and can resist physical contact), and
anxiety.
Zygel has been designated a Fast Track development
program by the U.S. Food and Drug Administration for treatment of
behavioral symptoms of FXS. Enrollment is ongoing in CONNECT-FX, a
multi-national, randomized, double blind placebo controlled pivotal
clinical trial of Zygel in FXS (https://www.connectfxtrial.com/);
topline data are expected in the second half of 2019. Additionally,
Zynerba expects topline data from its Phase 2 open label BELIEVE 1
trial of Zygel in developmental and epileptic encephalopathies
(DEE) in the third quarter of 2019. Zynerba has also initiated the
Phase 2 BRIGHT trial in Autism Spectrum Disorder and the Phase 2
INSPIRE trial in 22q11.2 Deletion Syndrome, with data expected from
both studies in the first half of 2020.
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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